Alnylam Pharmaceuticals, Inc. ALNY announced encouraging results from its phase II open-label extension (OLE) study on patisiran. Results are being presented at the annual meeting of the American Academy of Neurology. Alnylam’s shares gained 1.7% following the news.
Patisiran, one of the most advanced candidates at Alnylam, is a subcutaneously administrated ribo nucleic acid interference (RNAi) therapeutic being developed for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR) in patients suffering from familial amyloidotic polyneuropathy (FAP).
Results from the study revealed that a mean reduction of 2.5 point in modified Neuropathy Impairment Score (mNIS+7) was observed at 12 months in patients treated with patisiran which compared favorably to the 13 to 18 point increase in mNIS+7 at 12 months from analysis of historical data sets of untreated FAP patients.
Results further revealed a reduction in serum TTR protein levels by up to 88% for approximately 16 months. The mean levels of TTR reduction were around 80% for the period. The candidate was also generally well tolerated in this study.
Alnylam is continuing with the phase II open-label extension study on patisiran to evaluate the long-term safety and tolerability of patisiran in ATTR patients suffering from FAP. The initial results from this study have been encouraging. The company plans to report 18-month OLE data in late 2015.
Alnylam is also evaluating the candidate in the global phase III APOLLO study (n=200) in FAP patients. The study is comparing patisiran with placebo with a primary endpoint of change in the mNIS+7 measurements from baseline. The company is enrolling patients for the study.
We believe that the successful development of patisiran is crucial for the company, which currently has no marketed product. We expect investor focus to remain on the candidate going forward.
We remind investors that Alnylam is in collaboration with Sanofi SNY for the development and commercialization of RNAi therapeutics across the world.
Alnylam carries a Zacks Rank #2 (Buy). Among the better-ranked stocks in the health care sector are Amgen Inc. AMGN and Biogen Idec Inc. BIIB. Both carry a Zacks Rank #1 (Strong Buy).
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