Genetic Medicines Focused-Ionis Pharmaceuticals' Fatty Liver Candidate Lowers Excess Liver Fat, Midstage Study Data Shows

Ionis Pharmaceuticals Inc IONS released results from a Phase 2 study of ION224, an investigational DGAT2 antisense inhibitor for 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.

Also Read: Rare Disease Focused Ionis Pharmaceuticals Gears Up for FDA Submission After Donidalorsen Outperforms Placebo In Swollen Vessels Study.

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<0.001 (120 mg) and p=0.015 (90 mg)). 
•  Subgroup analysis indicated significant improvements in the primary endpoint were observed in patients with F2 and F3 (advanced) fibrosis.
•  ION224 achieved statistically significant MASH resolution without worsening of fibrosis, as measured by biopsy (p=0.039).
•  44% of patients treated with 120 mg achieved ≥50% relative reduction in liver steatosis as measured by MRI-PDFF compared to 3% for placebo.
•  32% of patients treated with 120 mg achieved a ≥1 stage improvement in fibrosis without worsening steatohepatitis as measured by biopsy compared to 12.5% for placebo.

ION224 was safe and well-tolerated in MASH participants. Those in the ION224 study arms did not experience any worsening of hepatic or renal function or gastrointestinal side effects, and there was a lower rate of early termination compared to placebo. 

Additionally, there were no on-study deaths or treatment-related serious adverse events. 

Price Action: IONS shares are up 1.34% at $43.75 on the last check Wednesday.

Photo by Christina Victoria Craft on Unsplash