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Note While This Document Is Obsolete

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NOTE: WHILE THIS DOCUMENT IS OBSOLETE A CURRENT VERSION OF THIS LISTING IS
AVAILABLE AT www.avarx.com.
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AVAILABLE PHARMACEUTICAL PRODUCTS
December 2011
TABLE OF CONTENTS
ACTIVE COMPANY TAKEOVER SITUATIONS ............................................................................................................... 4
ADDICTION MANAGEMENT ..................................................................................................................................... 10
ANTIBODIES ............................................................................................................................................................. 11
ANTI-INFECTIVES ...................................................................................................................................................... 15
BANKRUPTCY AND RESTRUCTURING SITUATIONS .................................................................................................. 18
BIG PHARMA / BIG BIOTECH PROGRAMS FOR OUTLICENSING ............................................................................... 19
BIGGER DEALS ($200MM+ IN VALUE) ..................................................................................................................... 20
BIODEFENSE ............................................................................................................................................................. 24
BIOSIMILARS ............................................................................................................................................................ 25
BONE AND SPINE / ORTHOBIOLOGICS..................................................................................................................... 25
CARDIOLOGY - PRIMARY CARE ................................................................................................................................ 27
CARDIOLOGY - SPECIALTY CARE .............................................................................................................................. 29
CNS - NEUROLOGY ................................................................................................................................................... 32
CNS - PSYCHIATRY .................................................................................................................................................... 38
CNS - SLEEP .............................................................................................................................................................. 40
CRITICAL CARE PRODUCTS ....................................................................................................................................... 41
DENTAL DRUGS ........................................................................................................................................................ 42
DERMATOLOGY - AESTHETICS ................................................................................................................................. 42
DERMATOLOGY - MEDICAL ...................................................................................................................................... 43
DIABETES - ORALS .................................................................................................................................................... 45
DIABETES - INSULINS / INJECTIBLES ......................................................................................................................... 46
DIAGNOSTICS ........................................................................................................................................................... 47
DRUG DELIVERY ....................................................................................................................................................... 48
EMERGING MARKETS - PARTICULARLY BRAZIL, CHINA, INDIA, TURKEY DEALS ....................................................... 48
ENDOCRINE .............................................................................................................................................................. 49
FIBROSIS................................................................................................................................................................... 50
GASTROINTESTINAL ................................................................................................................................................. 51
GENERICS ................................................................................................................................................................. 55
GOUT ....................................................................................................................................................................... 58
HAIR / SCALP ............................................................................................................................................................ 58
HEARING / EAR DISORDERS ..................................................................................................................................... 59
HEMATOLOGY .......................................................................................................................................................... 59
HEPATOLOGY ........................................................................................................................................................... 62
HOSPITAL PRODUCTS .............................................................................................................................................. 63
IMMUNOLOGY / INFLAMMATION / AUTOIMMUNE DISEASE ................................................................................. 65
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LATE STAGE ASSETS (PHASE 3 STARTED TO NEWLY APPROVED) ............................................................................ 68
MATURE BRANDED PRODUCTS ............................................................................................................................... 73
NEONATOLOGY ........................................................................................................................................................ 74
NUTRITIONALS AND NATURAL PRODUCTS .............................................................................................................. 74
OBESITY.................................................................................................................................................................... 75
ONCOLOGY - LIQUID TUMORS ................................................................................................................................. 75
ONCOLOGY - SOLID TUMORS .................................................................................................................................. 78
ONCOLOGY - SUPPORTIVE CARE ............................................................................................................................. 86
OPTHALMOLOGY ..................................................................................................................................................... 88
ORGAN TRANSPLANT ............................................................................................................................................... 89
ORPHAN PRODUCTS ................................................................................................................................................ 90
OTC PRODUCTS ........................................................................................................................................................ 94
PAIN PRODUCTS ...................................................................................................................................................... 95
PEDIATRICS ............................................................................................................................................................ 100
PROTEIN PLATFORMS ............................................................................................................................................ 102
RENAL / NEPHROLOGY .......................................................................................................................................... 102
RESPIRATORY ......................................................................................................................................................... 105
REVERSE MERGER CANDIDATES ............................................................................................................................ 108
RIGHTS IN ASIA ...................................................................................................................................................... 109
RIGHTS IN CANADA ................................................................................................................................................ 111
RIGHTS IN THE EU .................................................................................................................................................. 112
RIGHTS IN LATIN AMERICA .................................................................................................................................... 114
RIGHTS IN MIDDLE EAST / AFRICA ......................................................................................................................... 116
ROYALTIES .............................................................................................................................................................. 116
SMOKING CESSATION / SAFE CIGARETTES ............................................................................................................ 117
SPECIALTY PHARMA - MARKETED PRODUCTS ....................................................................................................... 117
STEM CELLS, RNAI AND CELL THERAPY .................................................................................................................. 120
UROLOGY AND MEN’S HEALTH ............................................................................................................................. 121
VACCINES ............................................................................................................................................................... 123
VIROLOGY .............................................................................................................................................................. 125
WOMEN’S HEALTH ................................................................................................................................................ 129
WOUND CARE AND TISSUE SEALANTS .................................................................................................................. 132
TORREYA PARTNERS PERSONNEL .......................................................................................................................... 134
RECENT TRANSACTIONS WHERE TORREYA ACTED AS FINANCIAL ADVISOR ......................................................... 135

