Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) inked a collaboration deal with The Alpha-1 Project (TAP), the venture philanthropy subsidiary of the Alpha-1 Foundation, for the continued development of ALN-AAT. ALN-AAT is a subcutaneously administrated ribo nucleic acid interference (RNAi) therapeutic being developed for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease.
TAP works with patients, academia, pharmaceutical and biotech companies and other public health organizations for cures and therapies for chronic obstructive pulmonary disease (COPD) and liver disease caused by AAT deficiency.
The research and development activities for ALN-AAT will be partially funded by TAP and the rest by Alnylam. The company intends to file an investigational new drug (IND) application for ALN-AAT in mid 2015. Results from pre-clinical studies have shown that treatment with ALN-AAT resulted in approximately 90% knockdown of serum AAT.
As per company sources, there are around 10,000 to 20,000 people in the U.S. and EU respectively suffering from AAT deficiency with associated liver pathology.
Alnylam has made significant progress with its pipeline so far. Patisiran and ALN-TTRsc are two of the most advanced candidates at Alnylam. The company is evaluating patisiran in the phase III APOLLO study in transthyretin-mediated amyloidosis patients suffering from familial amyloidotic polyneuropathy. Alnylam intends to file for regulatory applications for the candidate later in the year.
Meanwhile, ALN-TTRsc is in a phase II study in patients suffering from familial amyloidotic cardiomyopathy. ALN-TTRsc is also an RNAi therapeutic targeting the TTR gene being developed for the treatment of ATTR. The company has multiple pipeline related activities in the coming quarters. We expect investor focus to remain on the development of the pipeline.
Alnylam, a biopharmaceutical company, presently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the sector include Regeneron Pharmaceuticals, Inc. (REGN - Free Report) , Gilead Sciences Inc. (GILD - Free Report) and Alexion Pharmaceuticals, Inc. (ALXN - Free Report) . While Gilead and Regeneron are Zacks Rank #1 (Strong Buy) stocks, Alexion holds a Zacks Rank #2 (Buy).