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Alnylam Reports Positive Initial Phase I Data on ALN-AS1

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Alnylam Pharmaceuticals, Inc.ALNY announced positive initial data from an ongoing phase I study on ALN-AS1 for the treatment of acute hepatic porphyries.

The phase I study is being carried out in three parts - A, B and C - on asymptomatic high excreter (ASHE) patients, who carry the genetic mutation of acute intermittent porphyria (AIP) and have elevated levels of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic heme intermediates that mediate porphyria attacks.

Initial data showed that a single subcutaneous dose administration of ALN-AS1 resulted in a reduction of up to 82% in ALA and up to 93% in PBG. The candidate was found to be well-tolerated. These data were presented at the International Congress of Porphyrins and Porphyrias (ICPP).

Alnylam has now initiated part B of the study on ALN-AS1 and expects to present initial data in 2016. The company targets to initiate part C on AIP patients suffering from recurrent attacks in early 2016.

In addition, the company presented initial data from the EXPLORE study, which is being conducted on patients with hepatic porphyrias suffering from recurrent attacks. Initial data from the study provided meaningful insights into the natural history and substantial disease burden of patients. Additional data from the study is expected in 2016.

We note that Alnylam has a collaboration agreement with Genzyme, a Sanofi SNY company, for ALN-AS1.

Other players in acute hepatic porphyries treatment market include Panhematin and Normosang.

Meanwhile, the company continues to progress on its pipeline. Currently, Alnylam has two phase III candidates, patisiran and revusiran, for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropathy and familial amyloidotic cardiomyopathy, respectively.

Alnylam carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Gilead Sciences Inc. GILD and Regeneron Pharmaceuticals, Inc. REGN , each sporting a Zacks Rank #1 (Strong Buy).

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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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