Madrigal’s MGL-3196 Achieves Primary Endpoint in Patients with Heterozygous Familial Hypercholesterolemia

Madrigal Pharmaceuticals (NASDAQ:MDGL) announced positive results from a double-blind, placebo-controlled, multi-center, 12-week Phase 2 clinical trial in patients with heterozygous familial hypercholesterolemia (HeFH), a severe genetic dyslipidemia that causes early onset cardiovascular disease. MGL-3196 is a first-in-class, oral, once-daily, liver-directed, thyroid hormone receptor (THR) β-selective agonist medication. MGL-3196 is also being developed as a treatment for patients with non-alcoholic steatohepatitis (NASH).

As quoted in the press release:

MGL-3196 was well-tolerated with primarily mild and some moderate AEs, the numbers of which were balanced between placebo and drug-treatment groups. Fewer than 7% of patients did not complete the study, and patients who discontinued for AEs, all mild to moderate, were balanced between drug-treated and placebo patients. There were 2 serious adverse events in the study, both unrelated to treatment, one in a placebo and one in a drug-treated patient.

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