Arrowhead Pharmaceuticals Files for Regulatory Clearance to Initiate Phase 1/2a Study of ARO-DM1 for Type 1 Myotonic Dystrophy

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-DM1, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy.

ARO-DM1 is designed to reduce expression of the dystrophia myotonica protein kinase (DMPK) gene. There is currently no approved disease-modifying therapy for DM1. Treatments have focused on symptomatic management, including physical therapy, exercise, ankle-foot orthoses, wheelchairs, and other assistive devices.

James Hamilton, M.D., MBA, Chief of Discovery and Translational Medicine at Arrowhead, said: “ARO-DM1 is Arrowhead’s second clinical candidate utilizing our TRiM platform to deliver RNAi therapeutics to skeletal muscle. Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities and may become physically disabled and have a shortened life expectancy. ARO-DM1 represents a novel approach to treat DM1 by silencing aberrantly transcribed DMPK mRNA, which could lead to improvements in multiple symptoms, including muscle strength and function.”

An application for approval of the clinical trial was submitted to a local Ethics Committee and 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 ARODM1-1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DM1 in up to 48 subjects with DM1.

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.