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December 2011
AVAILABLE PHARMACEUTICAL PRODUCTS
This report is a periodic compilation of pharmaceutical products that are currently in licensing processes or where
actual or potential M&A discussions are taking place. We have divided opportunities by therapeutic area. Items
involving public companies are only listed where it is broadly understood that a licensing / sale process is
underway or where a product that is an excellent candidate for outlicensing is available but no known active
process is underway. This list is distributed to individuals with an interest in business development in the
pharmaceutical industry. Occasionally, we include products where the owner has asked that their identity remain
undisclosed. In most cases Torreya Partners can make an introduction to a party noted as “undisclosed”.
To assist you, we highlight listings with the following tags:
NEW items flagged are new since the last listing of products.
UPDATE denotes a change on an existing listing.
COMPLETED denotes that a transaction was completed on a previously listed available product.
H High interest or likely to end up as a large deal. An indicator of one of the higher quality available assets.
$ items denote marketed products
In many cases we also provide a web link to relevant information about a compound or company noted by (Link)
or, alternatively contact information (Contact) or a non-confidential presentation (Noncon).
Torreya Partners is pleased to be collaborating with
Deloitte Recap which offers comprehensive Biopharma
alliance information, deal values, and drug development
histories to support the development of alliance and
clinical development strategies. You will find some references to Deloitte Recap material and products in this
report.
ACTIVE COMPANY TAKEOVER SITUATIONS
NEW Abdi Ibrahim Ilac - largest Turkey drug maker with revenues over $800mm. Reported in May 2011 that was in
discussions to sell a strategic stake.
UPDATE Actelion - has received takeover approaches from several strategic bidders according to the Wall Street
Journal (10/7/2010). Activist shareholder in Elliott pushed for a change of control transaction. Wall Street Journal
Sep 28, 2011: “Elliott Advisors Cuts Actelion Stake in Wake of Control Battle”. Elliott failed to put its own directors
on Board and JP Garnier, formerly CEO of GSK, recently became Chairman. A takeover of Actelion appears to be
quite unlikely in light of these events.
Acusphere - Imagify, pending EMA filing for approval of this cardiac imaging agent. Differentiated from SPECT
agents on the market insofar as permits evaluating of myocardial perfusion, an important marker of coronary
artery disease (CAD) without radioactive markers. Potential first-to-market drug in $600 million and $2 billion
addressable market in E.U. and U.S. respectively. Recent SPA from FDA defines design of final U.S. trial needed for
approval. Company is assisted by Torreya Partners. For details contact [email protected]
(Link)
UPDATE Akebia - Positive Phase 2 with a HIF modulator for the treatment of anemia. Originally developed at P&G
Pharma. Open to a licensing deal in but could consider other possibilities including a change of control. Company
expects to report a second Phase 2 trial dataset in March 2012. (Link)
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UPDATE Allos Therapeutics - Oncology marketer with Folotyn® for liquid tumors. After a recently failed merger
attempt with AMAG, Allos is rumored to be continuing to explore strategic alternatives with the assistance of JP
Morgan. (Link)
UPDATE $ AMAG - Feraheme IV iron product - Recently approved. Company is commercializing on its own. AMAG’s
recent merger attempt with Allos was ended in November 2011. On Nov 17, 2011, AMAG announced that it had
hired Jefferies to explore all opportunities to enhance shareholder value. Frank Thomas, interim CEO of AMAG
indicated: “We will expeditiously complete this process, which will include a parallel review of a potential sale of
the company and other strategic merger and acquisition transactions.”
UPDATE Amarin - Developing a pure omega-3 for reduction of triglycerides. Phase 3 data reported out very strong.
Company has indicated that is has retained a financial advisor (Lazard) to explore a sale. Update: Nov 2011 - no
sale reported and company’s valuation has dropped from over $2bn to under $1bn. Amarin recently filed an NDA
for its lead product. Amarin appears to be a likely takeover candidate for the next year. (Link)
Amoun Pharmaceutical - An Egyptian company that manufactures off-patent branded generic formulations. It is
one of the largest pharmaceutical companies in Egypt. It sells over 135 human products in over 275 forms. Of
these products, 33 occupy the top 2 positions in their respective therapeutic categories and subcategories. Open
to a company sale or strategic stake purchase. Reuters - Dec 6, 2010: “CVCI is also preparing to sell Amoun, one of
Egypt's biggest drugmakers, people familiar with the matter told Reuters on Oct. 20. It owns Amoun with two
other co-investors.” Bloomberg reported in Feb 2011 that the company has been looking for $1 billion in a sale
price but that political upheaval in Egypt has hindered the sale.
COMPLETED Anadys - ANA598 is a low-nanomolar inhibitor of HCV genotype 1a and 1b replicons via N5b
polymerase. Has started a Phase 2 trial. Anadys also has ANA773, an oral inducer of endogenous interferons with
nice Phase 1 data. May 2010 - Lazard retained to act as advisor to Anadys to explore strategic alternatives. Nov
2010 press release: Anadys continues to work with Lazard Frères & Co. LLC. to explore potential strategic
transactions, in parallel with moving the ANA598 and ANA773 programs forward. Update: Oct 17, 2011 - Roche
acquires Anadys for $230mm - a 256% premium.
UPDATE $ Avanir - Launched Neudexta for the treatment of pseudobulbar effect and, potentially, other indications.
Positive data and good patent picture. Widely rumored to be an M&A candidate. $5.7 million in revenue in first
year of launch (soft numbers). Market cap of $248 million as of November 2011. EMA application recently
accepted.
COMPLETED $ Azur Pharma has hired Lazard to advise on funding options including a potential stake sale according
to the Sunday Times on May 22, 2011. Company has a solid franchise as a marketer of drugs in the CNS and
women’s health areas. A company sale could be possible. Update: Sep 19, 2011 - Jazz Pharma merges with Azur
Pharma as an Ireland domiciled company with former Jazz shareholders taking 80$ of the combined company. A
transaction closing is expected in Q1 2012.
UPDATE Biofarma - Biofarma for sale via JP Morgan. Reuters (12/6/2010): Citigroup's (C.N) venture capital arm and
two co-investors have begun an auction of Turkish copycat drugmaker Biofarma, three people familiar with the
matter said, in what could be Turkey's biggest healthcare deal.” Update: A number of parties rumored to have
looked at this asset but price ask was seen as prohibitive. As of Nov 2011 no sale had taken place.
UPDATE Biota - Developing a once weekly inhalable long-acting neuraminidase inhibitors for the treatment of flu.
Would compete against Relenza from GSK. Currently partnered with Daichi-Sankyo in Japan. Received a large
BARDA grant in Q2 2011 and has retained Piper Jaffray as financial advisor to maximize value of existing programs
and help to enter the U.S. market on a commercial basis. No transaction announced as of December 2011. (Link)
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NEW China Nuokang -i ts lead products include Baquting for bleeding control, Aiduo, a cardiovascular stress imaging
agent, and Aiwen, an anti-arrhythmic agent. The company has a value of $180mm. Biopharm Insight in November
reported that this company hired Lazard for a sale process.
$ Claris Life Sciences - Indian injectibles company has hired Rothschild to look at strategic options. Company has a
rich pipeline of hospital generic injectibles using novel delivery methods. (Link)
UPDATE Covidien Pharmaceuticals - According to the New York Times on June 7, 2011 “Covidien, the health care
company spun out from Tyco four years ago, may seek to sell its pharmaceutical unit…” This division of Covidien
(formerly Mallinckrodt) has a major business selling pain products (both branded and generics) and imaging
products. Revenues are around $2 billion. Update: As of December 2011 no sale has taken place. Company is
rumored to be interested in a sale of the whole business (rather than pieces) for a full price. YE 2011 numbers
reported on Nov 15, 2011 and were robust (sales up 9% yoy) with strong performance in generics.
UPDATE Exelixis - According to Bloomberg on April 12, 2011: “Exelixis Inc. is working with Goldman Sachs Group Inc.
to prepare for potential takeover offers after its experimental drug helped prostate-cancer patients in a study.”
Company’s XL-184 has reported dramatic data on reducing metastatic prostate lesions at ASCO. Update: company
has hired a Chief Commercialization Officer with intention to introduce cabozantinib to the U.S. market and has
reported strong data for cabozanitinib in the treatment of medullary thyroid cancer.
UPDATE Gen-Probe - Widely rumored to be for sale with interest from Novartis. Well known diagnostics company.
Process well underway but company viewed as expensive. Update: Wall Street journal reports on July 20, 2011
that “Novartis is no longer actively pursuing U.S. medical diagnostic-testing company Gen-Probe, meaning Gen-
Probe could end its sales process…”
UPDATE GlaxoSmithKline - running a process to divest non-core OTC brands with assistance from Goldman Sachs.
The products to be divested, which are primarily sold in Europe and the United States, had sales in 2010 of
approximately £500 million, 10% of GSK’s total Consumer Healthcare turnover. They include analgesics:
Solpadeine, BC and Goody’s; vitamin and supplement product Abtei; feminine hygiene treatment Lactacyd; and alli
for weight management. Reuters Update on Nov 14, 2011: “GlaxoSmithKline is assessing final bids for a clutch of
its non-prescription drugs, keeping the process on track for the selection of a buyer by the end of the year, people
familiar with the matter said on Monday.” (Link)
COMPLETED Graceway - Could consider sale of products from 3M and has hired Lazard to explore alternatives
through an ongoing process. Aldara for acitinic keratosis a key seller but was recently genericized. Update: Nov 23,
2011 - Medicis acquired Graceway for $455mm after the company filed for Chapter 11 bankruptcy on Sep 29,
2011.
NEW Guangxi Golden Throat (Guang Xi Jin Sang Zi), a privately held manufacturer of healthcare products, is
reportedly in sale talks. The company has annual revenues of around $47mm from its throat lozenges, which sell
under the 'Golden Throat' brandname.
UPDATE Hi-Tech Pharmacal - Market rumors in April 2011 that company could be purchased. As of December 2011
2011 no transaction had taken place of this manufacturing company of generic liquids and ointments. The
company has delivered strong earnings throughout 2011 and is trading at close to twice the price in Dec 2011 it
had earlier in 2011.
COMPLETED Inova Pharmaceuticals - Asian OTC and Pharma product company with revenues > $500mm. Ran a sale
process via Greenhill Caliburn. No transaction has taken place as of June 2011. Nov 21, 2011: Valeant buys iNova
for $625 million.
UPDATE Ipsen - Completed a strategic review in 2011 with the theme of increasing focus and growing the footprint.
The implications of this for potential partnerships or asset divestitures are threefold: (1) Ipsen looking for a partner
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in the French primary care arena, (2) Ipsen looking to find a buyer for its industrial site in Dreux France which
makes solid dose and liquid formulations and (3) “Ipsen will explore all options to maximize value (of its short
stature franchise) while meeting its obligations to patients and partners. It will be managed directly by regions and
countries.” Update: Ipsen sold off the rights to Apokyn in the U.S. for over 1X revenues on Nov 2, 2011.
COMPLETED Labopharm - Company has retained a financial advisor and is actively looking at alternatives. Press
Release (4/29/2011): “The Board and management are currently engaged in reviewing the Company's
technologies, products and product candidates to determine the appropriate path forward for each of its assets,
including consideration of opportunities to monetize the Company's approved products. Labopharm believes that
its INTELLITAB(TM) and POLYMERIC NANO-DELIVERY SYSTEMS(TM) (PNDS(TM)) technology platforms could have
potential in their respective fields of application and are evaluating strategies to capitalize on these assets. As part
of the strategic review process, Labopharm is also exploring potential business combinations that could create
value for shareholders.” Update: Labopharm acquired by Paladin Labs for $20mm on August 17, 2011.
$ Lipose - Viafill fat transfer system on the market with applications in aesthetics where traditional volumizers are
not well suited - particularly for the face and breasts. Company assisted by Torreya Partners. For details contact
[email protected].
UPDATE $ Medicure - May sell U.S. rights to Aggrastat, originally a Merck drug, for acute cardiac indications. Update:
company restructured its liabilities on July 18, 2011 in order to reduce debt. Beal Advisors will be working with the
company to find a partner or buyer for Aggrastat going forward. Revenues are currently at a run rate of $3mm a
year. (Link)
Meda - A Wall Street Journal Report on July 27, 2011 indicated that Valeant had approached Meda about a
takeover offer. Meda responded indicating that its board of directors had not received an approach of the kind
reported in the WSJ.
Merlion - Developing finafloxacin, a best in class 4th generation fluoroquinolone being targeted against severe,
life-threatening infections. Compound has successfully progressed through to mid-stage clinical development. Oral
formulation demonstrated in PoC studies compelling efficacy in uncomplicated UTI and the eradication of
Helicobacter pylori. IV formulation currently completing Phase I trials and Phase 2 studies in complicated UTI are
planned, as are clinical protocols in complicated respiratory tract infections. MerLion are exploring strategic
alternatives for this product with the assistance of Torreya Partners. For further details, contact
[email protected]. (Link)
Mimetica - Has developed MTC896 as a topical gel for the treatment of excessive sebum production in subjects
with acne and other skin conditions. MTC896 is a highly selective and potent antagonist ( $10mm. Hospital setting is where generally used.
University of Western Ontario - Novel antifungal agent with strong pre-clinical efficacy versus candidas. (link)
NEW Ventria - has developed a recombinant lactoferrin for the prophylaxis and treatment of infection in
prematurely born children. Positive POC dataset for this product.
Zurex - Zuragen used for prevention of catheterrelated bloodstream infections. Potentially of high value in the
hospital setting. (Link)
BANKRUPTCY AND RESTRUCTURING SITUATIONS
NEW AlteaTherapeutics - has agreed to shutter its operations according to a story published in the Atlanta Business
Chronicle on Dec 9, 2011 (Link). Atlanta-based Altea Therapeutics Corp. was developing a proprietary, noninvasive
method to deliver therapeutic proteins, conventional drugs and vaccines through the skin by creating
“micropores” on the skin’s surface.
Aryx Therapeutics - ATI-2042, an improved amiodarone like molecule, for reduction of atrial fibrillation. Also has
tecarfarin - an anticoagulant. GI candidate with promise. Update: Feb 19, 2010: Company retained Cowen to
explore strategic options. Dec 15, 2010: “Over the course of the strategic process ARYx initiated earlier this year,
the most significant interest, even in the absence of a binding offer, was shown in ARYx's gastrointestinal product
candidate, naronapride (ATI-7505), demonstrating the potential value of the asset. Also, interactions with the US
Food and Drug Administration ("FDA") in April 2010 substantially clarified the remaining clinical development
requirements for the compound. As a result, the lead investors in ARYx encouraged the company to seek
substantial additional funding to continue the development of naronapride internally, and such funding has been
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actively pursued since late summer without final resolution.” Company close to default on debt. Update: March
2011 - company went into a wind down of its operations. (Link)
Ceragenix - in Chapter 11 bankruptcy with a skin cream and assets in the anti-infective area. (Link)
NEW Commonwealth Biotechnologies – Richmond VA company has filed for bankruptcy court protection.
COMPLETED Graceway - Could consider sale of products from 3M and has hired Lazard to explore alternatives
through an ongoing process. Aldara for acitinic keratosis a key seller but was recently genericized. Update: Nov 23,
2011 - Medicis acquired Graceway for $455mm after the company filed for Chapter 11 bankruptcy on Sep 29,
2011.
Orbus Pharma - Canadian generic drug maker is in bankruptcy. Has a variety of generic CNS drugs on the market.
(Link)
NEW Peptimmune – has been in bankruptcy. MS assets recently sold to third party.
NEW Undisclosed player - dermatology company with more than $40mm in revenue has hired a financial advisor to
restructure its debt that likely exceeds intrinsic value of assets. Process has been underway for a month or two.
Vion Pharmaceuticals - Received an approvable letter for Onrigin, a late stage alkylating agent. Has > $15mm in
cash but $60mm in debt. Filed for Chapter 11 bankruptcy and is being advised by Roth Capital. FDA has made it
clear that a randomized Phase 3 trial will be required.
NEW Vyteris - has closed operations. Several programs are in development at this company including patch
products for women’s health and pain applications.
BIG PHARMA / BIG BIOTECH PROGRAMS FOR OUTLICENSING
Biogen Idec - Looking to outlicense Galiximab, an anti-CD80 antibody, which has shown activity in B-cell
lymphomas. Has gone through Phase 3 trials. Biogen looking to outlicense after a recent strategic review. (Link)
Biogen Idec - looking to outlicense BIIB021, an HSP90 modulator, targeted for GIST. (Link) (Link2). Also outlicensing
BIIB028, an HSP90 modulator that is in Phase 1 studies.
Biogen Idec - looking to outlicense Volociximab, a chimeric monoclonal antibody that inhibits the functional
activity of a5ß1 integrin, a protein found on activated endothelial cells. Blocking the activity of a5ß1 integrin has
been found to prevent angiogenesis. This product is jointly owned with Abbott’s Facet Biotechnology. (Link)
Biogen Idec - looking to outlicense BIIB015 which consists of Biogen Idec's Cripto-binding antibody and
Immunogen’s DM4 cell-killing agent. BIIB015 advanced into Phase I testing in the summer of 2008. This compound
was slated for Phase 2 in 2011 until Biogen Idec decided to exit oncology after a recent strategic review.
Biogen Idec - looking to outlicsense an anti IGF1-R antibody for solid tumors. Has progressed into Phase 2 studies.
(Link)
GlaxoSmithKline - running a process to divest non-core OTC brands with assistance from Goldman Sachs. The
products to be divested, which are primarily sold in Europe and the United States, had sales in 2010 of
approximately £500 million, 10% of GSK’s total Consumer Healthcare turnover. They include analgesics:
Solpadeine, BC and Goody’s; vitamin and supplement product Abtei; feminine hygiene treatment Lactacyd; and alli
for weight management. (Link)
Ipsen - Completed a strategic review in 2011 with the theme of increasing focus and growing the footprint. The
implications of this for potential partnerships or asset divestitures are threefold: (1) Ipsen looking for a partner in
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the French primary care arena, (2) Ipsen looking to find a buyer for its industrial site in Dreux France which makes
solid dose and liquid formulations and (3) “Ipsen will explore all options to maximize value (of its short stature
franchise) while meeting its obligations to patients and partners. It will be managed directly by regions and
countries.”
UPDATE Pfizer - has announced that it is selling vet medicines and its nutritional business. Both businesses are
leaders in their class. Update: process to sell infant nutrition business is rumored to be underway in November
2011. It is believed that the vet medicines business is most likely to be spun out.
Sanofi / Genzyme - Outlicensing RDP58 (Delmitide), a clinical-stage D-amino acid decapeptide with established
anti-inflammatory properties. Proof of concept for this product opportunity has been demonstrated in models of
inflammatory bowel disease (IBD).
Sanofi / Genzyme - Outlicensing tolevamer for CDAD which is in Phase 3 studies but missed the primary endpoint.
(Link)
Sanofi / Genzyme - Looking to outlicense Genz-29155, a novel, small molecule, orally bioavailable, 1x daily novel
inhibitor of TNF-? signaling. Proof of concept has been demonstrated in multiple models of transplantation
rejection, multiple sclerosis (MS), sepsis, inflammatory bowel disease (IBD) and lupus. (Link)
NEW $ Undisclosed - large pharma disposing of a marketed antibiotic with global rights. This product is off patent.
NEW $ Undisclosed - large pharma disposing of a marketed oncology drug for NHL with revenues of around $5mm.
Significant barriers to entry.
NEW $ Undisclosed player - approved hospital anesthetic in the United States with differentiation from existing
products. Also could be used in physician office setting where sedation required. Open to a product sale or other
value creating arrangement. Global rights available.
NEW $ Undisclosed - division of larger pharmaceutical company with approximately $30mm revenues in oncology
product sales. Active sale process underway.
Undisclosed - Large Pharma disposing of several late stage compounds for treatment of depression.
Undisclosed - Large Pharma disposing of several late stage compounds for treatment of hyperlipidemia.
Undisclosed - Pharma disposing of more than 20 compounds including several with commercial rights in EU and
Asia.
NEW Undisclosed - Pharma disposing of large mature product. Off patent with revenues over $80mm.
Undisclosed - pharma company is open to divesting a marketed specialty cardiology product with revenues >
$30mm per annum.
Undisclosed - Large pharmaceutical company interesting in outlicensing a late stage program in wound care.
Undisclosed player - Partnering a Phase 2 progesterone modulator for uterine fibroids and endometriosis.
BIGGER DEALS ($200MM+ IN VALUE)
NEW Abdi Ibrahim Ilac - largest Turkey drug maker with revenues over $800mm. Reported in May 2011 that was in
discussions to sell a strategic stake.
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UPDATE Actelion - has received takeover approaches from several strategic bidders according to the Wall Street
Journal (10/7/2010). Activist shareholder in Elliott pushed for a change of control transaction. Wall Street Journal
Sep 28, 2011: “Elliott Advisors Cuts Actelion Stake in Wake of Control Battle”. Elliott failed to put its own directors
on Board and JP Garnier, formerly CEO of GSK, recently became Chairman. A takeover of Actelion appears to be
quite unlikely in light of these events.
UPDATE Allos Therapeutics - Oncology marketer with Folotyn® for liquid tumors. After a recently failed merger
attempt with AMAG, Allos is rumored to be continuing to explore strategic alternatives with the assistance of JP
Morgan. (Link)
UPDATE $ AMAG - Feraheme IV iron product - Recently approved. Company is commercializing on its own. AMAG’s
recent merger attempt with Allos was ended in November 2011. On Nov 17, 2011, AMAG announced that it had
hired Jefferies to explore all opportunities to enhance shareholder value. Frank Thomas, interim CEO of AMAG
indicated: “We will expeditiously complete this process, which will include a parallel review of a potential sale of
the company and other strategic merger and acquisition transactions.”
UPDATE Amarin - Developing a pure omega-3 for reduction of triglycerides. Phase 3 data reported out very strong.
Company has indicated that is has retained a financial advisor (Lazard) to explore a sale. Update: Nov 2011 - no
sale reported and company’s valuation has dropped from over $2bn to under $1bn. Amarin recently filed an NDA
for its lead product. Amarin appears to be a likely takeover candidate for the next year. (Link)
Amoun Pharmaceutical - An Egyptian company that manufactures off-patent branded generic formulations. It is
one of the largest pharmaceutical companies in Egypt. It sells over 135 human products in over 275 forms. Of
these products, 33 occupy the top 2 positions in their respective therapeutic categories and subcategories. Open
to a company sale or strategic stake purchase. Reuters - Dec 6, 2010: “CVCI is also preparing to sell Amoun, one of
Egypt's biggest drugmakers, people familiar with the matter told Reuters on Oct. 20. It owns Amoun with two
other co-investors.” Bloomberg reported in Feb 2011 that the company has been looking for $1 billion in a sale
price but that political upheaval in Egypt has hindered the sale.
NEW Amylin - has purchased rights to Byetta® back from Eli Lilly. This GLP-1 inhibitor is expected to have $1 billion
or more in revenues and has substantial ex-U.S. revenue. Amylin is searching for a commercialization partner
outside of the United States.
COMPLETED Anadys - ANA598 is a low-nanomolar inhibitor of HCV genotype 1a and 1b replicons via N5b polymerase.
Has started a Phase 2 trial. Anadys also has ANA773, an oral inducer of endogenous interferons with nice Phase 1
data. May 2010 - Lazard retained to act as advisor to Anadys to explore strategic alternatives. Nov 2010 press
release: Anadys continues to work with Lazard Frères & Co. LLC. to explore potential strategic transactions, in
parallel with moving the ANA598 and ANA773 programs forward. Update: Oct 17, 2011 - Roche acquires Anadys
for $230mm - a 256% premium.
UPDATE $ Avanir - Launched Neudexta for the treatment of pseudobulbar effect and, potentially, other indications.
Positive data and good patent picture. Widely rumored to be an M&A candidate. $5.7 million in revenue in first
year of launch (soft numbers). Market cap of $248 million as of November 2011. EMA application recently
accepted.
COMPLETED $ Azur Pharma has hired Lazard to advise on funding options including a potential stake sale according
to the Sunday Times on May 22, 2011. Company has a solid franchise as a marketer of drugs in the CNS and
women’s health areas. A company sale could be possible. Update: Sep 19, 2011 - Jazz Pharma merges with Azur
Pharma as an Ireland domiciled company with former Jazz shareholders taking 80$ of the combined company. A
transaction closing is expected in Q1 2012.
UPDATE Biofarma - Biofarma for sale via JP Morgan. Reuters (12/6/2010): Citigroup's (C.N) venture capital arm and
two co-investors have begun an auction of Turkish copycat drugmaker Biofarma, three people familiar with the
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matter said, in what could be Turkey's biggest healthcare deal.” Update: A number of parties rumored to have
looked at this asset but price ask was seen as prohibitive. As of Nov 2011 no sale had taken place.
Chelsea Therapeutics - NORTHERA™ (droxidopa), is an orally active synthetic precursor of norepinephrine initially
being developed for the treatment of neurogenic orthostatic hypotension. Large potential in the Parkinson’s
market where there has been a reported 60% reductions in falls in PD patients with NOH - also could work in
fibromyalgia. On market already through Dainippon Pharma in four Asian countries. Company recently reported
that it is filing for an NDA based on studies that have already been completed and has recently completed its QTc
work. Anticipates approval by Q2 2012.
UPDATE Covidien Pharmaceuticals - According to the New York Times on June 7, 2011 “Covidien, the health care
company spun out from Tyco four years ago, may seek to sell its pharmaceutical unit…” This division of Covidien
(formerly Mallinckrodt) has a major business selling pain products (both branded and generics) and imaging
products. Revenues are around $2 billion. Update: As of December 2011 no sale has taken place. Company is
rumored to be interested in a sale of the whole business (rather than pieces) for a full price. YE 2011 numbers
reported on Nov 15, 2011 and were robust (sales up 9% yoy) with strong performance in generics.
Dyax - Has DX-88, for hereditary angioedema. Recently approved. No known sale process underway but company
appears to be an attractive takeover target.
UPDATE Exelixis - According to Bloomberg on April 12, 2011: “Exelixis Inc. is working with Goldman Sachs Group Inc.
to prepare for potential takeover offers after its experimental drug helped prostate-cancer patients in a study.”
Company’s XL-184 has reported dramatic data on reducing metastatic prostate lesions at ASCO. Update: company
has hired a Chief Commercialization Officer with intention to introduce cabozantinib to the U.S. market and has
reported strong data for cabozanitinib in the treatment of medullary thyroid cancer.
Gen-Probe - Widely rumored to be for sale with interest from Novartis. Well known diagnostics company. Process
well underway but company viewed as expensive.
GlaxoSmithKline - running a process to divest non-core OTC brands with assistance from Goldman Sachs. The
products to be divested, which are primarily sold in Europe and the United States, had sales in 2010 of
approximately £500 million, 10% of GSK’s total Consumer Healthcare turnover. They include analgesics:
Solpadeine, BC and Goody’s; vitamin and supplement product Abtei; feminine hygiene treatment Lactacyd; and alli
for weight management. (Link)
COMPLETED Graceway - Could consider sale of products from 3M and has hired Lazard to explore alternatives
through an ongoing process. Aldara for acitinic keratosis a key seller but was recently genericized. Update: Nov 23,
2011 - Medicis acquired Graceway for $455mm after the company filed for Chapter 11 bankruptcy on Sep 29,
2011.
UPDATE Hi-Tech Pharmacal - Market rumors in April 2011 that company could be purchased. As of December 2011
2011 no transaction had taken place of this manufacturing company of generic liquids and ointments. The
company has delivered strong earnings throughout 2011 and is trading at close to twice the price in Dec 2011 it
had earlier in 2011.
COMPLETED Inova Pharmaceuticals - Asian OTC and Pharma product company with revenues > $500mm. Ran a sale
process via Greenhill Caliburn. No transaction has taken place as of June 2011. Nov 21, 2011: Valeant buys iNova
for $625 million.
Intermune - Bloomberg reports on April 27: “Biotechnology company InterMune Inc known for its drug to treat
lung scarring, hired Goldman Sachs (GS.N) to help it weigh a possible sale... Goldman has been conducting an
auction of InterMune for more than a month and some potential bidders have been spooked by the biotechnology
company's expectations for a sale price, Bloomberg news reported.” Company commercializing Pirfenidone in
Europe. Company market cap over $2bn.
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Ipsen - has completed a strategic review in 2011 with the theme of increasing focus and growing the footprint. The
implications of this for potential partnerships or asset divestitures are threefold: (1) Ipsen looking for a partner in
the French primary care arena, (2) Ipsen looking to find a buyer for its industrial site in Dreux France which makes
solid dose and liquid formulations and (3) “Ipsen will explore all options to maximize value (of its short stature
franchise) while meeting its obligations to patients and partners. It will be managed directly by regions and
countries.”
Mannkind - Looking to partner Afresa, an inhalable insulin, that has an upcoming PDUFA date. Rumored to be in an
active M&A mode. Company received an approvable letter on Mar 15, 2010. The FDA asked for more information
on data designed to support the clinical utility of Afrezza, as well as information about how comparable the
commercial version of the product is to the version used in clinical trials.
Meda - A Wall Street Journal Report on July 27, 2011 indicated that Valeant had approached Meda about a
takeover offer. Meda responded indicating that its board of directors had not received an approach of the kind
reported in the WSJ.
Morton Grove Pharmaceuticals - Owned by Wockhardt and involved in the development, manufacture, and
marketing of prescription oral liquid and topical liquid pharmaceuticals and is one of the leading manufacturers
and marketers of prescription oral liquid pharmaceuticals in the United States. No announcement of an sale but
Wockhardt would likely consider a good price. Update: April 2010 - Wockhardt's USD 110m foreign currency
convertible bonds are at the core of a dispute between Wockhardt and its foreign lenders which has stopped a
recent sale from Wockhardt to Abbott on nutritionals.
NEW Onyx - rumored to be exploring strategic alternatives. Substantial value potential tied to a recently filed NDA
for carfilzomib, a protease inhibitor, for the treatment of liquid tumors including multiple myeloma. Bloomberg
reported on Nov 30, 2011 that Onyx is working with Centerview as a financial advisor. On Dec 12, FDA indicated
that an accelated review would not take place.
NEW Par Pharmaceuticals - Relational Investors filed a 13D showing 8.7% ownership of this company on November
25, 2011. In the 13D Relational indicated: “Despite these opportunities for improvement, the Reporting Persons
believe that the Company may continue to trade at discounted prices because of industry challenges and the
Company’s sub-optimal size and product scope. If the discount persists, the Reporting Persons believe that, in
keeping with sound stewardship principles, the Company’s board will be required to consider broad strategic
alternatives. Specifically, the Reporting Persons are confident that substantial cost savings could be achieved in a
transaction with a strategic buyer.” Note: there is no evidence that Par Pharmaceuticals has received offers or is
open to receiving such offers at present.
UPDATE Pfizer - has announced that it is selling vet medicines and its nutritional business. Both businesses are
leaders in their class. Update: process to sell infant nutrition business is rumored to be underway in November
2011. It is believed that the vet medicines business is most likely to be spun out.
Qualicaps - owned by Carlyle. Maker of gelcaps (like Capsugel) is up for auction. UBS is rumored to be sellside
advisor on business with approx. $350mm in EBITDA. As of June 2011 no sale had taken place.
NEW Rottapharm - for sale according to the Wall Street Journal. Company has two Phase 3 drugs in development
and a strong group of branded products in the market. Revenues over $850 million. Sale price could be over $2.5
billion. Company rumored to be using Credit Suisse to find a buyer.
NEW San Raffaele del Monte Tabor- privately-held Italian pharmaceutical company, is soliciting offers other than
the EUR 350m binding offer from Vatican bank IOR and Italian entrepreneur Vittoria Malacalza, according to Il Sole
24 on Dec 2, 2011.
UPDATE Savient Pharmaceuticals - FDA approved KRYSTEXXA (pegloticase) in Sep 2010, a PEGylated uric acid
specific enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy.
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Company is seeking a commercial buyer and is using JP Morgan and Lazard in its search for an acquisition partner.
Savient is now pursuing a launch of Krystexxa on its own and is building a 50-person plus sales force. Update: First
year sales were $2.6 million. Market Cap of $160mm. (Link)
UPDATE Sigma-Tau - Reuters (6/2/11): “Italy's Sigma-Tau is eyeing the sale of up to 49 percent in the family-owned
drugmaker to private equity, ahead of a possible IPO that could value it at more than $2 billion, people familiar
with the situation said.” Company has approximately €1bn revenue with a strong rare disease business and a well
established European brand business. It is believed that discussions regarding a stake sale are no longer active but
rather the company is focused on restructuring its Italian business.
Stada - Chief executive Hartmut Renttzlaff indicated an openness to takeover offers after the recent sale of
Ratiopharm.
UPDATE Thrombogenics - completed Phase 3 studies for Microplasmin in Phase 3 clinical development for the non-
surgical treatment of back of the eye diseases. Good evidence of efficacy with two positive Phase 3 trials reported.
Expected to be on market by end of 2012. Would consider a sale. (Link)
NEW Undisclosed Player - U.S. generic company with approximately $90mm in gross revenue is searching for a
buyer with the assistance of a financial advisor. Company has substantial presence in medicines for cough & cold,
womens health and pediatrics.
NEW Undisclosed - German vertically integrated marketer of generic and branded pharma products with strength in
cardiovascular, dental and veterinary medicines. Revenues of this company exceed €100mm.
NEW Undisclosed Player - U.S. generic company with more than $100 million in revenues is for sale. Company has a
significant branded business and a manufacturing facility.
NEW Undisclosed player - Division of Indian generic company that is focused on oral solid dose preparations is for
sale. Revenues over $300mm. Company has strength in formulation work and manufacturing. Ships product to
numerous global locations.
COMPLETED Undisclosed player - U.S. generic player with approximately $200mm in net revenues and strength in
drug delivery solutions would consider a company sale. Update: Par acquires Anchen Pharmaceuticals for $410mm
in cash on Aug 24, 2011.
COMPLETED Undisclosed player - Large pan-Asian drug company open to a sale. Update: Menarini acquires Invida
Group on Nov 16, 2011. Price not announced but Invida a substantial business with more than $200mm in
revenue.
UPDATE Vivus - Looking to partner Qnexa in Europe and other ROW territories. An approval is possible in the EMA
with long-term market exclusivity in mid-2012 (or sooner).
NEW Wockhardt - rumored to be in a process to sell its substantial nutrition business as part of a process to pay
down external debt.
BIODEFENSE
Cleveland Biolabs - Looking to partner a radiation antidote (CBLB502) with positive animal data.
Elusys - Has positive animal data for Anthim, a high-affinity humanized monoclonal antibody targeting the anthrax
toxin protective antigen. Recently received a U.S. government contract for up to $143 million. In active strategic
discussions. (Link)
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PharmAthene - Protexia - BChE functions as a natural bioscavenger, like a sponge, to absorb and degrade
organophosphate poisons (e.g. nerve agents) before they cause neurological damage. Phase 1 studies were
presented and good news so far. PharmAthene is developing Protexia in collaboration with the U.S. Department of
Defense as a broad spectrum prophylaxis for the U.S. military. Company recently indicated that it is open to
individual product deals despite not having a company level sale process. (Link)
Soligenix - The company is looking to divest biodefense assets including vaccines for ricin toxin botulinum toxin and
a product which protects against radiation exposure. Process via HealthPro BioVentures.
NEW Xoma - XOMA 3AB,is a biodefense anti-botulism antibody candidate in Phase 1 studies.
BIOSIMILARS
NEW Green Cross - In Phase 3 with a pegylated GCSF.
NEW Hanmi Pharma - developing long-acting versions of EPO, GSCF, HGH and an exendin analogue.
NEW ISU Abxis - Biosimilar to ReoPro for adjuvant of PCI (Percutaneous Coronary Intervention) procedure. Clotinabl
is anti-GPIIb/IIIa monoclonal antibody and Fab Protein which blocks platelet aggregation. On market in 7 countries.
Also has biosimilar for Cerezyme.
Undisclosed generic player - Looking for a partner for a portfolio generic biologics in development. Wants to retain
manufacturing rights.
Undisclosed player - a number of emerging markets players are developing portfolios of generic injectable drugs
including cytotoxics and anti-infectives.
NEW Undisclosed player - looking to partner a recombinant formulation of human serum albumin. Broad
applications and advantages over blood derived product. For further details contact
[email protected].
NEW USV - offering a biogeneric PTH. Teriparatide is a recombinant form of N-terminal 1-34 amino acids of human
parathyroid hormone which is a 84 AA protein. Approval pending in India. Also partnering a growth hormone and a
PDGF product.
Zelos - A better PTH analogue. Strong Phase 2 data. Company also pursuing an intranasal PTH and a potential
“biogeneric” PTH. (Link)
BONE AND SPINE / ORTHOBIOLOGICS
UPDATE Abiogen - Neridronate is an amino-bisphosphonate used in Metabolic Osteopathy and has gone into Phase
3 trials. Also being studies for patients with thalassemias. (Link)
Ablynx - Has positive Phase 1b safety and efficacy data for a “nanobody” targeting RANK-L (compare to Amgen’s
Prolia®). Dataset presented at EULAR in May 2011. (Link)
Accelalox - Use of locally delivered proteins to promote fracture healing. Promising osteobiologics company. (Link)
Anika - Introducing MONOVISC™ which is a single injection supplement to the synovial fluid of the osteoarthritic
joint in order to provide symptomatic relief of joint pain. Contains sodium hyaluronate. Company also focusing on
obtaining FDA clearance for three orthopedic products from FAB which are Hyaloglide, Hyalonect and Hyalofast by
the end of Q1 2011. Anika intends to launch its own sales force for the orthopedic market for all of the above
products. (Link)
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Bio3 Research - HMGB1 for cartilage repair with promising preclinical results. (Link)
Biomimetic - Rumored acquisition target with injectible bone graft line based upon rhPDGF. PMA application
pending and apparently in good shape. Looking for an ex-U.S. partnership. (Link) Ships augument bone graft to
Canada. (Link)
BioTime - Using HyStem® hydrogels in the development of therapeutic products for the treatment of
osteoarthritis, and plans to develop HyStem®-Rx as a cell delivery medical device to improve outcomes in cell
transplant procedures, including reconstructive and cosmetic surgery. (Link)
NEW Chiesi - CHF4227 - SERM for treatment of osteoporosis. Ready for Phase 2 studies.
Effrx - Effervescent alendronate. Solves problem of having to sit up for 30 minutes after taking Fosamax. Ex-US
partnership via Nycomed. Filed for market approval in Europe.
Enobia - Developing ENB-0040, a human recombinant tissue non-specific alkaline phosphatase, for the treatment
of hyperphosphatasia - a debilitating bone condition. Positive data in man. Update: Company has continued to
report positive data and may be looking at a rapid approval with FDA. As a result, has not proceeded with potential
M&A deal in light of value inflection ahead.
Ferring - Outlicensing a PTH analogue for osteoporosis. Pre-clinical. (Link)
NEW Kaken - KCB-1B is a Trafermin (rh-bFGF) product and under development for Bone Fracture in Japan.
Kuros Biosurgery - KUR-113 has shown strong efficacy in the treatment of open fractures in tibia patients. Study
was a Phase 2b trial with 200 patients that reported out in April 2011.
H Lexicon Pharmaceuticals - Pursuing LX1031 for IBS. Could a potent agent for treating osteoporosis. Lexicon
completed a Phase 2 clinical trial of LX1031 in patients with non-constipating IBS in November 2009. Company in
active discussions on this and two other molecules.
Medi-Post - CARTISTEM® has been developed to treat damaged articular cartilage on knee as a result of acute
traumatic injury or more chronic conditions such as osteoarthritis. Phase 3 clinical trial has shown efficacy in
cartilage regeneration. KFDA approval pending. U.S. IND filing in process. (Link)
Mesoblast - Preclinical trials showed that a single, low dose of Mesoblast’s allogeneic adult stem cells into severely
damaged intervertebral discs resulted in dramatic reversal of the degenerative process, regrowth of disc cartilage
and sustained normalization of disc pathology, anatomy and function. (Link)
NEW Ono Pharmaceutical - Has completed Phase 2 studies with ONO-5334, a cathepsin K inhibitor for the
treatment of osteoporosis. Open to an ex-Japan partnership transaction.
Sanos Bioscience - Promising Phase 2a data for a GLP-2 for the treatment of osteoporosis.
Tarsa Therapeutics announced that the patient enrollment has been completed in TAR01-201, a double-blind
Phase 2 study comparing Tarsa's oral recombinant salmon calcitonin (from Unigene) to placebo in approximately
120 postmenopausal women who have low bone mass (osteopenia) and are at increased risk of fracture. (Link)
$ Tigenix - Launching ChondroMimetic and ChondroCelect in the EU, a collagen based implant for the treatment of
small osteochondral lesions. In studies have seen restoration of the articular surface and integration with the
surrounding bone and cartilage at six months in patients treated with ChondroMimetic. U.S. approval by BLA will
require an additional study. (Link)
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Tokai Pharmaceuticals - Semparatide, a PTH, has been in Phase 2. Being developed for increasing osteoblasts in
stem cell transplantation.
University of South Carolina - Preclinical binder of PTH1r - promising alternative to use of parathyroid hormone in
treatment of bone loss. Supportive pre-clinical data. (Link)
University of Western Ontario - Preclinical peptides which bind collagen and promote bone repair and
regeneration in preclinical models. (Link1)
NEW USV - offering a biogeneric PTH. Teriparatide is a recombinant form of N-terminal 1-34 amino acids of human
parathyroid hormone which is a 84 AA protein. Approval pending in India.
Versalion Pharma - An osteotropic alendronate-b-cyclodextrin conjugate for treatment skeletal diseases,
particularly stress fractures. The conjugate shows very strong binding to hydroxyapatite (HA, main component of
the skeleton). Its ability in forming molecular inclusion complex with prostaglandin E1 (PGE1), a potent bone
anabolic agent) was confirmed by phase solubility experiments and differential scanning calorimetry (DSC). (Link)
Zelos - A better PTH analogue. Strong Phase 2 data. Company also pursuing an intranasal PTH and a potential
“biogeneric” PTH. (Link)
CARDIOLOGY - PRIMARY CARE
H Aegerion - MTP-1 Adjunct therapy to statins for persons with familial hypercholesterolemia. Similar products in
development at Japan Tobacco. Recently reported very strong Phase 3 data. High interest. Recently completed an
IPO. (Link)
H UPDATE Amarin - Developing a pure omega-3 for reduction of triglycerides. Phase 3 data reported out very
strong. Company has indicated that is has retained a financial advisor (Lazard) to explore a sale. Update: Nov 2011
- no sale reported and company’s valuation has dropped from over $2bn to under $1bn. Amarin recently filed an
NDA for its lead product. Amarin appears to be a likely takeover candidate for the next year. (Link)
Amulet Pharmaceuticals - Technology to deliver nitric oxide in conjunction with pharma treatments. Exploring a
sale transaction.
H Anthera Pharmaceuticals— Varespladib is an inhibitor of secretory phospholipase in Phase 2 for prevention of
arteriosclerosis. Promising data and other molecules. In an ongoing Phase 3 program. (Link)
H Ardelyx - developing non-absorbed compounds for the control of salt buildup and excess fluid retention. Related
compounds in development at Sorbent Therapeutics. The potential market for this class of drugs in persons
suffering from salt-related hypertension is very large.
BTG - Angiotensin Therapeutic Vaccine (ATV) for high blood pressure. This therapeutic vaccine (biologic) is in early
Phase 2 clinical development. Large potential market as alternative approach to blood pressure management.
(Link)
Carolus Therapeutics - CT-2008 is a peptide antagonist of the RANTES-PF4 heterodimer which enhances leukocyte
recruitment causing inflammation. Preclinical studies have demonstrated that therapeutics targeting the RANTES-
PF4 heterodimer can slow the development of plaque in atherosclerosis-susceptible murine models. Also see
Novimmune.
Cardax - Heptax, a preclinical compound, reduces cholesterol, triglycerides, blood pressure, liver enzymes, and
fasting blood sugar while raising HDL. (Link) Update: Have been awarded two patents, one including (CDX-085).
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Cerenis Therapeutics - Looking to partner its synthetic apoA-1HDL product and a PPAR-delta (with Phase 1 data).
Recently completed a financing to support further studies with the lead program.
Cerenis Therapeutics - Would consider partnering a non-flushing niacin that has been through Phase 2 studies.
Company recently reported positive data from these studies. (Link)
Essentialis - Diazoxide choline controlled release tablets for reduction of triglycerides and overall lipid profile.
Strong Phase 2 data in reducing triglycerides. Now going into Phase 3 with an SPA for a single study to get to
approval.
UPDATE Furiex Pharmaceuticals - developing a muscle sparing statin, PPD 10558. In a preclinical study comparing
PPD 10558 and a commonly prescribed statin, a high dose of the marketed statin caused severe muscle necrosis
and increases in liver and kidney function tests and muscle enzyme (CK) levels while PPD 10558 did not
demonstrate any such abnormalities. Update: Furiex discontinued development of this product on Dec 8, 2011
after this drug missed its primary endpoint. (Link)
Intekrin - completed Phase 2 trials with INT131 a PPAR gamma, the PPAR subclass predicted to be less likely to
cause the side effects of TZDs. Looking to partner or M&A.
NEW Japan Tobacco - Looking to outlicense JTT-130, an MTP inhibitor for cholesterol management, particularly
hypertriglyceridemia. This compound has completed Phase 2 studies and has shown rapid clearance and activity in
small intestine. These properties are likely to limit liver toxicity concerns associated with this drug class.
UPDATE KaroBio - Eprotirome is a novel, selective, thyroid hormone receptor agonist, developed for treatment of
dyslipidemia. Successfully showed reduction in LDL in a recent Phase 2 study. Karo is in a Phase 3 EU trial to
demonstrate a reduction on top of statins in famililial hypercholesterolemia. (Link).
Nicox - developing a promising nitric oxide donating statin. High potential to supplement statin therapy.
H Omthera - pursuing Epanova in a Phase 3 study. Epanova is a patent protected, novel, ultra-pure mixture of the
free fatty acid forms of eicosapentaenoic acid (EPA) and docosahexaenoic acid (DHA). Behind Amarin in
development but company claims to have a superior efficacy profile. (Link)
Quatrx - Several interesting drugs in dyslipidemia using TSH (thyromimetic) approach. Also see Karo.
H Relypsa - positive Phase 2 data achieved for a potassium binder to treat hyperkalemia which is prevalent in
persons with end stage renal disease and those with congestive heart failure. Company recently raised $70mm to
get through Phase 3 studies of this promising treatment. (Link)
Thorne Research - HEP-40 Cholesterol binder that reduced LDL cholesterol by 17 points over a statin, on average,
in a Phase 2 study. (Link)
Undisclosed player - has a promising non-flushing niacin.
$Undisclosed player - company with a marketed but not promoted cardiovascular product with 2011 revenues
around $9 million.
UCLA - Selective inhibitor of M-CSF to reduce risk of atherosclerosis (link)
NEW Undisclosed player - Phase 2 data on drug for raising HDL and lowering triglycerides. Novel mechanism and
impressive safety profile. Seeking ex-U.S. partnership.
Veloxis - AtorPhen. Phase 2 low dose fenofibrate with atorvastatin. Also looking to partner rights to Fenoglide
outside of the U.S.
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UPDATE Vitae Pharmaceuticals - Working on renin inhibitors for hypertension. Preparing to go into Phase 2b studies
in 2012. (Link)
CARDIOLOGY - SPECIALTY CARE
Ablynx - ALX-0081 is a Nanobody targeting von Willebrand Factor (vWF), to reduce the risk of thrombosis in
patients with acute coronary syndrome (ACS) and thrombotic thrombocytopenic purpura (TTP). Through Phase 1b.
Phase 2 data are expected.
Action Pharma - AP214 finished Phase 2, a modified dMSH-peptide analogue, for the treatment of post-surgical
kidney injury in the cardiac surgery context. The results demonstrate that AP214 is well tolerated and safe at all
three dose levels. At the highest dose level, AP214 prevents the increase in serum creatinine by 50-60%, and in the
IL-6 response by 30-40%, compared to placebo (trends based on blinded data). This is consistent with a robust
effect to prevent postsurgical acute kidney injury (AKI) and systemic inflammatory response.
Acusphere - Imagify, pending EMA filing for this cardiac imaging agent. Highly differentiated from SPECT agents on
the market insofar as permits evaluating of myocardial perfusion, an important marker of coronary artery disease
(CAD) without radioactive markers. Potential first-to-market drug in $600 million and $2 billion addressable market
in E.U. and U.S. respectively. Recent SPA from FDA in US. Will require one further trial for FDA approval. Company
is assisted by Torreya Partners. For details contact [email protected] (Link)
NEW Adventrx- ANX-188 is a novel, purified, rheologic and antithrombotic compound initially being developed as a
first-in-class treatment for pediatric patients with sickle cell disease in acute crisis. Ready for Phase 3 studies and
open to partnering deals.
Aldagen - ALD-401 is a type of adult stem cells called aldehyde dehydrogenase-bright (ALDHbr) stem cells isolated
from cord blood. ALD-401 is being developed for the post acute treatment of ischemic stroke and is in a Phase 2
study.
H AnGes MG - Japanese company is looking to partner Collategene, a therapeutic using hepatocyte growth factor
(an angiogenic factor) which creates new blood vessels for the treatment of ischemic diseases. Very positive Phase
3 data in treating PAD. An SPA has been agreed with the FDA for a Phase 3 trial of Peripheral Arterial Disease.
UPDATE ARCA - Gencaro (Bucindolol) is a nonselective beta-blocker which is being developed for treatment of heart
failure on a genetically-targeted basis. Very strong data in preventing death post-MI and currently purusing a trial
for this drug in AF. Approvable letter from FDA with guidance on a further trial required for approval. Open to a
partnership deal on Gencaro. (Link)
ARMGO Pharma - In Phase 2a with a product that stabilize ryanodine receptor/calcium release channel. ARMCO
hypothesizes that it may be possible to restore the strength of muscle contraction. (Link)
Biolex - BLX-155 is a direct acting thrombolytic (clot dissolver) which was superior to t-PA in a recent preclinical
study reported at ISTH. (Link)
NEW $ Canyon Pharmaceuticals - Canyon Pharmaceuticals is seeking to build a strategic alliance preferentially on a
worldwide basis to commercialize Desirudin (Iprivask® US-registration / Revasc® EU-registration), a first/best in
class anticoagulant drug which is approved by the FDA, the EMA and several of the rest of world authorities.
Desirudin is a direct thrombin inhibitor and the only subcutaneous direct thrombin inhibitor (DTI) with approval for
venous thromboembolism (VTE) prophylaxis following hip- and knee-replacement surgery.
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Cardiokine - has recently received rights to Lixivaptan for the treatment of hyponatremia. Has finished three Phase
3 studies but has not published the results to date. Two vaptans on the market but potential differentiation of this
product. Actively exploring a company sale. (Link)
Cardioxyl - CXL-1020 in development for an initial indication of Acute Decompensated Heart Failure, and is
completed a Phase I/IIa clinical trial with positive results. Company has recently launched a dose ranging Phase 2
study.
H Celladon - developing MYDICAR®, a genetically-targeted enzyme-replacement therapy intended to restore levels
of SERCA2a and in phase 2 development for the treatment of acute and chronic heart failure. Company reported
positive data at AHA in Nov 2010.
Cordex - ATPace™ has an approved SPA to enter a pivotal Phase 2b/3 clinical trial for the treatment of a common
heart arrhythmia called paroxysmal supraventricular tachycardia. Cordex believes that ATPace™ is a more reliable
and superior product than existing therapies because of its unique ability to recruit the vagus nerve. Cordex has
hired WBB Securities to explore strategic options for this product and, potentially, the company. (Link)
Corimmun - German company developing COR-1, a peptide drug for the treatment of immune-mediated heart
failure due to anti-ß-adrenergic auto-antibodies, entering phase IIa. Also developing Revacept, a biological for the
prevention and treatment of atherothrombosis, Key indications coronary heart disease, acute myocardial infaction
and transitory ischemic attacks and stroke. Entering phase 2a shortly. (Link)
Debiopharm - Debio 0614 inhibits the Na+K+ ATPase pump and acts as a calcium modulator without tachycardia,
Positive efficacy results from a recent Phase 2a study. Company would consider partnerships.
Diffusion Pharmaceuticals - Looking to partner trans sodium crocetinate for treatment of intermittent claudication
and diseases associated with hypoxia. Reported encouraging data from a Phase 1b study for claudication at AHA in
Nov 2010.
Endotis - Developing EP-42675, a synthetic glycol-drug as an anticoagulant. Reported positive Phase 1 data at AHA
in December 2009. Has now developed an antidote to the same drug and has reported promising data (also see
Polymedix and Portola). (Link)
Evolva - In Phase 1 for EV-077, an oral thromboxane inhibitor, for managing platelet aggregation and diabetic
nephropathy. In December 2010 reported that had carried out further formulation work and had confirmed that it
had found a well behaved formulation of this drug in a Phase 1 study. (Link)
FCB-Pharmicell - Cerecellgram is a bone marrow derived stem cell composition, containing mesenchymal stem cells
and cells specifically useful for brain regeneration. Cerecellgram is being developed for the treatment of acute
ischemic stroke. In a Phase 3 ramdonmized clinical trial. (Link)
Kai - KAI-1455 is a selective epsilon protein kinase C activator designed to reduce ischemic injury during planned
surgical procedures, such as coronary artery bypass grafting, vascular surgery and pediatric cardiac surgery. In
Phase 2b trials and open to a partnership transaction. (Link)
NEW Lacer - Spanish company looking for partner for LA-419, an oral eNOS regulator targeted at ischemic
cardiovascular disorders. (Link)
$ Medicure - May sell U.S. rights to Aggrastat, originally a Merck drug, for acute cardiac indications. Update:
company restructured its liabilities on July 18, 2011 in order to reduce debt. Beal Advisors will be working with the
company to find a partner or buyer for Aggrastat going forward. (Link)
Menarini - Would partner Amediplase, a novel thrombolytic agent that has completed Phase 2 testing. (Link)
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$ Microbix - Owns Urokinase, formerly of Abbott (Abbokinase). Did revenues of $10mm+ recently. Not formally for
sale but purchased for a low price due to manufacturing issues which have largely been fixed. (Link)
Milestone Pharmaceuticals - Has a novel and potent short-acting calcium channel antagonist in preclinical
development for the treatment of transient cardiovascular conditions such as angina and atrial arrhythmias. (Link)
NanoCor Therapeutics - Preparing for clinical trials of Carfostin™, an intracellular protein therapeutic for the
treatment of Chronic Heart Failure (CHF). Carfostin™ is comprised of the delivery of a therapeutic gene, protein
Phosphatase-1 Inhibitor-1 (I-1) with the use of Biological NanoParticles (BNPs™) and the Self-Complementary
Vector Technology. (Link)
Neurocrine - Looking to partner Urocortin 2 program for CHF. Can improve cardiac output without raising heart
rate. Will report out Phase 2 data in 2011. (link)
UPDATE Nile Therapeutics - Would consider outlicensing its chimeric natriuretic peptide in Phase 1b clinical studies
for the treatment of acute decompensated heart failure. Data from this trial were encouraging. (Link)
NoNO - seeking a partner with the capability to develop and commercialise NA-1 (a PSD95 inhibitor) for acute
ischemic stroke (AIS).
Oxford University - Has identified a novel receptor for anti-platelet drugs - G6B. (link)
Paion - Looking to outlicense Solulin, an improved recombinant version of thrombomodulin, a blood coagulant.
Has completed Phase 1 studies. Also in Phase 2 with flovagatran, a direct thrombin inhibitor. (Link)
H Pervasis - In a POC P1b trial to study PVS-10200 to prevent restenosis in patients with peripheral arterial disease
(PAD) who undergo an angioplasty and stent procedure in the superficial femoral artery. Very promising indication.
Actively exploring options.
UPDATE Polymedix - Finished Phase 1 with a Factor Xa antitode that reverse effect of heparin and associated
compounds. Proof of concept achieved. Now in Phase 2a trial for the reversal of anticoagulant activity of
enoxaparin. Also, see Portola for a related Factor Xa antitode. (Link)
UPDATE Regado Biosciences - Developing injectible antithrombotics. Impressive Phase 2b data reported in
November 2011 from REG1 Anticoagulation System, a novel two-component system comprised of a Factor IXa
inhibitor anticoagulant and its complementary control agent. (Link)
UPDATE Sembiosys - APOa1 is a injectible protein that leads to atherosclerotic plaque regression. Preclinical version
of protein is available with very strong proof of principal data. Update: Company enters into a licensing deal for
partial geographic rights with Tasly of China. (Link)
Syngis - Developing AX200 in acute stroke. In Phase 2 trials. (Link)
Thrombogenics - TB-402 is a novel human antibody which partially blocks Factor VIII, an essential blood clotting
factor. Reported positive Phase 2 data. ThromboGenics and its partner BioInvent plan to out-license TB-402 for its
later stage development and commercialization. Currently dosing a P2b study.(Link)
Trevena- TRV120027, is a ?-arrestin biased ligand of the angiotensin receptor (AT1R) in development for acute
heart failure. Ten months into a Phase 2a study. (Link)
$ Undisclosed - pharma company is open to divesting a marketed specialty cardiology product with revenues >
$30mm per annum.
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NEW $ Undisclosed - Pharma company with a product for heart failure and revenues over $20mm would consider a
sale.
Via Pharmaceuticals - VIA-2291 is a reversible inhibitor of 5-LO, a key enzyme in the biosynthesis of leukotrienes
that impact inflammation and atherosclerosis. Positive Phase 2 data in a recent ACS trial. Also in Phase 1 studies of
VIA-3196, a THR beta agonist for management of dyslipidemia.
Viron Therapeutics - Developing VT-111, a serine protease inhibitor. Reduces restenosis and increases plaque
stability in animal models. Has recently finished a Phase 2a study in PCI patients. (Link). Viron has been granted U.S
patents for organ transplant and arthritis drug candidates.
Xention - In a Phase 1 study for a novel treatment for atrial fibrillation (Link)
NEW Zensun - Phase 2 studies for treatment of CHF via remodeling of cardiac muscle cell sarcomeric and
cytoskeleton structure or cell-cell adhesion using rhNRG-1 protein.
CNS - NEUROLOGY
Acadia Pharmaceuticals - In Phase 3 studies of pimavanserin for the treatment of Parkinson’s disease psychosis.
This program had been partnered to Biovail but was returned after the merger with Valeant. (Link)
Acceleron - Latest stage unpartnered product is ACE-031 which works by inhibiting myostatin and other negative
regulators of muscle mass thereby freeing the body to rebuild muscle tissue. Phase 2 trials underway in DMD.
Partnered with Shire outside the U.S. Acceleron will retain all commercial rights in North America. (Link)
$ Accera - Axona, a medical food for treatment of Alzheimers. Related medical food treatments for Alzheimers in
development by Adeona, Bellus Health and Groupe Danone. Update: Oct 5, 2010: New CEO appointed. Company
remains open to a partnering or change of control transaction.
$Acorda - FDA approved AMPYRA® (dalfampridine) on Jan. 2010, which is indicated as a treatment to improve
walking in patients with MS. Launch of this product is now underway. Recent studies show increased improvement
in walking with use of AMPYRA. Partnered Europe rights to BiogenIdec.Company considered a top acquisition
candidate. (Link)
Adamas Pharmaceuticals - In Phase 2/3 with ADS?5102, a proprietary extended release (ER) formulation of
amantadine indicated for Levodopa-induced dyskinesia in Parkinson’s disease. (Link)
Adeona - in Mid-Phase 2 of a trial for oral estriol for MS. (Link).
Adeona - recently missed primary endpoint in trial for zinc therapy for the treatment of Alzheimer’s disease.
Company reported a retrospective subgroup analysis in June 2011 where the treatment was associated with an
improvement in symptoms. (Link)
UPDATE Addex - In Phase 2a for diplaglurant, an mGLUR5 NAM for the treatment of Parkinson’s Dyskinesia and
dystonia. This program is regarded as a very promising approach for PD. Additional data are expected in the
second half of 2012 and company could seek a partnership when these data are in. Company recently announced a
reorganization and CEO stepped down. Update: new CEO appointed in October 2011. (Link)
Aeoulus Pharmaceuticals - AEOL 10150 is an oral catalytic antioxidant that mimics and thereby amplifies the body’s
natural enzymatic systems for eliminating reactive oxygen species. Product has completed Phase 1 and is indicated
for ALS. Company recently received a $118 million research and development contract from BARDA. Company
reported significant survival advantage in non-himan primate study of AEOL 10150 as a treatment against lung
damage from radiation exposure.
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Allon Therapeutics - Allon Therapeutics is testing davenutide in a variety of CNS indications including Progressive
Supernuclear Palsy (PSP). Positive Phase 2 data in amnestic mild cognitive impairment (aMCI), a precursor to
Alzheimer’s disease (AD). (Link). Allon is currently a Phase 2/3 clinical trial in progressive supranuclear palsy (PSP),
a type of frontotemporal dementia (FTD). Phase 3 is underway. Allon intends to commercialize in and outside of
North America.
Allozyne - Long acting interferon beta for MS. Phase 2 data are upcoming. Update: Allozyne recently merged with
Poniard but remains interested in partnering its interferon beta program.
NEW Andalusian Initiative for Advanced Therapies - A research group from the Andalusian Public Health System
(SSPA) has developed a new composition for the treatment of cognitive disorders associated with Fragile X
Syndrome. Phase 1b trial results have shown that the composition counteracts the production of free radicals by
reducing oxidative stress, while improving behavior and learning in individuals affected with the Fragile X
Syndrome. (Link)
Avineuro Pharmaceuticals - has reported positive Phase 1 data on AVN-322, a 5-HT6 antagonist for Alzheimer’s
Disease. Is preparing to commence Phase 2 studies. (Link)
H Bluebird Bio - Positive Phase 1b type data for LentiGlobin® for gene therapy treatment in a young adult with
severe betathalassemia, a blood disorder that is one of the most frequent inherited diseases. Also has positive
data for a treatment for Adrenoleukodystrophy (ALD) is a rare, inherited neurological disorder.
Bial - BIA 9-1067 is currently being developed by BIAL to be used in addition to L-DOPA/carbidopa or L-DOPA/
benserazide preparations in PD patients. Currently in Phase 3 studies. Promising results have been obtained for BIA
9-1067 in previous studies. (Link)
Bionomics - BNC210is a novel anti-anxiolytic which has completed Phase 1 studies and displays good drug-like
properties.Two parallel Phase Ib trials of BNC210 were initiated in France in October 2010 and results exceeded
expectations. (Non-con)
Bioprojet - In Phase 3 studies of pitolisant for the treatment of excessive daytime sleepiness in Parkinson’s disease.
Being tested as an add-on to modafinil. Supportive Phase 2 data. (Link)
BTG - BTG-1531. This NCE is a proprietary EP4 antagonist with potential applications in inflammation, pain and CNS
(including MS). Clinical data is available from several studies. (Link)
BTG - Pleneva is an orally administered investigational compound, designed to restore the balance between pro-
inflammatory (e.g. tumour necrosis factor-? TNF?) and anti-inflammatory cytokines (e.g. transforming growth
factor-?1, TGF?1) in patients with multiple sclerosis (MS).In a small single-centre, double-blind, placebo-controlled
clinical pilot study, aPleneva prototype provided clinical benefits to patients with RRMS, including decreases in
both relapse rate and EDSS scores, with additional benefits seen on pain and cognitive endpoints. A European
multicenter Phase IIa study has completed patient recruitment. (Link)
Celtic Pharma - Has hired an investment bank to divest Xerecept, a novel agent in late Phase 3 studies for
peritumoral brain edema. See a related program at Eustralis Pharma.
CeNeRx BioPharma - CXB722 has potential as a novel anxiolytic, demonstrating a significant effect on both
endocrine and cardiovascular biomarkers associated with stress. CXB722 has been studied in approximately 800
patients, demonstrating safety and efficacy in a range of mood and anxiety disorders. (Link)
H Chelsea Therapeutics - NORTHERA™ (droxidopa), is an orally active synthetic precursor of norepinephrine
initially being developed for the treatment of neurogenic orthostatic hypotension. Large potential in the
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Parkinson’s market where there has been a reported 60% reductions in falls in PD patients with NOH - also could
work in fibromyalgia. On market already through Dainippon Pharma in four Asian countries. Company recently
reported that it is filing for an NDA based on studies that have already been completed and has recently completed
its QTc work. Anticipates approval by Q2 2012.
Chiesi - CHF1512, an oral, soluble form of carbidopa/L-dopa for Parkinson’s disease. SPA issued by FDA for Phase 3
study. Ready to start Phase 3 trials under protocols agreed with the FDA through the SPA process. Note: the rights
to this product were returned to Chiesi from Vernalis in 2010 after failing to find a partner. (Link)
$ CNS Therapeutics - has introduced Gablofen, an AP rated intrathecal version of baclofen for control of severe
spasticity among patients with movement disorders. This product has significant advantages over the existing
marketed product and is likely to have significant revenue traction over the next several years. (Link)
Colucid Pharmaceuticals - Lasmitidan, a novel drug for migraine, selectively targets 5HT1F receptors expressed in
the trigeminal nerve pathway is entering Phase 3 trials. Company open to partnering or sale transaction. (Link)
Cynapsus Therapeutics - Developing APL-130277, an apomorphine thin film strip formulation for the rescue of
patients experiencing “OFF” periods in Parkinson’s disease. Expects to enter its first human clinical trials of APL-
130277 in the second half of 2011. (Link)
Cytokinetics - According to a recent press release, Cytokinetics, Inc. announced opening of next phase 2 clinical
trial of CK-2017357, a fast skeletal muscle activator, in patients with amyotrophic lateral sclerosis. CK-2017357
selectively activates the fast skeletal troponin complex by increasing its sensitivity to calcium, leading to an
increase in skeletal muscle force.Saw activity in a recent Phase 2a trial Actively seeking partnerships.(Link)

