Press release
Arrowhead Pharmaceuticals Files for Regulatory Clearance to Initiate a Phase 1/2 Study of ARO-DUX4 for Facioscapulohumeral Muscular Dystrophy
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate
About this update from Arrowhead Pharmaceuticals, Inc.
[{"type":"text","content":" PASADENA, Calif.--(BUSINESS WIRE)--\nArrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2 clinical trial of ARO-DUX4, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD). ARO-DUX4 is the first clinical candidate utilizing Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform to target disease associated genes in skeletal muscle.\n\n\n“FSHD is a debilitating disease caused by the aberrant expression of the DUX4 gene in skeletal muscle. ARO-DUX4 engages the RNA interference pathway to selectively inhibit DUX4 expression and is a promising strategy to reverse the downstream myotoxicity of DUX4 and potentially enable stabilization or improvement in muscle function in patients. We believe ARO-DUX4 has the potential to be an impactful new medicine for patients,” said Chris Anzalone, Ph.D., president and CEO of Arrowhead. “As previously communicated, we are in discussions with external parties regarding the most appropriate path for the clinical development and commercialization of ARO-DUX4 with the goal of bringing this promising product candidate to patients in need as expeditiously as possible.”\n\n\nAn application for approval of the clinical trial was submitted to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials. Pending clearance, Arrowhead intends to proceed with ARODUX4-1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DUX4 in adult patients with FSHD type 1. The study is designed to enroll up to 52 subjects.\n\n\nAbout Facioscapulohumeral Muscular Dystrophy\n\n\nFacioscapulohumeral muscular dystrophy (FSHD) is a debilitating, muscle-wasting disease characterized by progressive asymmetrical weakness and fatty infiltration of skeletal muscles in the face, shoulders, upper arms, abdomen, or lower leg and is the third most common form of muscular dystrophy. There is currently no approved therapy for FSHD.\n\n\nAbout ARO-DUX4\n\n\nARO-DUX4 is an investigational RNAi therapeutic designed to reduce expression of the gene that encodes the human double homeobox 4 (DUX4) protein as a...