Madrigal Pharmaceuticals Announces the Initiation of a Phase 2 Study of MGL-3196 in Patients with Non-alcoholic Steatohepatit...
October 20 2016 - 07:30AM
-- Study expected to provide proof-of-concept
data in NASH for the first liver-directed, thyroid hormone receptor
(THR) ß-selective agonist –
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 non-alcoholic steatohepatitis (NASH). MGL-3196
is a first-in-class, oral, once-daily, liver-directed, thyroid
hormone receptor (THR) ß-selective agonist medication.
“Thyroid hormone, through activation of its ß receptor, plays a
central role in controlling lipid metabolism, impacting many health
parameters from levels of serum cholesterol and triglycerides to
the pathological buildup of fat in the liver,” said Paul A.
Friedman, M.D., Chairman and CEO of Madrigal. “The lack of
selectivity of older compounds limited their clinical utility, so
we designed this study to provide clinical proof-of-concept for the
role of highly-specific THR ß activation in regulating lipid
metabolism in the liver to treat the cause of NASH. It will also
allow us to confirm the value of assessing the early reduction of
liver fat with imaging as a meaningful and predictive treatment
endpoint.”
“NASH is the leading liver disease in the United States with a
growing prevalence for which no FDA approved treatment is yet
available,” said Dr. Stephen Harrison, Principal Investigator of
the study as well as Medical Director for Pinnacle Clinical
Research, San Antonio, Texas, and Visiting Professor of Hepatology,
Oxford University. “We are hopeful that this novel approach, by
targeting an underlying pathophysiologic mechanism of NASH, will
eventually address a significant unmet need in this patient
population.”
Becky Taub, M.D., CMO and Executive VP, Research &
Development of Madrigal added, “Madrigal’s 36-week NASH study has a
unique design based on the mechanism of action of MGL-3196 that
allows us to determine the primary endpoint after 12 weeks of
treatment, the percent change in hepatic fat fraction from baseline
as measured by MRI-PDFF (magnetic resonance imaging-estimated
proton density fat fraction), an imaging-based
biomarker.”
The randomized, double-blind, placebo-controlled, multi-center
Phase 2 study will enroll 117 patients 18 years of age and older
with biopsy-confirmed NASH. Patients are randomized to receive
either placebo or MGL-3196, twice as many receiving MGL-3196 as
placebo. Efficacy will be 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 NASHNASH (non-alcoholic steatohepatitis)
is a common liver disease in the United States and world-wide,
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 2-5% of American
adults,1 or more than 15 million people. 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 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. Based on evidence of broad
activity and a favorable safety profile from pre-clinical and Phase
1 studies, MGL-3196 is entering Phase 2 clinical trials for the
treatment of non-alcoholic steatohepatitis (NASH) and familial
dyslipidemias/hypercholesterolemias. 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.
1 https://www.niddk.nih.gov/health-information/health-topics/liver-disease/nonalcoholic-steatohepatitis/Pages/facts.aspx
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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