$ Daval International - Aimspro, orphan Status Designations have been awarded by the Therapeutic Goods
Administration (TGA) for the treatment of Krabbe Leukodystrophy and Amyotrophic Lateral Sclerosis. In June, 2011
completed Phase 2 trial study of AIMSPRO for treatment of bladder dysfunction in patients with secondary
Progressive MS. To report topline data in the Fall. Sold on a named patient basis.
EnVivo Pharmaceuticals - EVP-6124, a gamma secretase inhibitor, for Alzheimer’s disease showed signs of
cognitive improvement in a Phase 2a study. Currently in Phase 2b studies. Recent issues with Lilly Semagacestat
have raised questions about gamma secretase inhibitors as a class. Looking for a partnership. EnVivo currently has
partnered with Mitsubishi Tanabe Pharma Corporation (MTPC) for the further research, development, and
commercialization of its lead product, EVP-6124, in several Asian countries (most notably, in Japan). (Link)
ExonHit - Looking to partner EH202, which has completed Phase 2a studies in Alzheimer’s disease.
Galantos Pharma - Developing Memogain, whichis made up of galantamine, a nicotinic acetylcholine receptor
sensitizer marketed as Razadyne by J&J. In pre-clinical studies.
Green Cross - Open to international outlicensure of GCC1290K which is a prodrug of 3-hydroxymorphinan. This has
progressed through Phase 1 studies in Korea. (Link)
Green Cross - Developing Intravenous Immune Globulin (IVIG). In Phase 3 in Korea. Recently went forward with an
IND in the United States. Extensive evidence supports use of this product to treat Alzheimers disease. (Link)
Impax Laboratories - Impax is looking to partner the ex-US rights to IPX056 - a controlled release baclofen for
spasticity in multiple sclerosis. (Link)
Innate Therapeutics - Going into Phase 2a studies with MIS416, an immune system modulator, in multiple
sclerosis. Patent issued for MS in New Zealand in January, 2011 and pending in international markets.Began
recruiting patients for Phase 2 clinical trials in September 2010 and initiated trials in October 2010. (Link)
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Intec Pharma - An analysis of the results showed that the administration of the Levodopa drug with the Accordion-
Pill significantly improved its efficacy. Administration of the drug with the Accordion-Pill enabled a significant
reduction in the number of Levodopa doses per day in comparison to the currently marketed Levodopa products.
(Link)
Intellect Neurosciences - OX1 is a Phase 1b copper binding molecule that stabilizes non-toxic soluble forms of
amyloid and protects brain cells by blocking redox-mediated neurotoxicity. It has been tested for safety in healthy
elderly people in Phase1 clinical trials. (Link)
KemPharm - KP106 is a novel prodrug for the treatment of attention-deficit hyperactivity disorder (ADHD). KP106,
a new chemical entity (NCE), is composed of the active pharmaceutical d-amphetamine and a ligand and was
created through application of KemPharm’s proprietary Ligand Activated Therapy (LAT) approach. Positive Phase 1
data. (Link)
NEW Kyowa Hakko Kirin - looking to partner a Phase 3 Parkinson’s drug, Kw-6002. Istradefyline is an adenosine A2a
receptor antagonist. (Link)
Manhattan Pharmaceuticals - reported positive Phase 1b data for AST-915 for the treatment of essential tremor.
Compound associated with reductions in the severity of tremor. (Link)
MarcoPolo Pharmaceuticals - VLB-01 is a ML2 (MT3/ Quinone oxidoreductase 2 (QR2)) receptor agonist with a
unique receptor profile implying first-in-class anti-epileptic activity. VLB-01 is being developed for treatment of
epilepsy. Phase 2a results are pending. (Link)
Marinus Pharma - Positive phase 2a data for Ganalaxone, a novel epilepsy neurosteroid, in which statistics showed
significant reductions in seizure frequency. The compound is also beginning trials for its affect on PTSD patients
and UCD neurologists have been awared a grant to study the compound in treatment of Fragile-X syndrome
starting later in 2011. (Link)
Medesis Pharma - Medesis Pharma, announced the successful completion of a Phase I clinical trial of NP03 (lithium
citrate), a disease modifying treatment for Huntington's disease. (Link)
Medicinova - Running a partnership process for MN-166, indicated for the treatment of relapsing multiple
sclerosis. MediciNova is collaborating with Kyorin Pharmaceutical Co., Ltd. in the global development of MN-166.
(Link)
Memen Pharmaceuticals - Phase 2 data show improvement in memory from levo-amphetamine sulfate. Company
looking for a partner to conduct further Phase 2 work.
Mithridion - MCD-386, a muscarinic agonist, has completed phase I studies and seeks to halt or slow down the
progression of Alzheimer’s disease and improve memory and cognitive function. (Link)
Nelson Pharmaceuticals - Developing MagneVal, a reformulated, patented version of Valproate for epilepsy that is
less subject to carnitine deficiency. (Link)
Neuraltus Pharmaceuticals - In Q1 2011, started a Phase 2 randomized, double-blind, placebo-controlled,
multicenter study of NP001 in subjects with amyotrophic lateral sclerosis (ALS). Works by regulating macrophages.
(Link)
Neuren Pharma - developing Motiva for improvement of apathy and cognition after stroke. Missed endpoint in a
recent Phase 2b trial. (Link)
UPDATE Neurocrine - In Phase 1b with a VMAT2 inhibitor designed to slow the transport of monamines such as
dopamine. Intended for Phase 2 studies in tardive dyskinesia associated with Parkinson’s. In second quarter 2011,
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approached the FDA reagarding filing of IND in the US and have meeting schedule with the FDA for June 2011.
Enrolling second Phase 2b trial. Looking for ex-U.S. partners. (Link)
Neuroderm - In a Phase 2a study for ADHD with patch containing nicotine and other agents. (Link)
NEW Neuroderm - developing ND0611, a carbidopa patch for treating Parkinson’s disease that would largely
improve the bioavailability of orally administered levodopa. Reported positive Phase 2 data in November 2011.
(Link)
NeuroHealing Pharmaceuticals - dosing patients in a Phase II/III study to test the efficacy of NH001 (apomorphine)
in accelerating the recovery and improving the outcome of patients in a vegetative state following a severe
traumatic brain injury (TBI). (Link)
Neurologix - In Phase 2 with a gene therapy program for Parkinson’s Disease with positive data for a non-
dopamergic approach. Has hired MTS Partners to seek “strategic collaborations.” Update: No transaction reported
as of June 2011. (Link)
Neurotherapeutics Pharma - NTP-2014 has been evaluated in multiple animal models of epilepsy, neuropathic and
nociceptive pain, and migraine. In every one of these models, the compound exhibited a dramatic level of efficacy,
outperforming positive controls represented by some of the industry's most commercially successful therapies. An
IND is being filed to go forward in man with this compound.
Neurosearch - In Phase 1b with Ordopidine for Parkinson’s dyskinesia. (Link)
Neuronova - In June 2011 NeuroNova completed a Phase I/II, randomized, double-blind, placebo controlled, safety
and tolerability study of intracerebroventricular administration of sNN0031 (PDGF-BB) to patients with idiopathic
Parkinson's Disease of moderate severity, using an implanted catheter and a synchromed pump. Also in Phase 1b
for ALS therapy. (Link)
UPDATE Newron - has failed with ralfinamide in Phase 3 for lower back pain. Market cap around $45mm. Has a solid
pipeline of CNS candidates. Open to an M&A deal. Update: Had announced a merger with BioTie which was
terminated on Oct 28, 2011. Company looking for alternatives.
Noscira - Nypta® (tideglusib) is a GSK-3 enzyme inhibitor that has shown positive effects in experimental models
against the main lesions that arise in Alzheimer’s disease: tau protein hyperphosphorylation, amyloid plaques, and
neuron loss. In a Phase 2b trial. Also in a trial for the treatment of progressive supranuclear palsy. (Link)
Oxford Biomedica - In a Phase I/II trial of ProSavin for the treatment of Parkinson's disease (PD). This gene therapy
has been associated with improvements in patient symptons. (Link)
Pharmaneuroboost - looking to start a Phase 3 program for a fixed dose combination of citalopram and
pipamperone. (Link)
Prana - Actively partnering PTB2 for Alzheimers. Good Phase 2a data for novel aggregation inhibitor of amyloid
beta plaque. Prana has planned a Phase IIb study to determine the impact of PBT2 on progression of cognitive and
functional decline in patients suffering from mild to moderate Alzheimer’s Disease and is going forward with next
trial.
NEW Proximagen - 5-HT1a agonist for refractive epilepsy. Safe and well tolerated in >500 patients. In a Phase 2 trial.
Psychogenics - Pursuing eltoprazine in Phase 2 studies for ADHD. Supportive Phase 2a data. (Link)
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NEW Raqualia - RQ-00000009 is a preclinical 5Ht-4 partial agonist. Compound penetrated into brain effectively and
significantly improved memory and cognitive deficit in rodent models. In addition, RQ-00000009 decreased brain
cortex ?-amyloid proteins in Tg2576 mice.
Receptos - in a Phase 1 study for an S1P1 agonost for the treatment of multiple sclerosis. Expect to enter Phase 2
studies in 2012.
NEW Repligen - open to partnering RG3039 and RG2833. 3039 IS A Phase 1 drug for spinal muscular atrophy and
2833 is a Phase 1 ready drug for the treatment of Friedreich’s Ataxia.
NEW Siena Biotech - Selisistat is a first-in-class selective SirT1 inhibitor, a potentially disease-modifying mechanism
for Huntington’s disease. The compound is currently in Phase 2a clinical trials.
SK Biopharmaceuticals - YKP3089 is a novel compound with broad-spectrum anticonvulsant activity under clinical
development. Through recent a Phase IIa photosensitivity study, the potential for efficacy in humans was
confirmed. (Link)
NEW SK Pharmaceuticals - YKP509 - NCE that has been in 2000 patients for epilepsy and neuropathic pain. Looking
for a partner.
Sonexa - ST101 is a small, orally active molecule that is well absorbed and efficiently penetrates the blood-brain-
barrier. In development for Alzheimer’s disease with positive animal data. ST101 has completed Phase I studies
and is being evaluated in Phase 2 Proof-of-Concept trials. (Link)
NEW Spectrum Pharmaceuticals - looking for a licensee for SPi-339 a novel molecule for the treatment of ADHD.
(Link)
Stemcells - HuCNS-SC is well-characterized, normal human CNS stem cells (HuCNS-SC) from brain tissue, isolated
and purified using monoclonal antibodies against cell surface antigens. HuCNS-SC is being developed as
intracerebral injection for the treatment of myelin disorders such as Pelizaeus-Merzbacher Disease. Phase 1 data
upcoming in 2012. (Link)
UPDATE Supernus - NDA to be filed soon for Epliga for Refractory Partial Onset Epileptic Seizures. Once a day CR
version of oxcarbazepine. In addition, an NDA has filed for SPN-538, an extended release topiramate. Company has
a royalty that has been partially monetized. No active M&A process underway but the company is venture owned
implying an exit is possible. Company has an S-1 on file. (Link)
Suven Life Sciences - SUVN-502 is a potent, highly selective and active antagonist at 5-HT6 serotonin receptor.
SUVN-502 is being developed as once daily oral formulation for the treatment of memory and cognitive disorders.
Entering Phase 2a studies. (Link)
Sygnis AG - In Phase 2 with AX200 for stroke.
H TauRx Therapeutics - planning to initiate two Phase 3 trials testing a new reformulated version of its tau inhibitor
Rember for the treatment of Alzheimer’s disease. Very promising data obtained with this program. Company open
to a partnership transaction.
Transition Therapeutics - has finished a Phase 1 study for TT-301 for traumatic brain injury.
UPDATE Trophos - Olesoxime for ALS. In Phase 2/3. Active in preclinical models. No POC yet in man. Data expected
in Q4 2011. Actelion has a go/no go decision to make to acquire this company based on a previous option
agreement. (Link)
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Undisclosed party - Developing orphan neurology products based upon a compound designed to correct certain
mitochondrial defects.
H new Undisclosed party - Positive Phase 2 data for a compound for the treatment of Fragile X syndrome.
Company is seeking a buyer or global partnership with assistance of a financial advisor.
H new Undisclosed party - sale of marketed neurology product with U.S. rights, growing revenues and orphan
protection. Interested parties should contact Benj Garrett ([email protected]).
Vectura Group - positive data from a Phase 2b clinical study for its inhalation product, VR040 (apomorphine hcl),
for the treatment of off episodes in patients with fluctuating Parkinson's disease.
NEW Xenoport - XP19986 is a prodrug of the R-isomer of baclofen. Positive Phase 2 data for spasticity. XenoPort
reached agreement with the FDA on a Special Protocol Assessment (SPA) for a pivotal Phase 3 clinical trial of
arbaclofen placarbil (AP), previously known as XP19986, as a potential treatment of spasticity in multiple sclerosis
(MS) patients.
CNS - PSYCHIATRY
Abiogen - isoxazoline derivative which is being developed as an oral formulation for the treatment of generalized
anxiety disorders. (Link)
Acadia Pharmaceuticals - Preparing to file an IND for AM-831, a small molecule product candidate for the
improvement of cognition in persons with schizophrenia. This product is partnered with Meiji Seika Kaishia in Asia.
Also see Bioline. (Link)
UPDATE Alexza - Looking to partner several inhaled products including ADASUVE (Loxapine) which recently met the
primary endpoint in a Phase 3 Bipolar Disorder trial. A Phase 3 study in schizophrenia was successful. Product was
partnered to Biovail and recently returned. Promising opportunity. Update: Dec 8, 2011: FDA advisory committee
documents somewhat negative on pulmonary safety of this product but committee voted in favor its approval.
Auspex Pharma - SD-254 is a deuterated venlafaxine helps avoid multiple dose levels. Has completed Phase 2a
studies and shown superior PK. (Link)
UPDATE $ Avanir - Launched Neudexta for the treatment of pseudobulbar effect and, potentially, other indications.
Positive data and good patent picture. Widely rumored to be an M&A candidate. $5.7 million in revenue in first
year of launch (soft numbers). Market cap of $248 million as of November 2011. EMA application recently
accepted.
Avineuro Pharmaceuticals - positive topline results from a Phase 2a clinical proof of concept trial to assess AVN-
211 as an augmentation therapy to improve cognition in schizophrenia patients. In a double blind trial in 50
patients stabilized on an atypical antipsychotic therapy, AVN-211 met the protocol criteria for positive results on
the primary efficacy outcome measures. (Link)
Azeva Pharma - SRX246 and SRX251 are being developed as novel therapeutics for stress-related CNS disorders,
including depression and Intermittent Explosive Disorder. Both are ready for Phase 2 studies. (Link)
COMPLETED $ Azur Pharma has hired Lazard to advise on funding options including a potential stake sale according
to the Sunday Times on May 22, 2011. Company has a solid franchise as a marketer of drugs in the CNS and
women’s health areas. A company sale could be possible. Update: Sep 19, 2011 - Jazz Pharma merges with Azur
Pharma as an Ireland domiciled company with former Jazz shareholders taking 80$ of the combined company. A
transaction closing is expected in Q1 2012.
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BiolineRx - recently was returned rights to BL-1020 a late stage drug for cognitive improvement in patients with
schizophrenia. Positive Phase 2 data.
Braincells - BCI224 contains sabcomeline, a direct-acting cholinergic muscarinic receptor agonist. BCI224 is being
developed as a parenteral formulation for the treatment of major depressive disorders. In Phase 2 studies. (Link)
Braincells - also developing BCI-540. Results from the Phase 2a trial suggest BCI-540 has the potential to treat a
difficult-to-treat population, people with MDD who have failed previous treatments, and have a co-morbid anxiety
disorder. (Link) (Press release)
CeneRx - TriRima (CX 157) a reversible MAO-A inhibitor in Phase 2 for the treatment of depression. After phase 2 is
completed, CeneRx will look for a partner. (Link)
Cortex - Developing AMPAKINE compounds for depression and schizophrenia. Recently returned to company from
Merck for repartnering. Also received rights pack from Biovail in May 2011. Partnered on product, CX1632 via an
option deal to Servier in June 2011. (Link)
Cyrenaic Pharmaceuticals - CYR-101 is a small molecule drug with a unique mechanism of action. It has high affinity
for sigma-2 and 5-HT2A receptors and has been shown to be potentially effective in modulating dopamine
pathways without the negative dopamine-related side-effects that are associated with all current antipsychotics.
Promising Phase 2a data. (Link)
Envivo Pharmaceuticals - EVP-6124 is a potent, orally bioavailable and selective alpha-7 agonist, for patients with
schizophrenia. Recent Phase 2b data showed that EVP-6124 had a clinically meaningful and statistically significant
impact on patients’ overall cognition - the trial’s pre-specified primary endpoint - when taken in combination with
second-generation antipsychotics and as measured by the full CogState overall cognitive index, or “OCI” (p=0.05
for all patients treated with EVP-6124 versus placebo). Also see Bioline.
NEW Euthymics Bioscience - developing amitifadine for major depressive disorder. In a 318 patient Phase 2b/3 trial
at present. (Link)
Inmedix - Owns use patents for outlicensing to use Pramexipole (Phase 2 completed) and ropinorole’s phase I is
completed. (Requip) for fibromyalgia.
UPDATE $ Intelgenx - CPI-300 is a novel, high strength of Bupropion HCl, the active ingredient in Wellbutrin XL(R).
Indicated for depression. This product was approved in November 2011 and Intelgenx is in active licensing
discussions to find a commercial partner.
H Intracellular Therapeutics - Pursuing ITI-007 for schizophrenia and other compounds. This atypical antipsychotic
is in Phase 2 and has a differentiated profile from drugs on the market and promises higher efficacy with a better
side effect profile. (Link)
KemPharm - Developing a thin film strip with a conturolled release of Adderal. Less abuse potential than Vyvanse.
Looking to partner this drug which has completed Phase 1 studies. (Link)
MarcoPolo Pharmaceuticals - BC-19 is an eburnamine derivative, previously investigated for the indications of
cerebral insufficiency and cognitive deficits. As an antidepressant, BC-19 had shown beneficial properties when
administered to patients suffering from treatment resistant depression. (Link)
Medicure - In Phase 2 for the treatment of tardive dyskenesia (Tardoxal) as a side effect of schizophrenia
treatments. (Link)
Naurex - depression drug based upon Glycine-site Functional Partial Agonists (GFPAs), peptides which are designed
to achieve the strong efficacy of classic NMDA modulators without their limiting CNS side effects. Has completed
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Phase 1 studies with no safety issues. Recently completed an $18mm financing and is in Phase 2a studies for this
novel approach. (Link)
NoNO, Inc. - NA-1 is currently in a multicenter, placebo-controlled randomized Phase 2 trial testing its efficacy in
reducing damage produced by strokes incurred following neurointerventional procedures.
Repligen - Would consider a partnership for Uridine which is in Phase 2b for bipolar depression. This compound
recently failed its primary endpoint. (Link)
UPDATE Reviva Pharma - RP-5000 is a potent, selective NCE being developed for the treatment of schizophrenia and
is available as an oral formulation. Has finished Phase 1b studies with positive results. (Link)
NEW Spectrum Pharmaceuticals - looking for a licensee for SPi-376 a novel preclinical molecule for the treatment of
schizophrenia. (Link)
Synosia - Rufinamide (SYN-111) in Phase 2 in GAD. Eisai has the rights to market SYN-111 in the EU.
Transcept Pharma - pursuing ultra low dose ondansetron as an adjunctive treatment of obsessive compulsive
disorder. Phase 2 study coming up. (Link)
$ Undisclosed - ADHD marketed drug with revenues > $50 million from a pharma. Would consider a sale.
$ Undisclosed player - sale of company with over $10mm in revenue with largely genericized specialty products in
CNS and renal disease.
NEW Undisclosed - U.S. rights to a marketed drug for depression are available.
NEW $ Undisclosed player - availability of product with pysch and primary call point. $12mm product sales run rate.
University of Florida - Halogenated amino acids as a partial agonist of the glutamate-binding site of NMDA
receptors for schizophrenia. Research stage.
University of South Carolina - Homolog of Ritalin for ADHD. Preclinical. (link)
CNS - SLEEP
Alliance Pharmaceuticals - Looking to outlicense Posidorm, a surge formulation of melatonin. Alliance is seeking a
co-development and co-marketing partnership deal with a partner company to progress Posidorm through late
stage development to product registration and subsequent marketing. (Link)
NEW Aptalis - AdvaTab® Temazepam is the first ODT formulation of a highly prescribed benzodiazepine. In Phase 3
development. Looking for commercialization partner for this product.
Bioprojet - In Phase 3 studies of pitolisant for the treatment of excessive daytime sleepiness in Parkinson’s disease.
Being tested as an add-on to modafinil. Supportive Phase 2 data. (Link)
Evotec - Would like to outlicense EVT-201, a GABAa Receptor Positive Allosteric Modulator for Insomnia. The
results from Phase 2 have shown positive data, still looking for partners for further development. Update:
Recently licensed China rights to Jingxin Pharma.
Intec Pharma - Intec Pharma announced the Phase 2 clinical data with Accordion Pill Zaleplon. The analysis of the
intermediate results of the trial showed that taking the Zaleplon medication with the Accordion Pill resulted in
statistically significant improvement in its effectiveness versus placebo. (Link)
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Neurim Pharma - Circadin® 2 mg prolonged-release melatonin formulation, approved for use (up to 13 weeks) in
treatment of primary insomnia characterized by poor quality of sleep in patients aged 55 years or over. (Link)
NEW Somnus Therapeutics - developing SKP-1041, a delayed release formulation of Zaleplon for sleep. Key
indication is to prevent middle of the night wakening. Positive Phase 2 data. Company interested in finding a
partner before starting Phase 3 studies.
UPDATE Transcept Pharmaceuticals - Looking for an ex U.S. partner for middle of night insomnia drug, Intermezzo.
Transcept has formed a U.S. partnership with Purdue Pharma for Intermezzo. Drug was approved by FDA in
December 2011.
NEW Undisclosed - rights to a marketed sleep drug may be available.
Vanda - Tasimelteon, Melatonin agonist for sleep wake disorders, in phase III. Has orphan designation.
CRITICAL CARE PRODUCTS
Action Pharma - AP214 finished Phase 2, a modified dMSH-peptide analogue, for the treatment of post-surgical
kidney injury in the cardiac surgery context. The results demonstrate that AP214 is well tolerated and safe at all
three dose levels. At the highest dose level, AP214 prevents the increase in serum creatinine by 50-60%, and in the
IL-6 response by 30-40%, compared to placebo (trends based on blinded data). This is consistent with a robust
effect to prevent postsurgical acute kidney injury (AKI) and systemic inflammatory response. (Link)
UPDATE AM-Pharma - Has achieved highly positive Phase 2 results in the treatment of acute kidney failure with
alkaline phosphotase. Company advancing a backup program with stronger patent protection. (Link)
COMPLETED Artisan Pharma - ART-123 for sepsis in Phase 2b. Recently approved in Japan. Company was acquired in
November 2011 by Asahi Kasei Pharma.
Asklepion - L-Citruline for the 20 percent of children who have heart surgery with use of the bypass pump develop
pulmonary hypertension, or high blood pressure in the lungs. In early studies this product appears to prevent
pulmonary hypertension from developing. Now in Phase 3 studies. (Link)
NEW BCO Pharma - Phase 3 ready program for the treatment of hypotension in neonates. Novel presentation of
existing hypotensive agent.
Diffusion Pharmaceuticals - Looking to partner trans sodium crocetinate (TSC) for treatment of intermittent
claudication and diseases associated with hypoxia. Positive Phase 1b/2a data reported at AHA in 2010.
Discovery Labs - is reportedly engaged in partnership discussions with respect to licensing its neonatal franchise,
which includes Surfaxin, Sufaxin LS and Aerosurf. Want a partner to collaborate on clinical development for
Surfaxin LS and Aerosurf, and to play a central role in commercialization for all three products.Surfaxin is a
synthetic KL4 Surfactant for treatment of neonatal RDS. Company expects to refile for approval in Q1 2011. Has
granted development and marketing rights for Discovery Labs’ SRT products to Esteve in the key southern Europe
markets of Spain, Italy, Portugal, Greece, and Andorra.
DSX Therapeutics - Developing a Mab that targets inducible nitric oxide synthase, which is involved in sepsis
pathology. Pre-clinical program.
Focus Care Pharmaceuticals - Looking for partners in Europe and other geographical locations for its line of rapid
oral rehydration salts.
UPDATE Infacare - Developing Stannsoporfin (Stanate) for Neonatal Hyperbilirubinemia, which is in a Phase 2b trial.
Company expects to report out data soon.(Link)
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$ Microbix - Owns Urokinase, formerly of Abbott (Abbokinase). Did revenues of $10mm+ recently. Not formally for
sale but purchased for a low price due to manufacturing issues which have largely been fixed. (Link)
Oxygen Biotherapeutics - Entering Phase 2b trials for Oxycyte in traumatic brain injury (TBI) in Switzerland and
Israel. Compay has moved forward to its second cohort in the trial. (Link)
Polymedix - PMX-60056 is a universal anticoagulant reversing agent, active against both heparin, low molecular
weight heparin (LMWH) and pentasaccharide (Arixtra). Phase 1B complete with POC achivieved, looking for global
and local partners. (Link)
Serendex - inhaled rfVIIa for blast injury and lung bleeding. Six patient study showed high efficacy. Orphan
designation granted. (Link)
NEW Undisclosed player - looking to partner a recombinant formulation of human serum albumin. Broad
applications and advantages over blood derived product. For further details contact
[email protected].
DENTAL DRUGS
Array BioPharma - Oral p38 inhibitor has shown significant analgesic benefit in a recent Phase 2 trial in dental pain.
$ Pierrel Group - Received FDA approval in March 2010 for Articaine - a local dental anaesthetic that contains a
thiophene ring. The thiophene ring of Articaine increases its lipophilicity and is highly diffusible allowing an
effective tissue penetration.
NEW Sanifit - ASB 01 - OTC dental health / oral hygiene product inhibits formation of dental calcium deposits
(tartar). In NDA filing phase.
NEW Undisclosed - Marketed dental drug with applications in the treatment of oral mucositis is available.
Interestested parties should contact [email protected].
NEW Undisclosed - German vertically integrated marketer of generic and branded pharma products with strength in
cardiovascular, dental and veterinary medicines. Revenues of this company exceed €100mm.
DERMATOLOGY - AESTHETICS
$ Anika - Has rights to Elevess®, a hyaluronic acid dermal filler. Open to a partnering deal. The rights were recently
returned to Anika and company has been modifying filler to address historical issues with the product.
Biolab - interested in partnering a variety of aesthetic products that are sold in Brazil. (Link)
BTG - Varisolve, a Phase 3 treatment for the removal of varicose veins. BTG now looking at commercializing this
product in the United States. Other regions available. (Link)
$ Church & Dwight - Would sell some Del Pharma niche products.
Cosmo Pharma - CB-03-01, NCE anti-androgen for the topical treatment of Acne, Hirsutism and Androgenic
Alopecia. , Phase 2 studies showed a POC for acne. Recently showed nice POC in treatment of alopecia. (Link)
Fibrocell Sciences - Azficel-T is an autologous cell therapy for the treatment of moderate to severe nasolabial fold
wrinkles in adults. Drug has been approved and launched. (Link)
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$ Lipose - Viafill fat transfer system on the market with applications in aesthetics where traditional volumizers are
not well suited, particularly for the face and breasts. Company assisted by Torreya Partners. For details contact
[email protected].
Lithera - Announced positive results from a Phase IIb clinical study of LIPO-102, its novel injectable combination of
salmeterol xinafoate (SX) and fluticasone propionate (FP) for selective, non-ablative fat reduction. In a Phase 2b
study LIPO-102 was well-tolerated when administered weekly for 8 weeks into the subcutaneous abdominal fat of
healthy subjects and produced dose- and time-related reductions in mean abdominal volume and circumference.
Pharmena - bundle of dermatology products that are currently sold in Poland. These products are IP protected and
well suited to either an OTC or physician office promotion setting. Looking for an international partner to
commercialize. For further discussions contact Tom Bird of Torreya Partners at [email protected].
$ ScarGuard - Could consider a company sale. Markets ScarGuard MD, a widely used scar remedy used by plastic
surgeons. Also has OTC product.
Sinclair IS Pharma - SPHR913 is designed to restore the quality of the skin by reducing the breakdown of collagen
and elastin. (Link)
UPDATE $ Suneva Medical - Marketing Artefill, a long-acting dermal filler with impressive revenue growth. Open to
a partnership or even a company sale. Update 12/2011: Company reports substantial revenue growth for the year
and expects additional growth in 2012. (Link)
Topotarget - Avugane contains the HDAC inhibitor VPA for use as a topical treatment of inflammatory skin
diseases, including acne vulgaris (common acne), psoriasis and atopic dermatitis. Promising data from a double-
blinded placebo controlled Phase 2. Looking to partner.
$ Undisclosed player - Aesthetic dermatology business has been open to a sale with revenues over $80 million.
Valeant - in a mid-July investor call, the CEO of Valeant indicated that he might consider a sale of the company’s
aesthetic assets including the recently acquired Sculptra filler from Dermik. "Well, I think like everything, it
depends what it's worth to us versus what it’s worth to other people. I don’t think we are at critical mass in the
aesthetics area," the CEO said.
DERMATOLOGY - MEDICAL
Anacor - Looking for ex-U.S. partnerships for dermatology portfolio including preclinical and clinical compounds for
psoriasis, tinea pedis, acne and atopic dermatitis. AN2728 is past POC stage and is in a Phase 2 trial for psoriasis
and is expected to report in the first half of 2009. Based on these results, Anacor expects to initiate a Phase 2b
dose-ranging trial for AN2728 in mid-2010. Also going into an extensive Phase 3 program for AN2690 for the
treatment of onychomycosis. completed an IPO in Q4 2010. (Link)
Apricus Biosciences - Running a process to outlicense NM100060 for onychomychosis. Has been in multiple Phase
3 studies. (Link)
Biofrontera - BF-200 ALA, currently in European registration for the treatment of actinic keratosis. BF-200 ALA
combines a nanoemulsion with 5-aminolevulinic acid (ALA). The product is developed in photodynamic therapy of
precancerous skin lesions (actinic keratosis). Looking to partner. (Link)
NEW Burke Pharma - late stage product in development for the treatment of warts. Approved device to accompany
pharmaceutical approach.
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Creabilis - CT327 is a novel topically applied TrkA kinase inhibitor developed using Creabilis’ LSE (Low Systemic
Exposure) technology. CT327 contains pegylated K252a, which interacts at a nanomolar level with tyrosine kinase
receptor (TrkA), the receptor for nerve growth factor (NGF). It has strong analgesic activity and it acts by blocking
NGF activity. Good results in Phase 2a psoriasis study versus placebo. Also positive data in study versus atopic
dermatitis. High potential for the treatment of neuropathic pain. (Link)
$ Dusa Pharma - Main tool is PDT coupled with derm drugs. Revenues around $30mm. (Link)
Echo Therapeutics - 505b2 NDA application pending for Durhalieve (triamiconolone) for dermatoses. (Link)
Forward Pharma - Positive Phase 2 data on FP-187 for psoriasis. Would consider a sale. (Link)
Glenmark Pharma - Microsphere Adapalene + Clindamycin for acne was compared against Adapalene +
Clindamycin in a standard gel formulation and had less irritation. Available for outlicensing.
COMPLETED Graceway - Could consider sale of products from 3M and has hired Lazard to explore alternatives
through an ongoing process. Aldara for acitinic keratosis a key seller but was recently genericized. Update: Nov 23,
2011 - Medicis acquired Graceway for $455mm after the company filed for Chapter 11 bankruptcy on Sep 29,
2011.
Ibsa - Looking to outlicense betametasone valerate patch for treatment of psoriasis. A wide range of markets (EU,
USA, South America, Middle and Far East) are still available on exclusive or semi-exclusive basis. Could do a global
agreement. (Link)
$ Jina Pharma - Lipid based Amphotericin B Gel is Indicated in acute, chronic and recurrent type of skin fungal
infection and cutaneous leishmaniasis. Provides specificity to target fungal cells.
Labtec - LabiPatch adheres to the semi-moist environment of the lip and surrounding skin to deliver acyclovir for
herpes sores. Looking for a licensee. Also has a patch for atopic dermatitis.
NEW Laurantis Pharma - Cis-UCA emulsion cream is a phase 2 stage topical dermatology product indicated for the
treatment of atopic dermatitis. Preliminary efficacy has been seen in a controlled exploratory phase 2a study in
patients with mild to moderate atopic dermatitis. A proof-of-concept phase 2b study with up to 170 patients with
moderate to severe atopic dermatiris is ongoing. The study is expected to be completed in Q2/2012.
Mimetica - MTC896 as a topical gel for the treatment of excessive sebum production in subjects with acne and
other skin conditions. MTC896 is a highly selective and potent antagonist ( $40mm.
Vyteris - has indicated that it is interested in disposing of a portfolio of pain products in development. These
products include LidoSite®, an FDA approved product for the pretreatment of needle injection and venipuncture
sites with Lidocaine (a related product is at Nuvo Research); a Phase 1 zolmitriptan patch and an NSAID patch.
(Link)
EMERGING MARKETS - PARTICULARLY BRAZIL, CHINA, INDIA, TURKEY DEALS
NEW Abdi Ibrahim Ilac - largest Turkey drug maker with revenues over $800mm. Reported in May 2011 that was in
discussions to sell a strategic stake.
Amoun Pharmaceutical - An Egyptian company that manufactures off-patent branded generic formulations. It is
one of the largest pharmaceutical companies in Egypt. It sells over 135 human products in over 275 forms. Of
these products, 33 occupy the top 2 positions in their respective therapeutic categories and subcategories. Open
to a company sale or strategic stake purchase. Reuters - Dec 6, 2010: “CVCI is also preparing to sell Amoun, one of
Egypt's biggest drugmakers, people familiar with the matter told Reuters on Oct. 20. It owns Amoun with two
other co-investors.” Bloomberg reported in Feb 2011 that the company has been looking for $1 billion in a sale
price but that political upheaval in Egypt has hindered the sale.
UPDATE Biofarma - Biofarma for sale via JP Morgan. Reuters (12/6/2010): Citigroup's (C.N) venture capital arm and
two co-investors have begun an auction of Turkish copycat drugmaker Biofarma, three people familiar with the
matter said, in what could be Turkey's biggest healthcare deal.” Update: A number of parties rumored to have
looked at this asset but price ask was seen as prohibitive. As of Nov 2011 no sale had taken place.
NEW China Nuokang - ts lead products include Baquting for bleeding control, Aiduo, a cardiovascular stress imaging
agent, and Aiwen, an anti-arrhythmic agent. The company has a value of $180mm. Biopharm Insight in November
reported that this company hired Lazard for a sale process.
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$ Claris Life Sciences - Indian injectibles company has hired Rothschild to look at strategic options. Company has a
rich pipeline of hospital generic injectibles using novel delivery methods. (Link)
NEW Guangxi Golden Throat (Guang Xi Jin Sang Zi), a privately held manufacturer of healthcare products, is
reportedly in sale talks. The company has annual revenues of around $47mm from its throat lozenges, which sell
under the 'Golden Throat' brandname.
NEW Huanghai Pharmaceutical - has been approached by interested bidders according to Biopharm Insight (Nov 15,
2011). It has a well-established products brand and sales network in the local Chinese market, thanks to its star
product "Nifedipine" has 70% market share.
NEW Mustafa Nevzat - Turkish generic pharmaceutical maker with revenues of approximately $250mm. According
to Bloomberg, in talks to sell a strategic stake.
NEW Undisclosed - Chinese specialty pharmaceutical company with strength in anti-infectives. Good EBITDA and
revenue over $70mm in 2011. Torreya Partners assisting in sale of majority stake. For details please contact
[email protected].
NEW Undisclosed player - Division of Indian generic company that is focused on oral solid dose preparations is for
sale. Revenues over $300mm. Company has strength in formulation work and manufacturing. Ships product to
numerous global locations.
COMPLETED Undisclosed player - Large pan-Asian drug company open to a sale. Update: Menarini acquires Invida
Group on Nov 16, 2011. Price not announced but Invida a substantial business with more than $200mm in
revenue.
Undisclosed player - a number of emerging markets players are developing portfolios of generic injectable drugs
including cytotoxics and anti-infectives.
Undisclosed player - Large domestic generic player in emerging markets has expressed openness to a change of
control transaction.
ENDOCRINE
NEW Althea Technologies - has a promising phase 2 extended release version of human growth hormone in
development. Previously this compound was at Genentech.
Ambrilia Biopharma - Long acting version of ocreotide for acromegaly ready for market introduction in 2010. Large
market opportunity. Company in bankruptcy. (Link) Recently agreed to extend licensing option agreement. There
are still multiple partnerhip opportunities aviable.
Biopartners - In late stage trials with a sustained release version of human growth hormone. (Link)
Camurus - CAM2029 (octreotide chloride FluidCrystal® Injection depot) for treatment of acromegaly is in a Phase 2
trial against Sandostatin® LAR from Novartis.
Critical Pharma - CP016 is a long acting injection of somatropin (recombinant human growth hormone). CPO16 is in
its phase 1 of studies.
COMPLETED $ DuoCort - Plenadren contains hydrocortisone, a glucocorticoid receptor agonist. It delivers a more
physiological dose of the active substance, better mimicking the body's own glucocorticoid serum profile
combining rapid release and extended release characteristics. Plenadren is being developed as a dual-release once
a day tablet, for the treatment Addison's disease (adrenal insufficiency). Sold on a named patient basis by Clinigen.
Update: Company bought by Viropharma in November 2011. (Link)
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$ Ibsa - Tirosint (L-Thyroxin) in soft gel capsules for treatment of hypothyroidism. Approved in the U.S. but not yet
marketed. Company searching for a marketing partner. Ibsa - looking to outlicense betametasone valerate patch
for treatment of psoriasis. A wide range of markets (EU, USA, South America, Middle and Far East) are still
available on exclusive or semi-exclusive basis. Possibilities for a global agreement. (Link)
NEW $ Shunfeng Pharmaceutical, a Chinese topical skin care drug manufacturer is exploring a sale. Revenues
around $35mm.
FIBROSIS
H COMPLETED Amira Pharmaceuticals - AM152, has completed Phase 1 studies of a drug candidate for fibrosis based
on LPA1 target. Company interesting in pursuing IPF and scleroderma of the lung. Note: this company was bought
by BMS in July 2011 for $350mm upfront plus milestones.
Angion Biomedica - in Phase 2 studies for BB3, an HGF mimetic for the treatment of hepatic fibrosis and to
facilitate better outcomes in renal transplantation. The naturally-occurring cytokine hepatocyte growth factor
(HGF), also known as scatter factor, is active in numerous tissues throughout the body, participating in the
regulation of angiogenesis, organogenesis, tissue repair and neural induction. (Link)
Argentis - ARG201 in P2 for scleroderma. Some positive data but missed P2 endpoint at 12 months. (Link)
Capstone Therapeutics - AZX100 is a novel synthetic 24-amino acid peptide. Based on its demonstrated effects in
pre-clinical models and safety in clinical trials it is in Phase 2 development for hypertrophic and keloid scarring with
potential for treatment of pulmonary fibrosis and intimal hyperplasia. Data at year end 2010. Update: Dec 13, 2010
- company indicates that it missed the primary endpoint in the keloid scarring trial.
Catena - VPC51299, an orally available pre-clinical molecular, targets LPA GPCRs with high potency (also see
Amira). These GPCRs elicit a signaling cascade upon LPA binding the receptor leading to LPA’s biological effects,
such as proliferation, migration, angiogenesis and so on. (Link)
Conatus Pharmaceuticals - CTS-1027 an oral MMP inhibitor for liver disease with potential to treat liver fibrosis.
Phase 2 study on treatment of hepatitis C patients underway. Results from this Phase 2 trial are forthcoming.
UPDATE $ Daval International - In Oct 2011, announced positive results from a Phase 2 of AIMSPRO in patients with
Late Stage Established Diffuse Cutaneous Systemic Sclerosis (diffuse scleroderma). (Link)
H COMPLETED Excalliard Pharma - Has technology for topical application of antisense drugs to treat fibrotic diseases
like scleroderma. Recent Phase 2 data showed EXC 001 treatment significantly reduced scar severity in subjects
undergoing an elective abdominoplasty compared to placebo. Update: Company bought by Pfizer for an
unannounced amount on Nov 22, 2011.(Link)
H FibroGen - Would consider outlicensing FG-3019 anti-fibrosis compound. Novel mechanism based on CTGF.
(Link)
NEW Fibrotech - Has commenced manufacturing and non clinical toxicology for FT011, an antifibrotic for the
treatment of diabetic nephropathy. Clinical trial due to commence in Q1 2012. (Link)
NEW Galectin Therapeutics - developing drug candidates for the treatment of liver fibrosis based on Galectin
modulation. These candidates are currently in preclinical stage. Company planning to file IND. (Link)
UPDATE Intercept Pharma - INT-747, an FXR modulator, for treatment of liver disease including primary biliary
cirhossis and NASH. (Link) Phase 2 results were positive. Company in active partnership discussions.
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Intermune - Bloomberg reports on April 27: “Biotechnology company InterMune Inc known for its drug to treat
lung scarring, hired Goldman Sachs (GS.N) to help it weigh a possible sale... Goldman has been conducting an
auction of InterMune for more than a month and some potential bidders have been spooked by the biotechnology
company's expectations for a sale price, Bloomberg news reported.” Company commercializing Pirfenidone in
Europe. Company market cap over $2bn.
NEW Phenex Pharma - completed Phase 1 on an FXR agonist for liver fibrosis and NASH. See Intercept Pharma for a
similar program. Open to business development deals.
Promedior - Developing recombinant human Serum Amyloid P Component for the prevention and treatment of
fibrotic pathology. PRM-151 in Phase 2a trials for prevention of post-surgical scarring in glaucoma patients. Would
consider a corporate sale and is currently in active partnership talks after raising an additional $12 million.
H Stromedix - STX-100 is being developed for the treatment of chronic allograft dysfunction in kidney transplant
recipients. Also exploring IPF indication. Humanized monoclonal antibody to integrin ?v?6, going into Phase 2.
NEW Vernalis - V85546 - Phase 2-ready novel selective anti-inflammatory compound that selectively inhibits
MMP12 and has in-vivo efficacy in pre-clinical models of Chronic Obstructive Pulmonary Disease (COPD), Multiple
Sclerosis (MS) and liver fibrosis. Phase I SAD and MAD studies have been conducted. Substantial safety and tox
package would support up to 6 month dosing in Phase II. Worldwide rights available. (Link)
GASTROINTESTINAL
4SC - 4SC-101 in Phase 2a trials for Crohn’s disease. 4SC-101 contains vidofludimus, a novel, selective and orally
available, small molecule inhibitor of dihydroorotate dehydrogenase (DHODH), which interferes with cell
proliferation through blocking the synthesis pathway of pyrimidines, thereby halting the proliferation of rapidly
multiplying cells, in particular of lymphocytes which are involved in the pathology of autoimmune disorders.
Adeona Pharmaceuticals - outlicensing CORRECTA™, a retention enema formulation of the widely used topical
antifungal agent clotrimazole, for the treatment of acute refractory pouchitis. Completing a Phase 2 study. (Link)
Ajinimoto - AJM300 is an alpha-4 or beta-7 integrin inhibitor, which is being developed for the treatment of
Crohn's disease. This product is in Phase 2 studies.
H Albireo - Positive IBS-c Phase 2 data on A3309 which modulates the enterohepatic circulation of bile acids by
inhibiting the Intestinal Bile Acid Transporter (IBAT). Company starting Phase 3 studies. (Link)
Alevium - Long acting PPI. Solves a number of problems with omeprazole and Nexium.
H Alvine - Enzymatic treatment for celiac disease. Currently in a Phase 2a clinical trial. (Link)
AM-Pharma - Has achieved early POC results in the treatment of ulcerative colitis with alkaline phosphatase. (Link)
Ardelyx - RDX5791 is a systemic NHE3 (sodium/proton exchanger type 3) inhibitor which increases intestinal
sodium leading to enhanced interstitial fluid volume and transit. RDX5791 is being developed as capsule for the
treatment of constipation predominant irritable bowel syndrome (IBS-C).
Aryx Therapeutics - ATI-7505, an improved cisapride like molecule, for reduction of GERD, constipation and
dyspepsia. Update: Feb 19, 2010: Company retained Cowen to explore strategic options. Dec 15, 2010: “Over the
course of the strategic process ARYx initiated earlier this year, the most significant interest, even in the absence of
a binding offer, was shown in ARYx's gastrointestinal product candidate, naronapride (ATI-7505), demonstrating
the potential value of the asset. Also, interactions with the US Food and Drug Administration ("FDA") in April 2010
substantially clarified the remaining clinical development requirements for the compound. As a result, the lead
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investors in ARYx encouraged the company to seek substantial additional funding to continue the development of
naronapride internally, and such funding has been actively pursued since late summer without final resolution.”
Company close to default on debt. Update: March 2011 - company went into a wind down of its operations. (Link)
NEW Atlantic Healthcare - Novel Phase 2 product for treatment of pouchitis. On market in EU on a named patient
basis. Open to a U.S. partnership.
Bioprojet - Dexecadotril is selective inhibitors of neprilysin (NEP) which exhibits intestinal antisecretatory action.
Dexecadotril is being developed for the treatment of acute diarrhea in children and is in Phase 3 studies. (Link)
NEW Cancer Prevention Pharmaceuticals - seeking a ROW partner for its combo of eflornithine and sulindac. In
Phase 3 studies for familial adenomatous polyposis (FAP).
ChiRhoClin - Secretin is a polypeptide hormone having 27 amino acids produced by S cells of the duodenum.
Secretin is being developed as intravenous infusion for the treatment of pain due to chronic pancreatitis. (Link)
Cosmo Pharma - developing Naloxone MMX for the treatment of opioid induced constipation (OIC). Strong story
and competes against AZ/Nektar drug. In Phase 1 studies.
NEW Cosmo Pharma - looking for a partner for low molecular weight heparin derivative that is used to control
ulcerative colitis. Impressive Phase 2 dataset supports the story.
NEW Cubist - recently acquired Adolor and open to partnering rights to Adolor products in Asia. Included is
ADL5945 for the treatment of opioid induced constipation. This product has completed Phase 2 studies. Interested
parties should contact [email protected].
COMPLETED Cytokine Pharma Sciences - CPSI-2364 is an orally active form of the synthetic guanyl hydrazone
semapimod. CPSI-2364 inhibits signal transduction pathways that produce pro-inflammatory cytokines like TNF-
alpha, IL-1, IL-6 and nitric oxide. CPSI-2364 is being developed as an oral formulation for the treatment of ERCP-
induced pancreatitis and is in Phase 2 studies. Update: Company acquired by Ferring on October 24, 2011.
Edusa Pharmaceuticals - In Phase 2 studies with Pumosetrag, a 5-HT3 partial agonist that works locally within the
upper GI tract, is being developed to treat GERD symptoms in patients who continue to experience heartburn and
regurgitation while on PPIs or H2-antagonists. (Link)
Embil - Would outlicense Kortos cream for hemorrhoids and anal fissures. (Link)
EryDel - EryDex contains dexamethasone sodium phosphate. Dexamethasone is a glucocorticoid agonist which
binds with high affinity to specific cytoplasmic receptors. This results in modification of transcription hence,
protein synthesis in order to achieve inhibition of leukocyte infiltration at the site of inflammation. EryDex is being
developed as intravenous infusion using EryDel technology for the treatment of crohn's disease. In Phase 3 studies.
(Link)
Evoke Pharma - EVK-001, an intranasal metoclopramide, is currently in Phase 2b for the treatment of the
symptoms associated with diabetic gastroparesis. (Link) Product registration will occur following a pending Phase 3
clinical trial.
NEW Furiex - developing MuDelta which was recently returned by J&J. Company reached an agreement with FDA
for a Phase 3 trial of MuDelta in diarrhea-predominant irritable bowel syndrome (IBS-D) after an End-of-Phase 2
meeting with the agency. Interested parties should contact [email protected]. (Link)
Genzyme / Sanofi - Outlicensing RDP58 (Delmitide), a clinical-stage D-amino acid decapeptide with established
anti-inflammatory properties. Proof of concept for this product opportunity has been demonstrated in models of
inflammatory bowel disease (IBD).
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Hutchison MediPharma - HMPL-004 has completed two global phase 2 clinical trials in IBD and ulcerative colitis. A
223-patient global Phase IIb trial for the treatment of ulcerative colitis (“UC”) showed a decrease in rectal bleeding
and an excellent safety profile. Company actively looking for a partner to pursue Phase 3 studies. (Link)
Hyperion Therapeutics - Developing GT4P, an ammonium remover, for urea cyclic disorders and hepatic
encephalopathy - Positive Phase 3 data and a high likelihood of approval in 2012. (Link)
Index Pharmaceuticals - Kappaproct®, a nuclear factor NF-kappa-B p65 subunit oligonucleotide has completed its
third clinical phase 2 trial for the treatment of steroid resistent/dependent ulcerative colitis patients and is
entering a phase 3 trial during 2011. (Link)
H Lexicon Pharmaceuticals - Pursuing LX1031 for IBS. Lexicon completed a Phase 2 clinical trial of LX1031 in
patients with non-constipating IBS in November 2009. Update: Currently pursuing LX1033, a more potent
compound which is near the IND stage.
NEW Lipid Therapeutics - looking for a U.S. partner for LT-02 with strong positive Phase 2b data for
Phosphatidylcholine derivative to treat 5-ASA resistant ulcerative colitis. Potential for approval with a single Phase
3 study.
Menarini - Ibodutant, a tachykinin NK2 receptor antagonist for IBS. In phase I clinical studies in humans, the drug
was safe and well tolerated, showing good PK. Now in Phase 2 studies. (Link)
UPDATE Meritage Pharma - Positive Phase 2b data on effect of oral viscous budesonide (OVB) an oral formulation of
budesonide for the potential treatment of patients with eosinophilic esophagitis (EoE).
Moberg Derma - A-Fizz is in preclinical development for the treatment of chronic anal fissures.
UPDATE NPS Pharma - developing GATTEX for the treatment of short bowel syndrome. Well advanced in clinical
development. Recently saw some deaths of subjects on study. NPS has indicated interest in commercializing this
product on its own.
Ocera - Development of a novel carbon sorbent for mild hepatic encephalopathy and IBS, as well as the NCE, OCR-
002, for acute liver failure. Company to report out Phase 2b data for hepatic encephalopathy compound soon.
Active business development discussions underway.
NEW Ono Pharmaceutical - Has completed Phase 1 studies with ONO-2952, a TSPO antagonist for the treatment of
IBS. An ex-Japan partnership transaction is possible.
UPDATE OxThera - Oxazyme is recombinanate oxalate degrading enzyme for the treatment of kidney stones. No
data reported from a pending study in some time. A related compound is at Althea Technologies. (Link)
Paion - Looking to outlicense CNS7056 based on the available Phase 2 data for the drug as a short-acting
intravenous anesthetic/sedative for colonoscopy. Positive data were reported out in Nov 2009.
Palau Pharma - Currently undertaking a Phase 2 clinical trial with Derslazine sodium for mild to moderate
ulcerative colitis. Looking for a partner to assist with later stage trials.
Pharmos Corporation looking to partner Dextofisopam for diarrhea predominant and alternating diarrhea and
iconstipation irritable bowel syndrome. Positive in Phase 2a and missed endpoint in Phase 2b but activity seen.
Raqualia Pharma - In Phase 2 with a reversible inhibitor of gastric H+/K+-ATPase for GERD. Likely superior to
current proton pump inhibitor (PPI) therapies because of rapid onset, long duration and the absence of a food
effect. Going into Phase 2. (Link) (Noncon)
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NEW Rhythm Pharmaceuticals. RM-131, a novel ghrelin agonist, is effective in restoring normal gastric function in
animal models of delayed gastric emptying owing to a direct prokinetic effect. This compound is in Phase 1 trials
with a focus on gastroparesis.
NEW Ritter Pharma - RP-G28 for lactose intolerance has completed a Phase 2a study. A multi-center randomized,
double-blinded, placebo-controlled parallel group trial using a novel short-chain galacto-oligosaccharide (RP-G28)
enrolled 61 proven lactose intolerant maldigesters with the aim of improving lactose digestion and tolerance.
Lactose maldigestion (LM) was determined by breath hydrogen. Daily dosing with RP-G28 resulted in a significant
reduction in breath hydrogen production (p=0.01), peak hydrogen production (p=0.03) and in symptoms of
abdominal pain (p=0.01), flatulence (p=0.05), abdominal cramping (p=0.01) and gurgling (p=0.03) after a 25 g
lactose challenge.
Romark - Pursuing Nitazoxanide for C diff associated disease, rotavirus and hepatitis C. (link)
Rose Pharma - GLP1 for IBS. Positive data. Compound from Lilly. Company open to a sale transaction.
NEW $ Salix - looking for a European partner for RELISTOR, a subcutaneous treatment for opioid-induced
constipation. This was recently partnered in Asia to Link Healthcare.
UPDATE Shield Therapeutics - ST10-021 is a stable complex of ferric iron in an oral formulation for the treatment of
iron deficiency anemia associated with inflammatory bowel disease. This drug is in Phase 3 testing. IDA affects
approximately 73% of patients with ulcerative colitis or Crohn’s disease globally. (Link)
NEW Sigmoid Pharma - In Phase 2 for CyCol for Ulcerative Colitis. Targeted release of pre-solubilised
immunosuppressant for local GI release and activity. No systemic absorption. Therefore, a convenient, efficacious
and safe alternative to steroids, immunomodulators and biologics.
UPDATE SLA Pharma - Has developed a formulation of diltiazem for the treatment of anal fissures. This product is in
Phase 3 studies and is licensed to Ventrus Bioscience for the US market. Ventrus data likely in Q2 2012. SLA has
rest of world rights. (Link)
SLA Pharma - Has developed Eicosapentaenoic acid (EPA) is an omega-3 polyunsaturated fatty acid, taken as a
gastro-resistant oral capsule, for the treatment of Familial Adenomatous Polyposis (FAP). Phase 3 clinical study
data demonstrates a significant reduction in rectal polyp number in subjects with familial adenomatous polyposis
who had previously undergone colectomy and ileal-rectal anastomosis. (Link)
Soligenix - has partnered orBec/BDP with Sigma-Tau in the U.S. Looking to partner in ROW. Indication is GI
manifestation of acute GVHD, thereby reducing the need for systemic immunosuppressive drugs to treat GI GVHD.
Company is running a confirmatory trial and indicates a partnership is most likely after results are in. (Link)
UPDATE Synergy Pharma - Synergy presently has a drug, plecanatide, in clinical development to treat chronic
constipation (CC) and constipation-predominant irritable bowel syndrome. Has shown positive Phase 2a data.
Compare to Ironwood / Forest compound linaclotide. Update: Nov 2011 - raised $15mm for further trials. (Link)
Theravance - 5-HT
4
receptor agonist compound, TD-5108, is for the treatment of chronic constipation and other
disorders related to reduced gastrointestinal motility. Strong positive Phase 2 data. Update: “ (CEO indicated on
Sep 29) in talks with several companies regarding a partnership, but he is very open to speaking with additional
companies”
Thorne - PreBiox for the prevention of constipation and diahrrea associated with use of antibiotics. Extensive
Phase 2 data.
Tioga - Asimadoline from Merck KGaA. Phase 2b IBS data were positive. In Phase 3 studies. Rights to certain Asian
countries in-licensed by Ono in September 2009. (Link)
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Tranzyme - TZP-102 is an agonist of ghrelin receptors and acts as a GI prokinetic agent in Phase 2a studies. Strong
data were seen. Also, TZP-101, is an injectible ghrelin agonist being evaluated in two concurrent Phase IIb trials for
the treatment of acute indications, severe gastroparesis and post-operative ileus (POI) and are still looking for
partners.
Undisclosed player - two early stage GI drugs from a large pharma company are available for partnering.
NEW Undisclosed player - highly effective product for ulcerative colitis. 80%+ remission rates seen in recent clinical
study. Company looking for commercialization partner.
UPDATE Ventrus - Have iferanserin for hemorrhoids with a good data package. Enrolling a Phase 3 study which will
report out in Q2 2012. Company has gone public and raised over $70mm in capital to finance and obtain approval
for its top two programs. Product has potential revenues in excess of $1 billion. (Link)
Xenoport - XP19986 is a prodrug of the R-isomer of baclofen. Baclofen is a generic drug that has been shown in
investigator-led studies to be effective in the treatment of GERD. Interested in partnering this drug and is in phase
2.
NEW Yuhan Corporation - YH4808 is a Potassium-Competitive Acid Blocker (P-CAB) with characteristics of potent,
sustained inhibition of gastric acid secretion with faster onset. Unlike PPIs, YH4808 does not require acid-activation
which resulted in rapid increase in stomach pH. Therefore, fast symptom relief in patients expected. Positive Phase
1 data. (Link)
Zealand Pharma - Looking to partner ZP1848 for IBD which is completing Phase I development. ZP1848 is a SIP
modified novel GLP-2 peptide analogue with enhanced stability and efficacy. Company has excellent medicinal
chem and development capabilities. Would consider an M&A transaction. (Link)
GENERICS
Actavis - Has restricted its debt and company performing well. While no formal process is underway the company
could be open to a strategic transaction in next several years.
Amoun Pharmaceutical - An Egyptian company that manufactures off-patent branded generic formulations. It is
one of the largest pharmaceutical companies in Egypt. It sells over 135 human products in over 275 forms. Of
these products, 33 occupy the top 2 positions in their respective therapeutic categories and subcategories. Open
to a company sale or strategic stake purchase. Reuters - Dec 6, 2010: “CVCI is also preparing to sell Amoun, one of
Egypt's biggest drugmakers, people familiar with the matter told Reuters on Oct. 20. It owns Amoun with two
other co-investors.” Bloomberg reported in Feb 2011 that the company has been looking for $1 billion in a sale
price but that political upheaval in Egypt has hindered the sale.
NEW Apotex - looking for ex-Canada licensees / commercial partners for its large portfolio of generic products.
(Link)
Biofarma - Biofarma for sale via JP Morgan. Reuters (12/6/2010): Citigroup's (C.N) venture capital arm and two co-
investors have begun an auction of Turkish copycat drugmaker Biofarma, three people familiar with the matter
said, in what could be Turkey's biggest healthcare deal.” Update: A number of parties rumored to have looked at
this asset but price ask was seen as prohibitive.
$ Claris Life Sciences - Indian injectibles company has hired Rothschild to look at strategic options. Company has a
rich pipeline of hospital generic injectibles using novel delivery methods. (Link)
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$ CNS Therapeutics - has introduced Gablofen, an AP rated intrathecal version of baclofen for control of severe
spasticity among patients with movement disorders. This product has significant advantages over the existing
marketed product and is likely to have significant revenue traction over the next several years. (Link)
UPDATE Covidien Pharmaceuticals - According to the New York Times on June 7, 2011 “Covidien, the health care
company spun out from Tyco four years ago, may seek to sell its pharmaceutical unit…” This division of Covidien
(formerly Mallinckrodt) has a major business selling pain products (both branded and generics) and imaging
products. Revenues are around $2 billion. Update: As of December 2011 no sale has taken place. Company is
rumored to be interested in a sale of the whole business (rather than pieces) for a full price. YE 2011 numbers
reported on Nov 15, 2011 and were robust (sales up 9% yoy) with strong performance in generics.
UPDATE Hi-Tech Pharmacal - Market rumors in April 2011 that company could be purchased. As of December 2011
2011 no transaction had taken place of this manufacturing company of generic liquids and ointments. The
company has delivered strong earnings throughout 2011 and is trading at close to twice the price in Dec 2011 it
had earlier in 2011.
NEW Huanghai Pharmaceutical - has been approached by interested bidders according to Biopharm Insight (Nov 15,
2011). It has a well-established products brand and sales network in the local Chinese market, thanks to its star
product "Nifedipine" has 70% market share.
Ibsa - Tirosint (L-Thyroxin) in soft gel capsules for treatment of hypothyroidism. Approved in the U.S. but not yet
marketed. Company searching for a marketing partner. Ibsa - looking to outlicense betametasone valerate patch
for treatment of psoriasis. A wide range of markets (EU, USA, South America, Middle and Far East) are still
available on exclusive or semi-exclusive basis. Possibilities for a global agreement. (Link)
NEW Jubilant Pharma - has a list of generic oral solid pharmaceuticals in development that are available for
licensing for Europe and the U.S. (Link)
NEW Labormed - Romanian manufacturer of generic drugs is interested in finding global licensing partners for a
series of generic products (Link)
NEW Niche Generics - Looking for partners for its long list of generic products that have been developed. Subsidiary
of Unichem. (Link)
NEW Par Pharmaceuticals - Relational Investors filed a 13D showing 8.7% ownership of this company on November
25, 2011. In the 13D Relational indicated: “Despite these opportunities for improvement, the Reporting Persons
believe that the Company may continue to trade at discounted prices because of industry challenges and the
Company’s sub-optimal size and product scope. If the discount persists, the Reporting Persons believe that, in
keeping with sound stewardship principles, the Company’s board will be required to consider broad strategic
alternatives. Specifically, the Reporting Persons are confident that substantial cost savings could be achieved in a
transaction with a strategic buyer.” Note: there is no evidence that Par Pharmaceuticals has received offers or is
open to receiving such offers at present.
Stada - Chief executive Hartmut Retzlaff indicated an openness to takeover offers after the sale of Ratiopharm.
Undisclosed generic player - Looking for a partner for a portfolio generic biologics in development. Wants to retain
manufacturing rights.
Undisclosed player - a number of emerging markets players are developing portfolios of generic injectable drugs
including cytotoxics and anti-infectives.
Undisclosed player - Large domestic generic player in emerging markets has expressed openness to a change of
control transaction.
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Undisclosed player - U.S. company with one approved product and a rich pipeline of other high value injectable
generic products is looking for either a marketing partner or a company buyer.
COMPLETED Undisclosed player - U.S. generic player with approximately $200mm in net revenues and strength in
drug delivery solutions would consider a company sale. Update: Par acquires Anchen Pharmaceuticals for $410mm
in cash on Aug 24, 2011.
NEW Undisclosed - Chinese specialty pharmaceutical company with strength in anti-infectives. Good EBITDA and
revenue over $70mm in 2011. Torreya Partners assisting in sale of majority stake. For details please contact
[email protected].
NEW Undisclosed player - U.S. generic player with approximately $250mm in net revenues and strength in solid
dose manufacturing would consider a company sale.
$ Undisclosed player - sale of company with over $10mm in revenue with largely genericized specialty products in
CNS and renal disease.
NEW Undisclosed Player - U.S. generic company with approximately $90mm in gross revenue is searching for a
buyer with the assistance of a financial advisor. Company has substantial presence in medicines for cough & cold,
womens health and pediatrics.
NEW Undisclosed player - Division of Indian generic company that is selling its oral solid dose preparations business.
Revenues over $300mm. Company has strength in formulation work and manufacturing. Ships product to
numerous global locations.
NEW Undisclosed Player - U.S. generic company with more than $100 million in revenues is for sale. Company has a
significant branded business and a manufacturing facility.
NEW Undisclosed Player - U.S. generic company with more than $40 million in revenues is for sale via Torreya
Partners. Company has a significant pipeline, high growth and a fully developed operating platform. Interested
parties should contact [email protected].
NEW Undisclosed - German vertically integrated marketer of generic and branded pharma products with strength in
cardiovascular, dental and veterinary medicines. Revenues of this company exceed €100mm.
NEW Undisclosed player - U.S. generic company with revenues over $100mm but weak profibability is considering a
sale transaction.
Undisclosed player - several portfolios of ANDAs including a group of six solid dose ANDAs on infrequently
genericized products (generally through one bio study) and a portfolio of around 20 solid dose/liquid ANDAs on
more frequently genericized products.
Undisclosed player - U.S. generic player with approximately $25mm in revenues and an interesting pipeline is
running a sale process.
Undisclosed player - U.S. generic player with approximately $30mm in revenues and some manufacturing capacity
would consider a company sale deal.
NEW USV - offering a biogeneric PTH. Teriparatide is a recombinant form of N-terminal 1-34 amino acids of human
parathyroid hormone which is a 84 AA protein. Approval pending in India.

