Pipeline Probe: Alnylam Pharmaceuticals, Inc. (ALNY)

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Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) again has two drugs in trials, one intravenous and one subcutaneous. ALN-TTR02 is expected to report top-line phase 2 results mid-year. ALN-TTRsc should report phase 1 results around the same time. Encouraging data would lead to the next phase trials to begin at the end of the year.

The company formed an alliance last fall with Sanofi SA (ADR) (NYSE:SNY) subsidiary Genzyme. Alnylam reserved most rights to develop and commercialize the ALN-TTR products but gave Genzyme the Japanese and Asian markets. Genzyme paid $22.5 million upfront and may pay out undisclosed milestone payments along the way. Alnylam would receive double-digit tiered royalties if approval comes in Genzymes territories.


Finishing the 5

ALN-AT3 is a pre-clinical hemophilia treatment that should launch a phase 1 trial in late 2013. Alnylam’s seeking a Asian-market partner for the drug.

ALN-AS1 treats acute intermittent porphyria — a rare metabolic disorder that can cause abdominal pain, motor weakness, and cognitive impairments. The drug’s only in the discovery phase but the company’s hoping to move the process into the clinic next year.

ALN-TMP is in the pre-clinical stage and Alnylam hopes to find a partner before moving into phase 1. The drug treats beta thalassemia, which is a genetic blood condition caused by improperly produced hemoglobin that leads to anemia.

Foolish final thoughts

How healthy are Alnylam’s pipes? It’s too soon to say. But the picture will continue to become clearer in the coming year, especially if the company sticks to its 5×15 plans.

For now, I’m adding Alnylam to my watchlist. Want to keep up with the latest news from the company? Add Alnylam to your free, personalized watchlist.


Brandy Betz has no position in any stocks mentioned. The Motley Fool has no position in any of the stocks mentioned.
Brandy is a member of The Motley Fool Blog Network — entries represent the personal opinion of the blogger and are not formally edited.

The article Pipeline Probe: Alnylam originally appeared on Fool.com is written by Brandy Betz.

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