Ionis announces results from Phase 2 study of ION224

Ionis Pharmaceuticals announced positive results from a Phase 2 study of ION224, an investigational DGAT2 antisense inhibitor in development for the treatment of metabolic dysfunction-associated steatohepatitis, or MASH, previously referred to as nonalcoholic steatohepatitis, or NASH. The study met its primary endpoint at both doses, achieving liver histologic improvement, and also met the important secondary endpoint of MASH resolution. Key highlights from the 160-patient study at 51 weeks included: ION224 achieved statistically significant liver histologic improvement as measured by at least a 2-point reduction in NAFLD Activity Score, or NAS; dubgroup analysis indicated that significant improvements in the primary endpoint were observed in patients with both F2 and F3 fibrosis; ION224 achieved statistically significant MASH resolution without worsening of fibrosis, as measured by biopsy; 44% of patients treated with 120 mg achieved greater than or equal to50% relative reduction in liver steatosis as measured by MRI-PDFF compared to 3% for placebo; 32% of patients treated with 120 mg achieved a greater than or equal to1 stage improvement in fibrosis without worsening steatohepatitis as measured by biopsy compared to 12.5% for placebo; ION224 was safe and well-tolerated in the study. ION224 is an investigational LIgand-Conjugated Antisense medicine designed to reduce the production of diacylglycerol acyltransferase 2 to treat patients with MASH.