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Alnylam's Extension Study on Patisiran Reveals Positive Data

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Alnylam Pharmaceuticals, Inc.ALNY announced encouraging new 12-month data from an ongoing phase II open-label extension (OLE) study on patisiran for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) patients with familial amyloidotic polyneuropathy (FAP).

Patisiran was found to reduce pathogenic, misfolded TTR monomers and oligomers in FAP patients. A rapid and sustained reduction of about 90% in serum non-native conformations of TTR (NNTTR) was observed. Moreover, the results continue to support the hypothesis that patisiran has the potential to stop neuropathy progression in ATTR amyloidosis. Moreover, patisiran was generally well-tolerated out to 21 months of treatment.

The company expects to report initial 18-month data in early Nov 2015.

We note that Alnylam has a collaboration agreement with Genzyme, a Sanofi SNY company, for the development and commercialization of RNAi therapeutics. Under the terms of the agreement, Alnylam will be responsible for patisiran in North America and Western Europe, while Genzyme will be responsible for the rest of the world.

Patisiran is being evaluated in the phase III APOLLO study in ATTR patients with FAP.

According to information provided by the company, approximately 10,000 people are affected by FAP worldwide with a life expectancy of 5 years to 15 years from the onset of symptoms.

Meanwhile, Alnylam also announced a favorable ruling in an inventorship dispute related to Tuschl II patents.

Alnylam carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the health care sector are Emergent BioSolutions, Inc. EBS and Regeneron Pharmaceuticals, Inc. REGN , each sporting a Zacks Rank #1 (Strong Buy).

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REGENERON PHARM (REGN): Free Stock Analysis Report

SANOFI-AVENTIS (SNY): Free Stock Analysis Report

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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