Alnylam Pharmaceuticals, Inc.
) has progressed on its pipeline in the recent past, especially
with its transthyretin-mediated amyloidosis (ATTR) program.
Alnylam's share price has been exhibiting an upward trend over
the last few days primarily due to pipeline related news.
Alnylam has ALN-TTR02, ALN-TTR01 and ALN-TTRsc in its ATTR
program, which are sub cutaneous ribo nucleic acid interference
(RNAi) therapeutic targeting the transthyretin (TTR) gene that is
being developed for the treatment of ATTR.
Earlier this week, the company announced that it has completed
enrollment for its phase II study on ALN-TTR02. Interim results
from the study revealed that treatment with ALN-TTR02 resulted in
robust and statistically significant reduction in TTR - the
disease-causing protein - by up to 93%. The candidate was
generally safe and well tolerated in this study as well.
Alnylam also initiated an open-label extension (OLE) study on
ALN-TTR02. The OLE study will be evaluating the safety and
tolerability of the candidate along with the measurement of
effects of treatment toward several clinical endpoints. Initial
data from this study is expected in 2014.
Moreover, the company remains on track to initiate a phase III
study on ALN-TTR02 in familial amyloidotic polyneuropathy (FAP)
patients by Dec 31, 2013. The primary objective of the study is
to measure the difference in the change in the mNIS+7 score from
baseline between patients under ALN-TTR02 treatment compared to
those under placebo.
We note that Alnylam has a development and commercialization
) for Alnylam's RNAi therapeutics, which include ALN-TTR02 and
ALN-TTRsc. The agreement is for the development and
commercialization of these candidates in Japan and the broader
Asian-Pacific region. Alnylam intends to develop and
commercialize the ALN-TTR program in other countries as well.
We are encouraged by the pipeline progress at Alnylam. However,
most of the candidates at the company's pipeline are in the early
or middle stages of development. We expect investor focus to
remain on Alnylam's pipeline going forward.
Alnylam presently carries a Zacks Rank #3 (Hold). Stocks such as
Jazz Pharmaceuticals Public Limited Company
) currently look better positioned with a Zacks Rank #1 (Strong
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