Alnylam (ALNY) Files CTA to Commence ALN-TTRsc Phase I in U.K.

January 3, 2013 8:04 AM EST
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). ALN-TTRsc is a subcutaneously administered RNAi therapeutic that comprises an siRNA conjugated to a triantennary N-acetylgalactosamine (GalNAc) ligand. GalNAc-siRNA conjugates are a proprietary Alnylam delivery platform and are designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor. ALN-TTRsc is the first GalNAc-siRNA to enter clinical development stages. Following approval of the CTA, Alnylam expects to initiate a Phase I study with ALN-TTRsc early in 2013 with data expected to be reported in mid-2013.

“RNAi therapeutics hold great promise for the treatment of ATTR due to their ability of achieving rapid, potent, and durable knockdown of TTR, the disease-causing protein. At Alnylam, we are committed to advancing an industry leading effort for patients with ATTR. Accordingly we are advancing both ALN-TTR02, an intravenously administered RNAi therapeutic currently in a Phase II clinical trial in patients, and now ALN-TTRsc, a subcutaneously administered RNAi therapeutic,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “This CTA filing for ALN-TTRsc marks our first for an RNAi therapeutic that utilizes our proprietary GalNAc conjugate delivery platform enabling subcutaneous dose administration. Our pre-clinical studies with ALN-TTRsc have confirmed the ability of achieving over 80% TTR knockdown at single digit mg/kg doses with a very wide therapeutic index. We very much look forward to the continued advancement of ALN-TTRsc, including the start of this Phase I clinical trial in healthy volunteers early in the year with data expected mid-year.”

As per the filed CTA, the Phase I trial of ALN-TTRsc is planned to be conducted in the U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects. The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc. Secondary objectives include assessment of clinical activity of the drug as measured by serum TTR levels.

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