Alnylam Pharmaceuticals, Inc. ALNY presented new positive data from its ongoing phase I study on ALN-AT3, the company’s investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), at the International Society on Thrombosis and Haemostasis 2015 meeting.
As per new interim data from the ongoing phase I study, ALN-AT3 showed potent and dose-dependent knockdown of AT of up to 86% in 12 hemophilia A and B patients. Additionally, AT knockdown was associated with statistically significant increases in thrombin generation with a mean increase of up to 350% with marked improvements in whole blood clotting.
Meanwhile, in an exploratory post-hoc analysis, it was found that ALN-AT3 reduced the frequency of bleeding at higher AT knockdown levels including a maximum bleed-free interval of 114 days. The candidate was generally well tolerated.
Based on encouraging data on the candidate, the company expects to expedite the advancement of ALN-AT3 and initiate pivotal studies in mid-2016.
The ongoing phase I study is being conducted in three parts. The company has already completed Part A and Part B of the study, while Part C is ongoing. The primary objective of the third part of the study is to evaluate the safety and tolerability of multiple doses of subcutaneously administered ALN-AT3 in hemophilia subjects.
We note that Alnylam has a collaboration agreement with Genzyme, a Sanofi SNY company, for ALN-AT3. As per the terms of the agreement, Alnylam retains rights to the candidate in North America and Western Europe, while Genzyme holds the right in rest of the world.
Alnylam currently has no approved product in its portfolio. We expect investor focus to remain on the company’s pipeline.
Alnylam carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are Valeant Pharmaceuticals International, Inc. VRX and Gilead Sciences Inc. GILD. Both sport a Zacks Rank #1 (Strong Buy).
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