-- Primary endpoint is the reduction of liver
fat, assessed by MRI-PDFF, at 12 weeks --
Madrigal Pharmaceuticals, Inc. (Nasdaq:MDGL) today announced that
it has completed patient enrollment of 125 patients, exceeding its
targeted enrollment of 117 patients, in its Phase 2
proof-of-concept study evaluating MGL-3196 for the treatment of
non-alcoholic steatohepatitis (NASH). MGL-3196 is a first-in-class,
oral, once-daily, liver-directed, thyroid hormone receptor (THR)
β-selective agonist medication.
“I am pleased to be participating in this important study as the
results will confirm if MGL-3196 is safe and well-tolerated
and shows efficacy in NASH patients,” stated Stephen A. Harrison,
M.D., Medical Director of Pinnacle Clinical Research, San Antonio,
Texas and Principal Investigator of the study. “Additionally, since
the study incorporates both magnetic resonance imaging-estimated
proton density fat fraction (MRI-PDFF), a non-invasive measure of
liver fat, along with liver biopsy, there is the potential to
provide additional evidence for a correlation between improvement
in non-invasive imaging and the histopathologic components
associated with NASH on biopsy.”
“As we completed patient recruitment within the timeframe we had
anticipated, we are on track to release top-line results for the
primary endpoint, the reduction of liver fat assessed by MRI-PDFF
at 12 weeks, by the end of this year,” stated Rebecca Taub, M.D.,
Chief Medical Officer, Executive Vice President and Founding
Scientist of Madrigal. “This also means that we will be on track to
provide top-line 36 week results, which include a final MRI-PDFF
and an end-of-study liver biopsy in the second quarter of
2018.”
“Data from this study will help us better design our phase 3
trial, planning for which is already underway,” stated Paul
Friedman, M.D., Chief Executive Officer of Madrigal. “I also point
out that top-line data from our phase 2 study with MGL-3196 in
heterozygous familial hypercholesterolemia will also become
available by year end or very early in 2018.”
About the Phase 2 NASH StudyThe randomized,
double-blind, placebo-controlled, multi-center Phase 2 study
enrolled 125 patients 18 years of age and older with liver
biopsy-confirmed NASH and included approximately 25 clinical sites
in the United States. Patients were randomized to receive either
placebo or MGL-3196 with twice as many patients receiving MGL-3196
as placebo.
The primary endpoint of the study is the reduction of liver fat
at 12 weeks, assessed by MRI-PDFF, with efficacy confirmed at the
end of the trial (36 weeks) by repeat MRI-PDFF and conventional
liver biopsy to examine histological evidence for the resolution of
NASH. Recent published data show a high correlation of reduction of
liver fat measured by MRI-PDFF to NASH scoring on liver biopsy.
Other secondary endpoints include changes in clinically relevant
biomarkers at 12 and 36 weeks, improvement in fibrosis by at least
one stage with no worsening of steatohepatitis, and safety and
tolerability. Additional information about the study [NCT02912260]
can be obtained at www.ClinicalTrials.gov.
About MGL-3196Among its many functions in the
human body, thyroid hormone, through activation of its beta
receptor, plays a central role in controlling lipid metabolism,
impacting a range of health parameters from levels of serum
cholesterol and triglycerides to the pathological buildup of fat in
the liver. Attempts to exploit this pathway for therapeutic
purposes in cardio-metabolic and liver diseases have been hampered
by the lack of selectivity of older compounds for the thyroid
hormone receptor (THR)-β, chemically-related toxicities and
undesirable distribution in the body.
Madrigal recognized that greater selectivity for thyroid hormone
receptor (THR)-β and liver targeting might overcome these
challenges and deliver the full therapeutic potential of THR-β
agonism. Madrigal believes that MGL-3196 is the first orally
administered, small-molecule, liver-directed, truly β-selective
thyroid THR agonist. MGL-3196 has demonstrated the potential for a
broad array of therapeutically beneficial effects, improving
components of both metabolic syndrome, such as insulin resistance
and dyslipidemia, and fatty liver disease, including lipotoxicity
and inflammation. These pleiotropic actions, coupled with an
excellent safety profile, suggest that MGL-3196 could be an ideal
drug for NASH. MGL-3196 has the unique potential to address the
root causes of the underlying disease process in NASH and
ultimately, the accompanying liver fibrosis, while also lowering
associated cardiovascular risk.
About NASH Non-alcoholic steatohepatitis (NASH)
is a common liver disease unrelated to alcohol use, characterized
by a build-up of fat in the liver, inflammation and increasing
fibrosis. Although people with NASH may feel well and often do not
know they have the disease, NASH can lead to permanent damage,
including cirrhosis and impaired liver function. According to the
National Institutes of Health (NIH), NASH affects approximately six
percent of American adults.1 It is the fastest growing reason
for liver transplants and is also associated with an increasing
incidence of liver cancer. There are currently no treatments
approved by the U.S. Food and Drug Administration (FDA) for
NASH.
About Madrigal PharmaceuticalMadrigal
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.
1 National Institutes of Diabetes and Digestive and Kidney
Disorders. Defintion and Facts of NAFLD and NASH. Available at
https://www.niddk.nih.gov/health-information/health-topics/liver-disease/nonalcoholic-steatohepatitis/Pages/facts.aspx
Accessed July 31, 2017.
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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