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Alnylam Advances Development Candidate For ALN-AS1, A Subcutaneously Administered RNAi Therapeutic Targeting Aminolevulinate Synthase 1 (ALAS-1) For The Treatment Of Hepatic Porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP). The new pre-clinical research findings, presented at the 9 th Annual Meeting of the Oligonucleotide Therapeutics Society being held October 6 – 8, 2013 in Naples, Italy, show that subcutaneous administration of a GalNAc-siRNA targeting ALAS-1 leads to rapid, dose-dependent, and long-lasting knockdown of ALAS-1 mRNA and complete inhibition of the toxic intermediates that mediate the symptoms and pathology of AIP. Based on these findings, including results in non-human primate studies, the company has selected its ALN-AS1 Development Candidate and expects to file an Investigational New Drug (IND) application for this RNAi therapeutic in 2014. ALN-AS1 is part of the company’s “Alnylam 5x15” product development and commercialization strategy, in which the company aims to advance five genetic disease target programs into clinical development, including programs in late stages, by the end of 2015. In addition, the company presented new pre-clinical data with its proprietary, clinically validated GalNAc-siRNA conjugate delivery platform for subcutaneous delivery of RNAi therapeutics with a wide therapeutic index.

“We are very pleased to advance ALN-AS1 as a new Development Candidate in our ‘Alnylam 5x15’ pipeline, with the goal of filing an IND in 2014. Our new pre-clinical data show that subcutaneous administration of ALN-AS1 results in complete suppression of the toxic heme biosynthesis intermediates that cause the symptoms and pathology of AIP,” said Rachel Meyers, Ph.D., Vice President of Research and RNAi Lead Development at Alnylam. “ALN-AS1 now becomes our third RNAi therapeutic utilizing our GalNAc-siRNA conjugate delivery platform to enter development stages, extending our progress with ALN-TTRsc for the treatment of transthyretin-mediated amyloidosis, where we intend to soon initiate a Phase II trial, and ALN-AT3 for the treatment of hemophilia, where we plan on filing our IND by the end of this year. With the recent clinical validation of our GalNAc-siRNA conjugate delivery platform, we have increased confidence that ALN-AS1 could become a transformative therapy for patients with AIP, an ultra-rare genetic disease with enormous unmet medical need. We very much look forward to filing our IND for this program in 2014.”

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