Ionis and AstraZeneca Partner on Fourth Drug Candidate, ION455, for the Treatment of NASH

On November 23, Ionis Pharmaceuticals announced that AstraZeneca had obtained a collaborative development license for its investigational antisense oligonucleotide drug, ION455, which is intended for the treatment of non-alcoholic steatohepatitis (NASH). Ionis will receive a $30 million licensing fee from AstraZeneca and is eligible for up to $300 million in milestone payments as well as royalties on net sales. AstraZeneca will be responsible for the further development of ION455, including regulatory filings and commercialization.

ION455 is the fourth drug co-developed by Ionis and AstraZeneca. Previously, Ionis and AstraZeneca have also been collaborating on researching potential treatments for kidney diseases, cardiovascular diseases, and cancer. ION455 is also the second potential NASH treatment developed in collaboration between Ionis and AstraZeneca, the other being ION839 (AZD2693), an antisense oligonucleotide designed to inhibit the production of patatin-like phospholipase domain-containing protein 3 (PNPLA3). In addition, Ionis is independently developing another NASH drug, ION224, an antisense oligonucleotide aimed at inhibiting the production of diacylglycerol O-acyltransferase 2 (DGAT2). ION224 is one of more than 20 drugs that the company is prioritizing and preparing for commercialization.

NASH is a chronic, progressive liver disease characterized by hepatic fat accumulation and inflammation, which can lead to scarring or fibrosis and impair liver function. Patients with NASH have a higher risk of progressing to advanced liver disease—including liver failure and hepatocellular carcinoma—than the general population. Currently, no pharmacological therapies are approved globally for the treatment of NASH, leaving patients with very limited treatment options.

Source: Jike Yao Wen

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