-- HeFH is the most common dominantly inherited disease, present in up to 1 in 200 people, in which there is a life-long burden of high LDL cholesterol build up requiring aggressive lipid lowering treatment --
-- Primary endpoint is the reduction of LDL cholesterol, with secondary endpoints including reduction of Lp(a), a highly atherogenic lipid particle --
“Because individuals with HeFH have a life-long burden of cholesterol buildup in their bodies, current standard of care employs aggressive therapy, often combinations of drugs, to attempt to lower this burden. Despite such aggressive therapy, more than one third of HeFH patients do not reach their cholesterol reduction goals,” said Dr.
“MGL-3196 has demonstrated impressive LDL cholesterol lowering in Phase I; MGL-3196 acts by mechanisms distinct from and complementary with statins, as our studies to date have suggested, and should readily combine with high dose statins and ezetimibe, thus having the potential to provide significant additional LDL cholesterol lowering. The data we have generated with MGL-3196 as well as what is known about THR-β agonism, gives us a high degree of confidence that MGL-3196 will perform well in these patients,” said
“Madrigal is conducting the Phase 2 study in
About the Study
The 12-week, randomized, double-blind, placebo-controlled, multi-center Phase 2 study will enroll 105 patients with HeFH in several European countries. Patients will be randomized in a 2:1 ratio to receive either MGL-3196 or placebo, in addition to their current drug regimen (including high dose statins and/or ezetimibe). The primary endpoint of the study is reduction of LDL cholesterol, with secondary endpoints including reductions in triglycerides, Lp(a), and ApoB, as well as safety. Lp(a) is a severely atherogenic lipid particle, commonly elevated in familial hypercholesterolemia patients, the levels of which are not adequately reduced by existing lipid lowering therapies. THR-ß agonism is one of the few therapeutic approaches that can substantially lower Lp(a).
Heterozygous familial hypercholesterolemia (HeFH), and a much rarer form called homozygous familial hypercholesterolemia (HoFH), are severe genetic dyslipidemias typically caused by inactivating mutations in the LDL receptor. Both forms of FH lead to early onset cardiovascular disease. HeFH, the most common dominantly inherited disease, is present in up to 1 in 200 people; the disease is found in higher frequencies in certain more genetically homogenous populations. Treatments exist for both HeFH and HoFH but many patients (as many as 40% of HeFH patients) are not able to reach their cholesterol (LDL-C) reduction goals on these therapies, reflecting the lifetime burden of cholesterol buildup in their bodies.
This communication contains “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to, the company's clinical development of MGL-3196, the timing and outcomes of clinical studies of MGL-3196, and the uncertainties inherent in clinical testing. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Madrigal undertakes no obligation to update any forward looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events. Please refer to Madrigal's filings with the
Marc Schneebaum, Madrigal Pharmaceuticals, Inc.IR@madrigalpharma.com Media Contact: Mike Beyer, Sam Brown Inc.firstname.lastname@example.org 312-961-2502