Ionis Pharmaceuticals, Inc. announced on Wednesday that its Phase 2 study, which is evaluating the drug ION224 for its effectiveness in treating metabolic dysfunction-associated steatohepatitis (MASH), successfully achieved its primary goal.
The primary goal of the study was to determine the percentage of patients showing MASH histologic improvement. This improvement was defined as achieving a minimum 2-point reduction in the Nonalcoholic Fatty Liver Disease Activity Score (NAS), along with at least a 1-point improvement in hepatocellular ballooning or lobular inflammation, without any worsening of fibrosis by the end of the treatment period.
According to the study, 44 percent of patients treated with ION224 showed a 50 percent or higher relative reduction in liver steatosis, compared to just 3 percent for placebo. Furthermore, 32 percent of patients treated with the drug saw an improvement of at least 1 stage in fibrosis without an exacerbation of steatohepatitis. This is in stark contrast to the 12.5 percent seen for placebo.
ION224 is formulated to limit the production of diacylglycerol acyltransferase 2 (DGAT2) in patients diagnosed with MASH. By cutting down the production of the DGAT2 enzyme, the overproduction of harmful triglycerides, which contribute to excess liver fat resulting in potential liver damage and inflammation, can be curtailed.
