Arrowhead files for clearance to begin study of ARO-DM1 for DM1 treatment

Arrowhead Pharmaceuticals 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.