Arrowhead Pharmaceuticals has submitted an application seeking approval to initiate a Phase I clinical trial of ARO-SOD1 to treat amyotrophic lateral sclerosis (ALS) harboring superoxide dismutase 1 (SOD1) mutations.

The company submitted the application to an ethics committee, in accordance with the Australian Department of Health and Ageing, Therapeutic Goods Administration’s clinical trial notification process.

The dose escalation, placebo-controlled, randomised study is designed to assess the safety and tolerability of the RNAi-based investigational medicine ARO-SOD1 in adults with ALS and SOD1 mutations.

It intends to enrol up to 24 subjects and also evaluate the pharmacodynamics and pharmacokinetics of ARO-SOD1.

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

ARO-SOD1 reduces expression of SOD1 in the central nervous system.

Arrowhead is also developing various therapeutics that utilise the natural pathway of gene silencing. They trigger the RNA interference mechanism and induce durable knockdown of target genes.

In April this year, Arrowhead reported interim results from a Phase I/II study of ARO-RAGE, designed for lowering the production of the receptor for advanced glycation end products (RAGE).

Interim results demonstrated a reduction in soluble RAGE, as measured in serum (after singular and two dose regimes) and bronchoalveolar lavage fluid (after a single dose).