Arrowhead Pharmaceuticals Initiates Phase 1/2 Study...
Arrowhead Pharmaceuticals Initiates Phase 1/2 Study of ARO-HSD in Normal Healthy Volunteers and Patients with NASH or Suspected NASH
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has dosed the first patients in AROHSD1001, a Phase 1/2 clinical study 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: “Genetic studies have recently pointed to HSD17B13 as a compelling target for multiple forms of liver disease. No other therapeutics against this target have reached clinical studies, so ARO-HSD is the first investigational medicine using any modality to initiate a clinical program to study whether HSD17B13 inhibition in the liver leads to a clinical benefit for patients. Further, based on our preclinical work and key findings from various studies with our other product candidates, we believe that an RNAi-based intervention that utilizes our proprietary TRiMTM platform has the potential to be highly active at silencing the expression of HSD17B13.”
AROHSD1001 (NCT04202354) is a Phase 1/2 single and multiple dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-HSD in up to 74 normal healthy volunteers and patients with NASH or suspected NASH. Additional exploratory objectives include the assessment of various measures of drug activity using liver biopsy.
HSD17B13 is a member of the hydroxysteroid dehydrogenase family involved in the metabolism of hormones, fatty acids, and bile acids. Published human genetic data indicate that a loss of function mutation in HSD17B13 provides strong protection against alcoholic hepatitis, cirrhosis, and NASH, with approximately 30-50% risk reduction compared to non-carriers.1