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Alnylam Submits CTA Application for ALN-HSD, an Investigational RNAi Therapeutic for the Treatment of Nonalcoholic Steatohepatitis (NASH)
– Company Expects to Initiate a Phase 1 Study in Late 2020 – CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Company Expects to Initiate a Phase 1 Study in Late 2020 –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company has submitted a clinical trial authorization (CTA) application to The Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom to initiate a Phase 1 study of ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 for the treatment of nonalcoholic steatohepatitis (NASH). The Company plans to initiate a Phase 1 study in late 2020, upon obtaining MHRA approval.\n\n\n“We are excited to advance our ALN-HSD program to the clinic in partnership with Regeneron, particularly given the mass prevalence and high unmet need in NASH, a progressive disease associated with high morbidity and mortality. Given the genetic validation of the target, we believe ALN-HSD has the potential to change the course of this serious and potentially life-threatening disease for which there are currently no approved pharmacologic treatment options,” said David Erbe, Ph.D., Senior Distinguished Investigator, Program Leader, ALN-HSD program at Alnylam. “Pending feedback from the MHRA, we look forward to initiating the Phase 1 study to evaluate the safety and preliminary pharmacodynamic activity of ALN-HSD in healthy volunteers and patients with NASH.”\n\n\nAbout ALN-HSD\n\n\nALN-HSD is an investigational, subcutaneously administered RNAi therapeutic targeting HSD17B13 in development in collaboration with Regeneron Pharmaceuticals for the treatment of nonalcoholic steatohepatitis. ALN-HSD utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables subcutaneous dosing with increased selectivity and a wide therapeutic index. The safety and efficacy of ALN-HSD have not been evaluated by the FDA, EMA or any other health authority.\n\n\nAbout NASH\n\n\nNonalcoholic steatohepatitis (NASH) is a highly prevalent chronic liver disease in which inflammation and liver cell injury are caused by accumulation of hepatic fat. NASH is a subset of a group of conditions called nonalcoholic fatty liver disease (NAFLD) that can lead to progressive fibrosis, cirrhosis, and hepatocellular carcinoma. Comorbidities include obesity, metabolic syndrome, and type 2 diabetes. Approximately 16 mi...