Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage
biopharmaceutical company pursuing novel therapeutics for
nonalcoholic steatohepatitis (NASH), today announced that the
Company’s Marketing Authorization Application (MAA) for resmetirom
for the treatment of NASH/metabolic dysfunction-associated
steatohepatitis (MASH) with liver fibrosis has been validated and
is now under evaluation with the European Medicines Agency’s (EMA)
Committee for Medicinal Products for Human Use (CHMP).
NASH is a leading cause of liver-related mortality and an
increasing burden on healthcare systems globally. Resmetirom is a
once-daily, oral, liver-directed THR-β agonist designed to target
key underlying causes of NASH. The clinical development program for
resmetirom is comprised of 18 clinical studies supporting the MAA:
twelve Phase 1 studies, two Phase 2 studies, and four Phase 3
studies. The pivotal MAESTRO-NASH trial of resmetirom is the only
Phase 3 trial in NASH to achieve both fibrosis reduction and NASH
resolution primary endpoints, an efficacy standard for NASH
therapies described in the EMA’s Reflection Paper on Regulatory
Requirements for the Development of Medicinal Products for Chronic
Non-infectious Liver Diseases.
“NASH with fibrosis represents a serious burden for both
patients and health systems. Without treatment, the disease can
lead to cirrhosis, liver failure, liver cancer and premature
death,” said Becky Taub, M.D., Chief Medical Officer and President
of Research & Development of Madrigal. “Based on the positive
results from our Phase 3 MAESTRO trials, we believe resmetirom has
the potential to become the first therapy for patients with NASH
with liver fibrosis to receive approval in Europe.”
In the United States, resmetirom received Breakthrough Therapy
designation from the FDA and was granted Priority Review with a
Prescription Drug User Fee Act (PDUFA) date of March 14, 2024, the
target date by which FDA intends to complete its review.
“As we near an FDA decision on accelerated approval of
resmetirom in the U.S. and advance our application in Europe,
Madrigal is generating new momentum for the NASH field,” said Bill
Sibold, Chief Executive Officer of Madrigal. “The results from our
pivotal Phase 3 trial recently published in the New England Journal
of Medicine provide a robust dataset to support regulatory
evaluation globally, and our two ongoing outcomes trials carry the
potential to reinforce Madrigal’s long-term leadership position in
NASH. Our regulatory, R&D and commercial activities continue to
accelerate, and each day brings us one step closer to delivering
the first foundational therapy to patients with this serious
disease.”
About NASH
Nonalcoholic steatohepatitis (NASH) is a more advanced form of
nonalcoholic fatty liver disease (NAFLD). NASH is a leading cause
of liver-related mortality and an increasing burden on healthcare
systems globally. Additionally, patients with NASH, especially
those with more advanced metabolic risk factors (hypertension,
concomitant type 2 diabetes), are at increased risk for adverse
cardiovascular events and increased morbidity and mortality.
Once patients progress to NASH with significant fibrosis
(consistent with fibrosis stages 2 and 3), the risk of adverse
liver outcomes increases dramatically. NASH is rapidly becoming the
leading cause of liver transplantation in the U.S.
Madrigal estimates that approximately 1.5 million patients have
been diagnosed with NASH in the U.S., of which approximately
525,000 have NASH with significant fibrosis. Madrigal plans to
focus on approximately 315,000 diagnosed patients with NASH with
significant fibrosis under the care of the liver specialist
physicians during the launch of resmetirom.
There are currently no FDA-approved therapies available for the
treatment of NASH.
NASH is also known as “metabolic dysfunction-associated
steatohepatitis (MASH)” following a change in disease nomenclature
introduced by hepatology medical societies in 2023.
About Madrigal Pharmaceuticals
Madrigal Pharmaceuticals, Inc. (Nasdaq: MDGL) is a
clinical-stage biopharmaceutical company pursuing novel
therapeutics for nonalcoholic steatohepatitis (NASH), a liver
disease with high unmet medical need. Madrigal’s lead candidate,
resmetirom, is a once-daily, oral, liver-directed THR-β agonist
designed to target key underlying causes of NASH. For more
information, visit www.madrigalpharma.com.
Forward Looking Statements
This communication includes “forward-looking statements” made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995, that are based on Madrigal’s beliefs
and assumptions and on information currently available to it, but
are subject to factors beyond its control. Forward-looking
statements reflect management’s current knowledge, assumptions,
judgment and expectations regarding future performance or events.