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GOUT
UPDATE Ardea Biosciences - RDEA806 oral for HIV and gout with good Phase 1b efficacy data. RDEA594 is the
metabolite that reduces uric acid. RDEA594 has completed Phase 2 studies with positive results and has been
evaluated in over 250 subjects. Company proceeding with Phase 3 trials.
UPDATE Biocryst - BCX4208 is a next generation purine nucleoside phosphorylase (PNP) inhibitor with the potential
for once-a-day dosing suitable for chronic administration. With its novel mechanism of action, BCX4208 has the
potential to address unmet medical needs across a broad spectrum of inflammatory diseases, including gout with
positive Phase 2 data. Company reported additional positive Phase 2 data in November 2011.
3SBIO / EnzymeRx - Developing Uricase-PEG 20 to treat gout (see Savient). In a Phase 1 study.Company in a sellside
process. Update: Company sold global rights to 3SBIO of China for $6.25 million in November 2010. Note that
3SBIO is planning to find a development partner for this product outside of China. (Link)
Metabolex, Inc., a biopharmaceutical company focused on the discovery and development of proprietary novel
medicines for the treatment of metabolic diseases, announced in May 2011 that it has initiated a Phase 2 clinical
trial of arhalofenate (MBX-102), its product candidate for the management of hyperuricemia in patients with gout.
Nuon Therapeutics - Developing tranilast for gout with strong positive Phase 2 data reported at EULAR 2010. (Link)
Pharmos - Data from Phase I studies in healthy volunteers using S-Tofisopam showed lowering of uric acid.
Company open to a change of control transaction. Also developing a Phase 2 product for GI disorders.
UPDATE Savient Pharmaceuticals - FDA approved KRYSTEXXA (pegloticase) in Sep 2010, a PEGylated uric acid
specific enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy.
Company is seeking a commercial buyer and is using JP Morgan and Lazard in its search for an acquisition partner.
Savient is now pursuing a launch of Krystexxa on its own and is building a 50-person plus sales force. Update: First
year sales were $2.6 million. Market Cap of $160mm. (Link)
Topotarget - Inflammasome Inhibitor is a protein scaffold and an interleukin 1 (IL-1) inhibitors are being developed
for the treatment of gout.
Undisclosed - Private company has a number of promising NCEs in development for the management of
hyperuricemia.
HAIR / SCALP
Advagen - Marketing OTC products that stimulate hair growth by inhibiting FGF-5 on the scalp. Based on research
from Australian company Cellmid, Ltd. (Link)
Cosmo Pharma - CB-03-01, NCE for the topical treatment of Acne, Hirsutism and Androgenic Alopecia, is in phase 2
studies. Recently showed nice Phase 2 POC in treatment of alopecia. Open to a partnership. (Link)
Follica - Company is developing technology that stimulates development of hair follicles. (Link)
Histogen - Histogen, Inc. announced the one year data findings of its Hair Stimulating Complex (HSC) pilot clinical
trial. Statistically significant new hair growth was seen in HSC-treated subjects at this follow-up timepoint, one year
after their single treatment with HSC.
Jina Pharma - MORR-F solution contains minoxidil and finasteride, which on topical application reduces falling of
hair and stimulate hair growth in individuals with alopecia. Efficacy shown. (Link)
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Manhattan Pharmaceuticals - Likely 2011 approval for Hedrin, a treatment for head lice. Hedrin is the top selling
head lice product in Europe. (Link)
Moberg Derma - K301 for seborrheic dermatitis has shown benefit in two Phase 3 clinical trials. Recently the
clinical studies were completed and now are looking for licensing.
Nidus Laboratories - Developing a short Fas?disabling peptide mimetic for topical therapy of hair loss.
Piedmont Pharmaceuticals - Have a late stage product for treatment of head lice. Partnered and marketed in
Europe. RESULTZ is sold internationally through licensing partners. Looking for other partners.
R-Tech Ueno - Announced that the Phase IIa clinical study of RK-023 to treat androgenetic alopecia has been
completed by January 21, 2011.
Topaz Pharma - completed two Phase 3 studies for the treatment of head lice with Ivermectin. Planning to submit
NDA to FDA in 2011. (Link)
NEW $ Undisclosed player - seeking buyer for approved product for the treatment of head lice in the United States.
Significant commercial traction. Interested parties should contact [email protected].
HEARING / EAR DISORDERS
13Therapeutics - Developing P13 as a treatment for Acute Otitis Media (AOM). Many colds in young children are
accompanied by ear infections. P13 is an orally available anti-inflammatory 20 amino acid peptide derived from a
viral regulatory protein. (Link)
Adherex - In Phase 3 development of sodium thiosulfate for treatment of hearing loss associated with Cisplatin
use. And is still looking for a partner. (Link)
UPDATE Auris Medical - AM101 is a small molecule, non-competitive antagonist of NMDA receptors that selectively
blocks NMDA receptors in the cochlea. AM101 is being developed as parenteral formulation for the treatment of
acute inner ear tinnitus. Phase 3 study planned for 2012 following positive data. (Link)
Desitin - studying Travistal in tinnitus. On market in U.S. but potential for exclusivity surrounding indication.
Foresight Biotherapeutics - Has recently completed a Phase 3 trial of FST-201 (dexamethasone 0.1% ) Otic
Suspension vs. the FDA-approved drug Ciprodex (ciprofloxacin 0.3%, dexamethasone 0.1%) Otic Suspension (Alcon
Laboratories, Inc.) in the treatment of acute otitis externa. (Link)
NEW Octoplus - developing OP-145, a therapeutic peptide for the treatment of chronic middle ear infection.
Otonomy - Searching for an ex-U.S. partner for a dexamethasone gel for Meniere’s disease which affects 600,000
people in the U.S. Going into Phase 1 with POC data expected in mid-2011. (Link)
Sound Therapeutics - going into Phase 2 studies of a product to reduce hearing loss from chemotherapy. (Link)
HEMATOLOGY
UPDATE Abiogen - Neridronate is an amino-bisphosphonate used in Metabolic Osteopathy and has gone into Phase
3 trials. Also being studies for patients with thalassemias. (Link)
Ablynx - ALX-0081 is a Nanobody targeting von Willebrand Factor (vWF), to reduce the risk of thrombosis in
patients with acute coronary syndrome (ACS) and thrombotic thrombocytopenic purpura (TTP). Through Phase 1b.
Phase 2 data are expected.
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NEW Adventrx- ANX-188 is a novel, purified, rheologic and antithrombotic compound initially being developed as a
first-in-class treatment for pediatric patients with sickle cell disease in acute crisis. Ready for Phase 3 studies and
open to partnering deals.
AesRx - Aes-103, is a small molecule (Da 126) discovered by researchers at Virginia Commonwealth University. It
works by increasing the affinity of sickle hemoglobin for oxygen. (Link)
H UPDATE Akebia - Positive Phase 2 with a HIF modulator for the treatment of anemia. Originally developed at P&G
Pharma. Open to a licensing deal in but could consider other possibilities including a change of control. Company
expects to report a second Phase 2 trial dataset in March 2012. (Link)
H Alder Biopharmaceuticals - Phase 2 data from ALD518 investigational antibody therapeutic that targets
interleukin-6 (IL-6) demonstrate a reversal of anemia in patients with advanced non-small cell lung cancer (NSCLC).
After 12 weeks of treatment with the anti-inflammatory therapeutic, 58 percent of patients who received ALD518
experienced hemoglobin level increases from less than 11 g/dL to more than 12 g/dL, while no patients receiving
placebo experienced this increase. Also saw improvements in lean body mass and reductions in fatigue. Note: this
antibody is partnered to BMS for all indications except cancer. (Link)
UPDATE Amsterdam Molecular Therapeutics (AMT) - Hemophilia B - very promising results from a gene therapy trial
to treat Hemophilia B which is due to a deficiency of Factor IX. Update: In Nov 2011 the company indicated that it
will be focusing resources on the development of this product. (Link)
AOP Orphan Pharma - P-1101 contains pegylated interferon alfa-2b (PEG-P-INF alpha-2b), a conjugation of
recombinant interferon alfa-2b with monomethoxy polyethylene glycol which shows antiviral and
immunomodulatory effects. P-1101 is being developed for the treatment of polycythemia vera. A Phase 1b study is
underway.
$ Biotest - a major global player in plasma proteins (IGs, coagulation factors) and antibodies (for inflammation) had
revenues in 2009 of 440mm EUR. Company was reported by Barron’s as a takeover target in Feb 2010.
H Bluebird Bio - Positive Phase 1b type data for LentiGlobin® for gene therapy treatment in a young adult with
severe betathalassemia, a blood disorder that is one of the most frequent inherited diseases. Also has positive
data for a treatment for Adrenoleukodystrophy (ALD) is a rare, inherited neurological disorder.
Catalyst Biosciences - developing improved proteases for hemophelia and inflammation. A Factor VII program has
been partnered to Wyeth but a Factor IX program which is in a preclinical stage is company owned at present.
Cellerant - Developing a novel, cell-based medicine (Myeloid Progenitors / CLT-008) as a treatment for
chemotherapy- and radiation-induced neutropenia as well as for Acute Radiation Syndrome. (Link)
Cleveland Biolabs - Open to partnering a preclinical stem cell mobilizer. Partnered in China. (Link)
Clinuvel - In Phase 3 for European approval for Afamelanotide, a photoprotectant to be used in Erythropoietic
Protoporphyria. Update: Company looking to partner in EU first and then the U.S.
$ Emisphere - has developed a rapid release Vitamin B12 which is on the market. Upcoming studies to show
relative efficacy of this product which would likely be marketed as a medical food.
COMPLETED GlycoMimetics - GMI-1070 is active in several models of diseases in which leukocyte adhesion and
activation plays a key role, including vaso-occlusive crisis of sickle cell disease. In Phase 2 studies. Company will
consider strategic options after completion of studies. Update: In October 2011 Pfizer entered into a licensing pact
for up to $340 million with Glycomimetics. (Link)
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Green Cross - Developing Intravenous Immune Globulin (IVIG). In Phase 3 in Korea. Recently went forward with an
IND in the United States. Extensive evidence supports use of this blood product to treat a variety of conditions
including Alzheimers disease. (Link)
UPDATE Hemaquest - Like Prometic below, exploring use of fatty acids and derivatives for the treatment of
hematologic disease. In a randomized multi-dose Phase 2 study of HQK-1001 in patients with sickle cell disease
with an interim data read expected soon. Recently appointed a new CEO and moved company to San Diego. (Link)
Incode BioPharmaceutics - HC3-1496 results in enzymatic depletion of the complement protein C3, the key
component for all three pathways of complement activation. Preclinical and applicable for oncology, PNH and RA.
NEW Japan Tobacco - Has Phase 1 HIF inhibitor for the treatment of anemia. Prefers to partner non-Japan rights
after achieving proof of concept.
UPDATE Medgenics - Medgenics has EPODURE with promising Phase 1b data showing efficacy in controlling anemia
without the cost of EPO. At ASN in Nov 2011 reported data which showed EPODURE treatment elevated
hemoglobin levels and maintained >9 g/dl for >3 months in 12/13 patients and >6 months in 7 /13 patients, with
the longest >30 months. Open to a partnership transaction.
UPDATE Noxxon - Developing NOX-A12, an inhibitor of SDF-1 or CXCR12. This is a chemokine that is a key regulatory
element in the homing and retention of hematopoietic stem cells in the bone marrow. SDF-1 binds with high
affinity to the chemokine receptors CXCR4 and CXCR7. The CXCR4/SDF-1 axis has been shown to play a role in stem
cell mobilization, vasculogenesis, tumor growth, and metastasis. In Phase I studies reported on Dec 12, 2011 with
healthy volunteers single doses of NOX-A12 up to 10.8 mg/kg and daily doses up to 2 mg/kg for five days were
found to be safe and well tolerated and resulted in dose-dependent mobilization of white blood cells and CD34+
cells as predicted by preclinical studies. (Link)
Palkion - Developing HIF modulators for anemia using molecules developed by Crystal Genomics. (Link)
UPDATE Polyphor - CXCR4 antagonist, POL6326 for hematological stem cell mobilization. In a Phase II clinical trial
for transplantation of autologous hematopoetic stem cells in multiple myeloma patients after chemotherapy.
Outlicensing opportunity. Similar to AnorMed Mozobil now controlled by Genzyme. (link)
Prometic - Exploring partnership options for PBI-1402 for treatment of anemia.
UPDATE Shield Therapeutics - ST10-021 is a stable complex of ferric iron in an oral formulation for the treatment of
iron deficiency anemia associated with inflammatory bowel disease. This drug is in Phase 3 testing. IDA affects
approximately 73% of patients with ulcerative colitis or Crohn’s disease globally. (Link)
SuppreMol - Developing soluble Fcy-Receptors (sFcyRs) for autoimmune disease. These are recombinant
autologous proteins with strong immunosuppressive potential. SM101, SuppreMol’s main product is a
recombinant, soluble, non-glycosylated version of the human Fcy receptor FyRIIb which is has completed a Phase 1
trial for ITP. In Phase 2 studies in ITP and going into further studies for lupus. (Link)
UPDATE Symphogen - Rozrolimupab (Sym001) is a recombinant polyclonal composition of 25 different Rhesus D
specific antibodies for the treatment of primary Immune Thrombocytopenia and for Anti-RhD prophylaxis (ADP) in
prevention of Hemolytic Disease of the Newborn. This product was returned to Symphogen from Biovitrum
Swedish Orphan for strategic reasons on Dec 30, 2010. Phase 2 data presented at ASH on Dec 12, 2011 showed
that rozrolimupab exhibited a favorable safety profile and induced a rapid increase in blood platelets in patients
with Immune Thrombocytopenia Purpura (ITP). (Link)
TaiGen - G-0054 is a potent and selective CXCR4 receptor antagonist. This molecule rapidly mobilizes stem cells
and progenitor cells from the bone marrow into peripheral circulation. (Link). Related drugs in development at
Chemokine Therapeutics and Cleveland Biolabs.
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UPDATE Tarix Pharmaceuticals – Positive Phase 2 data with TXA-127 for the prevention of chemotherapy induced
thrombocytopenia. Ongoing Phase 1b studies of this drug in hematological transplant. TXA-127 has much broader
applications in hematology, respiratory disease and fibrosis.
Therapure - TBI 304 is a monoclonal antibody that mimics the natural ability of hemoglobin to stimulate stem cells
to produce red blood cells. TBI 304 is in the preclinical stage. (Link)
Thrombogenics - TB-402 is a novel human antibody which partially blocks Factor VIII, an essential blood clotting
factor. Reported positive Phase 2 data and a Phase 2b study is underway. ThromboGenics and its partner BioInvent
plan to out-license TB-402 for its later stage development and commercialization once new data arrive. (Link)
HEPATOLOGY
Alfact Innovation - ALF-5755 has been shown to promote cell survival after apoptotic or oxidative stress, and liver
cell regeneration in primary cultures. ALF-5755 is being developed as slow intravenous infusion for the treatment
of acute liver failure. Recently granted orphan designation. (Link)
Alfama - In a preclinical stage of testing CORMs to treat acute liver failure. (Link)
UPDATE Antipodean Pharma – Phase 2b study for MitoQ which contains mitoquinone mesylate, a synthetic form of
coenzyme Q10. It is a mitochondria targeted antioxidant that selectively blocks mitochondrial oxidative damage
and prevents cell death. MitoQ is being developed by MitoQ Technology as an oral formulation for the treatment
of liver inflammation with the intention of use for treatment to lower AST’s and ALT’s.
NEW Asklepion - Cholic acid is an orphan drug that is a form of replacement therapy for children with a series of
inborn errors of bile acid metabolism manifesting as otherwise fatal cholestatic liver disease. This product is at the
Phase 3 stage.
UPDATE Biolex - Locteron reported Phase 2b studies in March 2011 for a long-acting interferon for Hepatitis C.
Because of its lower dosing, less depression was noted than with Peg-INTRON. On Mar 15, 2010 reported data
indicating that flu-like symptoms were much reduced with this compound versus Peg-INTRON and also showed
improved antiviral efficacy. (Link)
Conatus Pharmaceuticals - CTS-1027 an oral MMP inhibitor for liver disease with potential to treat liver fibrosis.
Phase 2 study on treatment of hepatitis C patients underway, so far the results have been positive.
NEW Genfit - FXR agonist in Phase 2. Proof of efficacy and safety of GFT505 have been obtained in Phase I and
Phase IIa trials (400 patients or healthy volunteers treated with GFT505 to date). The absence of safety concern
has been confirmed in a full toxicological package up to 2-year carcinogenicity studies.
HAC Biomed - a cell therapy company has very promising data for a treatment for the repair of damaged liver
tissue. Commercial stage in Germany with market entry in other territories soon. Assisted by Torreya Partners.
Interested parties should contact [email protected].
Hyperion Therapeutics - Developing GT4P, an ammonium remover, for urea cyclic disorders and hepatic
encephalopathy - Positive Phase 3 data and a high likelihood of approval in 2011/12 timeframe. Would consider a
partnership or sale transaction. (Link)
Intercept Pharma - INT-747, an FXR modulator, for treatment of liver disease including PBC and NASH. (Link) Phase
2 results were positive.
NasVax Ltd. - In a Phase 2a clinical trial in 36 subjects with NASH (Nonalcoholic steatohepatitis) or “fatty liver” and
the metabolic syndrome, oral aCD3 antibody immunotherapy was generally very safe and induced positive trends
in blood levels of two enzymes that are biomarkers for liver inflammation. (Link)
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Norgine - NRL972 is a liver disease detection diagnostic in Phase 2 clinical development in the US. NRL972 is a liver
staging tool, currently in Phase 3 clinical development in Europe and under an IND in Phase 2 clinical development
in the US and are looking for partnering options in US and Japan.
Ocera - Development of a novel carbon sorbent for mild hepatic encephalopathy and IBS (AST-120), as well as the
NCE, OCR-002, for acute liver failure. Phase 2b data for hepatic encephalopathy support application of product.
Active business development discussions underway.
NEW Phenex Pharma - completed Phase 1 on an FXR agonist for liver fibrosis and NASH. See Genfit and Intercept
Pharma for similar programs. Open to business development deals.
HOSPITAL PRODUCTS
Acusphere - Imagify, pending EMA filing for this cardiac imaging agent. Highly differentiated from SPECT agents on
the market insofar as permits evaluating of myocardial perfusion, an important marker of coronary artery disease
(CAD) without radioactive markers. Potential first-to-market drug in $600 million and $2 billion addressable market
in E.U. and U.S. respectively. Recent SPA from FDA in US. Will require one further trial for FDA approval. Company
is assisted by Torreya Partners. For details contact [email protected] (Link)
Adamis Pharmaceuticals - Launching Epinephrine Injection USP 1:1000 (0.3mg Pre-Filled Single Dose Syringe) (i.e.:
Epinephrine Injection PFS) to compete as a low cost alternative to the well known brand EpiPen®. Company was
recently unsuccessful in a “go public” merger with La Jolla Pharmaceuticals.
UPDATE $ AMAG - Feraheme IV iron product - Recently approved. Company is commercializing on its own. AMAG’s
recent merger attempt with Allos was ended in November 2011. On Nov 17, 2011, AMAG announced that it had
hired Jefferies to explore all opportunities to enhance shareholder value. Frank Thomas, interim CEO of AMAG
indicated: “We will expeditiously complete this process, which will include a parallel review of a potential sale of
the company and other strategic merger and acquisition transactions.”
COMPLETED Artisan Pharma - ART-123 for sepsis in Phase 2b. Recently approved in Japan. Company was acquired in
November 2011 by Asahi Kasei Pharma.
Asklepion - L-Citruline for the 20 percent of children who have heart surgery with use of the bypass pump develop
pulmonary hypertension, or high blood pressure in the lungs. In early studies this product appears to prevent
pulmonary hypertension from developing. Now in Phase 3 studies. (Link)
Avera Pharma - Gantacurium, a short-acting neuromuscular blocker, has successfully completed a Phase 2 clinical
study, where it was used as an adjunct to general anesthesia to enable rapid intubation of patients undergoing
surgery. Looking to partner.
NEW BCO Pharma - Phase 3 ready program for the treatment of hypotension in neonates. Novel presentation of
existing hypotensive agent.
Cara Therapeutics - CR845, a selective kappa opiod agonist, has completed a Phase 1 study. Phase 2 studies were
positive. Specificially, CR845 provided evidence of analgesic efficacy when administered as a single intravenous
dose to women following laparoscopic hysterectomy. In addition to decreases in reported pain levels, patients
receiving CR845 required substantially lower amounts of postoperative opioids for 16 hours, and showed a
significant reduction in the incidence of postoperative nausea. See a similar molecule in testing by Tioga
Pharmaceuticals (GI section). (Link)
$ Claris Life Sciences - Indian injectibles company has hired Rothschild to look at strategic options. Company has a
rich pipeline of hospital generic injectibles using novel delivery methods. (Link)
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$ Cohera Medical - Developing TissueGlu, a deep wound adhesive for use in surgical applications.
DSX Therapeutics - Developing a Mab that targets inducible nitric oxide synthase, which is involved in sepsis
pathology. Preclinical program.
FCB-Pharmicell - Cerecellgram is a bone marrow derived stem cell composition, containing mesenchymal stem cells
and cells specifically useful for brain regeneration. Cerecellgram is being developed for the treatment of acute
ischemic stroke. In a Phase 3 randomized clinical trial. (Link)
Focus Care Pharmaceuticals - Looking for partners for its line of rapid oral rehydration salts. (Link)
Genervon Biopharmaceuticals - Recruiting participants for a Phase 2 double blinded, randomized, placebo
controlled dose escalation study to evaluate the efficacy and the safety of GM602 in patients with acute middle
cerebral artery ischemic stroke within an 18-hour treatment window. GM602 is an endogenous six amino acid
neuropeptide with trophic (regeneration) and tropic (guidance) effects on the nervous system. (Link)
Maruishi Pharma - MR04A3 is a novel isoindoline hypnotic, binds to the central benzodiazepine (BZD) binding site
of the GABA A receptor (gamma-aminobutyric acid) and acts as an agonist. It is a sedative and analgesic which has
antinociceptive effect. MR04A3 is being developed as an intravenous formulation to induce anesthesia during
surgery and is in a Phase 1b trial.
NoNO - seeking a partner with the capability to develop and commercialise NA-1 (a PSD95 inhibitor) for acute
ischemic stroke (AIS).
Novabay Pharmaceuticals - Aganocide compounds (broad spectrum antimicrobial activity) for a variety of topical
applications like treatment of acne, decolonization of MRSA from the nares, and catheter-associated urinary tract
infections. NovaBay Pharmaceuticals and Galderma SA tweaked a March development deal for skin condition
dErugs, defining terms and fees and providing for an additional tolerance study. (Link)
UPDATE Optimer - Promising Phase 3 data for Prulifloxacin in infectious diarrhea. Favorable comparison versus
ViroPharma’s vancomycin. Open to a partnership transaction for this drug following the recent approval of Dificid
(fidaxomicin) by the FDA.
UPDATE $ Pacira Pharmaceuticals - Looking to partner Exprarel, a long acting bipuvicaine, outside of the U.S. This
product was approved by the FDA on Oct 31, 2011. Interested parties should contact Darren Pincus at
[email protected].
PharmaSurgics - In Phase 2 development for a pharmaceutical for anti-adhesion treatment after surgery. Very
promising approach. (Link)
Polymedix - Finished Phase 1 with a Factor Xa antitode that reverse effect of heparin and associated compounds.
Proof of concept achieved. (Link) Also, see Portola for a related Factor Xa antitode. (Link)
ProFibrix - Developing a surgical tissue sealant that stops acute and severe bleeding. FibroCaps are a novel
powdered mixture of fibrinogen and thrombin. Phase 2 clinical testing is currently ongoing in the Netherlands
where Fibrocaps is used for the treatment of the mild to moderate bleeding during liver surgery. ProFibrix expects
to finalize the study in the beginning of 2010.
NEW $ Salix - looking for a European partner for RELISTOR, a subcutaneous treatment for opioid-induced
constipation. This was recently partnered in Asia to Link Healthcare.
$ Undisclosed - Company with a portfolio of marketed hospital injectibles has been in an M&A sellside process.
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NEW Undisclosed player - looking to partner a recombinant formulation of human serum albumin. Broad
applications and advantages over blood derived product. For further details contact
[email protected].
$ Undisclosed - marketed hospital product available on a co-promotion basis for the prevention of acute allergy.
$ Undisclosed player - approved hospital anesthetic in the United States with differentiation from existing
products. Also could be used in physician office setting where sedation required. Open to a product sale or other
value creating arrangement. Global rights available.
Undisclosed - Phase 2 hospital product available for licensure for the treatment of acute allergy in the ER setting.
$ Undisclosed - antifungal with revenues > $10mm. Hospital setting is where generally used.
$ Undisclosed - pharma company is open to divesting a marketed specialty cardiology product with revenues >
$30mm per annum.
$ Undisclosed - Several products for oncology supportive care in the hospital setting.
NEW Ventria - has developed a recombinant lactoferrin for the prophylaxis and treatment of infection in
prematurely born children. Positive POC dataset for this Phase 2 product.
Zurex - Zuragen. Prevention of catheter related bloodstream infections. Phase 3 with FDA discussions ongoing.
IMMUNOLOGY / INFLAMMATION / AUTOIMMUNE DISEASE
4SC - 4SC-101 in Phase 2b trials for RA. This oral IL-17 inhibitor will see data in 2011. Multiple indications possible.
AB Science - In Phase 2 studies for Masitinib in in RA. Promising efficacy data in a single arm study but some
challenging side effects. (Link)
NEW Ablynx - reported on November 5, 2011 that it regained rights ATN-103 a Phase 2 nanobody treatment for
rheumatoid arthritis based on TNF-alpha modulation. Ablynx may develop this program itself or partner out. (Link)
Active Biotech - Licensing ABR-215757, for the treatment of systemic lupus erythematosus. In Phase 2.
Allostera - Developing oral peptides that block signals replacing certain antibodies. Preparing Phase 1 studies of an
allosteramer which targets IL-23R for the treatment of inflammation and psoriasis. Similar mechanism as
Ustekinumab.
Anaphore - ATX3105, blocks the receptor complex engaged by interleukin-23 (IL-23), an immunoregulatory protein
that has become a key target in strategies to develop better therapies for autoimmune disorders. This preclinical
drug candidate is being prepared for an IND. (Link)
Angelini - Bindarit inhibits mcp-1/CCL2. A Phase 2 clinical pilot study in lupus nephritis (LN) patients demonstrated
that subject treated with bindarit showed a significant reduction of urinary albumin excretion (UAE) and urinary
MCP-1/CCL2 levels. Promising data in diabetic nephropathy. Going into Phase 3 studies. (Link)
Anthera Pharmaceuticals— In Phase 2b for A623, an anti-BLyS compound for treating lupus. Has passed POC stage
but company is now dealing with a technical problem with some of its vials used in the study. (Link)
Aquinox Pharma - AQX-1125 modulates SHIP which controls PI3K for the treatment of cancer and inflammatory
disease. Late preclinical.
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Ardea Biosciences - RDEA806 oral for HIV and gout with good Phase 1b efficacy data. RDEA806 has successfully
completed Phase 1 and Phase 2a studies and has been evaluated in over 250 subjects. RDEA594 is the metabolite
that reduces uric acid.
H UPDATE Avila - In mid-Phase 1 for a BTK inhibitor that is highly selective and orally dosed. Btk plays a critical role
in the development and activation of B cells, and its inhibition will be of therapeutic significance in the treatment
of both of B cell-related hematological cancers (e.g. non-Hodgkin lymphoma (NHL) and B cell chronic lymphocytic
leukemia (B-CLL), and autoimmune diseases (e.g. rheumatoid arthritis). High interest in this company, confirmed
by the recent partnering deal between J&J and Pharmacyclics. Reported some Phase 1 data at ASH on Dec 13,
2011. Among the CLL patients in the highest dose arm completed in Phase 1 studies (4 of the 6 subjects), there
changes in circulating lymphocyte levels were noted that are consistent with the clinical response seen by the
Pharmacyclics product. (Link)
Biocon - Would consider a partnership for its anti-CD6 humanized antibody, T1h. Has completed a Phase 1 study in
RA and has two Phase 2 studies underway.
Biotest - looking to partner BT-061 once it has Phase IIb proof of concept (POC) data in the lead rheumatoid
arthritis. A 300 patient Phase 2b trial is slated to start shortly. Biotest is interested in retaining EU rights.
Biotie - Biotie initiated two clinical studies in rheumatoid arthritis and psoriasis patients with its fully human VAP-1
monoclonal antibody. Phase 1b studies were supportive of efficacy showing improvements in DAS28 versus
placebo. (Link)
Catalyst Biosciences - developing improved proteases for hemophelia and inflammation. A Factor VII program has
been partnered to Wyeth but a Factor IX program which is in a preclinical stage is company owned at present.
Cellzome - Has several PI3K kinase modulators in development to address RA and asthma. PI3K? is an emerging
target for inflammatory diseases. PI3K? is a dual lipid-protein kinase, which represents one of four isoforms of class
I phosphatidylinositol-3-Kinases. Company in active partnership discussions.
Chelsea Therapeutics - In talks to partner antifolate drug CH-4051 for RA.
H Forward Pharma - Positive Phase 2 data on FP-187 for psoriasis. Would consider a sale. (Link)
Genzyme - Looking to outlicense Genz-29155, a novel, small molecule, orally bioavailable, 1x daily novel inhibitor
of TNF-? signaling. Proof of concept has been demonstrated in multiple models of transplantation rejection,
multiple sclerosis (MS), sepsis, inflammatory bowel disease (IBD) and lupus. (Link)
H NEW ImmuPharma In October 2011, ImmuPharma regained worldwide rights to Lupuzor™ for lupus due to the
merger of Cephalon with Teva Pharmaceutical Industries. ImmuPharma intends to advance discussions with
pharmaceutical companies for the licensing of Lupuzor™. Lupuzor™ has completed successfully phase I, phase IIa
and phase IIb and has recently been granted approval by the FDA to commence phase 3 and has also been
designated "Fast Track". (Link)
Isotechnika - Voclosporin, an oral Calcineurin inhibitor, for transplant and psoriasis has achieved good POC.
Recently did a China deal.(Link)
NEW Italfarmaco - Givinostat is a Phase 2 HDAC inhibitor for inflammatory diseases and cancer. Has been in Phase 2
for juvenile arthritis, myeloproliferative diseases, Hodgkin’s disease and multiple myeloma.
Kiadis - ATIR is a personalized cell based therapy of donor T-lymphocytes depleted of alloreactive T-cells and is
infused after a patient receives a mismatched bone marrow transplantation to reduce Transplant Related
Mortality. Has shown effectiveness in a recent Phase 2a study.
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Lexicon Pharmaceuticals - In Phase 2 studies in RA with a LX-2931, a S1P lyase modulator. Strong pre-clinical data
package and a desire to partner this compound in 2010.
Micromet - Willing to outlicense a preclinical IL-2 inhibitor (MT-204).
Morphosys - MOR103 is a fully human HuCAL antibody against GM-CSF that is in Phase 1 studies. Also see a similar
antibody in development at Kalobios. (Link)
Neovacs - TNFalpha Kinoid in Phase 2a study in RA. TNF-alpha Kinoid is an immunotherapy designed to elicit
natural polyclonal antibody responses. Positive data were reported out.
Novimmune - In Phase 2 with an anti-CD3 antibody for Crohn’s disease. Exploring potential as an
immunomodulator in Type 1 diabetes and transplant rejection as well.
Nuon Therapeutics - Developing tranilast for gout and RA. In discussions with potential partners. (Link)
Opsona Therapeutics - In preclinical development of an anti-TLR Mab for treatment of inflammatory diseases such
as RA and lupus. (Link)
H NEW Phenex Pharma - in preclinical work for a ROR - gamma - t program for treatment of inflammation. High
interest in this program.
Phytomedics - Partnering PMI-001 for RA based upon a botanical. Phase 3 development plans have commenced.
Solid ACR50 scores in a Phase 2 study. JSB Partners advising.
COMPLETED H Portola - Early stage SYK inhibitor program (PRT062607) for cancer and immunology. Initiated a Phase
1 ascending dose trial in March, 2011 of PRT062607, an oral Syk-specific kinase inhibitor to treat chronic
inflammatory diseases and certain cancers, including non-Hodgkin’s lymphoma and chronic lymphocytic leukemia.
Drawing high interest after Rigel data.
Resolve Therapeutics - in preclinical studies for a biologic, RSLV-125, that targets the interferon pathway. This
molecule acts at the most proximal point in the interferon cascade. Potential to be an efficacious and safer
therapy than other products in development. (Link)
Rigel - partnering R348 is a selective, potent inhibitor of Janus Tyrosine Kinase 3 (JAK3). JAK3 is critical to immune
system activation and is an attractive target because its expression is limited to key cells in the immune system,
particularly T-cells, which mediate these diseases.
UPDATE Savient Pharmaceuticals - FDA approved KRYSTEXXA (pegloticase) in Sep 2010, a PEGylated uric acid
specific enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy.
Company is seeking a commercial buyer and is using JP Morgan and Lazard in its search for an acquisition partner.
Savient is now pursuing a launch of Krystexxa on its own and is building a 50-person plus sales force. Update: First
year sales were $2.6 million. Market Cap of $160mm. (Link)
SuppreMol - Developing soluble Fcy-Receptors (sFcyRs) for autoimmune disease. These are recombinant
autologous proteins with strong immunosuppressive potential. SM101, SuppreMol’s main product is a
recombinant, soluble, non-glycosylated version of the human Fcy receptor FyRIIb which is has completed a Phase 1
trial for ITP. (Link)
Syntrx Biosystems - As a folate analogue, aminopterin competes for the folate binding site of the enzyme
dihydrofolate reductase, thereby blocking tetrahydrofolate synthesis. Aminopterin is being developed as an oral
dosage form for the treatment of psoriasis and is in Phase 2 studies.
Taligen - Developing a Factor H (TT30) replacement, part of the complement system, for orphan diseases.
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NEW Undisclosed - highly promising early stage JAK3 program.
Veloxis - LCP-Tacro has completed Phase 2 studies with positive data in kidney transplant recipients. 505b2 with
better PK of tacrolimus. Other transplant products available.
Western States Biopharmaceuticals - Developing novel treatments for autoimmune disease. (Link)
Y’s Therapeutics - YSPSL is fused P-selectin glycoprotein ligand (PSGL) and human IgG1. It acts as an antagonist of
P-selectin. This is currently in Phase 2 studies for prevention of delayed graft function and prevention of IRI in
transplant patients.
Zalicus - Synavive® low dose glucocorticoid treatment for RA without previously noted side effects of this class. Has
shown ACR20 score of 63% in a Phase 2 trial with this oral treatment. Potentially an attractive treatment. (Link)
LATE STAGE ASSETS (PHASE 3 STARTED TO NEWLY APPROVED)
$ Access Pharmaceuticals - undertaking a commercial launch of Mugard which was approved in 2010. This product
is used for treating oral mucositis. Access is actively looking for a co-promotional partner for this product. June,
2011 received acceptance letter from Chinese SFDA citing all documentation for MuGard, indicated for treatment
of oral mucositis a side effect of anti-cancer treatments, has been submitted and accepted. (Link)
Acusphere - Imagify, pending EMA filing for this cardiac imaging agent. Highly differentiated from SPECT agents on
the market insofar as permits evaluating of myocardial perfusion, an important marker of coronary artery disease
(CAD) without radioactive markers. Potential first-to-market drug in $600 million and $2 billion addressable market
in E.U. and U.S. respectively. Company is assisted by Torreya Partners. For further details please contact
[email protected] (Link)
UPDATE Alexza - Looking to partner several inhaled products including ADASUVE (Loxapine) which recently met the
primary endpoint in a Phase 3 Bipolar Disorder trial. A Phase 3 study in schizophrenia was successful. Product was
partnered to Biovail and recently returned. Promising opportunity. Update: Dec 8, 2011: FDA advisory committee
documents somewhat negative on pulmonary safety of this product but committee voted in favor its approval.
UPDATE Amarin - Developing a pure omega-3 for reduction of triglycerides. Phase 3 data reported out very strong.
Company has indicated that is has retained a financial advisor (Lazard) to explore a sale. Update: Nov 2011 - no
sale reported and company’s valuation has dropped from over $2bn to under $1bn. Amarin recently filed an NDA
for its lead product. Amarin appears to be a likely takeover candidate for the next year. (Link)
Amsterdam Molecular Therapeutics (AMT) - Glybera for lipoprotein deficiency. This disease is serious, often
resulting in death and quite rare (one in a million). Assuming 500 patients in the Western world with the disease
and a treatment cost of $200k per annum per patient, this drug would generate annual revenues of approximately
$100 million. AMT has not yet filed this product with the FDA. Has filed an EMA registration for Glybera to treat
lipoprotein lipase deficiency. Update Update: Recently failed to get an EMA approval for this product. May need to
run an additional trial. (Link)
Anthera Pharmaceuticals— Varespladib is an inhibitor of secretory phospholipase for prevention of
arteriosclerosis. Promising data and other molecules. In an ongoing Phase 3 program. Also in Phase 2b with a
different compound for lupus. (Link)
AP Pharma - Would outlicense APF530 in Phase 3 for CINV. Positive Phase 3 data. The FDA has accepted for review
the NDA and, based on the Prescription Drug User Fee Act (PDUFA), has issued an action date of March 18, 2010.
Update: AP received a complete response letter that required additional studies on bioavailability and metabolism.
Company preparing a complete response in mid 2011. (Link)
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ARCA - Gencaro (Bucindolol) is a nonselective beta-blocker which is being developed for treatment of heart failure
on a genetically-targeted basis. Very strong data in preventing death post-MI. Approvable letter from FDA with
guidance on a further trial required for approval. Company open to alternatives. (Link)
Archimedes Pharma - Is seeking licensees for North America and Japan for PecFent, a nasally-delivered fentanyl
product for breakthrough cancer pain, currently in Phase 3 development. Archimedes Pharma is seeking licensees
outside of Europe for PecFent®. Has gained a positive opinion from the CHMP, and has been filed for regulatory
approval in the US. Preparing for a U.S. launch.
NEW $ BioAlliance Pharma - searching for a U.S. Partner for Oravig. Oravig is miconazole buccal tablets for the
treatment of oropharyngeal candidiasis (OPC), more commonly known as thrush, in adults and children age 16 and
older. This drug was returned by Strativa Pharmaceuticals recently to BioAlliance.
UPDATE BioDelivery Sciences - running a fully enrolled Phase 3 clinical trial assessing the efficacy and safety of
BEMA Buprenorphine for the treatment of moderate to severe chronic pain. In September 2011, BDSI reported
that it had missed the primary endpoint of improving mean pain intensity scores vs. placebo in the Phase 3 BUP-
301 trial to treat moderate to severe chronic low back pain. As a result, BioDelivery plans to conduct a second
Phase 3 trial for the product, which the company said will delay an NDA submission to FDA by about one year.
(Link)
Biosante - LibiGel (testosterone gel) for female sexual dysfunction is in Phase III, and is designed to be quickly
absorbed through the skin after a once-daily application.
Biovest International - Company emerged from Chapter 11 bankruptcy. Has several assets including BiovaxID
which has recently had positive Phase 3 data in NHL.
Camurus - Episil® (CAM2028) is a protective, bioadhesive intra-oral liquid for treatment of oral mucositis. The
product is registered in Europe as a Medical Device, Class I, with an expected market launch in Q2 2010. Note: This
product was licensed to IS Pharma in Europe after its recent approval. A 510k registration submission in the US is
under way.
NEW $ Canyon Pharmaceuticals - Canyon Pharmaceuticals is seeking to build a strategic alliance preferentially on a
worldwide basis to commercialize Desirudin (Iprivask® US-registration / Revasc® EU-registration), a differentiated
anticoagulant drug which is approved by the FDA, the EMA and several of the rest of world authorities. Desirudin is
a direct thrombin inhibitor and the only subcutaneous direct thrombin inhibitor (DTI) with approval for venous
thromboembolism (VTE) prophylaxis following hip- and knee-replacement surgery.
Chelsea Therapeutics - NORTHERA™ (droxidopa), is an orally active synthetic precursor of norepinephrine initially
being developed for the treatment of neurogenic orthostatic hypotension. Large potential in the Parkinson’s
market where there has been a reported 60% reductions in falls in PD patients with NOH; also could work in
fibromyalgia. On market already through Dainippon Pharma in four Asian countries. Company recently reported
that it is filing for an NDA based on studies that have already been completed and has recently completed its QTc
work. Anticipates approval by Q2 2012.
Clinuvel - In Phase 3 for European approval for Afamelanotide, a photoprotectant to be used in Erythropoietic
Protoporphyria. Data expected in Q1 2011. May 12, 2011 Clinuvel Pharmaceuticals Limited., announced that it
held a constructive Pre-Submission Meeting with the European Medicines Agency (EMA) on May 5. After reviewing
the proposed content of the registration dossier for afamelanotide (Scenesse), the EMA acknowledged that
Clinuvel would meet all filing requirements.
Collegium Pharma - NDA pending COL-003, a tamper resistant, abuse-deterrent, sustained release oxycodone
formulation. Seeking a partnership.
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Corcept Therapeutics - As an orphan designation for Corlux, in development for Cushing’s Syndrome - a condition
of hypercortisolism with approx. 20K persons in US. Positive Phase 3 data released in December 2010. In June,
2011 FDA accepted NDA submission from Corcept for Corlux. Anticipated completion of review set for Feb. 17,
2012.
Durect - Transdur Sufentanil for the treatment of pain. Longer duration than fentanyl patches and smaller patch
size. On May 5, 2011 the company wrote: “We continue discussions with potential partners regarding licensing
development and commercialization rights to this program to which we hold worldwide rights.”
Elite Pharmaceuticals - Phase 3 abuse resistant oxycodone for OA under an SPA. Company exploring strategic
options.
H UPDATE Exelixis - According to Bloomberg on April 12, 2011: “Exelixis Inc. is working with Goldman Sachs Group
Inc. to prepare for potential takeover offers after its experimental drug helped prostate-cancer patients in a
study.” Company’s XL-184 has reported dramatic data on reducing metastatic prostate lesions at ASCO. Update:
company has hired a Chief Commercialization Officer with intention to introduce cabozantinib to the U.S. market
and has reported strong data for cabozanitinib in the treatment of medullary thyroid cancer.
NEW Endoceutics - developing Femivia for the treatment of issues associated with hormone deficiency in post-
menopausal women (e.g., memory loss, diabetes, muscle loss). This product is in Phase 3. (Link)
Fibrocell Sciences - Emerged from Chapter 11 bankruptcy proceedings on Oct 6, 2009 (formerly known as
Isolagen). Company received a complete response letter from FDA requesting additional histopathological data
from the trial of azficel-T for the treatment of wrinkles. Azficel-T is an autologous cell therapy for the treatment of
moderate to severe nasolabial fold wrinkles in adults.
Foresight Biotherapeutics - Has recently completed a Phase 3 trial of FST-201 (dexamethasone 0.1% ) Otic
Suspension vs. the FDA-approved drug Ciprodex (ciprofloxacin 0.3%, dexamethasone 0.1%) Otic Suspension (Alcon
Laboratories, Inc.) in the treatment of acute otitis externa (Swimmer’s ear). (Link)
Hyperion Therapeutics - Developing GT4P, an ammonium remover, for urea cyclic disorders and hepatic
encephalopathy - Positive Phase 3 data and a high likelihood of approval in 2011/12 timeframe. Would consider a
partnership or sale transaction. (Link)
$ Ibsa - Tirosint (L-Thyroxin) in soft gel capsules for treatment of hypothyroidism. Approved in the U.S. but not yet
marketed. Company searching for a marketing partner. Ibsa - looking to outlicense betametasone valerate patch
for treatment of psoriasis. A wide range of markets (EU, USA, South America, Middle and Far East) are still
available on exclusive or semi-exclusive basis. Possibilities for a global agreement. (Link)
UPDATE $ Intelgenx - CPI-300 is a novel, high strength of Bupropion HCl, the active ingredient in Wellbutrin XL(R).
Indicated for depression. This product was approved in November 2011 and Intelgenx is in active licensing
discussions to find a commercial partner.
Intermune - Bloomberg reports on April 27: “InterMune known for its drug to treat lung scarring, hired Goldman
Sachs (GS.N) to help it weigh a possible sale... Goldman has been conducting an auction of InterMune for more
than a month and some potential bidders have been spooked by the biotechnology company's expectations for a
sale price, Bloomberg news reported.” Company commercializing Pirfenidone in Europe. Company market cap over
$2bn.
Keryx - In Phase 3 with Zerenex, a ferric citrate for treatment of hyperphosphatemia in ESRD. Expects to file an
NDA assuming positive data by mid-2012.
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Mannkind - Looking to partner Afresa, an inhalable insulin, that has an upcoming PDUFA date. Rumored to be in an
active M&A mode. Company received an approvable letter on Mar 15, 2010. The FDA asked for more information
on data designed to support the clinical utility of Afrezza, as well as information about how comparable the
commercial version of the product is to the version used in clinical trials.
MAP Pharma - Would look at partnership for its late stage inhaled migraine drug, Levadex. In Jan 2010, announced
that the U.S. Food and Drug Administration (FDA) has informed the Company that a second pivotal efficacy study is
not required for this drug. (Link)
NEW Mayne Pharma - Lozanoc™ (SUBA®-itraconazole) is an improved patent protected formulation of itraconazole
to treat fungal infections. The bioavailability of SUBA®-itraconazole is twice that of the orginator product
(Sporanox®) and shows reduced intra- and inter-subject variation. A Marketing Authorisation Application (MAA) in
the EU has been submitted (November 2010) and discussions with the FDA are underway regarding further
requirements for 505(b)2 filing and US registration. Interested parties should contact
[email protected].
Moberg Derma - K301 for seborrheic dermatitis has shown benefit in two Phase 3 clinical trials.
Molecular Insight Pharmaceuticals - Azedra in Phase 2 for the treatment of metastatic neuroendocrine tumors
such as pheochromocytoma, carcinoid and neuroblastoma that are not amenable to treatment with surgery or
conventional chemotherapy. Very positive recent data that highlight potential for rapid approval. SPA in place with
FDA. Company has emerged well capitalized from a bankruptcy process with a new board of directors.
$ Neurogesx - Qutenza is a patch that delivers synthetic capsaicin for PHN on the market in U.S.. Recently
approved. Partnered in the EU to Astellas in June 2009. Company looking for partnerships in Asia and Latin
America. (Link)
UPDATE NPS Pharma - developing GATTEX for the treatment of short bowel syndrome. Well advanced in clinical
development. Recently saw some deaths of subjects on study. NPS has indicated interest in commercializing this
product on its own.
UPDATE NuPathe - Recently went public with Zelrix, a promising late stage candidate for migraine (iontophoretic
sumatriptan patch). NDA filing in Oct 2010 with PDUFA date of August 29, 2011. FDA issued a complete response
letter requesting additional CMC and early stage clinical work. Company is open to strategic discussions.
UPDATE Optimer - Promising Phase 3 data for Prulifloxacin in infectious diarrhea. Favorable comparison versus
ViroPharma’s vancomycin. Open to a partnership transaction for this drug following the recent approval of Dificid
(fidaxomicin) by the FDA.
OPKO Health - Will report Phase 3 data later this year for Bevasiranib, a siRNA drug designed to silence VEGF for
treatment of AMD.
OxiGene - ZYBRESTAT is currently being evaluated in a pivotal registration study in anaplastic thyroid cancer (ATC)
under a Special Protocol Assessment agreement with the U.S. Food and Drug Administration (FDA). In active
partnership dialogue. Update: Oxigene merger with Vaxgen was declined by Vaxgen Shareholders on Feb 3, 2010.
UPDATE $ Pacira Pharmaceuticals - Looking to partner Exprarel, a long acting bipuvicaine, outside of the U.S. This
product was approved by the FDA on Oct 31, 2011. Interested parties should contact Darren Pincus at
[email protected].
UPDATE Pharmaxis - Preparing to launch Bronchitol® for the treatment of patients with cystic fibrosis in Q1 2011.
Update: company has had regulatory setback in EMA and subsequent reversal. On track now. Bronchitol launched
in Australia.
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Piedmont Pharmaceuticals - Have a late stage product for treatment of head lice. Partnered and marketed in
Europe. RESULTZ is sold internationally through licensing partners. Looking for other partners.
UPDATE Protein Sciences - Has FluBlok for treatment of influenza. Pending BLA. Agreement of licensing signed by
TM and UMN for Asian rights. Update: thinking of an IPO following approval by FDA of FluBlok in 2012. (Link)
UPDATE Raptor Pharmaceutical - likely to be able to file for NDA of DR Cysteamine, which is delayed-release,
enteric-coated microbead formulation of cysteamine bitartrate for the treatment of cystinosis. In June 2011,
Raptor reported positive data from a pivotal, Phase 3 clinical trial, examining the safety and tolerability of every
12-hour DR Cysteamine compared to immediate-release cysteamine bitartrate (the current standard of care) in
nephropathic cystinosis patients. Company intends to self-commercialize this product. (Link)
NEW Rempex - planning to file an NDA in 2012 for an undisclosed antibiotic. (Link)
SciClone - ZADAXIN in Phase 3 for the treatment of hepatitis C.
Savient Pharmaceuticals - FDA approved KRYSTEXXA (pegloticase) in Sep 2010, a PEGylated uric acid specific
enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy. Company
is seeking a commercial buyer and is using JP Morgan and Lazard in its search for an acquisition partner. Update:
Savient is now pursuing a launch of Krystexxa on its own and is building a 50-person plus sales force. (Link)
Stallergenes - Stalair® line of allergy products including ones aimed at rhinitis, asthma and mites. Late stage
opportunity for a large market. Both specialty and potential primary care call points. Company recently indicated
that it intends to pursue U.S. commercialization on its own. Currently looking for a China partner. (Link)
H UPDATE Syndax - Entinostat (SNDX-275), an oral class I- selective HDAC inhibitor with a long half life, for cancers.
In registration for breast cancer 2
nd
line treatment. Company would be most likely to consider either a global
partnership deal, an IPO or a change of control transaction. (Link)
Thrombogenics - completed Phase 3 studies for Microplasmin in Phase 3 clinical development for the non-surgical
treatment of back of the eye diseases. Good evidence of efficacy with two positive Phase 3 trials reported. Would
consider a sale. (Link)
UPDATE Titan Pharmaceuticals - Probuphine is a novel, subcutaneous implant formulation of buprenorphine
designed to deliver six months of medication following a single treatment. This product has demonstrated strong
positive results in a controlled Phase 3 study for the treatment of opioid addiction and an NDA filing is in
preparation. (Link)
Topaz Pharma - completed two Phase 3 studies for the treatment of head lice with Ivermectin. Planning to submit
NDA to FDA soon. (Link)
Toyama - T-705, a viral RNA polymerase inhibitor, is in Phase 3 studies in Japan and Phase 2 in the United States as
a highly promising anti-viral drug for the treatment of influenza. (Link)
Undisclosed - neurology oriented marketer looking for a primary care marketer to co-promote a rapidly growing
and recently approved product for migraine attacks.
UPDATE Vivus - Would be interested in partnering avanafil, a PDE V inhibitor in development for erectile
dysfunction. Recently reported out second Phase 3 program also with strong data. Vivus submitted an NDA for
this drug on June 30, 2011 and has a PDUFA date in the U.S. of April 29, 2012. Cowen predicts this drug will take
8% market share with $800mm in U.S. revenue. (Link)
UPDATE Vivus - Looking to partner Qnexa in Europe and other ROW territories. An approval is possible in the EMA
with long-term market exclusivity in mid-2012 (or sooner).
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Winston Pharmaceuticals - Rheumaderm cream for the treatment of OA. NDA pending. Marketed in Canada by
Sanofi. Based on civamide which acts on type-C neurons by specific binding to a membrane receptor, the TRPV-1
receptor. Looking for licensing partners in other parts of the world. (Link)
Winston Pharmaceuticals - Also pursuing Civamide, a TRPV-1 modulator, for the treatment of episodic cluster
headaches. Late stage with approval pending in Canada. (Link)
Zogenix - Sumavel, a needlefree transdermal delivery of sumatriptan for migraine. Recent FDA approval with
product launch using Astellas as a co-promotion partner. Company could consider an M&A transaction. Note:
Raised $30mm from Cowen Healthcare Royalty Partners in July 2011.
Zurex - Zuragen is for prevention of catheter-related bloodstream infections. Hospital application available.
MATURE BRANDED PRODUCTS
$ Medicure - May sell U.S. rights to Aggrastat, originally a Merck drug, for acute cardiac indications. Update:
company restructured its liabilities on July 18, 2011 in order to reduce debt. Beal Advisors will be working with the
company to find a partner or buyer for Aggrastat going forward. (Link)
$ Microbix - Owns Urokinase, formerly of Abbott (Abbokinase). Did revenues of $10mm+ recently. Not formally for
sale but purchased for a low price due to manufacturing issues which have largely been fixed. Signed a marketing
agreement with Kinlytic. (Link)
COMPLETED $ Undisclosed - marketed bronchodilator with revenues > $20mm. Company open to a product sale.
Note: Graceway Pharma was sold to Medicis in Nov 2011, inclauding its Maxair auto-inhaler.
$ Undisclosed player - process for sale of company with a marketed but not promoted cardiovascular product with
2011 revenues around $9 million.
NEW $ Undisclosed - large pharma disposing of a marketed antibiotic with global rights. This product is off patent.
NEW $ Undisclosed - large pharma disposing of a marketed oncology drug with revenues of around $5mm.
Significant barriers to entry.
$ Undisclosed player - sale of company with over $10mm in revenue with largely genericized specialty products in
CNS and renal disease.
NEW $ Undisclosed - two marketed antibiotics in U.S.. One is facing generic competition. Revenues total around
$10mm.
$ Undisclosed player - selling a group of marketed hormone products with the assistance of Torreya Partners.
Interested parties should contact Tom Bird ([email protected]).
$ Undisclosed player - selling off $20mm revenue+ commercial product for narrow market with pediatric
applications.
NEW $ Undisclosed player - mature oncology product for treatment of breast cancer with revenues of
approximately $8mm. Product is highly profitable as it is not promoted.
NEW $ Undisclosed - Pharma disposing of large mature product. Off patent with revenues over $80mm.
$ Undisclosed - pharma company is open to divesting a marketed specialty cardiology product with revenues >
$30mm per annum.
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NEW $ Undisclosed player - selling off portfolio of $50mm area specialty pharma products included. Process well
advanced via Torreya Partners. Interested parties should contacted Benj Garrett
([email protected]).
COMPLETED $ Undisclosed party - looking to sell a portfolio of marketed prenatal vitamins with assistance from
Torreya Partners. Interested parties should contact Tom Bird ([email protected]).
NEONATOLOGY
NEW BCO Pharma - Phase 3 ready program for the treatment of hypotension in neonates. Novel presentation of
existing hypotensive agent.
Discovery Labs - is reportedly engaged in partnership discussions with respect to licensing its neonatal franchise,
which includes Surfaxin, Sufaxin LS and Aerosurf. Want a partner to collaborate on clinical development for
Surfaxin LS and Aerosurf, and to play a central role in commercialization for all three products.Surfaxin is a
synthetic KL4 Surfactant for treatment of neonatal RDS. Company expects to refile for approval in Q1 2011. Has
granted development and marketing rights for Discovery Labs’ SRT products to Esteve in the key southern Europe
markets of Spain, Italy, Portugal, Greece, and Andorra.
UPDATE Infacare - Developing Stannsoporfin (Stanate) for Neonatal Hyperbilirubinemia, which is in a Phase 2b trial.
Company expects to report out data soon. (Link)
NEW Premacure - Lack of IGF-1 in premature babies can lead to severe complications. A Phase I clinical trial for of
IGF-I/IGFBP-3 (Premiplex®) found that levels of IGF-I were increased to within physiological levels and that
administration to preterm infants is safe and well tolerated. A Phase II, safety and efficacy multi-center clinical trial
has started in Sweden and is currently recruiting patients.
Symphogen - Rozrolimupab (Sym001) is a recombinant polyclonal composition of 25 different Rhesus D specific
antibodies for the treatment of primary Immune Thrombocytopenia and for Anti-RhD prophylaxis (ADP) in
prevention of Hemolytic Disease of the Newborn. The ongoing phase 2 clinical trial evaluates the safety and
efficacy, and explores the dose range of Sym001 in immune thrombocytopenia patients. This product was returned
to Symphogen from Biovitrum Swedish Orphan for strategic reasons on Dec 30, 2010. (Link)
NEW Undisclosed - company developing special type of milk for the treatment of babies born prematurely. On the
market.
NEW Ventria - has developed a recombinant lactoferrin for the prophylaxis and treatment of infection in
prematurely born children. Positive POC dataset for this product.
NUTRITIONALS AND NATURAL PRODUCTS
$ Accera - Axona, a medical food for treatment of Alzheimers. Related medical food treatments for Alzheimers in
development by Adeona, Bellus Health and Groupe Danone. Update: Oct 5, 2010: New CEO appointed. Company
remains open to a partnering or change of control transaction.
$ Bellus Health - Would consider partnering US rights to Vivimind for Alzheimers. Active partnering discussions
ongoing in Europe. In April, 2011 announced that it has signed an exclusive license and distribution agreement
with Agahan Ayandeye Pars Inc. for this product in the MidEast.
$ Enzymatic Therapy - A natural product distributor sells 250+ natural products. Considering a sale. (Link)
KV Pharma - Licensing rights to prenatal vitams, PrimaCare, and hematinics, Repliva, via Robert W. Baird. (Link)
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NEW Rottapharm - for sale according to the Wall Street Journal. Company has two Phase 3 drugs in development
and a strong group of branded products in the market. Revenues over $850 million. Sale price could be over $2.5
billion.
COMPLETED $ Undisclosed party - looking to sell a portfolio of marketed prenatal vitamins with assistance from
Torreya Partners. Interested parties should contact Tom Bird ([email protected]).
NEW Wockhardt - rumored to be in a process to sell its substantial nutrition business as part of a process to pay
down external debt.
OBESITY
7TM - TM38837 is a CB1 which has been shown to be inactive in the CNS. This suggests a promising approach given
the history with endocannabinoids. It will be important next to show that such a compound is efficacious. (Link)
BHV Pharma - BHV091009 is a highly selective sodium glucose co-transporter 2 inhibitor ("SGLT2i") being
developed for the treatment of diabetes and obesity. Two supportive Phase 2b studies. Also pursuing an obesity
indication. (Link)
Fasgen - Obesity and metabolic disease modification programs have progressed and company is looking for a
partnership. Strong IP and set of compounds in the fatty acid synthase area including GPAT and CPT-1 inhibitors.
Preclinical program entering the lead generation phase. (Link)
Lithera - Announced positive results from a Phase IIb clinical study of LIPO-102, its novel injectable combination of
salmeterol xinafoate (SX) and fluticasone propionate (FP) for selective, non-ablative fat reduction. In a Phase 2b
study LIPO-102 was well-tolerated when administered weekly for 8 weeks into the subcutaneous abdominal fat of
healthy subjects and produced dose- and time-related reductions in mean abdominal volume and circumference.
Neurosearch - Looking to partner obesity program (Tesofensine) going into Phase 3. July, 2011 signed agreement
with Biolin Scientific over sale of company’s 30.1% shareholding in Sophion BioScience and all other owners will
sell in Sophion will see ownership share.
University of Strathclyde - Galegine derivatives as anti-obesity compounds. Similar to metformin and likely work
through AMP kinase activation. (Link).
UPDATE Vivus - Looking to partner Qnexa in Europe and other ROW territories. An approval is possible in the EMA
with long-term market exclusivity in mid-2012 (or sooner).
Yonsei University - Peptides which reduce obesity. Preclinical. (link)
H Zafgen - in Phase 1 studies of a novel treatment for obesity in bariatric patients. This drug potentially
circumvents many of the problems that have arisen with recent late stage candidates in obesity, including pursuing
a sicker patient population with a product that can’t be widely used. July, 2011 signed a $33 million series C
financing with Atlas Venture and Third Rock Ventures. (Link)
ONCOLOGY - LIQUID TUMORS
4SC - developing resminostat for Hodgkin’s Lymphoma. Phase 2a data showed a few partial responders and some
cases of stable disease. (Link)
Acetylon Pharma - Taking ACY-1215 a next-generation Class II-selective histone deacetylase (HDAC) inhibitor - into
Phase I/II clinical testing for patients with relapsed and relapsed-refractory multiple myeloma. No POC data yet.
(Link)
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Affimed - Taking AFM13, with a novel tetravalent bispecific antibody structure, for the treatment of Hodgkin's
disease (HD) into Phase I. No approved treatments on market. Partnership discussions underway.
UPDATE Allos Therapeutics - Oncology marketer with Folotyn® for liquid tumors. After a recently failed merger
attempt with AMAG, Allos is rumored to be continuing to explore strategic alternatives with the assistance of JP
Morgan. (Link)
Antisoma - Partnering AS1413, formerly Xanafide, is a DNA intercalator in phase 3 development in secondary AML
outside the U.S. Expect to report out data in Q1 2011. Would consider an M&A deal.
H Ariad - AP24534, multitargeted kinase inhibitor in Phase 1 for hematological cancer. Appears to address a form
of CML caused by a mutant T315I which is resistant to currently available drugs. M&A not likely.
H UPDATE Avila - In mid-Phase 1 for a BTK inhibitor that is highly selective and orally dosed. Btk plays a critical role
in the development and activation of B cells, and its inhibition will be of therapeutic significance in the treatment
of both of B cell-related hematological cancers (e.g. non-Hodgkin lymphoma (NHL) and B cell chronic lymphocytic
leukemia (B-CLL), and autoimmune diseases (e.g. rheumatoid arthritis). High interest in this company, confirmed
by the recent partnering deal between J&J and Pharmacyclics. Reported some Phase 1 data at ASH on Dec 13,
2011. Among the CLL patients in the highest dose arm completed in Phase 1 studies (4 of the 6 subjects), there
changes in circulating lymphocyte levels were noted that are consistent with the clinical response seen by the
Pharmacyclics product. (Link)
Benitec Limited - starting a small Phase I/II study using Benitec's DNA-directed RNA interference (ddRNAi)
technology in lymphoma patients carrying the HIV virus.
Biocryst - Phase 2 open label study with Forodesine for CLL showed 6 of 23 PRs. In May 2011 presented positive
data from its two completed, randomized, double-blind, placebo-controlled Phase 2 studies of BCX4208in patients
with gout at the Annual European Congress of Rheumatology. Partnered with Mundipharma for Europe, Asia,
Australia and certain neighboring countries.
Biogen Idec - Looking to outlicense Galiximab, an anti-CD80 antibody, which has shown activity in B-cell
lymphomas. Has gone through Phase 3 trials. Biogen looking to outlicense after a recent strategic review. (Link)
BioInvent - In Phase 2 studies in multiple myeloma with an anti-CD54 antibody. Encouraging Phase 1 data.
H Celator Pharmaceuticals - Strong Phase 2 data for CPX-351 for AML shown at ASH in Dec 2010. In June 2011
announced positive results in elderly patients diagnosed with secondary acute myeloid leukemia treated with CPX-
351. Observations included high-risk patients with secondary AML showed greater improvements with CPX-351.
(Link)
Celleron Therapeutics - Developing CXD101, an HDAC inhibitor from AZ with a novel biomarker strategy. (Link)
Chroma Therapeutics - Tesedostat has completed a Phase 2a study in AML. 10 of 38 patients with a poor prognosis
achieved either a partial or a complete response to the drug. August, 2010 announced that the Company had
advanced its novel macrophage-targeted HDAC inhibitor, CHR-5154, into preclinical development and received
undisclosed milestone payment from partner GlaxoSmithKline.(Link)
Clavis Pharma - Elycatarabine 9/61 CRs in a Phase 2 trial in late stage AML. Large clinical trials undertaken. Recently
outlicensed second program for pancreatic cancer to Clovis Oncology. In June, 2011 announces that Clovis
Oncology, Inc, its development partner for CP-4126, has expanded the ongoing observational study to determine
the patient stratification parameters in the pivotal LEAP clinical trial with CP-4126. (Link)
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NEW Cytrx - Looking to partner Bafetinif, a dual Bcr-Abl and Lyn-kinase inhibitor for CML where a mutation exists
which creates resistance to imatinib and dasatinib. Has completed a Phase 1b study. A Phase 2 study in relapsed
CLL is underway.
Deciphera - developing switch inhibitors which control kinase shape. DCC-2036 in Phase 1 for CML. (Link)
Epicept - Ceplene approved for AML in Europe. Active partnership process underway. Jan 11: EpiCept announces
commercial licensing agreement for Ceplene(R) with Meda in Europe and Pacific Rim. Receives $3mm upfront and
royalties and milestones. U.S. rights available but company received a refusal to file on Aug 23, 2010 from FDA. In
June, 2011 Ceplene received written response from FDA for application of Special Protocol Assessment of Ceplene.
FDA invited company to request meeting to discuss response to application. (Link)
NEW Erytech - Graspa® is an innovative formulation of asparaginase. Graspa® demonstrated positive results in
Phase 2 trials in acute lymphoblastic leukaemia (ALL) and excellent safety profile compared to control treatment.
Graspa® is currently in Phase II/III clinical trial in relapsed and ALL patients in Europe.
NEW Il-Yang Pharmaceuticals - looking to partner radotinib, a novel treatment for leukemia based on bcr-ABL
modulation. In Phase 3 studies in Korea. (Link)
H Intellikine - Very promising dual Pi3k / m-tor compounds, entering Phase 1 clinical trials. Company in active
partnership process across all compounds. A partnership is in place witih Infinity Pharma. Intellikine remains an
exciting player in its space and is open to a change of control transaction.
NEW Italfarmaco - Givinostat is a Phase 2 HDAC inhibitor for inflammatory diseases and cancer. Has been in Phase 2
for juvenile arthritis, myeloproliferative diseases, Hodgkin’s disease and multiple myeloma.
NEW KAEL-GemVax - Phase 3 results anticipated for GV1001 in pancreatic cancer in May 2012. GV1001 is a
therapeutic peptide cancer vaccine targeting telomerase. The vaccine has been tested in clinical trials for
pancreatic cancer, lung cancer, liver cancer, melamona and chronic lymphomytic leukemia (CLL).
Lorus - looking to partner antisense approach to AML. Promising Phase 1b data.
Nereus Pharmaceuticals - Developing marizomib (NPI-0052), is a second-generation inhibitor of the 20S
proteasome. Patient data coming up and a potential strategic transaction under consideration. Company also has a
Phase 2 vascular disrupting agent with positive interim data in NSCLC. In June 2010 this company received
additional funding from its investors. (Link)
OncoNova - Estybon inhibits the PI-3 Kinase, ERK (growth) and AKT (pro-survival) pathways. Entering a Phase 2
study for the control of MDS. July 2011, announced license agreement with SymBio Pharmaceuticals to collaborate
and commercialize Rigosertib in Japan and Korea. Conducting late-stage clinical trials in the US, Europe and India
for treatment of MDS and solid tumors. (Link)
NEW Onyx - rumored to be exploring strategic alternatives. Substantial value potential tied to a recently filed NDA
for carfilzomib, a protease inhibitor, for the treatment of liquid tumors including multiple myeloma. Bloomberg
reported on Nov 30, 2011 that Onyx is working with Centerview as a financial advisor. On Dec 12, FDA indicated
that an accelated review would not take place.
S*Bio - going into Phase 2 with novel HDAC inhibitor. In Dec 2010 reported positive safety and tolerability results
from Phase 1.
S*Bio - In June, 2011 announced results from multiple Phase I/II clinical studies of JAK2 inhibitor SB1518 which
confirmed safety and efficacy. S*Bio has received the rights back to this program from Onyx Pharma. S*Bio is open
to a change of control transaction.
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Stemline Therapeutics - A cancer stem cell targeting agent, SL-401, saw Phase 2 data at ASH which demonstrated
efficacy, including two durable complete remissions (CRs), multiple blast reductions and disease stabilizations in
relapsed/refractory AML, poor risk elderly AML, and high risk MDS. (Link)
Sunesis - Vosaroxin in Phase 2 for AML. Data reported in Nov 2010 indicated meaningful response rates and
prolonged survival times. Entering a Phase 3 study known as VALOR. Compound intercalates DNA and inhibits
topoisomerase II, resulting in replication-dependent, site-selective DNA damage. Company would consider
partnership and, potentially, a sale. (Link)
H UPDATE Syndax - Entinostat (SNDX-275), an oral class I- selective HDAC inhibitor with a long half life, for cancers.
In registration for breast cancer 2
nd
line treatment. Company would be most likely to consider either a global
partnership deal, an IPO or a change of control transaction. (Link)
Telik - In June, 2011 announced the initiation of Phase 2 clinical trial to evaluate oral Telintra in patients with
Revlimid refractory or resistant, deletion 5q MDS. Trial will enroll up to 117 patients and employ a sequential
design with two interim analyses.
Topotarget - Would out license HDAC product, Belinostat, in larger indications. Recently picked up from Curagen.
Company considered a takeover target. Positive Phase 1 data reported at ASH 2010. Recently finished recruitment
for Phase I trial of oral belinostat in patients with refractory or relapsed Hodgkin’s or non-Hodgkin’s lymphoma as
second part of development program.
NEW $ Undisclosed - large pharma disposing of a marketed oncology drug with revenues of around $30mm.
Significant barriers to entry.
NEW Undisclosed - Large Pharma disposing of a promising drug for the treatment of NHL.
Xencor - Developing an anti-CD30 (XmAB 2513) antibody for Hodgkin’s disease and T-cell lymphoma. Has finished
Phase 1. (Link)
YM Biosciences - CYT387 in Phase 2 study in myelofibrosis. Recently successful completion of Phase 1 and Phase 2a
studies. YM argues that this has the potential to be the best in class JAK inhibitor. (Link)
NEW Ziopharm Oncology - Darinaparsin in Phase 2 for PTCL. Favorable results to date.
ONCOLOGY - SOLID TUMORS
4SC - 4SC-201 developing HDAC in hepatocellular carcinoma. Preparing for Phase 2. Reported positive data from
clinical trials for Phase I/II in June 2011. Expected to conclude in 2012.
AB Science - In Phase 3 studies of Masitinib against imatinib (Gleevec®) in GIST. A recent Phase 2 trial saw
prolonged PFS and OS. (Link)
Access Pharmaceuticals - Searching for a partner for a platinum prodrug, ProLindac, similar to Eloxatin. Currently in
a Phase 2 study in ovarian cancer. Company is also interested in partnering an anti-proliferative antibody called
Angiolix which targets Lactadherin. (Link)
Active Biotech - Successful Phase 2 study showed tasquinimod’s ability to impede disease progression in symptom-
free patients with metastatic, castrate-resistant, prostate cancer. Drug works by attacking blood vessels. April,
2011 entered partnership to co-develop TASQ with Ipsen. Phase 3 trial was recently initiated and patient
recruitment is ongoing. Rights are available in North America, South America and Japan. (Link)
Adaptimmune - Has opened a Phase I/II, two cohort, open label clinical trial in metastatic melanoma to investigate
the safety, bioactivity and anti-tumor effect of patients' own T cells that have been genetically modified to express
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a high affinity T cell receptor (TCR) specific for a type of tumor antigen (protein) known as a cancer testis antigen
(CT antigen).
Adherex - Eniluracil enhances 5-FU effect. Open to partnership. Phase 1 data positive. Enrolling patients in a Phase
2 trial for breast cancer. Was at GSK before. (Link)
Advantagene - Phase 2 positive in prostate cancer for AdV-tk (ProstAtak). Going into Phase 3 trial with SPA.
Advenchen Laboratories - YN-968D1 contains apatinib, a novel small molecule angiogenesis inhibitor that shows
selective protein tyrosine kinase (PTK) inhibition on VEGFr2 and Ret and mild inhibition on c-Kit and c-SRC. In Phase
2 studies in China.
Agennix - Open to partnering Talactoferrin which is a recombinant form of human lactoferrin - for the indication of
non-small cell lung cancer. It is currently in clinical phase 3 trials (75% enrolled) and has shown good efficacy in
Phase 2 in NSCLC and renal cell carcinoma. Company formed from a recent merger with GPC Biotech AG. (Link)
Alchemia - Starting a Phase 3 program of its HA-irinotecan with the intention of partnering after data are in. Plans
to seek a 505b(2) approval in the United States.
Ambrx - very exciting platform technologies which allow engineering of proteins developed in both eukaryotic and
prokaryotic cells including an antibody drug conjugate program for oncology. (Link)
AmpliMed - Phase 2 trial of Amplimexon in 142 patients with advanced metastatic pancreatic cancer will report
out soon. Will partner before starting Phase 3 trial.
Anaphore - pioneering Altimers®, a new class of protein therapeutics that has a trivalent structure. Better ability to
lock on to a target. Working on a number of targets including a TRAIL-R antibody for oncology that is pre-clinical.
(Link)
Agenus - Prophage, a personalized cancer vaccine tested in 800+ patients - 8 cancers - in over 15 clinical trials
spanning Phase 1, 2, and 3 - market approval in Russia. Update: Results recently announced from Phase 2 clinical
trials testing Prophage Series G-200 vaccine in recurrent brain cancer, primary objective to asses survival rate for
26 weeks. Results showed 93% of patients were alive after 26 weeks and had a median overall survival of 11
months.
Angstrom Pharmaceuticals - completed a placebo-controlled Phase 2 study evaluating Å6 in recurrent ovarian
cancer. The time to tumor progression was doubled in patients receiving Å6 over that of placebo (p $500mm. Ran a sale
process via Greenhill Caliburn. No transaction has taken place as of June 2011. Nov 21, 2011: Valeant buys iNova
for $625 million.
$ Lipose - Viafill fat transfer system on the market with applications in aesthetics where traditional volumizers are
not well suited - particularly for the face and breasts. Company assisted by Torreya Partners. For details contact
[email protected].
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$ Pacira Pharmaceuticals - Looking to partner Exprarel, a long acting bipuvicaine, outside of the U.S. Interested
parties should contact Darren Pincus at [email protected].
NEW $ Rottapharm - for sale according to the Wall Street Journal. Company has two Phase 3 drugs in development
and a strong group of branded products in the market. Revenues over $850 million. Sale price could be over $2.5
billion. Company rumored to be using Credit Suisse to find a buyer.
NEW $ San Raffaele del Monte Tabor - privately-held Italian pharmaceutical company, is soliciting offers other than
the EUR 350m binding offer from Vatican bank IOR and Italian entrepreneur Vittoria Malacalza, according to Il Sole
24 on Dec 2, 2011.
UPDATE $ Savient Pharmaceuticals - FDA approved KRYSTEXXA (pegloticase) in Sep 2010, a PEGylated uric acid
specific enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy.
Company is seeking a commercial buyer and is using JP Morgan and Lazard in its search for an acquisition partner.
Savient is now pursuing a launch of Krystexxa on its own and is building a 50-person plus sales force. Update: First
year sales were $2.6 million. Market Cap of $160mm. (Link)
UPDATE Sigma-Tau - Reuters (6/2/11): “Italy's Sigma-Tau is eyeing the sale of up to 49 percent in the family-owned
drugmaker to private equity, ahead of a possible IPO that could value it at more than $2 billion, people familiar
with the situation said.” Company has approximately €1bn revenue with a strong rare disease business and a well
established European brand business. It is believed that discussions regarding a stake sale are no longer active but
rather the company is focused on restructuring its Italian business.
$ Suneva Medical - Marketing Artefill, a long-acting dermal filler with impressive revenue growth. Open to a
partnership or even a company sale. (Link)
$ Undisclosed player - selling off $20mm revenue+ commercial product for narrow market with pediatric
applications.
$ Undisclosed player - selling off portfolio of $50mm area specialty pharma products with some pediatric products
included.
$ Undisclosed player - selling a $10mm marketed primary care cardiovascular product.
$ Undisclosed player - approved hospital anesthetic in the United States with differentiation from existing
products. Also could be used in physician office setting where sedation required. Open to a product sale or other
value creating arrangement. Global rights available.
Undisclosed player - open to a merger or sale of $35mm revenue company with commercial presence in the U.S.
pain market. Torreya Partners advising.
NEW Undisclosed - German vertically integrated marketer of generic and branded pharma products with strength in
cardiovascular, dental and veterinary medicines. Revenues of this company exceed €100mm.
Undisclosed player - dermatology company with more than $40mm in revenue has hired a financial advisor to
restructure its debt that likely exceeds intrinsic value of assets. Process has been underway for a month or two.
COMPLETED Undisclosed player - Large pan-Asian drug company open to a sale. Update: Menarini acquires Invida
Group on Nov 16, 2011. Price not announced but Invida a substantial business with more than $200mm in
revenue.
NEW $ Undisclosed player - availability of product with pysch and primary call point. $12mm product sales run rate.
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NEW $ Undisclosed - Pharma company with a product for heart failure and revenues over $20mm would consider a
sale.
NEW Undisclosed - U.S. rights to a marketed drug for depression are available.
Zogenix - Needlefree transdermal delivery of sumatriptan for migraine. Has 80 sales reps. Recent FDA approval
with upcoming product launch using Astellas as a co-promotion partner. Update: company filed for an IPO in Sep
2010 - had $7mm in revenues in first half of 2010 with a substantial operating loss.
STEM CELLS, RNAI AND CELL THERAPY
Aldagen - ALD-401 is a type of adult stem cells called aldehyde dehydrogenase-bright (ALDHbr) stem cells isolated
from cord blood. ALD-401 is being developed for the post acute treatment of ischemic stroke and is in a Phase 2
study.
Calando Pharmaceuticals - Has a promising nanotechnology approach for delivery of RNAi. Looking for a company
sale. (Link)
Cardio3 - Developing a stem cell transplant method to address CHF after MI using cardiopoetic cells. (Link) Cardio3
BioSciences SA signs a co-development agreement with Artelis SA for the industrialization of the manufacturing
process of C-Cure and inaugurate its new premises in Mont-Saint-Guibert. (Link)
Cellerant - Developing a novel, cell-based medicine (Myeloid Progenitors / CLT-008) as a treatment for
chemotherapy- and radiation-induced neutropenia as well as for Acute Radiation Syndrome. Update: Sep 1, 2010:
received a substantial BARDA grant. On March 22, 2011, Cellerant Therapeutics initiates a Phase I/II clinical trial of
CLT-008 for Chemotherapy induced neutropenia in acute Leukemia patients.
NEW Geron - has ceased development of its embryonic stem cell development program and is open to partnering
this program to a third party.
HAC Biomed - a cell therapy company has very promising data for a treatment for the repair of damaged liver
tissue. Commercial stage in Germany. Assisted by Torreya Partners. Interested parties should contact
[email protected].
NEW Innovacell - Positive Phase IIb data for ICES13, a personalised cell therapy for SUI. The primary endpoint met
on the trial was a reduction in incontinence episode frequency across 32 centres in four EU countries. (Link)
Ipierian - Working on reprogramming of pluripotent stem cells for various therapeutic applications. (Link)
Kiadis Pharma - Reviroc is being developed as a treatment that eliminates blood cancer cells from autologous
transplants for patients with end stage blood cancer. Granted orphan by FDA which is in Phase 3. (Link)
MaxCyte - transfection cell platform with ability to program cells to attack a variety of major diseases.
Medi-Post - CARTISTEM® has been developed to treat damaged articular cartilage on knee as a result of acute
traumatic injury or more chronic conditions such as osteoarthritis. Phase 3 clinical trial has shown efficacy in
cartilage regeneration. KFDA approval pending. U.S. IND filing in process. (Link)
Oxford Biomedica - In a Phase I/II trial of ProSavin for the treatment of Parkinson's disease (PD). This gene therapy
has been associated with improvements in patient symptons. (Link)
Pervasis - Developing Vascugel, a cell therapy product, to accelerate vein remodeling for patients receiving an AV
fistula (AVF) in end stage renal disease. Compare to Proteon Therapeutics which has optioned a product for the
same indication to Novartis and is in phase 2 studies.
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Quintessence Biosciences - RNA degrading Ribonucleases being developed for oncology applications.
NEW Santaris Pharma - Phase 2a data of miravirsen, a microRNA-targeted drug, shows dose-dependent, prolonged
viral reduction of 2-3 logs HCV RNA after four-week treatment in Hepatitis C patients. (Link)
Silence Therapeutics - Disclosed on Sep 6
th
2010 that it had received an approach that could lead to an offer.
Company is a leader in RNAi delivery and associated therapeutics. Silence has retained a financial advisor and is
pursuing its duties as a UK listed company to maximize shareholder value. Dec 21, 2010: “Further to the
announcement dated 6th September 2010, Silence confirms that it remains in an offer period. The Company
continues to explore a variety of strategic opportunities.” Feb 7, 2011: The Board of Silence received a number of
indicative proposals but it is the view of the Board that none of these were sufficiently compelling to pursue
further in the context of the continued success of the Company. The Board has therefore terminated these
discussions to allow management to focus all of its efforts on the ongoing business for the purpose of creating
long-term shareholder value.”
Stemcells - HuCNS-SC is well-characterized, normal human CNS stem cells (HuCNS-SC) from brain tissue, isolated
and purified using monoclonal antibodies against cell surface antigens. HuCNS-SC is being developed as
intracerebral injection for the treatment of myelin disorders such as Pelizaeus-Merzbacher Disease. Phase 1 data
upcoming in 2012. (Link)
Sylentis - focused on topical RNAi therapy with a Phase 1 trial of a therapy for glaucoma and dry eye.
Tengion - Leading regenerative medicine company open to partnership deals for the development of both an
artificial bladder and an artificial kidney. Tengion has recently gone through a leadership transition and plans an
FDA meeting to discuss the kidney program before the end of 2011. (Link)
UROLOGY AND MEN’S HEALTH
Afferent Pharmaceuticals - Developing P2X3 receptor antagonist for OAB. Supportive preclinical data. (Link)
Ampio - Zertane is a 5-Hydroxytryptamine Inhibitor (5-HT) and mu-opioid receptor agonist. Zertane is being
developed as fast dissolving oral tablets for the treatment of premature ejaculation in men. Very strong Phase 3
data reported in a recent trial in the prevention of premature ejaculation. (Link)
NEW Apricus Bio - has filed for market authorization to sell its erectile dysfunction drug, Vitaros®, in Latin America.
Open to a commercial partnership deal.
Aptys - looking to outlicense transdermal surfactant-free testosterone gel. Phase 3 ready.
Camurus - Prosenze® Depot (CAM2032) is a ready-to-use LHRH agonist depot designed for easy and painless
subcutaneous administation being developed for treatment of prostate cancer and endometriosis. The product has
shown positive Phase 2a data.
Celek Pharmaceuticals - CEL-031, is a Phase 2 drug in development for the treatment of non-muscle invasive
bladder cancer. Works by inducing tumor cell apoptosis and inhibiting cell proliferation. (Link)
Clarus Therapeutics - OriTex, a proprietary oral testosterone product being developed in Phase 2 for use in men
with low testosterone levels.
Conjugon - (C-1205) in Phase 1b studies for e. coli donor technology to prevent UTI’s in men with a urinary
catheter. (Link)
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GP Pharm - developing long-acting injectibles in the oncology area. Looking for licensors for Leuprorelin 1 month,
Leuprorelin 3 months, Triptorelin 1 month, Triptorelin 3 months, Gosorelin 3 months, Octreotide LAR and
Octretide MAR. Also has developed desmopressin drops.
NEW Innovacell - Positive Phase IIb data for ICES13, a personalised cell therapy for SUI. The primary endpoint met
on the trial was a reduction in incontinence episode frequency across 32 centres in four EU countries.
Intelgenx - INT007 found to be bioequivalent to a leading branded tablet containing a phosphodiesterase type 5
(PDE-5) inhibitor for the treatment of erectile dysfunction. INT007 has been developed using IntelGenx'
proprietary immediate release "VersaFilm" drug delivery technology. Market ready in 2012 as a 505(b)2. (Link)
Japan Tobacco - TRPV1 antagonist in Phase 1 development. Rights available. (Link)
King’s College - Reversible, non-hormonal, oral delivery approach to male contraception. Preclinical. (Link)
Merlion - Developing finafloxacin, a best in class 4th generation fluoroquinolone being targeted against severe,
life-threatening infections. Compound has successfully progressed through to mid-stage clinical development. Oral
formulation demonstrated in PoC studies compelling efficacy in uncomplicated UTI and the eradication of
Helicobacter pylori. IV formulation currently completing Phase I trials and Phase 2 studies in complicated UTI are
planned for late 2011, as are clinical protocols in complicated respiratory tract infections. MerLion are exploring
strategic alternatives for this product with the assistance of Torreya Partners. For further details, contact
[email protected]. (Link)
Merrion Pharma - Acyline is an oral GnRH antagonist for treatment of prostate cancer. Today’s products are
injectible. (Link)
Novadel - Developing Duromist (a lingual mist version of sildenafil) for introduction to the market after the Viagra®
patent expiration in 2012. Can complete pivotal 505b(2) trial in 2011. Recently filed an IND to start studies.
UPDATE OxThera - Oxazyme is recombinanate oxalate degrading enzyme for the treatment of kidney stones. No
data reported from a pending study in some time. A related compound is at Althea Technologies. (Link)
Palatin Technologies - Bremelanotide is a melanocortin agonist that has shown positive results in four Phase 2
efficacy for erectile dysfunction studies enrolling more than 1,300 men. Was partnered with King and after a
formulation change is intended for further Phase 2/3 studies in the next several years. Also showed positive results
in a Phase 2a pilot study for female sexual dysfunction (FSD). Company announced commencement of enrollment
in a Phase 2b study for FSD on June 28, 2011. (Link)
Pierre Fabre - Outlicensing North American rights to Javlor (vinflunine) - a newly approved bi-fluorinated MTI
(Microtubule inhibitor) for bladder cancer. Licensing process is making substantial progress. (Link)
H Protox Therapeutics - PRX302 has completed a promising open-label Phase 2a study in BPH. Topline data
pending on Phase 2b study in Q1 2010 and were quite positive. Warburg Pincus recently made a $35 million
investment which will facilitate registrational trials for this drug. Rights to this drug in Japan were licensed to Kissei.
(Link)
Quatrx - Fispemifene similar to GTX. Would partner this drug. (Link) Fispemifene has completed two Phase I clinical
studies in Europe and one Phase 2 clinical study in the US.
Stellar Pharma - has retained U.S. rights to Uracyst® from Watson. This is an investigational drug/device
combination for the treatment of interstitial cystitis. Watson indicated that the product did not meet its endpoint
in a double-blinded placebo controlled study. There may be an opportunity to redefine the patient population in a
further study with this drug and still achieve approval in the U.S. Note: On June 8, 2011, Stellar announced that it
had retained an advisor for its U.S. market products.
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UPDATE Taris Biomedical - Has recently completed a Phase 1 study showing the ability of an intravesical delivery
system to get drug to the bladder for the treatment of interstitial cystitis. Raised $18mm in April 2011. Recenty
started enrollment of a Phase 2 trial. (Link)
Telormedix - Developing a promising TLR9 modulator that is in a Phase 1 / 2 trial for the treatment of bladder
cancer. (Link)
Tengion - Leading regenerative medicine company open to partnership deals for the development of both an
artificial bladder and an artificial kidney. Tengion has recently gone through a leadership transition and plans an
FDA meeting to discuss the kidney program before the end of 2011. (Link)
UPDATE Theravida - Combination of oxybutynin and an agonist to improve tolerability and efficacy of oxybutynin for
treatment of overactive bladder (OAB). Will report out Phase 2b data in early 2012.
Toray Industries - TRK-130 provides new mechanism of action which relates to CNS, and controls lower urinary
tract function. Indicated for OAB. Phase IIa study has been completed and efficacy has been confirmed.
$ Undisclosed - mature, off-patent urology product.
Undisclosed - Pharma Company exploring sale of certain early clinical compounds in the urogenital area.
Urigen - In Phase 2 with URG101 for the treatment of acute flares of Interstitial Cystitis (IC/BPS). (Link)
UPDATE Vantia - Positive Phase 2a data with VA106483, a vasopressin agonist, for treatment of nocturia, excessive
trips to urinate at night. Likely to be effective based on mechanism. VA106483 is currently being investigated as a
new treatment for nocturia in a 30 patient trial to find the dose in older men with BPH. Data from this trial are
expected shortly. Recently raised $6mm. (Link)
UPDATE Vivus - Would be interested in partnering avanafil, a PDE V inhibitor in development for erectile
dysfunction. Recently reported out second Phase 3 program also with strong data. Vivus submitted an NDA for
this drug on June 30, 2011 and has a PDUFA date in the U.S. of April 29, 2012. Cowen predicts this drug will take
8% market share with $800mm in U.S. revenue. (Link)
VACCINES
Agenus - Adjuvant QS-21 has consistently stimulated strong cell-mediated and humoral immune responses when
combined with a variety of vaccine antigens. Approximately 15 product candidates utilizing QS-21 are currently in
clinical development by Agenus’ commercial licensees and collaborators.
Archivel Farma S.L - running a double-blind, randomized, placebo-controlled phase 2 clinical trial to investigate the
safety, tolerability, and immunogenicity of the novel antituberculous vaccine RUTI following one month of
isoniazid treatment in subjects with latent tuberculosis infection.
BioDiem - Working on a cell-based influenza vaccine. In Phase 2 studies. Open to a global partnership deal. Also
working on an intranasal Live Attenuated Influenza Vaccine (LAIV). The LAIV is now on market in India and has
been licensed for other emerging markets to Merck/WHO. (Link)
BTG - Angiotensin Therapeutic Vaccine (ATV) for high blood pressure. This therapeutic vaccine (biologic) is in early
Phase 2 clinical development. Large potential market as alternative approach to blood pressure management.
(Link)
BTG - CoVaccine HT. This is a proprietary vaccine adjuvant platform that generates very powerful antibody
responses. It has been used in a number of studies and diseases by groups around the world with impressive
performance data available.
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CureVac - upregulates mRNA to create specific autoantigen responses to disease. Has recently shown a nice proof
of principle in man for a therapeutic vaccine aimed at prostate cancer in a Phase 1b study.
H UPDATE Cytos - looking to partner CYT003 for allergic rhinoconjunctivitis. Vaccine has finished Phase 2b studies.
Asthma symptoms decreased by 33% under QbG10 treatment despite corticosteroid withdrawal, while they
increased by 29% under placebo treatment (p=0.01). Use of relief medication doubled in the placebo group, while
it remained stable in the QbG10 group (p=0.01). Update: Company has been exploring strategic options with aid of
a financial advisor. Recent attempt to restructure bonds has not succeeded. (Link)
Cytos - In preclnical development with Qb-Flu. This is a bacterially produced subunit vaccine composed of the
globular domain of the Influenza A hemagglutinin surface protein conjugated to Cytos’ virus-like particle Qb. Has
shown strong antibody titres achieved with this vaccine. What is important is that this approach avoids the
manufacturing issues associated with both cell culture and egg-based flu vaccines. (Link)
UPDATE Dynavax - HEPLISAV is an investigational adult hepatitis B vaccine that was formerly partnered with Merck.
In two Phase 3 trials, HEPLISAV demonstrated increased, rapid protection with fewer doses than current licensed
vaccines. Positive data reported on July 20, 2011 and regulatory approval pending. (Link)
GlycoVaxyn - Strong Phase 1 data for a novel vaccine for Shigella. Open to partnering. (Link)
Inovio - Looking for partnerships based upon its Syncon vaccine platform. Syncon facilitates better vaccines that
address changing strains of diseases, including HIV, HCV, HPV, and influenza. Planning to complete a Phase 1 for a
universal flu vaccine in 2011. Has shown positive results in recent HIV vaccine Phase 1 study. Starting a Phase 2
study in 2011 in cervical cancer. (Link) (noncon)
Intercell - rumored takeover candidate. Strong Japanese encephalitis vaccine marketed by Novartis and CSL.
H NEW Inviragen - DENVax, a promising clinical development candidate for dengue fever and other tropical
diseases. Phase 2 data coming up for this candidate which has a substantial commercial market. (Link)
JN International Medical - NmVac4-A/C/Y/W-135 is a lyophilized preparation of purified polysaccharides from
Neisseria meningitidis (meningococcus) of groups A, C, W135 and Y. Highly effective in a recent Phase 3 trial. (Link)
COMPLETED Juvaris - JVRS-100 is a highly potent toll-like receptor-directed cationic lipid-DNA complex that is being
developed as a therapeutic and an adjuvant to treat multiple diseases including influenza. In Phase 2 for vaccine
adjuvant for influenza in elderly. Note: In September 2011, this asset was sold to Colby Pharmaceutical Company.
UPDATE LigoCyte - has a Phase 2 vaccine is designed to treat norovirus, the leading worldwide cause of infectious
gastroenteritis, or inflammation of the stomach and intestines. Positive Phase 1b data. The company is exploring
strategic alternatives with assistance from Montgomery Marhsall (now Sagent). As of Dec 2011, no transaction had
taken place.
Okairos - Hepatitis C vaccine in development has shown T-cell response in Phase 1. (Link) A second Phase I study is
going to be started soon, in order to evaluate an alternative heterologous prime-boost regimen based on the
AdCh3, for the priming, and a Modified Vaccinia Ankara vector encoding NSmut, for the boosting (HCV003).
Update: Company completed a 16mm EUR financing in Sep 2010 led by Versant Ventures.
Pevion Biotech AG - In Nov 2010 reported positive preliminary results from a Phase I study of PEV7, the first
vaccine against recurrent vulvovaginal candidiasis (RVVC). Also known as chronic recurrent thrush. Occurs in 3 to
6% of women. (Link)
H VaxInnate - Developing Universal flu vaccines. Strong Phase 2 data. Company is open to a sale.
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Virionics - VLPs for HPV and HCV. Therapy and diagnostics story. MediGene holds exclusive marketing rights to
CVLP products in Germany and co-exclusive marketing rights for the rest of Europe. (Link)
Xiamen Innovax Biotech - Has developed a Hepatis E vaccine that has recently been shown to be completely
effective in the prevention of Hepatitis E in a 110,000 patient clinical trial. Approximately 30% of the world’s
population has Hepatitis E. Xiamen open to licensing rights to this product outside of China. (Link)
VIROLOGY
UPDATE Achillion - Phase 2b 12-week study underway for ACH-1625, for hepatitis C. Very positive Phase 2a data.
The drug candidate is an inhibitor of NS3 protease. Phase 1b data were quite promising showing a 4 log10
reduction in viral load. In preclinical studies ACH-1625 has demonstrated potency, unique pharmacokinetic
properties and a safe in vivo profile even at very high doses. (Link)
NEW Achillion - On Dec 4, 2011 reported proof-of-concept data from its Phase 1b clinical trial of ACH-2928, a first-
generation NS5A inhibitor, demonstrating that patients treated with ACH-2928 achieved a mean maximum 3.68
log10 reduction in HCV RNA after three-day monotherapy of 60 mg once daily. The compound also demonstrated
good safety and tolerability both in healthy volunteers and in patients with chronic hepatitis C (HCV).

