Arrowhead Pharmaceuticals announced that it entered into an exclusive license agreement with GlaxoSmithKline under which GSK will develop and commercialize ARO-HSD, Arrowhead’s investigational RNA interference (RNAi) therapeutic in a Phase 1/2 trial that is currently being developed as a treatment for patients with non-alcoholic steatohepatitis (NASH).

“Genetic studies have shown that HSD17B13 is a compelling therapeutic target for multiple forms of liver disease. Based on the clinical results generated to date, including those recently presented at the 2021 AASLD Liver Meeting, ARO-HSD could have the potential to be the first investigational therapeutic to achieve robust reductions in mRNA and protein levels of hepatic HSD17B13, leading to reductions in ALT, a liver enzyme typically elevated in liver diseases such as NASH,” said Christopher Anzalone, Ph.D., Arrowhead’s president and CEO. “GSK has a global reach and impressive capabilities in the clinical development and commercialization of important medicines. As the work continues towards progressing further Phase 2 studies and planning Phase 3 studies for ARO-HSD, we believe this agreement with GSK furthers the potential of ARO-HSD as a promising new investigational therapeutic for patients with NASH and other liver diseases. We look forward to enabling GSK to advance ARO-HSD towards the millions of patients worldwide who do not have adequate treatment options.”

“NASH can be a life-threatening disorder in which excessive fat build-up in the liver causes inflammatory damage and fibrosis. The compelling genetic evidence linking HSD17B13 variants with protection of the liver from inflammatory injury suggests that there is an opportunity to produce a first-in-class medicine to reduce the clinical consequences of NASH. It’s exciting to advance Arrowhead’s promising RNAi approach into Phase 2 studies as an important potential new medicine for patients with NASH,” said John Lepore, SVP and Head of Research, GSK.