Press release
Arrowhead Pharmaceuticals Gains Full Rights to NASH Candidate ARO-PNPLA3 with Promising Phase 1 Results
- Achieved Up to 40% Mean Reduction in Liver Fat in PNPLA3 I148M Homozygotes After Single-Dose PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals
About this update from Arrowhead Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Achieved Up to 40% Mean Reduction in Liver Fat in PNPLA3 I148M Homozygotes After Single-Dose\n\n PASADENA, Calif.--(BUSINESS WIRE)--\nArrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) announced today that it has gained rights to ARO-PNPLA3, formerly called JNJ-75220795, which was part of a 2018 research collaboration and option agreement between Arrowhead and Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson. ARO-PNPLA3 is an investigational RNA interference (RNAi) therapeutic developed using Arrowhead’s proprietary TRiMTM platform and designed to reduce liver expression of patatin-like phospholipase domain containing 3 (PNPLA3) as a potential treatment for patients with non-alcoholic steatohepatitis (NASH). ARO-PNPLA3 is currently in a Phase 1 clinical study. PNPLA3 has strong genetic and preclinical validation as a driver of fat accumulation and damage in the livers of patients who carry the common I148M mutation.\n\n“With no FDA approved therapies, NASH remains an area of significant unmet medical need. PNPLA3 is a unique NASH drug target as the I148M disease associated variant increases the risk of NAFLD, NASH, and hepatocellular carcinoma by 4 to 12-fold for homozygotes. The vast human genetic data in support of this target are compelling and supported by a strong pathophysiologic mechanistic understanding,” said Rohit Loomba, M.D., MHSc, Professor of Medicine, Director of the NAFLD Research Center at University of California at San Diego. “The Phase 1 data on ARO-PNPLA3 are extremely encouraging and I look forward to seeing this molecule progress to a later stage clinical study.”\n\nAfter a single dose in a Phase 1 clinical study, ARO-PNPLA3 achieved encouraging results, including:\n\n\nA dose-dependent mean reduction in liver fat of up to 40% in patients homozygous for the I148M mutation\n\n\nNo apparent treatment emergent increases in triglycerides or LDL-cholesterol\n\n\nSafety and tolerability\n\n\nNo clinically meaningful changes or trends in any safety parameters\n\n\nNo tolerability issues reported\n\n\nMostly mild adverse events (AE) reported\n\n\nNo serious or severe AEs\n\n\nNo AEs leading to treatment or study discontinuation\n\n\n\n\n“We are impressed by the initial Phase 1 clinical data for JNJ-75220795, now called ARO-PNPLA3. We understand that Janssen is undergoi...