Adamas Pharmaceuticals - Pursuing Triple Combination Therapy for Influenza (ADS-8902). Adamas
Pharmaceuticals, Inc., a privately held company, announced today that it has expanded its Phase 2 clinical study of
a proprietary investigational triple-combination antiviral drug therapy for influenza to include centers in the United
States, Canada and Europe. (Link)
Aicuris - Good Phase 2 data in treatment of human CMV. Looking to outlicense this compound. Recently granted
orphan designation by COMP of the EMA.
Aicurus - Phase 2 data for AIC-316, herpes simplex product. Looking to partner global rights before running Phase 3
trial. Large market with data to be released in early 2011.
COMPLETED Anadys - ANA598 is a low-nanomolar inhibitor of HCV genotype 1a and 1b replicons via N5b
polymerase. Has started a Phase 2 trial. Anadys also has ANA773, an oral inducer of endogenous interferons with
nice Phase 1 data. May 2010 - Lazard retained to act as advisor to Anadys to explore strategic alternatives. Nov
2010 press release: Anadys continues to work with Lazard Frères & Co. LLC. to explore potential strategic
transactions, in parallel with moving the ANA598 and ANA773 programs forward. Update: Oct 17, 2011 - Roche
acquires Anadys for $230mm - a 256% premium.
Agenus - AG-707 is an investigational vaccine consisting of recombinant human heat shock protein-70 complexed
with 32 distinct synthetic antigens from the HSV-2 proteome. Has finished Phase 1 studies.
Avexa - In Phase 3 for apricitabine with positive data, a next generation NRTI for HIV (origin Biochem Pharma /
Shire). Announced on July 6, 2011 that it had retained Pharma Ventures to assist in outlicensing this compound.
Avila Therapeutics - Developing AVL-181 which silences proteins. Works as a pan-genotype protease inhibitor for
Hepatitis C with promising pre-clinical data. Update: Nov 29, 2010 - Avila publishes further work in Nature showing
increased selectivity, potency and duration of action of this NS3 inhibitor. (Link)
Avir Green Hills Biotechnology - deltaFLU is a live attenuated influenza virus with an optimal balance between
attenuation and immunogenicity. Produced in CHO cells. deltaFLU is applied intranasally with a spray device.
Positive Phase 1 data. (Link)
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Beech Tree Labs - In Phase 2 for an agent to treat herpes simplex. The agent BTL-TML-HSV is a broad antiviral and
inhibits viral reassembly after infection and viral uncoating. Update: company recently completed a financing
which will facilitate completion of the clinical trial. (Link)
BioAlliance Pharma - Looking to partner acyclovir Lauriad for herpes labialis. (Link) BioAlliance Pharma , today
announces final positive results of its pivotal phase 3 clinical study in immunocompetent patients with recurrent
herpes labialis (LIP Study) treated with acyclovir Lauriad. Primary and secondary endpoints have been met with
marked efficacy and good tolerance. (Link)
NEW $ BioAlliance Pharma - searching for a U.S. Partner for Oravig. Oravig is miconazole buccal tablets for the
treatment of oropharyngeal candidiasis (OPC), more commonly known as thrush, in adults and children age 16 and
older. This drug was returned by Strativa Pharmaceuticals recently to BioAlliance.
NEW BioCryst Pharma - developing peramivir, a Phase 3 IV antiviral treatment for influenza. Has substantial
promise. (Link)
BioDiem - Working on a cell-based influenza vaccine. In Phase 2 studies. Open to a global partnership deal. (Link)
Biolex - Finishing up Phase 2 for a long-acting interferon for Hepatitis C (Loctemapron).
Bionor Immuno - (Vacc-4x)HIV vaccine generates a positive immunologic response in Phase 2 trials. Ongoing Phase
2b study with data to report out in the next six months. Update: product failed to show any effect in a Phase 2
trial.
Biota - Looking to partner a once weekly inhalable long-acting neuraminidase inhibitors for the treatment of flu.
Would compete against Relenza from GSK. Currently partnered with Daichi-Sankyo in Japan. One of two Phase 3
studies have reported out with positive data - large market. Recently received a large BARDA grant and has
retained Piper Jaffray as financial advisor to maximize value of existing programs and help to enter the U.S. market
on a commercial basis. (Link)
Biota Holdings - Looking to partner a product to treat the common cold. Specifically, canyon-like clefts on
rhinovirus surface attach to the receptor allowing infection. Biota achieved proof of concept in a Phase IIa
(challenge study) in 2009. Now in Phase 2b studies for this product. (Link)
Biotron - BIT225 represents a first-in-class drug for treatment of HCV, targeting the p7 protein of HCV. In a Phase
2a study with data reporting out in September 2011. (Link)
Chimerix - CMX001 is a lipid mimic of cidofovir that more easily passes into cells and then targets double viruses
including herpes viruses and orhoproxviruses. Current in Phase 2 against CMV and BK virus. A recent trial showed
that this drug works better than existing antivirals in persons with impaired renal function. In active partnership
talks. Also in development for smallpox. (Link)
Eiger BioPharmaceuticals - Developing Clemizole as a NS4B-RNA binding inhibitor for the treatment of hepatitis C.
In Phase 1. Eiger BioPharmaceuticals Acquires Exclusive License to Novel Hepatitis C Virus (HCV) Technology From
Stanford University.
NEW Endeavor Vision - LMV-601 is the first in a new class of drugs known as PC-PLC inhibitors. It has outstanding
antiviral properties resulting from the inhibition of early viral gene transcription. It is currently in clinical
development (Phase I on infected patients) for the treatment of Human Papillomavirus (HPV) infection of the
cervix.
Epiphany Bio - Developing EPB-348. Good results so far for Herpes Zoster through Phase 2b trial. No partnership
announced thus far. Also developing a compound for mono. Licensed from Medivir. (Link)
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Flamel - Looking to license a long-acting IFN-alpha (compare to PEG-INTRON) for Hepatitis C which is currently in
phase II. Better AE profile and strong efficacy in trials to date. Flamel Technologies Announces the Initiation of a
Phase 2a Clinical Trial of Flamel's IFN-Alpha-2b XL in Patients with Chronic Hepatitis C Virus Infection.
GlobeImmune - Strong efficacy in treating Hepatitis C with GI-5005 a viral fusion protein. Phase 2b data show that
the HCV vaccine increased sustained virologic response by 12 percent in patients who previously failed therapy
with standard of care. (Link)
Idera - looking to partner IMO-2125, its TLR9 agonist for hepatitis C (HCV) after positive Phase 1 data.
Mechanistically, this is a DNA-based compound that stimulates an innate immune response, including the
induction of high levels of interferon-alpha and other cytokines that work to suppress HCV viral replication. Phase
2 studies have been delayed. (Link)
H UPDATE Inhibitex - Reported Phase 2 data in December 2010 from a clinical trial of FV-100, its antiviral compound
in clinical development for the treatment of herpes zoster (shingles). While the results showed improvements in
the reduction in the severity and duration of shingles-associated pain of 3% and 7% for the 200mg and 400mg
patient cohorts, it was far from the 25% reduction called for as the primary endpoint, and not statistically
significant. Open to partnering this compound.
Inhibitex - Pursuing a nucleotide polymerase inhibitor for HCV which has shown high promise in Phase 1b data
reported on November 29, 2011. INX-189 with a substantial reduction in HCV RNA. (Link)
Innate Pharma - Looking to partner IPH-1201, a gamma-delta agonist immune modulator, for Hepatitis C. Solid
Phase 2a data reported at EASL.
Inovio - Looking for partnerships based upon its Syncon vaccine platform. Syncon facilitates better vaccines that
address changing strains of diseases, including HIV, HCV, HPV, and influenza. Planning to complete a Phase 1 for a
universal flu vaccine in 2011. Has shown positive results in recent HIV vaccine Phase 1 study. Starting a Phase 2
study in 2011 in cervical cancer. (Link) (noncon)
NEW Japan Tobacco - looking to partner JTK-853, an RNA polymerase inhibitor for the treatment of Hepatitis C. Has
completed POC in a Phase 1 study.
NEW LG Life Sciences - LB80380 - Phase 2 novel nucleotide polymerase inhibitor for chronic HBV infection. An ester
prodrug of phosphonate nucleotide analogue of guanosine monophosphate with potent activity against hepatitis B
virus (HBV). Designed to overcome problems of currently approved HBV drugs such as resistance and renal tox.
UPDATE Marinomed Biotechnologie - The privately-held Austrian biopharmaceutical company is in talks with
pharma players to outlicense its lead compound MAM-05.101, is an antiviral nasal spray for the treatment of the
common cold - particularly in childen. This product is a GRAS botanical and has shown good efficacy in a Phase 2
type trial. Can likely be introduced to the market now as either an OTC product or medical food. On market in EU.
Looking for licensing partner for U.S., Canada and Japan. In Austria the nasal spray has been marketed since 2008,
the product was licensed to Boehringer Ingelheim for Europe, Russia and CIS, South America, parts of Asia and
Australia in 2010.
Mymetics - Has hired Lazard to look at its strategic alternatives. Company developing mucosal antibodies to
prevent HIV infection. Phase 1 data show safe and well tolerated. Also has a preclinical RSV vaccine. (Link)
Myrexis - Portfolio includes Vivecon, a Phase 2 maturation inhibitor for HIV. Company also has additional pre-
clinical HIV products. Has suspended development of this product and is seeking an external partner. (Link)
NanoViricides - Early stage treatments for influenza and avian flu. (Link)
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UPDATE Neurotune - Positive top-line results from its Phase IIa study of dimiracetam (NT-11624) for treatment-
induced neuropathic pain in HIV patients receiving anti-retroviral medication. Phase 2b study to start in 2012
under recently granted IND. (Link)
Okairos - Hepatitis C vaccine in development has shown T-cell response in Phase 1. (Link) A second Phase I study is
going to be started soon, in order to evaluate an alternative heterologous prime-boost regimen based on the
AdCh3, for the priming, and a Modified Vaccinia Ankara vector encoding NSmut, for the boosting (HCV003).
COMPLETED Pharmasset - Positive results for PSI-7851 in Phase 1 studies, a second generation polymerase inhibitor
for Hepatitis C. Has also seen positive Phase 1 data with PSI-938, a guanosine nucleotide analog polymerase
inhibitor. Recently entered a combination study with Tibotec. Update: company bought by Gilead for $14bn on
Nov 22, 2011.
Presidio Pharmaceuticals - (PPI-802) is less subject to resistance for HIV treatment. In Phase 2. (Link)
Presidio Pharmaceuticals - PPI-461, preclinical small-molecule NS5A inhibitor for hepatitis C. Has shown favorable
data and is well-suited to be a part of an oral combination drug for HCV. Reported Phase 1a data that were
favorable in July 2010. Also presented further positive clinical data at AASLD 2010. Announced positive Phase 1b
data on March 30, 2011.
UPDATE Protein Sciences - Has FluBlok for treatment of influenza. Pending BLA with an approval likely. Company is
exploring an IPO.
NEW Progenics - looking to outlicense its virology pipeline. Included is PRO-140, a Phase 2 antibody for HIV. Unlike
small-molecule CCR5 antagonists, PRO 140 inhibits HIV entry at concentrations that in vitro do not appear to block
CCR5's natural activity of directing the migration of immune cells towards sites of inflammation in the body. (Link)
NEW Quantum Pharmaceuticals - Matrix M1 protein inhibitors for the management of influenza. (Link)
Redox - Phase 2 trials of Doxovir, a non-nucleoside analogue, for Herpes labialis, have been successful. (Link)
NEW Santaris Pharma - Phase 2a data of miravirsen, a microRNA-targeted drug, shows dose-dependent, prolonged
viral reduction of 2-3 logs HCV RNA after four-week treatment in Hepatitis C patients. (Link)
SciClone - ZADAXIN in Phase 3 for the treatment of hepatitis C. Partnered in Europe to Sigma-Tau.
UPDATE Scynexis - SCY-635 targets cyclophilin for treatment of Hepatitis C. Novel mechanism and promising Phase 1
data. Currently in Phase 2 studies.
Shire - Would outlicense Gene-Activated

