Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) recently announced positive top-line results from a phase I study on ALN-TTRsc. The news positively impacted Alnylam share price by 15.8%.
ALN-TTRsc is a 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 reducing serum TTR protein levels to more than 80% in healthy volunteers who were enrolled in the study. The recent study results were at par with the previously reported ALN-TTRsc results in non-human primates.
Results from the phase I study further revealed that ALN-TTRsc was generally safe and well tolerated. The randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study is still ongoing. Alnylam will assess the safety and tolerability of the candidate (single or multiple ascending subcutaneous doses) along with other parameters like assessment of clinical activity of the candidate as measured by serum TTR levels.
Alnylam plans to initiate a phase II study on ALN-TTRsc later this year depending on the successful completion of the phase I study. Positive results from the phase II study will lead to the initiation of a phase III study on the candidate in 2014.
We remind investors that Alnylam also has ALN-TTR02 in its pipeline that is being developed for the treatment of ATTR. Alnylam is currently evaluating the safety and tolerability of the candidate in a phase II study.
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 pipeline progress at Alnylam. The company is expecting several study results on its RNAi therapeutic candidates in the coming quarters. Positive pipeline related news will boost the stock.
Alnylam, a biopharmaceutical company, presently carries a Zacks Rank #1 (Strong Buy). Other biopharmaceutical stocks such as Jazz Pharmaceuticals Public Limited Company (JAZZ - Free Report) and Santarus, Inc. also look well positioned with both carrying a Zacks Rank #1.