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Today, Arrowhead Pharmaceuticals announced that it has reached a cooperation agreement with GlaxoSmithKline (GSK) to jointly develop and promote its research RNAi therapy ARO-HSD for the treatment of non-alcoholic steatohepatitis (NASH)
.
Currently, it is being tested in phase 1/2 clinical trials
The excessive accumulation of fat in the liver in NASH patients leads to inflammatory damage and liver fibrosis
.
At present, there are more than 100 million NASH patients in the world, and their risk of liver cirrhosis and liver cancer is significantly increased
Genetic studies have shown that a loss-of-function variant in a gene called HSD17B13 is associated with a reduced risk of chronic liver disease and a reduced risk of steatosis progressing to steatohepatitis
.
This means that reducing the expression of this gene may prevent non-alcoholic fatty liver disease from further progressing to NASH and prevent further liver fibrosis and cirrhosis in patients with NASH
ARO-HSD is an RNAi therapy designed for this target, which can inhibit the production of HSD17β13 protein expressed by this gene
.
The preliminary results of the Phase 1/2 clinical trial showed that ARO-HSD dose-dependently reduced the mRNA level of HSD17B13 in all patients.
In addition, in patients treated with ARO-HSD at doses greater than 100 mg, a decrease in liver alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels was observed
.
Among the 18 patients receiving treatment, 9 patients observed a decrease in liver fat levels, which was 4%-41%
.
According to the agreement, Arrowhead will receive an upfront payment of US$120 million and is eligible for development and commercialization milestone payments of more than US$1 billion
.
GSK will obtain the development and commercialization rights of ARO-HSD outside of Greater China
Reference materials:
[1] Arrowhead Pharmaceuticals Enters Exclusive License Agreement with GSK for ARO-HSD.
[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