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Alnylam Reports Positive Data on Fitusiran, Givosiran

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Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) announced new positive results from an ongoing phase II open-label extension (OLE) study on fitusiran in the treatment of patients with hemophilia A or B, without inhibitors.

A look at Alnylam’s year-to-date share price movement shows that the stock has underperformed the Zacks classified Medical-Biomed/Genetics industry. Share price tumbled 55.8% year to date, compared with the industry’s 25.6% decline.



Coming back to the latest news, data from the study revealed that once-monthly subcutaneous administration of fitusiran led to consistent reduction of antithrombin (AT) and increase in thrombin generation, resulting in a median-estimated annualized bleeding rate (ABR) of 1.0. The candidate was generally well tolerated with no thromboembolic events reported. The company expects to report data from the study at least once every year.

Results were presented at the annual meeting of the American Society of Hematology.

Alnylam also presented positive interim results from Part D of its ongoing phase I study on fitusiran in patients with hemophilia with inhibitors.

The company expects to begin a phase III clinical program on the candidate in early 2017.

We note that Alnylam has a collaboration agreement with Genzyme, a Sanofi (SNY - Free Report) company, for fitusiran.

Moreover, Alnylam announced positive initial results from Cohorts 1 and 2 of Part C of a phase I study on givosiran (ALN-AS1) for the treatment of acute hepatic porphyrias. Findings demonstrated robust and durable reduction of aminolevulinic acid and porphobilinogen, the toxic heme intermediates that are believed to mediate porphyria symptoms and acute attacks.

The company plans to meet with regulatory authorities with the goal of advancing givosiran into a phase III study in late 2017.

Alnylam currently has Zacks Rank #3 (Hold).

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