Alnylam Pharmaceuticals, Inc.
) 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.
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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
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
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
) for its RNAi therapeutics is also encouraging.
Alnylam presently carries a Zacks Rank #4 (Sell). Stocks such as
) currently look better positioned with a Zacks Rank #1 (Strong