PASADENA, CA, USA I December 16, 2019 I Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has filed an application for approval to begin a Phase 1 clinical trial of ARO-HSD, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with alcohol related and nonalcohol related liver diseases, such as nonalcoholic steatohepatitis (NASH).

Javier San Martin, M.D., chief medical officer at Arrowhead, said: “HSD17B13, a member of the hydroxysteroid dehydrogenase family involved in the metabolism of hormones, fatty acids, and bile acids, has emerged as a potentially interesting target for multiple forms of liver disease. Published human genetic data indicate that a loss of function mutation in HSD17B13 provides strong protection against alcoholic hepatitis, cirrhosis, and nonalcoholic steatohepatitis, or NASH, with approximately 30-50% risk reduction compared to non-carriers. This genetic validation gives us confidence about the target gene as we embark on what we believe is the first clinical program using any modality against HSD17B13.”

Pending approval, Arrowhead intends to proceed with AROHSD1001, a Phase 1 single and multiple dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-HSD in normal healthy volunteers as well as in patients with NASH or suspected NASH. Additional exploratory objectives include assessment of various measures of drug activity using liver biopsy.

The application for approval of the clinical trial is being 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.

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.

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SOURCE: Arrowhead Pharmaceuticals