Alnylam Pharmaceuticals, Inc. (ALNY) revealed encouraging initial results from an ongoing phase II open-label extension study (n=27) on one of its most important pipeline candidates, patisiran (ALN-TTR02). The stock soared almost 19% following the announcement.
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 decrease in modified Neuropathy Impairment Score (mNIS+7) was observed at six months in patients treated with patisiran compared to an increase in mNIS+7 in untreated FAP patients.
Results further revealed a robust and statistically significant reduction in serum TTR protein levels by up to 90% for more than nine months. The mean levels of TTR reduction were around 80% for the period. The candidate was also generally well tolerated in this study. These results are being presented at the 2014 annual meeting of the American Neurological Association.
Alnylam is continuing with the two-year 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.
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 objective of mNIS+7. 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.
Alnylam carries a Zacks Rank #2 (Buy). Some other stocks in the healthcare sector that are worth considering include Medivation, Inc. (MDVN), Emergent BioSolutions, Inc. (EBS) and Ligand Pharmaceuticals Incorporated (LGND). All three stocks hold a Zacks Rank #1 (Strong Buy).
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