Alnylam Pharmaceuticals, Inc. (ALNY - Analyst Report) recently presented complete results from a phase IIb trial on ALN-RSV01 at the European Respiratory Society’s (ERS) Annual Meeting. The candidate failed to achieve the primary end point of the phase IIb trial.
However, the candidate demonstrated significant treatment effect. Alnylam will provide an update on its development plans for ALN-RSV01’s by the end of 2012 after discussing the complete results from the trial with regulatory authorities.
The randomized, double-blind, placebo-controlled phase IIb trial enrolled 87 patients. The reduction in the occurrence of new or progressive bronchiolitis obliterans syndrome (BOS) at 180 days was the primary endpoint of the trial, which the candidate failed to achieve. However, the candidate showed a statistically significant reduction in BOS at 90 days and was found to be generally safe and well tolerated in the trial.
Alnylam has collaborated with Kyowa Hakko Kirin Co. for the development of ALN-RSV01 in Asia and with Cubist Pharmaceuticals Inc. in the rest of the world.
Alnylam makes use of the potentially revolutionary RNAi technology, a natural cellular process for selectively turning off the activity of genes, to build a pipeline to treat a wide array of diseases. In January 2011, Alnylam launched its “Alnylam 5X15” strategy aimed at developing RNAi therapeutic products for the treatment of genetically defined diseases addressing major unmet medical needs.
Alnylam expects to have five such RNAi therapeutic programs in advanced clinical development by the end of 2015. We expect investor focus to remain on the company’s pipeline progress.
The major programs under development are ALN-PCS (hypercholesterolemia), second generation ALN-TTR (transthyretin/TTR-mediated amyloidosis/ATTR), ALN-HPN (refractory anemia), ALN-AT3 (hemophilia) and ALN-TMP (hemoglobinopathies).
Alnylam also has collaborations or deals with players, such as Novartis AG (NVS - Analyst Report) , Biogen Idec Inc. (BIIB - Analyst Report) and Roche Holding AG (RHHBY - Analyst Report) to further develop and utilize its core technology.
The stock carries a Zacks #3 Rank (Hold rating) in the short run.