Pipeline updates are highly awaited events in the pharma/biotech sector as they play an important role in deciding whether or not to invest in a particular company. These updates provide information on experimental drugs and at times provide an insight into the commercial potential of the candidate once it is successfully developed and commercialized.
Earlier in the week, Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) initiated a phase I study on ALN-AT3 for the treatment of hemophilia and rare bleeding disorders (RBD). ALN-AT3 is a subcutaneously administered ribo nucleic acid interference (RNAi) therapeutic targeting antithrombin (AT).
The phase I study on ALN-AT3, to be conducted in the U.K., is a single- and multi-dose, dose-escalation study consisting of two parts. The randomized, single-blind, placebo-controlled, single-dose initial part of the study (n~24) is evaluating the safety and tolerability of a single low dose of ALN-AT3.
The latter part of the trial is 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 is assessing the safety and tolerability of multiple doses of ALN-AT3 in these patients. Alnylam is expecting initial results from the study late this year.
ALN-AT3 was granted orphan drug designation for both hemophilia A and B by the U.S. Food & Drug Administration (FDA) in August last year. Earlier in the month, Alnylam entered into a broad multi-product, geographic alliance with Sanofi (SNY - Free Report) . As per the deal, Sanofi has the option to either co-develop and co-promote the candidate in North America and Western Europe with Alnylam or to commercialize the candidate in the rest of world.
We note that ALN-AT3 is one of the important programs under “Alnylam 5x15”. Earlier in the month Alnylam announced that it intends to move six to seven genetic medicine programs in the clinic by 2015 instead of five genetic medicine programs. ALN-AT3 is one of these programs.
Alnylam has progressed well with its pipeline in the past few quarters. We expect investor focus to remain on the company’s pipeline going forward.
Alnylam presently carries a Zacks Rank #2 (Buy). Some other stocks worth considering include Actelion Ltd. and Affymetrix Inc. . Both stocks carry a Zacks Rank #1 (Strong Buy).