Human Growth Hormone (GA-IFN?) - for Hepatitis C.
UPDATE Starpharma - positive data for VivaGel. VivaGel® is being developed for the prevention of HIV and HSV-2
(genital herpes). It has also shown very promising activity against the human papillomavirus (HPV). Starpharma
commenced a dose ranging Phase 2 trial in 2010 in 132 patients. Two Phase 3 trials (200 patients in each trial) that
would aim to establish the effectiveness of Vivagel in preventing recurrence of the infection, are also planned.
Symphogen - Novel polyclonal antibody technology platform with a promising antibody in development for RSV
(Sym003), still in the pre clinical stage.
Toyama - T-705 (favipiravir), a viral RNA polymerase inhibitor, is in Phase 3 studies in Japan and Phase 2 in the
United States as a highly promising anti-viral drug for the treatment of influenza. (Link)
H VaxInnate - Developing Universal flu vaccines. Strong Phase 2 data. Company is open to a sale. Recently received
a major U.S. government contract.
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Virionics - VLPs for HPV and HCV. Therapy and diagnostics story. (Link) Moreover, MediGene holds exclusive
marketing rights to CVLP products in Germany and co-exclusive marketing rights for the rest of Europe.
Xiamen Innovax Biotech - Has developed a Hepatis E vaccine that has recently been shown to be completely
effective in the prevention of Hepatitis E in a 110,000 patient clinical trial. Approximately 30% of the world’s
population has Hepatitis E. Xiamen open to licensing rights to this product outside of China. (Link)
WOMEN’S HEALTH
Acrux - regained rights in May 2011 to Luramist®, a testosterone spray for women to enhance female sexual
function. This program has progressed through Phase 2 studies with Vivus. Also has a promising Phase 1 completed
progestin solution for birth control. (Link)
$ Acrux - rights outside of the U.S. are available for Estradiol MDTS® is a novel Estradiol Metered Dose Transdermal
Spray (MDTS®) hormone replacement product for the treatment of moderate to severe symptoms due to
menopause. This product was recently approved in Sweden and has pending approvals in a number of other
countries.
Adamis - successful completion of a Phase 3 contraceptive trial of the company's contraceptive gel product
candidate named Savvy (C31G) in Dec 2010. Looking to outlicense this product.
Agile Therapeutics - AG20015 is a low-estrogen dose once-weekly contraceptive patch containing levonorgestrel
(100-120mcg) and ethinyl estradiol (25-30mcg) as active ingredients. This product has successfully progressed
through Phase 2 studies and has high promise. Company is preparing to go into Phase 3 studies. Agile is also in
Phase 2 with a contraceptive product that does not use estrogen. (Link)
Alliance Pharma - Isprelor contains misoprostol as an active ingredient. It is a synthetic PGE1 analogue and serves
on both to ripen the cervix and augment labor as a one-step labor induction agent. Isprelor is being developed as
vaginal tablets (25mg) for the induction of labor and has completed Phase 3 studies. (Link)
Antares Pharma - has published successful results from a dose-finding Phase 2 trial for a novel contraceptive gel
containing the progestin Nestorone and estradiol (NES/E2) utilizing the Antares ATD (advanced transdermal
delivery) gel system. This program is in Phase 3 studies. (Link)
Anterion Therapeutics - has finished Phase 2 studies for Annuelle III, a contraceptive product that lasts for three
years. (Link)
Aoxing Pharmaceutical Company - Has enrolled over 120 patients in its on-going Phase 3 clinical studies of oral TJSL
Capsules, a novel investigational drug to treat primary dysmenorrhea ("PD"), or menstrual pain, in adult women.
This product is based upon Chinese herbs. (Link)
Apricus Bio - Femprox contains alprostadil which is a synthetic version of prostaglandin E1 (PGE1), a naturally
occurring vasodilating agent which increases blood flow to the female genitalia. Apricus Biosciences has completed
one Phase 2 trial in the United States and one Phase 3 study in close to 400 women in China, which achieved a 44%
positive response rate as compared to a placebo. Looking for a development partner. (Link)
NEW Athena DDS - Has a one shot treatment for bacterial vaginosis. Secnidazole 2gm single dose is comparable to 7
days treatment with Metronidazole tablet. Sachets are well accepted by patients and easy to use. Has been
approved in France.
NEW Ausio Pharma - AUS-131 is a first-in-class, nonsteroidal, nonhormonal estrogen receptor ? (ER?) agonist that
offers a potentially safer alternative to estrogen for the treatment of menopausal symptoms and is currently in
Phase 2a clinical trials. (Link)
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Bionovo - Looking to partner a SERM with promising Phase 2 data for hot flashes. This is based upon a botanical
product. Does not bind to estrogen receptor alpha. (Link)
UPDATE Biosante - LibiGel (testosterone gel) for female sexual dysfunction is fully enrolled in a pivotal Phase 3
study, and is designed to be quickly absorbed through the skin after a once-daily application. Company expects to
announced topline results from its pivotal study program shortly.
Bio-Term Pharmaceuticals - developing a drug candidate for the prevention of pre-term birth. (Link)
Burdica - Zestica Fertility™, a personal lubricant using the fertility enhancing properties of HA. The product range
was subsequently augmented by the introduction of Zestica Moisture™, designed to alleviate the dryness and
discomfort which is experienced after menopause. (Link)
Chiesi - Would outlicense CHF4227 program (SERM) that has completed Phase 1 testing.
Concert Pharmaceuticals - CTP-347 for hot flashes in Phase 1 which is completed. Good efficacy story. (Link)
COMPLETED $ Cytokine Pharmasciences - A number of women’s health products including applications in the OB
area - Misodel for labor induction, c-vad for bacterial vaginosis and Premis for cervical softening. Update: Company
acquired by Ferring on October 24, 2011. (Link)
Dilafor - DF-01 (tafoxiparin) is being developed as a subcutaneous injection for the prevention and treatment of
protracted labor (prolonged labor) during child birth. In a randomized, double-blind, placebo-controlled clinical
trial on 263 women showed that treatment with tafoxiparin provided a statistically and clinically significant
reduction in the incidence of protracted labor and caesarean sections. (Link)
$ Dong-A Pharma - Outlicensing Gonadopin® - a recombinant human follicle stimulating hormone (rec-hFSH) for
controlled ovarian hyperstimulation. Launched in Korea. Note: a similar product was recently in-licensed from Itero
to Watson Pharmaceuticals. Other similar products available for license from Ibsa and LG.
Easton Pharmaceuticals - VIORRA, is an over-the-counter aid for the treatment to restore and improve vaginal
moisture and elasticity which has a very positive effect on women’s sexual desire and arousal. Regulatory approval
in Mexico pending. (Link)
Embil - Would outlicense Kortos cream for hemorrhoids and anal fissures. (Link)
NEW Endoceutics - developing Femivia for the treatment of issues associated with hormone deficiency in post-
menopausal women (e.g., memory loss, diabetes, muscle loss). This product is in Phase 3. (Link)
FemmePharma - Running an open-label, multicenter Phase 2a evaluation of the use of topically administered
FP1198 for the treatment of moderate to severe cyclic breast pain (cyclic mastalgia). (Link)
GlycoTope - FSH-GEX is a Phase 1 follicle-stimulating hormone produced recombinantly with a fully human
glycosylation. FSH-GEX is being developed as subcutaneous formulation based on GlycoExpress technology for the
treatment of infertility. (Link)
Hisamitsu / Noven - positive Phase 2 data for low dose oral paroxetine mesylate for treatment of hot flashes
(Mesafem). May be interested in finding a partner for this product.
HRA Pharma - Two UPA contraceptive products (UPAC-ring and UPAC-IUS) based on the molecule ulipristal
acetate, a new chemical entity which modulates the activity of the progesterone receptor. This is in Phase 2 trials.
(Link)
NEW IBSA - Fostimon is a highly purified human-derived FSH, i.e. the natural pituitary follicle stimulating hormone.
Approved or close to approval in most territories.
Page | 131

