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Ionis announces positive results from Phase 2 study of ION224, an investigational medicine demonstrating clinical efficacy in the treatment of NASH/MASH

Published: 2024-03-13 11:00:00 ET
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  • Significant improvement in steatohepatitis with >2 point improvement in NAS score without worsening fibrosis, the primary endpoint of the study
  • Achieved key secondary endpoint of MASH resolution without worsening of fibrosis
  • More ION224 treated patients had an improvement of >1 stage fibrosis compared to placebo
  • ION224 was safe and well-tolerated in this study with once-monthly subcutaneous dosing

CARLSBAD, Calif., March 13, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) 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 (MASH), previously referred to as nonalcoholic steatohepatitis (NASH). The study met its primary endpoint at both doses (120 mg and 90 mg), achieving liver histologic improvement, and also met the important secondary endpoint of MASH resolution.

Ionis logo (PRNewsfoto/Ionis Pharmaceuticals, Inc.)

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 (NAS)* (p