Arrowhead doses first patient in Phase I/II trial of ARO-HSD

4th March 2020 (Last Updated March 4th, 2020 12:36)

US-based biopharmaceutical firm Arrowhead Pharmaceuticals has started dosing subjects in a Phase I/II clinical trial (AROHSD1001) of ARO-HSD for the treatment of patients suffering from alcohol and non-alcohol related liver diseases, such as nonalcoholic steatohepatitis (NASH).

Arrowhead doses first patient in Phase I/II trial of ARO-HSD
Photomicrograph of a liver biopsy from a patient with alpha-1 antitrypsin deficiency. Credit: Jerad M Gardner.

US-based biopharmaceutical firm Arrowhead Pharmaceuticals has started dosing subjects in a Phase I/II clinical trial (AROHSD1001) of ARO-HSD for the treatment of patients suffering from alcohol and non-alcohol related liver diseases, such as nonalcoholic steatohepatitis (NASH).

NASH is a type of non-alcoholic fatty liver disease (NAFLD). It occurs due to inflammation and damage caused by a build-up of fat in the liver.

The single and multiple dose-escalating trial AROHSD1001 will evaluate the safety and efficacy of the company’s first investigational RNA interference (RNAi) medicine ARO-HSD in about 74 healthy volunteers identified with NASH or suspected NASH.

It will also evaluate the pharmacokinetics and pharmacodynamic effects of ARO-HSD.

Other exploratory objectives of the study include the analysis of several measures of drug activity using liver biopsy.

Arrowhead Pharmaceuticals chief medical officer Javier San Martin 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 TRiM platform has the potential to be highly active at silencing the expression of HSD17B13.”

HSD17B13 falls under the hydroxysteroid dehydrogenase (HSD) family, which is involved in the fatty acids and bile acids metabolism.

In August last year, Arrowhead Pharmaceuticals initiated a potentially pivotal Phase II/III clinical trial of ARO-AAT, its second-generation subcutaneously administered RNAi therapeutic.