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13th Mar, 2024 12:00 AM
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Ionis Pharma's Fatty Liver Disease Drug Succeeds in Mid-stage Trial

(Reuters) -Ionis Pharmaceuticals said on Wednesday its experimental drug to treat a type of fatty liver disease met the main goal of reducing inflammation of the critical organ in a mid-stage trial.

The results come as companies race to bring the first approved treatment for the disease and tap into a global market expected to surpass $16 billion by 2030, according to market research firm Vision Research Reports.

Akero Therapeutics last week reported its experimental drug significantly reduced scarring after nearly two years in a mid-stage study while the U.S. Food and Drug Administration is expected to decide on Madrigal Pharmaceuticals' treatment by Thursday.

The trial enrolled 160 metabolic dysfunction-associated steatohepatitis (MASH) patients with scarring or fibrosis at severity of stage two or three, Ionis said.

The company said 32% of patients who received a higher dose of the drug, called ION224, saw improved scarring in the liver by at least one stage as measured by a biopsy, or tissue examination, compared to 12.5% patients on placebo.

Ionis said 44% of patients treated with a higher dose version achieved more than 50% relative reduction in abnormal retention of fat when measured by a quantitative imaging biomarker compared to 3% for placebo.

The company studied the drug - designed to reduce the production of an integral membrane protein - for 51 weeks in a two-part trial.

The condition, earlier called non-alcoholic steatohepatitis (NASH), affects around 5% of the U.S. population.

Ionis said a subgroup analysis indicated significant improvements in the main trial goal in patients with advanced liver scarring.

The company's shares were up 4% before the bell on Wednesday.

(Reporting by Pratik Jain in Bengaluru; Editing by Savio D'Souza and Sriraj Kalluvila)

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