The share price of
Alnylam Pharmaceuticals, Inc.
) shot up around 10.2% on Sep 23, 2013 to close the trading
session at $63.05. The stock was buoyed by the positive interim
results from a phase I study on its pipeline candidate ALN-TTRsc.
Alnylam also touched a 52-week high of $63.84 on Sep 23.
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ALN-TTRsc is a sub cutaneous ribo nucleic acid interference
(RNAi) therapeutic targeting the transthyretin (TTR) gene that is
being developed for the treatment of TTR-mediated amyloidosis
(ATTR). Results from the study revealed that ALN-TTRsc was
successful in robust, consistent, and statistically significant
reduction in serum TTR protein levels up to 94% in healthy
volunteers enrolled in the study.
Interim results also revealed that ALN-TTRsc was generally safe
and well tolerated in the study. Moreover, no serious adverse
effects were found during the study.
Alnylam mentioned in its press release that it is on track to
initiate a pilot phase II study on ALN-TTRsc in patients
suffering from familial amyloidotic cardiomyopathy (FAC) by year
end. Positive results from the phase II study will allow the
company to initiate a phase III study on the candidate by the end
We remind investors that Alnylam also has ALN-TTR02 in its
pipeline that is being developed for the treatment of ATTR
patients with FAP. Alnylam is currently evaluating the safety and
tolerability of the candidate in a phase II study. The company
plans to initiate a phase III study on the candidate in
polyneuropathy patients by Dec 31, 2013.
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 positive results from the phase I study.
Alnylam has progressed well so far with its RNAi therapeutics
candidates. 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
Alnylam presently carries a Zacks Rank #3 (Hold). Stocks such as
Biogen Idec Inc.
) currently look better positioned with a Zacks Rank #1 (Strong