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Alnylam Pharmaceuticals (ALNY) Completes Enrollment of Phase 2 Clinical Trial and Initiates Open-Label Extension (OLE) Study with ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR)


9/30/2013 9:41:26 AM

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today that it 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). Recent interim results from this Phase II study showed that ALN-TTR02 achieved up to 93% knockdown of TTR – the disease-causing protein in ATTR. ALN-TTR02 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 also announced today that its open-label extension (OLE) study with ALN-TTR02 is open for enrollment. The OLE study will evaluate the long-term safety and tolerability of ALN-TTR02 and will 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.

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