|Healthcare Review: Alnylam, Ariad Pharmaceuticals, Myriad Genetics, Rosetta Genomics, MannKind Corporation|
|By Staff and Wire Reports|
|Wednesday, 02 April 2014 14:01|
EMA's Committee for Orphan Medicinal Products recommends orphan drug status for Alnylam's ($ALNY -4.9%) transthyretin-mediated amyloidosis treatment ALN-TTRsc. The product is currently in a 15-patient Phase 2 clinical trial to evaluate its tolerability, assess its preliminary clinical activity in terms of knockdown TTR levels and additional exploratory tests. European orphan drug status applies to treatments for diseases affecting no more than 5 in 10,000 people where no effective treatment is available.
Ariad Pharmaceuticals ($ARIA) has appointed pharmaceutical veteran Hugh Cole as Senior VP and Chief Business Officer, responsible for global business and corporate development, licensing and strategic planning. Cole has over 25 years of experience in the industry, including as a senior VP at Shire Pharmaceuticals.
Myriad Genetics ($MYGN) shares jump 13.2% to $39.78 after news that Medicare payments for its genetic screening tests will be cut, but not nearly as much as initially expected. As a result, Jefferies analyst Brandon Couillard has lifted his price target on Myriad to $33 from $30, although he's maintained a Hold rating.
Pico caps Rosetta Genomics ($ROSG) and Marina Biotech ($MRNA) establish a strategic collaboration to identify and develop microRNA-based products targeting neuromuscular diseases and dystrophies. Initial focus will be Becker and Duchenne muscular dystrophies and myotonic dystrophy. Rosetta will identify microRNAs associated with the diseases and, if correlative, will develop the diagnostic products. Marina will develop the therapeutics.
MannKind Corporation (NASDAQ:MNKD)’s shares surged 75.05% to $7.04. The company on Apr. 1 announced that the Endocrinologic and Metabolic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) voted 13 to 1 to recommend that AFREZZA(R) (insulin human [rDNA origin]) Inhalation Powder be granted marketing approval by the FDA to improve glycemic control in adults with type 1 diabetes and voted 14 to 0 to recommend that AFREZZA be granted marketing approval by the FDA to improve glycemic control in adults with type 2 diabetes. If approved, AFREZZA would be the first ultra rapid-acting mealtime insulin therapy available in the United States.