Alnylam Pharmaceuticals, Inc. (ALNY) filed a Clinical Trial Application (:CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (:MHRA) for ALN-AT3. Alnylam intends to initiate a phase I study on ALN-AT3 for the treatment of hemophilia, including people with inhibitors, in early 2014.
ALN-AT3 is a subcutaneously administered ribo nucleic acid interference (RNAi) therapeutic, which targets antithrombin (AT). The phase I study on ALN-AT3, to be conducted in the U.K., will be a single- and multi-dose, dose-escalation study consisting of two parts. The initial part of the study will be a randomized, single-blind, placebo-controlled, single-dose, (n=24) and will be primarily evaluating the safety and tolerability of a single dose of ALN-AT3.
The latter part of the study will be an open-label, multi-dose, dose-escalation study (n=18) in patients suffering from moderate to severe hemophilia A or B. This part of the study will primarily assess the safety and tolerability of multiple doses of ALN-AT3 in these patients. Alnylam is expecting results from this study by the end of next year.
In Aug 2013, ALN-AT3 was granted orphan drug designation for both hemophilia A and B by the U.S. Food & Drug Administration (:FDA).
We note that ALN-AT3 is one of the important programs under “Alnylam 5x15”. Alnylam intends to move five RNAi therapeutic programs, including ALN-AT3, into clinical development, including programs in advanced stages by 2015.
Alnylam has progressed well with its pipeline in the past few quarters. Although most of the candidates at Alnylam are in their early-mid stages of development, we expect investor focus to remain on the company’s pipeline. Meanwhile, the company’s collaboration with companies like Novartis (NVS) and Roche (RHHBY) for its RNAi therapeutics is also encouraging.
Alnylam presently carries a Zacks Rank #4 (Sell). Stocks such as Actelion Ltd. (ALIOF) currently look better positioned with a Zacks Rank #1 (Strong Buy).