Madrigal Pharmaceuticals Announces Positive Outcome from Pre-Planned DSMB Safety Review and Completion of Enrollment of Phase...
September 19 2017 - 07:30AM
-- Second DSMB review includes data from both
Phase 2 studies in NASH and HeFH --
Madrigal Pharmaceuticals, Inc. (Nasdaq:MDGL) today announced that
the Company’s independent Data Safety Monitoring Board (DSMB) held
its second prespecified meeting to evaluate both the heterozygous
familial hypercholesterolemia (HeFH) and non-alcoholic
steatohepatitis (NASH) Phase 2 clinical trials and recommended that
both studies continue without protocol modifications. Madrigal also
announced that the Phase 2 study of MGL-3196 for the treatment of
HeFH, a severe genetic dyslipidemia that causes early onset
cardiovascular disease, has enrolled 113 patients, thereby
exceeding the target patient enrollment of 105 patients, and will
continue enrollment for the next several days. Top-line results
from the study are expected in early 2018.
“Individuals with HeFH suffer from a life-long burden of
cholesterol buildup and are at high risk of early coronary
artery disease. While treatable with LDL-C lowering strategies, we
estimate that more than one-third of HeFH patients do not
achieve their cholesterol reduction goals,” said John J. P.
Kastelein, MD, Ph.D., FESC, 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. “We are hopeful that results from this Phase 2 study will
support further development of MGL-3196 as a much needed additional
oral agent to treat people with HeFH.”
MGL-3196 is a first-in-class, oral, once-daily, liver-directed,
thyroid hormone receptor (THR) β-selective agonist medication that
has demonstrated significant LDL lowering in both clinical and
preclinical studies. MGL-3196 is also in a fully enrolled Phase 2
clinical trial for the treatment of NASH.
“We are encouraged by the recommendation of our DSMB to continue
both the NASH and HeFH clinical trials with no modifications to
either protocol. In the HeFH study, we have exceeded target
enrollment of patients who have not met treatment goals despite
high intensity statin therapy, including up to 40 mg of
rosuvastatin and 80 mg of atorvastatin, thus, providing opportunity
for additional LDL cholesterol lowering in HeFH,” stated Rebecca
Taub, M.D., CMO and Executive VP, Research & Development, and
founding scientist of Madrigal.
“We anticipate that the data from both the HeFH and NASH Phase 2
studies will confirm the potential therapeutic value of MGL-3196,”
stated Paul A. Friedman, M.D., Chairman and CEO of Madrigal. “We
look forward to the potentially exciting opportunities for Madrigal
as we advance through clinical development of this drug.”
About the Phase 2 HeFH StudyThe 12-week,
randomized, double-blind, placebo-controlled, multi-center Phase 2
study was designed to enroll 105 patients with HeFH in three
European countries. Patients are 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-β 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 percent 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) β-selective agonist that is currently in Phase 2
development for NASH and heterozygous familial hypercholesterolemia
(HeFH). For more information, visit www.madrigalpharma.com.
Forward-Looking Statements 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 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.com312-961-2502
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