GSK Partners with Arrowhead Pharmaceuticals in Over $1 Billion Deal to Develop RNAi Therapy ARO-HSD for NASH

Today, Arrowhead Pharmaceuticals announced that it has reached a cooperation agreement with GSK to jointly develop and promote its investigational RNAi therapy ARO-HSD for the treatment of non-alcoholic steatohepatitis (NASH). It is currently being tested in a phase 1/2 clinical trial.

NASH Patients Experience Inflammatory Damage and Liver Fibrosis Due to Excessive Fat Accumulation in the Liver. There Are Already More Than 100 Million NASH Patients Worldwide, with a Significantly Increased Risk of Cirrhosis and Liver Cancer. Currently, There Are No Approved Therapies for NASH.

Genetic studies show that loss-of-function variants in a gene called HSD17B13 are associated with a reduced risk of chronic liver disease and a decreased risk of steatosis progressing to steatohepatitis. This suggests that reducing the expression of this gene may prevent non-alcoholic fatty liver disease (NAFLD) from progressing to NASH, as well as prevent further liver fibrosis and cirrhosis in NASH patients.

ARO-HSD is an RNAi therapy designed to target this site, inhibiting the production of the HSD17β13 protein encoded by this gene. Preliminary results from the Phase 1/2 clinical trial show that ARO-HSD dose-dependently reduced mRNA levels of HSD17B13 in all patients, with mRNA levels decreasing by more than 90% at a dose of 200 mg.

In addition, decreases in liver alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels were observed in patients receiving ARO-HSD doses above 100 mg.

Among 18 patients receiving treatment, 9 patients showed a decrease in liver fat levels, with reductions ranging from 4% to 41%.

Under the agreement, Arrowhead will receive an upfront payment of $120 million and is eligible for over $1 billion in development and commercialization milestone payments. GSK will obtain the rights to develop and commercialize ARO-HSD outside of Greater China.

References:

[1] Arrowhead Pharmaceuticals Enters Exclusive License Agreement with GSK for ARO-HSD. Retrieved November 22, 2021, from https://ir.arrowheadpharma.com/news-releases/news-release-details/arrowhead-pharmaceuticals-enters-exclusive-license-agreement-gsk

(Original text has been abridged)

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