Instead Sciences - Amphora is a lubricating vaginal gel that has demonstrated efficacy in preventing conception
and STIs in extensive in-vitro studies and human clinical trials.
Kade - Would outlicense Cliovelle - a low dose Estradiol / Progestin combination product for treatment of
menopausal disorders.
Meditrina - has hired Robert W. Baird to explore strategic options. Company working with aromatase inhibitors,
MPI-676 and MPI-674 candidates which are headed to Phase 3 trials. (Link)
Mithra Pharmaceuticals - In Phase 2 for the treatment of cervical dysplasia with cidofovir as an active agent. (Link)
NEW Pantec - FSH Patch in Phase 2 testing for infertility treatment.
PearTree Women’s Healthcare - PT-201 and 01 for treatment of urogenital atrophy. PT-100 is a fixed dose combo
of estriol and progesterone as a vaginal suppository. PT-101 is a vaginal suppository containing a SERM indicated
for prevention of urigenital atrophy associated with menopause. (Link)
Pevion Biotech AG - In Nov 2010 reported positive preliminary results from a Phase I study of PEV7, the first
vaccine against recurrent vulvovaginal candidiasis (RVVC). Also known as chronic recurrent thrush. Occurs in 3 to
6% of women. (Link)
Repros Therapeutics - Proellex off clinical hold in Phase 3 for uterine fibroids. Has resumed studies and has
enrolled the fourth cohort in a low dose Proellex study as of June 2011.
$ Semprae Laboratories - markets Zestra, an OTC product for female sexual arousal enhancement. This product is
backed by clinical studies showing its efficacy. Company is open to promotional or partnership deals.
Starpharma - VivaGel® is being developed for the prevention of HIV and HSV-2 (genital herpes). It has also shown
very promising activity against the human papillomavirus (HPV). In August, reported positive data from a Phase 2
trial. The study showed that treatment with VivaGel (containing 1% of the active, SPL7013), once daily for seven
days, resulted in 74% of patients achieving Clinical Cure of BV 2 to 5 days after completion of therapy compared
with just 22% in the placebo group (P=0.0002). (Link)
Trimel Biopharma - Phase 2 data were positive for an intranasal testosterone product for FSD.
Undisclosed player - Partnering a Phase 2 progesterone modulator for uterine fibroids and endometriosis.
Undisclosed player - Partnering a Phase 3 ready drug for bacterial vaginosis. Differentiated from current
treatments.
COMPLETED $ Undisclosed party - looking to sell a portfolio of marketed prenatal vitamins with assistance from
Torreya Partners. Interested parties should contact Tom Bird ([email protected]).
$ Undisclosed player - selling a group of marketed hormone products with the assistance of Torreya Partners.
Interested parties should contact Tom Bird ([email protected]).
UPDATE Vantia - Pursuing VA111913, a vasopressin antagonist for dysmenorrhea, menstrual cramping pain. An
exploratory Phase 2 trial of VA111913 showed promising evidence of reduced pain levels in patients receiving the
drug candidate. (Link)
Vyteris - has indicated that it is interested in disposing of a portfolio of products in development. These products
include a patch that delivers a fertility enhancing hormone. (Link)
Zelos - A better PTH analogue. Strong Phase 2 data. Company also pursuing an intranasal PTH and a potential
“biogeneric” PTH. (Link)
Page | 132

