The stock price at Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) surged significantly last week following positive top-line data on its pipeline candidate ALN-TTRsc.
ALN-TTRsc is a ribo nucleic acid interference (RNAi) therapeutic targeting the transthyretin gene that is being developed for the treatment of TTR-mediated amyloidosis. The therapeutic is administered subcutaneously.
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.
Data from the study also 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.
ALN-TTR02 is another promising candidate in Alnylam’s pipeline. Last month, Alnylam announced positive interim results from a phase II study on ALN-TTR02. ALN-TTR02 is another RNAi therapeutic targeting the transthyretin gene for treating TTR-mediated amyloidosis.
We are encouraged by the pipeline progress at Alnylam. We are positive on the biopharmaceutical company despite the Zacks Rank #3 (Hold) carried by it. We believe that the 8% dip in Alynam’s price in yesterday’s trading on the Nasdaq has made it more affordable for investors after the lofty prices it touched last week. Consequently, we view the current price as an attractive entry point for long-term investors.
Biopharmaceutical stocks such as Jazz Pharmaceuticals Public Limited Company (JAZZ - Free Report) , Medivation, Inc. and Sarepta Therapeutics, Inc. (SRPT - Free Report) appear to be very attractive in the short-run with a Zacks Rank #1(Strong Buy).