The U.S. Food & Drug Administration (FDA) recently granted
Orphan Drug Designation to
Alnylam Pharmaceuticals, Inc.
) ALN-AT3 for the treatment of hemophilia B. ALN-AT3 is a
subcutaneously administered ribo nucleic acid interference (RNAi)
therapeutic, which targets antithrombin (AT). The candidate is
being developed for the treatment of hemophilia (hemophilia A,
hemophilia B, and hemophilia A or B) and other rare bleeding
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Alnylam now plans to file an investigational new drug (IND)
application for the candidate by the end of this year and also
intends to initiate a phase I study on ALN-AT3 by early next
We note that the Orphan Drug Designation is given to those drugs
and biologics that are meant for the safe and effective
treatment, diagnosis or prevention of rare diseases that affect
not more than 200,000 people in the U.S.
We are impressed by Alnylam's efforts to develop its pipeline. In
Jul 2013, the company announced positive top-line results from a
phase I study on ALN-TTRsc. ALN-TTRsc is a RNAi therapeutic
targeting the transthyretin (TTR) gene that is being developed
for the treatment of TTR-mediated amyloidosis (ATTR).
Alnylam plans to initiate a phase II study on ALN-TTRsc (familial
amyloidotic cardiomyopathy) later this year depending on the
successful completion of the phase I study. Positive results from
the phase II study will lead to the initiation of a phase III
study on the candidate in 2014. Alnylam also reported positive
data from a phase II study on ALN-TTR02, another candidate in its
pipeline being developed for the treatment of ATTR.
Moreover, the company is expecting several study results based on
its RNAi therapeutic candidates in the coming quarters. We expect
investor focus to remain on the development of its pipeline.
Alnylam, a biopharmaceutical company, presently carries a Zacks
Rank #3 (Hold). Other biopharma stocks, such as
Alexion Pharmaceuticals, Inc.
Anthera Pharmaceuticals, Inc.
) are comparatively well placed. While Actelion carries a Zacks
Rank #1 (Strong Buy), Alexion and Anthera carry a Zacks Rank #2