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Arrowhead Pharmaceuticals Readying to Kick Off Phase I SOD1-ALS Drug Trial

NEW YORK – Arrowhead Pharmaceuticals on Wednesday said it is seeking permission from regulators in Australia to begin studying an investigational RNA interference-based (RNAi) therapy in patients with a genetically distinct form of amyotrophic lateral sclerosis (ALS).

Arrowhead is gearing up to conduct a Phase I global trial of the investigational drug ARO-SOD1 in ALS patients in whom the neurodegenerative disease is caused by mutations in the superoxide dismutase 1 (SOD1) gene. ARO-SOD1 is designed to trigger RNAi to silence the SOD1 gene and reduce its expression in the central nervous system.

There are around 6,400 new cases of ALS, also known as Lou Gehrig's disease, every year worldwide, and around 2 percent of patients have SOD1-ALS. Pending clearance from the Australian Department of Health and Aged Care's Therapeutic Goods Administration, Arrowhead plans to enroll 24 patients with SOD1-ALS into the randomized, placebo-controlled, dose escalation Phase I study, dubbed AROSOD1-1001. 

A spokesperson for the Pasadena, California-based company said that in addition to Australia, it will seek regulatory permission to launch additional study sites in the US, Canada, and New Zealand and is considering other countries as well. 

Earlier this year, the US Food and Drug Administration greenlighted the first treatment targeting SOD1-ALS when it granted accelerated approval to Biogen for Qalsody (tofersen), an antisense oligonucleotide that binds to and helps degrade SOD1 mRNA to reduce the production of the SOD1 protein.