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Ionis Pharma reports positive results from the phase 2 study of ION224, demonstrating clinical efficacy in treating NASH/MASH

Ionis Pharmaceuticals, Inc. has released positive outcomes from a phase 2 trial of ION224, an investigational DGAT2 antisense inhibitor targeting metabolic dysfunction-associated steatohepatitis (MASH), formerly known as nonalcoholic steatohepatitis (NASH). Both doses (120 mg and 90 mg) achieved liver histologic improvement, the primary endpoint, and MASH resolution, a crucial secondary endpoint.

Key findings from the 160-patient, 51-week study include:

  • Significant liver histologic improvement with ION224, marked by ≥2-point reduction in NAFLD Activity Score (NAS) (p<0.001 for 120 mg, p=0.015 for 90 mg).
  • Primary endpoint improvements observed in F2 and F3 fibrosis subgroups.
  • Statistically significant MASH resolution without fibrosis worsening (p=0.039).
  • 44% of patients on 120 mg experienced ≥50% liver steatosis reduction vs. 3% on placebo.
  • 32% on 120 mg saw ≥1 stage fibrosis improvement without worsening steatohepatitis vs. 12.5% on placebo.

ION224 demonstrated good safety and tolerability. MASH, a severe form of metabolic liver disease, can lead to liver fibrosis and cirrhosis. ION224, a Ligand-Conjugated Antisense (LICA) medication, aims to reduce diacylglycerol acyltransferase 2 (DGAT2) production to address MASH.