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Alnylam Pharmaceuticals Inc recently announced its goal for the next two years (2013 and 2014) regarding its key “Alnylam 5x15” pipeline and partner program.

Alnylam’s core product strategy, ‘Alnylam 5x15,’ was introduced in January 2011. It aims to develop RNA (ribo nucleic acid) interference (RNAi) therapeutic products for the treatment of genetically defined diseases addressing major unmet medical needs.

Alnylam plans to advance its familial amyloidotic polyneuropathy (FAP) candidate, ALN-TTR02, into phase III clinical trials. We note that the candidate is currently in a phase II study, with results expected in mid-2013. Positive results from the phase II study would allow Alnylam to move the candidate into phase III development later this year.

Alnylam also intends to commence an open-label extension study on ALN-TTR02 in mid-2013 as a long-term treatment for patients participating in the ongoing phase II study. Periodic updates on the results from the extension study will be provided from 2014.

The company has a partnership with Genzyme, a Sanofi company, to commercialize the candidate in Japan and other countries in Asia, whereas Alnylam will commercialize the candidate in other parts of the world.

The company expects to complete phase I study on another candidate ALN-TTRsc, being developed for the treatment of patients suffering from familial amyloidotic cardiomyopathy (FAC). The company plans to move the candidate into pilot phase II study in late 2013. The company expects to initiate a phase III study on the candidate in 2014 after the successful phase II results.

Alnylam also plans to expand its ‘Alnylam 5x15’ pipeline with ALN-AT3 and ALN-AS1, which are being developed for the treatment of hemophilia and rare bleeding disorders (RBD) and acute intermittent porphyria (AIP), respectively. Alnylam also has plans to advance additional programs in ‘Alnylam 5x15’ through various partnership deals.

The company also plans to advance its support partner based programs in RNAi therapeutics. The company is developing ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection and ALN-VSP for the treatment of liver cancer, through partnerships.

Our Take

We remain positive on the company’s effort to develop its RNAi therapeutic products. We expect investor focus to remain on Alnylam’s pipeline. The company has collaborated with several companies including GlaxoSmithKline plc , Sanofi, AstraZeneca and Biogen Idec for microRNA therapeutics.

We currently have a Neutral recommendation on Alnylam. The stock carries a Zacks #3 Rank (Hold). However, other pharma stocks such as Targacept, Inc and Repligen Corporation carry a Zacks #1 Rank (Strong Buy).

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