Ionis reports positive data from Phase II MASH treatment trial

ION224 was found to be safe and well-tolerated without any treatment-associated serious adverse events.

Vishnu Priyan March 14 2024

Ionis Pharmaceuticals has announced positive data from a Phase II clinical trial of ION224, an investigational DGAT2 antisense inhibitor, for metabolic dysfunction-associated steatohepatitis (MASH).

The two-part, adaptive, multicentre, randomised, double-blind, placebo-controlled trial analysed the safety, efficacy, and pharmacokinetics of varying doses of ION224 when administered subcutaneously once a month in adult MASH patients.

It enrolled 160 subjects who received either ION224 or a placebo over 49 weeks.

The percentage of subjects who attained histologic improvement in MASH was the primary endpoint of the study.

According to the findings, the trial met the primary endpoint at both the 120mg and 90mg doses. It also achieved the key secondary endpoint of MASH resolution.

Notably, 44% of patients treated with 120mg of ION224 achieved a ≥50% relative reduction in liver steatosis, compared to 3% for the placebo group.

ION224 also demonstrated a statistically significant improvement in fibrosis stages without exacerbating steatohepatitis.

It was also found to be safe and well-tolerated without any treatment-associated serious adverse events or on-study mortality reported.

Ionis senior vice-president and chief medical officer Sanjay Bhanot said: “Reducing the production of DGAT2 enzyme decreases the overproduction of triglycerides that contribute to excess liver fat, which can result in liver damage and inflammation.

“We are encouraged by these ION224 data, showing that a monthly subcutaneous medicine targeting DGAT2 has the potential to improve MASH and prevent its progression to more severe stages, including advanced liver fibrosis and cirrhosis.

“The inhibition of DGAT2 represents a novel approach for MASH, a progressive disease in need of better treatment options.”

The latest development comes after Ionis announced plans to submit a new drug application to the US Food and Drug Administration following its Phase III OASIS-HAE study of donidalorsen in hereditary angioedema patients, which met its primary endpoint. 

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