The share price of Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) 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.
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 next year.
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 agreement with Sanofi (SNY - Free Report) 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 forward.
Alnylam presently carries a Zacks Rank #3 (Hold). Stocks such as Biogen Idec Inc. (BIIB - Free Report) and Actelion Ltd. currently look better positioned with a Zacks Rank #1 (Strong Buy).