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Alnylam completes Phase II enrollment for ATTR

Will begin Phase III in FAP later this year

By: Gil Roth

President, Pharma & Biopharma Outsourcing Association

Alnylam Pharmaceuticals has completed enrollment in its Phase II trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). Interim results from this Phase II study showed that ALN-TTR02 achieved as mich as 93% knockdown of TTR. The candidate’s activity was found to be rapid, dose dependent, and durable, with similar levels of TTR knockdown observed toward both wild-type and mutant protein. In addition, ALN-TTR02 was found to be generally safe and well tolerated in this study.

Alnylam has opened for enrollment an open-label extension (OLE) study with ALN-TTR02 to evaluate the long-term safety and tolerability of the candidate and also measure effects of treatment toward a number of clinical endpoints, including a Neuropathy Impairment Score, or “NIS.” The company intends to report clinical data from this study about once per year, with initial data in 2014.

Alnylam plans to begin a Phase III study of ALN-TTR02 in familial amyloidotic polyneuropathy (FAP) patients by the end of this year

In 2012, Alnylam entered into an exclusive alliance with Genzyme, a Sanofi company, to develop and commercialize RNAi therapeutics, including ALN-TTR02 and ALN-TTRsc, for the treatment of ATTR in Japan and the broader Asian-Pacific region. Alnylam plans to develop and commercialize the ALN-TTR program in North and South America, Europe, and rest of the world.

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