BioMarin Initiates Phase 3 Trial for PEG-PAL; Elan To Proceed with Formal Sale Process Print E-mail
By Staff and Wire Reports   
Friday, 14 June 2013 18:38

Below is a look at some of the headlines for companies that made news in the healthcare sector on June 13, 2013. BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has initiated the Phase 3 program for PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase) for the treatment of phenylketonuria (PKU).

"In the Phase 2 trial, PEG-PAL was shown to dramatically reduce blood Phe levels, and we are hopeful that we will achieve the same outcome with the Phase 3 program," stated Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "Adult patients with PKU and patients on the severe end of the disease spectrum still represent a very high unmet medical need. With PEG-PAL, it may be possible to provide a treatment benefit to this population."

The Phase 3 study (165-301) is an open-label, multi-center study to assess the safety and tolerability of an induction, titration and maintenance dose regimen of PEG-PAL self-administered by approximately 90 naïve adult PKU subjects. The primary objective of the 165-301 study is to characterize the safety and tolerability of PEG-PAL during induction, titration, and maintenance dosing. The secondary objective of the study is to evaluate blood Phe levels during induction, titration, and maintenance dosing.

After completion of the open label 165-301 study, subjects are expected to enroll in 165-302, a Phase 3 double-blind, placebo-controlled, randomized discontinuation study to evaluate the efficacy and safety of PEG-PAL self-administered by adults with PKU. The study will also enroll approximately 60 subjects from the Phase 2 program who are currently being treated with PEG-PAL. The primary objective of the 165-302 study is to evaluate blood Phe levels. The secondary objective of this study is to evaluate changes in neuropsychiatric assessments as measured by the Inattentive portion of the Attention Deficit and Hyperactivity Disorder Rating Scale (ADHD-RS) and the Profile of Mood States (POMS). These will be administered at baseline, four and eight weeks.


Elan Corporation, plc (NYSE:ELN)
announced it is proceeding with a formal sale process in light of the expressions of interest received to date. As part of this process, Royalty Pharma will be invited to participate if they so wish. Accordingly, shareholders are urged not to tender into the current Royalty Pharma hostile offer.

As previously stated, the Elan Board and management are aligned in maximizing the full value potential of the business on behalf of its shareholders. We will update the market as appropriate.

Elan’s financial advisors are Citigroup, Davy Corporate Finance, Morgan Stanley and Ondra Partners. Its legal advisors are A&L Goodbody and Cadwalader, Wickersham & Taft LLP.

Also Friday:

today announced that the U.S. Food and Drug Administration (FDA) has approved a new indication for XGEVA® (denosumab) for the treatment of adults and skeletally mature adolescents with giant cell tumor of bone (GCTB) that is unresectable or where surgical resection is likely to result in severe morbidity.

Astex Pharmaceuticals, Inc. (Nasdaq:ASTX)
, a pharmaceutical company dedicated to the discovery and development of novel small molecule therapeutics, today announced that updated clinical results of its novel hypomethylating agent, SGI-110, were presented in a poster session at the 18th Congress of the European Hematology Association (EHA) being held June 13-16, 2013 in Stockholm, Sweden.

AstraZeneca (NYSE: AZN)
and Bristol-Myers Squibb Company (NYSE: BMY) announced today that 33 abstracts on the companies’ research in diabetes have been accepted for presentation at the 73rd Scientific Sessions® of the American Diabetes Association (ADA) in Chicago, June 21-25.

Biozoom (OTC QB: BIZM)
exercised its option, under a joint venture agreement, to acquire 19.9% of Opsolution, a research and development company dedicated to advancing spectroscopic technology.

Bristol-Myers Squibb Company (NYSE: BMY)
and Simcere Pharmaceutical Group (NYSE: SCR), a leading pharmaceutical company in China, today announced that the companies have expanded their strategic relationship formed in 2010.

Cempra, Inc. (Nasdaq: CEMP)
, a clinical-stage pharmaceutical company focused on developing differentiated antibiotics to meet critical medical needs in the treatment of bacterial infections, today announced its intention to offer and sell shares of its common stock in an underwritten public offering pursuant to its existing shelf registration statement.

Cerner (Nasdaq:CERN)
, a developer of information management systems designed to achieve better efficiencies and better outcomes in health care, recently announced the winners of the Cerner Advancing Clinical Excellence (ACE) Awards 2013 during an award ceremony in Toronto.

Codexis (NASDAQ: CDXS)
, a developer of engineered enzymes for pharmaceutical, biofuel and chemical production, and Chemtex, a leader in chemical engineering and renewable processes, today announced the successful scale-up in the production of CodeXol® detergent alcohols using cellulosic sugars.

, a global leader in therapeutic ultrasound, announced today the full redemption of its $8.0 million outstanding long-term debt by using a portion of the net proceeds from the $12.0 million private placement executed on May 28, 2013.

Eli Lilly and Company (NYSE: LLY)
today announced it has stopped its Phase II study (BACC) for LY2886721, a beta secretase (BACE) inhibitor being investigated as a once daily treatment for its potential to slow the progression of Alzheimer's disease.

