Alnylam Provides Pipeline Update, Growth Strategy – Analyst Blog

Alnylam Pharmaceuticals, Inc. ( ALNY ) announced its pipeline growth strategy for the development and commercialization of RNAi therapeutics across three strategic therapeutic areas (STArs) - genetic medicines, cardio-metabolic disease and hepatic infectious disease.

Alnylam's genetic medicine STAr consists of a broad pipeline of RNAi therapeutics including patisiran (phase III - APOLLO) and revusiran (phase III - ENDEAVOUR), being developed for the treatment of transthyretin-mediated amyloidosis. Additionally, the company reported positive initial data from a phase I study on ALN-AT3 last week.

The company is advancing ALN-AT3 for the treatment of hemophilia and rare bleeding disorders. Further, the company plans to initiate a phase I/II study on ALN-CC5 for paroxysmal nocturnal hemoglobinuria.

Meanwhile, Alnylam intends to commercialize its genetic medicine products in the U.S. and EU, while Genzyme, a Sanofi ( SNY ) company, will develop and commercialize in the rest of the world.

In its cardio-metabolic disease STAr, Alnylam recently initiated a phase I study on ALN-PCSsc (RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia) with initial data expected in mid-2015. Alnylam has an agreement with The Medicines Company ( MDCO ) for ALN-PCSsc. Alnylam is also advancing other candidates including ALN-AC3 (hypertriglyceridemia), ALN-ANG (hypertriglyceridemia and mixed hyperlipidemia) and ALN-AGT (hypertensive disorders of pregnancy including preeclampsia) among others.

Finally, Alnylam's hepatic infectious disease STAr includes ALN-HBV for the treatment of hepatitis B viral infection. The company intends to file an investigational new drug (IND) application or an IND equivalent in late 2015.

Alnylam is looking for partnerships for programs in its cardio-metabolic disease and hepatic infectious disease STArs. At the same time it intends to retain significant product commercialization rights in the U.S. and EU.

Alnylam expects to provide additional guidance on pipeline programs in its three STArs in Jan 2015. We expect investor focus to remain on Alnylam's pipeline.

Alnylam currently carries a Zacks Rank #2 (Buy). A better-ranked stock in the health care sector is Amgen Inc. ( AMGN ) carrying a Zacks Rank #1 (Strong Buy).

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Alnylam Provides Pipeline Update, Growth Strategy - Analyst Blog

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