Madrigal lead candidate shows promise for NASH, cholesterol disorder

Madrigal Pharmaceuticals announced completed enrollment for two phase 2 trials of the company’s lead candidate MGL-3196, according to a press release. The trials are for nonalcoholic steatohepatitis and heterozygous familial hypercholesterolemia, or HeFH, respectively.

“Because safety is such an important requirement for patients with these diseases, we are encouraged by the latest recommendation from our [data and safety monitoring board] to continue both the NASH and HeFH clinical trials with no modifications to either protocol,” Rebecca Taub, MD, chief medical officer and executive vice president of research and development at Madrigal said in the release. “We look forward to the upcoming data readouts which, if positive, will enable us to initiate phase 3 trials in 2018.”

MGL-3196 is a first-in-class, orally administered, small-molecule, liver-directed, thyroid hormone receptor beta-selective agonist.

The ongoing phase 2 trial of MGL-3196 for NASH includes 125 patients aged 18 years or older with biopsy-confirmed NASH and more than 10% liver fat, confirmed by a magnetic resonance imaging-proton density fat fraction (MRI-PDFF).

At 12 weeks, results showed a high correlation of liver fat reduction by MRI-PDFF compared with liver biopsy. The researchers anticipate confirmed efficacy at 36 weeks, or end of trial.

Based on evidence of reduced low-density lipoprotein cholesterol in a phase 1 trial of MGL-3196 for HeFH, Madrigal initiated a phase 2 proof-of-concept trial for patients with HeFH in February 2017. The researchers completed enrollment in September 2017 with a total of 116 patients.

“With patient enrollment completed for the phase 2 NASH and HeFH clinical studies, we are on track to report top-line results for NASH late this year and HeFH early in 2018,” Paul Friedman, MD, chief executive officer of Madrigal, said in the release. – by Talitha Bennett

Reference: www.madrigalpharma.com

Editor's note: This report has corrected information from a company representative.