Alnylam Files Clinical Trial Application for ALN-PCS, an RNAi Therapeutic Targeting PCSK9 for the Treatment of Severe Hypercholesterolemia

Alnylam Pharmaceuticals, Inc. ALNY announced today that it has filed a Clinical Trial Application with the Medicines and Healthcare products Regulatory Agency to initiate a Phase I clinical trial with ALN-PCS, an RNAi therapeutic for the treatment of severe hypercholesterolemia. Upon receiving clearance of the CTA, Alnylam plans to initiate the Phase I trial and expects to present initial safety, tolerability, and clinical activity data from this study by the end of this year. “The filing of this CTA marks an important milestone in our Alnylam 5x15 product efforts, as it is the first program using our second generation lipid nanoparticle technology to enter clinical testing, where we aim to have important safety, tolerability, and clinical activity data by year's end. Moreover, we are excited about the potential for ALN-PCS to make an impact in the treatment of severe hypercholesterolemia as this RNAi therapeutic targets both intracellular and extracellular PCSK9, a target validated by human genetics that is known to play a central role in LDL cholesterol metabolism,” said Akshay K. Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. “We continue to execute on our Alnylam 5x15 initiative, which is focused on advancing innovative RNAi therapeutics that address genetically defined targets and diseases with high unmet medical need. ALN-PCS represents the second of such programs, following ALN-TTR01 which is currently in a Phase I trial with data expected later this quarter.”