Forward-looking statements include: all statements that are not
historical facts; statements referenced by forward-looking
statement identifiers, including the examples in the paragraph
below; prospects for, and uncertainties associated with, obtaining
EMA/European Commission approval of our MAA submission for
resmetirom; estimates of patients diagnosed with and/or under care
for NASH; the relationship between NASH progression and adverse
patient outcomes; the estimated clinical burden of uncontrolled
NASH; analyses for patients with NASH with significant fibrosis
concerning potential progression to cirrhosis, decompensated
cirrhosis, liver transplant or death, and cardiovascular risks,
comorbidities and outcomes; health economics assessments or
projections; resmetirom’s potential to be the first specialty
therapy for NASH patients with significant liver fibrosis, in the
U.S. and/or Europe; projections or objectives for obtaining
accelerated or full approval for resmetirom, including all
statements concerning potential clinical benefit to support
potential accelerated approval and/or approval; and statements or
references concerning - the potential efficacy and safety of
resmetirom for noncirrhotic NASH patients and cirrhotic NASH
patients, possible or assumed future results of operations and
expenses, business strategies and plans (including ex-US.
Launch/partnering plans), research and development activities, and
the timing and results associated with the future development of
resmetirom, the timing and completion of projected future clinical
milestone events, including enrollment, additional studies,
top-line data and open label projections, plans, Madrigal’s primary
and key secondary study endpoints for resmetirom and the potential
for achieving such endpoints and projections, the potential to
support an additional indication for resmetirom in patients with
well-compensated NASH cirrhosis, optimal dosing levels for
resmetirom, projections regarding potential NASH or NAFLD and
potential patient benefits with resmetirom, including future NASH
resolution, safety, fibrosis treatment, cardiovascular effects,
lipid treatment, and/or biomarker effects with resmetirom, and
strategies, objectives and commercial opportunities, including
potential prospects or results.
Forward-looking statements can be identified by terms such as
“accelerate,” “achieve,” “allow,” “anticipates,” “appear,” “be,”
“believes,” “can,” “confidence,” “continue,” “could,”
“demonstrates,” ”design,” “estimates,” “expectation,” “expects,”
“forecasts,” “future,” “goal,” “help,” “hopeful,” “inform,”
“intended,” “intends,” “may,” “might,” “on track,” “planned,”
“planning,” “plans,” “positions,” “potential,” “powers,”
“predicts,” ”predictive,” “projects,” “seeks,” “should,” “will,”
“will achieve,” “will be,” “would” or similar expressions and the
negatives of those terms.
Forward-looking statements are subject to a number of risks and
uncertainties including, but not limited to: the assumptions
underlying the forward-looking statements; general risks of
obtaining and maintaining regulatory approvals, including, but not
limited to, potential regulatory delays or rejections; specific
risks of, and uncertainties concerning, obtaining EMA regulatory
approval; our belief that resmetirom could meet the efficacy
standard for NASH therapies described in the EMA’s Reflection Paper
on Regulatory Requirements; risks associated with meeting the
objectives of Madrigal’s clinical studies, including, but not
limited to Madrigal’s ability to achieve enrollment objectives
concerning patient numbers (including an adequate safety database),
outcomes objectives and/or timing objectives for Madrigal’s
studies; any delays or failures in enrollment, and the occurrence
of adverse safety events; risks related to the effects of
resmetirom’s mechanism of action; the achievement of enrollment
objectives concerning patient number, safety database and/or timing
for Madrigal’s studies; enrollment and trial conclusion
uncertainties; market demand for and acceptance of our products;
the potential inability to raise sufficient capital to fund ongoing
operations as currently planned or to obtain financings on terms
similar to those arranged in the past; the ability to service
indebtedness and otherwise comply with debt covenants; outcomes or
trends from competitive studies; future topline data timing or
results; our ability to prevent and/or mitigate cyber-attacks,
unauthorized exfiltration of data or other security incidents; the
risks of achieving potential benefits in studies that includes
substantially more patients, and patients with different disease
states, than prior studies; the timing and outcomes of clinical
studies of resmetirom; the uncertainties inherent in clinical
testing; and uncertainties concerning analyses or assessments
outside of a controlled clinical trial. 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
submissions filed with the U.S. Securities and Exchange Commission,
or SEC, 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. Madrigal
specifically discusses these risks and uncertainties in greater
detail under the captions “Cautionary Note Regarding
Forward-Looking Statements” and “Risk Factors Summary” and within
Part I, Item 1A of its Annual Report on Form 10-K for the year
ended December 31, 2023, filed with the SEC on February 28, 2024,
and as updated from time to time by Madrigal’s other filings with
the SEC.
Investor Contact Tina Ventura, Madrigal
Pharmaceuticals, Inc., IR@madrigalpharma.com
Media ContactChristopher Frates, Madrigal
Pharmaceuticals, Inc., media@madrigalpharma.com
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