WOUND CARE AND TISSUE SEALANTS
Adocia - In a Phase 2 trial for the treatment of diabetic foot ulcers against Regranex with rhPDGF-BB spray. Interim
results show non-inferiority. (Link)
UPDATE $ Alliqua - Hydress +Plus™ received FDA approval for a silver-based wound dressing. Company open to
using an external marketing partner. (Link)
Auxano Biomedical / Emergent Bio - Developing SP-1 for wound repair along with some wound diagnostic tools.
(Link)
UPDATE $ Cohera Medical - Developing TissueGlu, a deep wound adhesive for use in surgical applications. This
product is currently approved in Europe and on the market in Germany. A PMA approval process is underway in
the United States.
$ Coloplast - Rumored to be considering disposal of wound care product line including Antimicrobials, foam
dressings and a fast gelling alginate.
UPDATE Derma Sciences - completed Phase 2 studies in diabetic foot ulcers of DSC127 which appears to stimulate
production of dermal adult progenitor cells, including mesenchymal stem cells, following injury. In June 2011,
Derma raised $29mm in a private placement financing. The company is now preparing to go into Phase 3 studies.
(Link)
FirstString Research - Bioengineered peptide based on a naturally occurring protein that accelerates wound healing
and tissue regeneration with significantly reduced scarring. Positive Phase 1 data reported in Sep 2010. (Link)
First Texas Medical - innovative portfolio of wound care and dermatology products.
UPDATE Glycotex - GLYC-101 is being developed to stimulate and modulate the natural cascade of wound healing
activities of several cell populations (including upregulation of TNF-alpha). The product candidate is a topical gel to
be applied directly on the wound surface. In May 2006, Glycotex completed a Phase 2 clinical trial of GLYC-101 in
Australia, in which GLYC-101 produced a statistically significant rate of wound area reduction versus combined
placebo and standard care in patients with chronic venous ulcers. In June 2011, positive results of another Phase 2
study were reported. (Link)
UPDATE $ Innocoll - CollaRx GENTAMICIN TOPICAL is a biodegradable and fully resorbable Gentamicin-Collagen
Sponge formulated and manufactured using Innocoll's proprietary collagen-based drug delivery technology,
CollaRx. Treats wound infections and shown in one Phase 2 trial to be superior to the current standard of care in
diabetic wounds. This product was recently approved as a surgical adhesion barrier in Europe. Company beginning
Phase 3 trials of this product in 2011. Note: Distribution partnerships have been initiated in 2011 for both Canada
and China. (Link)
UPDATE NB Therapeutics - Nitric oxide gas can be used for wound healing. Has been in a Phase 2 study but no
recent updates on clinical development of this product. (Link)
Pervasis - Developing Vascugel, a cell therapy product, to accelerate vein remodeling for patients receiving an AV
fistula (AVF) in end stage renal disease. Compare to Proteon Therapeutics which has optioned a product for the
same indication to Novartis. Update: In a Phase 2 trial. Results pending.
PharmaSurgics - in Phase 2 development for a pharmaceutical for anti-adhesion treatment after surgery. Very
promising approach. The Pergamum Operating unit located in Gothenburg - has received approval from the
German Regulatory Authority and the Independent Ethics Committee to begin a Phase 2 clinical trial in Germany.
(Link)
Page | 133

UPDATE ProFibrix - Developing a surgical tissue sealant that stops acute and severe bleeding. FibroCaps are a novel
powdered mixture of fibrinogen and thrombin. Phase 2 clinical testing is currently ongoing in the Netherlands
where Fibrocaps is used for the treatment of the mild to moderate bleeding during liver surgery. ProFibrix expects
to finalize the study shortly. Update: Entered into a component supply agreement with CSL Behring in June 2010.
Update: In July 5, 2011, ProFibrix raises US $11 million in series B financing for Fibrocaps. On Nov 15, 2011
reported that its Phase 2 trial met its primary endpoint (50% reduction in mean time to hemostatis versus actie
control). Company planning to start a Phase 3 study shortly.
Third Stream Bioscience - Developing a novel skin antimicrobial based upon a chemical composition developed by
Procter & Gamble. Promising data in dermatology (acne) and a variety skin cleansing applications.
Undisclosed - Large pharmaceutical company interesting in outlicensing a late stage program in wound care.

Page | 134

Torreya Partners LLC is a leading boutique investment banking firm that provides strategic advice and
assistance with Mergers & Acquisitions, Partnering and Financings to life science companies worldwide.
Torreya Partners has offices located in London, New York, Philadelphia and San Francisco.
TORREYA PARTNERS PERSONNEL
Name Title* Focus Area Geography Mobile Phone
Yonathan Belilty Analyst Pharmaceuticals London (44) 7500 780-305
Tom Bird Principal Specialty Pharmaceuticals New York (201) 893-1296
John Bradley Principal Specialty Pharmaceuticals New York (917) 648-3913
Nicole Clemenza Assistant Administrative Support New York (212) 331-7851
John Cullity, M.D. Principal Pharmaceuticals New York (646) 732-1023
Charles Dimmer III Principal Pharmaceuticals / Asia New York (646) 623-2966
Andrew Fineberg Principal Financings New York (917) 519-9521
Kristen Frederick CFO Administration New York (917) 912-0051
Wilder Fulford, Ph.D. Principal M&A / Pharmaceuticals London (44) 7979-603-498
Peter Garrambone Principal M&A / Pharmaceuticals New York (917) 742-5017
Benj Garrett Associate Analytical Support New York (412) 965-6008
Jeremy Goldberg Mng Dir Specialty Pharmaceuticals Philadelphia (800) 574-1741
Rodolphe Grepinet Principal M&A / Pharmaceuticals London (44) 7786-276-280
David Holbrook Principal Pharmaceutical Services New York (203) 246-2629
Stephanie Leouzon Advisor Biotechnology & Pharma London (44) 7974 566-739
Monica Karn MA Assistant Administration London (44) 7500-778-976
Tim Opler Ph.D. Principal Pharmaceuticals New York (650) 862-3029
Biliana Rajevic Vice-Pres Financings New York (917) 455-9841
Alan Selby, M.D., Associate Pharmaceuticals / Europe London (44) 7500-781-222
Mark Simon Principal Biotechnology New York (917) 796-0111
Tom Stack-Babich Analyst Analytical Support New York (646) 919-5797
Tim Wilson Ph.D. Principal Biotechnology London (44) 7966 156-143
Page | 135

RECENT TRANSACTIONS WHERE TORREYA ACTED AS FINANCIAL ADVISOR

Client

Month

Value

Transaction Description
Fund group December 2011 $25mm Acquisition of royalty-linked securities
U.S. Specialty
Pharma
November 2011 Undisclosed Acquisition of a dermatology Rx product
IT / Healthcare
Company
November 2011 Undisclosed Completion of bridge financing
U.S. Specialty
Pharma
October 2011 Undisclosed Sale of pre-natal vitamin line
Biotech
company
October 2011 Undisclosed Pain product licensing transaction
Amorcyte October 2011 $20mm Sale of cardiac cell therapy company to Neostem Inc.
Ventrus Bio July 2011 $52mm Financial Advisor in a follow-on equity financing
Predictive Edge July 2011 Undisclosed Equity financing for a novel IT company with significant
pharmaceutical industry applications.
Two fund
groups
June 2011 $110mm Sale of pharmaceutical product royalty interests on behalf of two
fund groups.
Global Pharma
Player
June 2011 Undisclosed Sale of an approved hospital drug with global distribution
Specialty
Pharma Player
June 2011 Undisclosed Co-promotion agreement in the pain area for a marketed
product in the U.S.
Aegera
Therapeutics
May 2011 Undisclosed Sale of the company to Pharmascience
Thorne
Research
May 2011 Undisclosed Strategic alliance in oncology with Helsinn
Global fund
group
April 2011 $487.5 mm Sale of Cubicin and Lexiscan royalty interests to Royalty Pharma.
Cynapsus
Therapeutics
April 2011 Undisclosed Fairness opinion in the acquisition of Adagio Pharmaceuticals
Lpath, Inc. December 2010 Up to $504mm
(14mm upfront)
License of iSonep antibody to Pfizer for ophthalmology using an
option structure.
Quinnova
Pharma
December 2010 Undisclosed Sale of company to Amneal LLC for an undisclosed price.
Financial
Players
Nov / Dec 2010 Undisclosed Multiple secondary sales of royalty bonds.
Three Rivers
Pharma
October 2010 $100mm + Sale of company to Kadmon, Inc.
Actient Pharma
/ GTCR
July 2010 Undisclosed Purchase of products with $54mm in revenue from UCB to forma
new specialty pharma company.
Columbia
Laboratories
July-10 $108mm +
royalties
License of progesterone business to Watson Pharmaceuticals
Dov Pharma July 2010 $2mm Fairness opinion in sale to Euthymics
DNP Health June 2010 Undisclosed Merger with Thorne Research, a $30mm revenue player in
neutraceuticals.
Undisclosed
Biotech
January-10 $100+mm License of anti-infective program to global player with profit split.
Athyrium
Capital
December-09 $15mm Purchase of royalty on approved cardiovascular drug.
Undisclosed December-09 $400+mm In-license of an investigational hospital drug.
Ikonisys December-09 Undisclosed Partnership of OncoFISH Cervical with Enzo
Page | 136

Nabi Biopharma November-09 $48mm Sale of Pentastaph to GlaxoSmithKline (with milestones)
Athyrium
Capital
September-09 $10mm Purchase of Telaprevir Milestone Payments from Vertex
Introgen
Therapeutics
September-09 $31 + mm
royalties
Sale of rights to drug pipeline to asset management company.
Teikoku Pharma

May-09 Undisclosed Purchase of Travanti Pharma
Introgen
Therapeutics
April-09 Undisclosed Sale of ITS to Western General (later sold to Lonza) and IP
Portfolio to Crucell.
Investor Group March-09 $47,000mm Advisor to Genentech minority investor group in Roche
acquisition.
Aradigm February-09 $4.5mm Financial Advisor in Registered direct financing.
AGI Dermatics September-08 Undisclosed Sale fo Estee Lauder Companies
Portola
Pharmaceuticals
July-08 $60mm Financial Advisor in Series D Preferred Financing
Navitas Pharma May-08 Undisclosed Sale to Gilead
ProEthic
Pharma
March-08 60mm Sale to Kowa
Midlothian
Laboratories
December-07 7mm Sale to Hi-Tech Pharmacal

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