Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) has progressed on its pipeline in the recent past, especially with its transthyretin-mediated amyloidosis (ATTR) program. Alnylam’s share price has been exhibiting an upward trend over the last few days primarily due to pipeline related news.
Alnylam has ALN-TTR02, ALN-TTR01 and ALN-TTRsc in its ATTR program, which are sub cutaneous ribo nucleic acid interference (RNAi) therapeutic targeting the transthyretin (TTR) gene that is being developed for the treatment of ATTR.
Earlier this week, the company announced that it has completed enrollment for its phase II study on ALN-TTR02. Interim results from the study revealed that treatment with ALN-TTR02 resulted in robust and statistically significant reduction in TTR – the disease-causing protein – by up to 93%. The candidate was generally safe and well tolerated in this study as well.
Alnylam also initiated an open-label extension (OLE) study on ALN-TTR02. The OLE study will be evaluating the safety and tolerability of the candidate along with the measurement of effects of treatment toward several clinical endpoints. Initial data from this study is expected in 2014.
Moreover, the company remains on track to initiate a phase III study on ALN-TTR02 in familial amyloidotic polyneuropathy (FAP) patients by Dec 31, 2013. The primary objective of the study is to measure the difference in the change in the mNIS+7 score from baseline between patients under ALN-TTR02 treatment compared to those under placebo.
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. 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 Jazz Pharmaceuticals Public Limited Company (JAZZ - Free Report) and Actelion Ltd. currently look better positioned with a Zacks Rank #1 (Strong Buy).