Ionis announces third investigational antisense medicine to treat nonalcoholic steatohepatitis (NASH) enters development

IndustryPRwire -- Ionis Pharmaceuticals announced that the biopharmaceutical company AstraZeneca has licensed ION455, an investigational antisense medicine being developed as a potential treatment for nonalcoholic steatohepatitis (NASH). ION455 is the second medicine for the treatment of NASH that Ionis has partnered with AstraZeneca. The companies have also partnered on ION839 (AZD2693), which is designed to inhibit the production of patatin-like phospholipase domain-containing 3 (PNPLA3) protein, a major genetic determinant of NASH progression. Separately, Ionis is also developing a wholly owned NASH program. ION224 is designed to reduce the production of DGAT2, or diacylglycerol acyltransferase 2, for treating patients with NASH. ION224 is one of more than 20 medicines in the growing Ionis-owned pipeline that the company is prioritizing and preparing for commercialization.

NASH is the most severe form of nonalcoholic fatty liver disease (NAFLD). It is related to the epidemic of obesity, pre-diabetes and diabetes. Unlike liver disease caused by alcohol consumption, NAFLD is the result of an accumulation of fat in the liver, which can lead to inflammation and cirrhosis, an advanced scarring of the liver that prevents the liver from functioning normally. About 20 percent of NASH patients are reported to develop cirrhosis and 30 to 40 percent of patients with NASH cirrhosis experience liver-related death.i Currently, a liver transplant is the only treatment for advanced cirrhosis and liver failure. Because of the high prevalence of NASH, it has recently become the third most common indication for liver transplantation in the U.S.

Brett P. Monia, Ph.D., Ionis' chief executive officer Commented "Today, there are no FDA-approved medicines to specifically treat nonalcoholic steatohepatitis. However, due in large part to the progress made by our cardio-metabolic franchise, three Ionis-discovered novel medicines are now in development. These are encouraging advances that we hope will one day bring therapeutic benefit to patients who have limited treatment options."

ION455 is the fourth medicine to reach development in partnership with AstraZeneca. Ionis earned $30 million from AstraZeneca for licensing ION455 and is eligible to earn up to $300 million in milestone payments plus royalties on net sales. AstraZeneca will have responsibility for further development of ION455, including regulatory filings, and commercialization.

In addition to NASH, Ionis and AstraZeneca are collaborating on potential treatments for kidney disease, cardiovascular disease and cancer.

Related Posts

Subscribe Our Newsletter