PASADENA, CA, USA I June 27, 2023 I Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has filed an application for approval to initiate a Phase 1 clinical trial of ARO-SOD1, an RNAi-based investigational medicine designed to reduce expression of superoxide dismutase 1 (SOD1) in the central nervous system (CNS) as a potential treatment for patients with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations. ARO-SOD1 is the first therapeutic candidate designed for delivery to the CNS to enter clinical studies that leverages Arrowhead’s Targeted RNAi Molecule (TRiMTM) platform.

“The ARO-SOD1 program entering clinical studies represents the further expansion of our proprietary TRiMTM platform, which now includes product candidates addressing a multitude of diverse disease areas that target the liver, lung, muscle, and CNS,” said Chris Anzalone, Ph.D., president and CEO of Arrowhead.

Pending regulatory clearance, Arrowhead intends to proceed with AROSOD1-1001, a Phase 1 randomized, placebo-controlled, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-SOD1 in adult patients with ALS harboring a SOD1 mutation considered to be causative of ALS. The study is designed to enroll up to 24 subjects.

An application was submitted to an ethics committee in compliance with the Clinical Trial Notification process of the Australian Department of Health and Ageing, Therapeutic Goods Administration.

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

For more information, please visit, or follow us on Twitter @ArrowheadPharma. To be added to the Company’s email list and receive news directly, please visit

SOURCE: Arrowhead Pharmaceuticals