Press release
Alnylam and Regeneron Report Promising Data from Ongoing Phase 1 Study of ALN-HSD in NASH Patients and Healthy Volunteers
Target knockdown and safety results support continued clinical development Alnylam and Regeneron intend to initiate a Phase 2 study in late 2022 Detailed
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\nTarget knockdown and safety results support continued clinical development\n\nAlnylam and Regeneron intend to initiate a Phase 2 study in late 2022\n\nDetailed results to be presented at an upcoming medical congress\n\n CAMBRIDGE, Mass. & TARRYTOWN, N.Y.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) and Regeneron Pharmaceuticals (Nasdaq: REGN) announced today preliminary Phase 1 data supporting the clinical advancement of ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 in development for the treatment of nonalcoholic steatohepatitis (NASH).\n\nAfter single-dose evaluation in healthy adult volunteers (Part A), multiple doses of ALN-HSD are being studied in adult patients with NASH (Part B). Patients in the first two Part B cohorts (200 and 400 mg quarterly) have completed at least 6 months on the study; remaining cohorts are exploring a lower dose or a later biopsy time point. In the first two Part B cohorts, ALN-HSD was associated with robust target knockdown and numerically lower liver enzymes and biopsy-derived nonalcoholic fatty liver disease (NAFLD) Activity Score (NAS)* over six months in patients receiving ALN-HSD (N=20) relative to placebo (N=4). The study was not powered to achieve statistical significance on these endpoints, and the primary outcome measure is frequency of adverse events. ALN-HSD has exhibited an encouraging safety and tolerability profile to date; the most common treatment-emergent adverse event in healthy subjects treated with ALN-HSD (N=44) was injection site reaction in five patients; all injection site reactions were mild in severity. No treatment-related serious adverse events have been reported in either healthy volunteers or patients with NASH to date. Based on these results, the companies plan to initiate a Phase 2 study in adult patients with NASH in late 2022.\n\n“We are excited to share these initial results, indicating what we believe to be a favorable profile for ALN-HSD and supporting continued clinical development of this investigational medicine, particularly given the significant prevalence and unmet need in NASH – a progressive liver disease and a leading cause of liver transplant,” said Kevin Sloan, Ph.D., Vice President, Development Programs and Program Leader for the ALN-HSD program at Alnylam. “Our RNAi platform is ideally suited for this geneti...