Madrigal Pharmaceuticals Announces the Initiation of a Phase 2 Study of MGL-3196 in Patients with Heterozygous Familial Hyper...
February 23 2017 - 8:16AM
-- 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 --
Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL) today announced that
the first patient has been dosed in its Phase 2 study of MGL-3196
for the treatment of 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 in a Phase 2
clinical trial for the treatment of non-alcoholic steatohepatitis
(NASH).
“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. John J. P. Kastelein, Professor of Medicine in the
Department of Vascular Medicine at the Academic Medical Center
(AMC) of the University of Amsterdam and the principal investigator
of the study. “Confirming the potential for MGL-3196 to safely
lower LDL cholesterol and Lp(a) with this study will bring us
closer to adding a much-needed new treatment option for this
population.”
“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 Paul A. Friedman, M.D., Chairman and CEO of
Madrigal. “We expect to have topline data from this study near year
end.”
“Madrigal is conducting the Phase 2 study in Europe, with the
first patient dosed in Denmark. The European patient registries for
HeFH should help support rapid trial recruitment by our clinical
trial sites,” said Becky Taub, M.D., CMO and Executive VP, Research
& Development of Madrigal. “In addition to the efficacy and
safety endpoints, the results of this study will provide additional
confirmation that we have selected the appropriate dose of MGL-3196
for these patients.”
About the StudyThe 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).
About HeFHHeterozygous 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.
About Madrigal PharmaceuticalsMadrigal
Pharmaceuticals, Inc. (Nasdaq: MGDL) is a clinical-stage
biopharmaceutical company pursuing novel therapeutics that target a
specific thyroid hormone receptor pathway in the liver, which is a
key regulatory mechanism common to a spectrum of cardio-metabolic
and fatty liver diseases with high unmet medical need. The
company’s lead candidate, MGL-3196, is a first-in-class, orally
administered, small-molecule, liver-directed, thyroid hormone
receptor (THR) ß-selective agonist that is currently in Phase 2
development for NASH and HeFH. For more information, visit
www.madrigalpharma.com.
Forward-Looking StatementsThis 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 U.S. Securities
and Exchange Commission for more detailed information regarding
these risks and uncertainties and other factors that may cause
actual results to differ materially from those expressed or
implied.
Investor Contact:
Marc Schneebaum, Madrigal Pharmaceuticals, Inc.
IR@madrigalpharma.com
Media Contact:
Mike Beyer, Sam Brown Inc.
mikebeyer@sambrown.com
312-961-2502
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