Ionis Pharma's Phase 2 Study Of ION224 In MASH Meets Primary Goal

(RTTNews) - Ionis Pharmaceuticals, Inc. (IONS) Wednesday said its Phase 2 study evaluating ION224 in metabolic dysfunction-associated steatohepatitis (MASH) met its primary goal.

The primary endpoint of the study was the percentage of patients who achieved MASH histologic improvement, defined as achieving at least a 2-point reduction in Nonalcoholic Fatty Liver Disease Activity Score (NAS) with at least 1-point improvement in hepatocellular ballooning or lobular inflammation, and without worsening of fibrosis at end of the treatment period.

In the study, 44 percent of patients treated with ION224 achieved greater than or equal to 50 percent relative reduction in liver steatosis compared with 3 percent for placebo. Thirty two percent of patients treated with the drug candidate achieved a greater than or equal to 1 stage improvement in fibrosis without worsening steatohepatitis compared to 12.5 percent for placebo.

ION224 is designed to reduce the production of diacylglycerol acyltransferase 2 (DGAT2) in patients with MASH. Reducing the production of DGAT2 enzyme decreases the overproduction of triglycerides that contribute to excess liver fat, which can result in liver damage and inflammation.