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Alnylam Pharmaceuticals, Inc. (ALNY - Analyst Report) earned a $7 million milestone payment from partner, Sanofi (SNY - Analyst Report) following the successful completion of a phase II study on their candidate, patisiran (ALN-TTR02).

Patisiran is a sub-cutaneous ribo nucleic acid interference (RNAi) therapeutic targeting the transthyretin (TTR) gene that is being developed for the treatment of transthyretin-mediated amyloidosis (ATTR).

Results from the study revealed that multiple doses of patisiran helped in a robust and statistically significant reduction of serum TTR protein levels by up to 96%. The mean levels of TTR reduction were more than 85%. The candidate was generally safe and well tolerated in this study.

Alnylam is currently evaluating patisiran in the phase III APOLLO study in ATTR patients suffering from familial amyloidotic polyneuropathy (FAP). The primary objective of the study is to measure the difference in the change in mNIS+7 score from baseline between patients under patisiran treatment compared to those under placebo.

We note that Alnylam has a development and commercialization agreement with Sanofi 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 candidates under the ALN-TTR program in other countries as well.

We are encouraged by the pipeline progress at Alnylam, especially with the company’s ATTR program. 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, a biopharmaceutical company, carries a Zacks Rank #4 (Sell). Some better-ranked stocks in the same sector include Actelion Ltd. (ALIOF) and Jazz Pharmaceuticals (JAZZ - Analyst Report). Both stocks hold a Zacks Rank #1 (Strong Buy).

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