Enanta Pharmaceuticals, Inc., (NASDAQ: ENTA)
, a research and development-focused biotechnology company dedicated to creating small molecule drugs in the infectious disease field, today announced that Jay R. Luly, Ph.D., President and Chief Executive Officer, will present a corporate overview followed by a Q&A session and participate in a Hepatitis C panel discussion on June 18, 2013 at the Wells Fargo Securities 2013 Healthcare Conference in Boston, MA.

Hydromer, Inc. (OTC Pink: HYDI)
and LATECBA S.A., an Argentinean company, have entered into a Supply and Support Agreement.

Immunomedics, Inc. (Nasdaq:IMMU)
, a biopharmaceutical company primarily focused on the development of monoclonal antibody-based products for the targeted treatment of cancer, autoimmune and other serious diseases, today announced that the Company will present at the Wells Fargo Securities 2013 Healthcare Conference on Wednesday, June 19, 2013 at 1:50 pm Eastern Time at The InterContinental Hotel in Boston, MA.

InterMune, Inc. (NASDAQ: ITMN) today announced that the Board of the Italian Drug Agency (AIFA) has approved the pricing and reimbursement conditions for Esbriet® (pirfenidone), and that the agreement has been published in the Official Gazette, the official journal of the government of Italy.

LED Medical Diagnostics Inc. (TSX VENTURE:LMD) (OTCQX:LEDIF) (FRANKFURT:LME) announces that it has completed its previously announced non-brokered private placement of 17,000,000 units at an issue price of $0.15 per unit for gross proceeds of $2.55 million.

Mast Therapeutics, Inc. (NYSE MKT: MSTX)
today announced the pricing of an underwritten public offering of 50,000,000 units, each unit consisting of a fixed combination of one share of common stock and one warrant to purchase 0.5 of a share of common stock, at a public offering price of $0.50 per unit.

NPS Pharmaceuticals, Inc. (NASDAQ: NPSP)
, a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced that president and chief executive officer Francois Nader, M.D. has received the Ernst & Young Entrepreneur Of The Year® 2013 Award in the Life Sciences category in New Jersey.

pSivida Corp. (NASDAQ: PSDV)
, a leader in developing sustained release, drug delivery products for treatment of back-of-the-eye diseases, today announced that the United Kingdom’s National Institute for Health and Care Excellence (NICE) issued draft guidance recommending ILUVIEN® for the treatment of pseudophakic patients (those who have undergone prior cataract surgery) with chronic diabetic macular edema (DME) considered insufficiently responsive to available therapies.

PAREXEL International Corporation (NASDAQ: PRXL)
, a leading global biopharmaceutical services provider, today announced that Ingo Bank has been appointed Senior Vice President and will become Chief Financial Officer on September 1, after fiscal year-end audited financial statements are filed.

Pressure BioSciences, Inc. (OTCQB: PBIO)
announced that it has closed the third and final tranche of its Series J Private Placement of Convertible Preferred Stock and Warrants (the "Series J Private Placement").

PURE Bioscience, Inc.(OTCQB: PURE), creator of the patented silver dihydrogen citrate (SDC) antimicrobial, reported results for its fiscal third quarter ended April 30, 2013.

QLT Inc. (Nasdaq:QLTI) (TSX:QLT) is pleased to announce that at the annual general and special meeting (the "Meeting") held today, QLT's shareholders approved a special cash distribution in the amount of US$200 million, by way of a reduction of the capital of the Company's common shares (the "Shares").

REVA Medical, Inc. (ASX:RVA)
is pleased to announce that its Chairman and CEO, Robert Stockman, is presenting at the Wells Fargo Securities 2013 Healthcare Conference at 1:50 p.m. US EDT on Wednesday, June 19, 2013.

Royalty Pharma notes today's announcement by Elan Corporation, plc (NYSE: ELN) that it intends to explore a sale of the Company. Royalty Pharma believes it to be a clear frustrating tactic that offers shareholders no certainty that a deal will ever be reached.

Teva Pharmaceutical Industries Ltd. (NYSE: TEVA)
announced today that a number of abstracts will be presented during the 17th Annual International Congress of Parkinson’s Disease and Movement Disorders in Sydney, Australia, June 16-20, 2013, also known as the Movement Disorders Society (MDS).

TherapeuticsMD, Inc. (NYSE MKT: TXMD)
, a women’s healthcare company focused on developing and commercializing products targeted exclusively for women, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's Investigational New Drug (IND) application for TX12-004HR, a vaginal estradiol suppository.

Titan Medical Inc. (TSX VENTURE:TMD) (OTCQX:TITXF) announced today that effective June 1, 2013 it has entered into an agreement with OpX Consulting, LLC ("OpX").

"Featured Content" profiles are meant to provide awareness of these companies to investors in the small-cap and growth equity community and should not in any way come across as a recommendation to buy, sell or hold these securities. BiomedReports is not paid or compensated by newswires to disseminate or report news and developments about publicly traded companies, but may from time to time receive compensation for advertising, data, analytics and investor relation services from various entities and firms. Full disclosures should be read in the 'About Us Section'.

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