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.
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Earlier in the week,
Alnylam Pharmaceuticals, Inc.
) 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
). 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
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
). Both stocks carry a Zacks Rank #1 (Strong Buy).