Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage
biopharmaceutical company pursuing novel therapeutics for
nonalcoholic steatohepatitis (NASH), today provides a summary of
corporate updates and reports first quarter 2023 financial results.
Paul Friedman, M.D., Chief Executive Officer of Madrigal,
stated, “In the first quarter of 2023, the Madrigal team made
significant progress advancing our regulatory preparations, disease
education initiatives, and market access strategy. Additionally, we
continue to execute against our clinical development objectives,
and have completed enrollment of the MAESTRO-NASH trial. We are
working diligently on the resmetirom approval process and recently
announced that we obtained Breakthrough Therapy designation for
resmetirom, reinforcing our confidence in our regulatory strategy
and the planned NDA filing in Q2 2023.”
Becky Taub, M.D., Chief Medical Officer and President of
Research & Development of Madrigal, stated, “The MAESTRO-NASH
primary trial data will be the opening presentation in the first
general session of EASL’s International Liver Meeting in June; we
look forward to sharing additional results from the study and
engaging in scientific exchange at this important scientific forum.
In addition to our primary oral presentation focused on the
MAESTRO-NASH biopsy results, we’ll be presenting several abstracts
from the MAESTRO program that provide a broader view of resmetirom
treatment response.”
Recent Corporate Highlights
- Madrigal announced that resmetirom has received Breakthrough
Therapy designation from the FDA for the treatment of patients with
NASH with liver fibrosis. A drug that receives Breakthrough Therapy
designation is eligible for more intensive guidance on an efficient
drug development program and organizational commitment involving
senior managers from FDA.
- The MAESTRO-NASH registrational trial in noncirrhotic NASH has
completed enrollment.
- Patient recruitment remains focused on MAESTRO-NASH-OUTCOMES, a
noninvasive Phase 3 clinical outcome trial evaluating resmetirom in
patients with well-compensated NASH cirrhosis with goals to support
an additional indication in NASH cirrhosis and support long-term
clinical benefit and full approval for noncirrhotic NASH.
- The Institute for Clinical and Economic Review (ICER) published
an updated Evidence Report for its value assessment of resmetirom
and obeticholic acid. The Evidence Report indicates that resmetirom
has the potential to be a cost-effective treatment for patients
with at-risk NASH.
Resmetirom Data Presentations at EASL
Multiple resmetirom abstracts have been accepted at EASL’s
International Liver Congress taking place June 21-24 in Vienna:
- Oral presentation: “Primary results from MAESTRO-NASH a pivotal
phase 3 52-week serial liver biopsy study in 966 patients with NASH
and fibrosis” [Thursday, June 22 at 10:30 AM. Presenter: Stephen
Harrison]
- Poster: “Characterizing the histologic implications of
resmetirom-induced liver volume reduction using artificial
intelligence-powered digital pathology” [Presenter: Janani
Iyer]
- Poster: “Resmetirom helps regulate thyroid hormone levels
within the liver in patients with nonalcoholic fatty liver disease”
[Presenter: Stephen Harrison]
- Poster: “Resmetirom improves the lipid/lipoprotein profile in
patients with nonalcoholic fatty liver disease” [Presenter: Naim
Alkhouri]
- Poster: “Imaging and biomarker thresholds to accurately
diagnose NASH cirrhosis in a 180 patient biopsy confirmed cohort”
[Presenter: Rohit Loomba]
NASH Epidemiology and Health Economics Outcomes Research
Presentations at ISPOR
Five Madrigal posters focused on NASH epidemiology and health
economics outcomes research are being presented at the ISPOR 2023
meeting taking place this week (May 7-10) in Boston:
- “NASH Progression Rates Based on Fibrosis and Inflammation
(NAS): A Paired Biopsy Analysis from a Natural History Cohort in
the US” [Presenter: Jesse Fishman]
- “The Impact of Treatment-related Changes in Lipids on the
Cost-effectiveness of Resmetirom and Obeticholic Acid for Treatment
of Nonalcoholic Steatohepatitis” [Presenter: Jesse Fishman]
- “Impact of Different Non-invasive Tests on Estimated Prevalence
of Presumed Nonalcoholic Steatohepatitis Among US Adults, NHANES
2017-2020” [Presenter: Tom O'Connell]
- “Systematic Literature Review, Network Meta-analysis, and
Cost-effectiveness Analysis of Resmetirom for the Treatment of
Nonalcoholic Steatohepatitis” [Presenter: Jesse Fishman]]
- “Primary Cardiovascular Event Risk Associated With Nonalcoholic
Steatohepatitis Among US Adults, NHANES 2017-2020” [Presenter: Tom
O'Connell]
Financial Results for the Three Months Ended March 31,
2023
As of March 31, 2023, Madrigal had cash, cash equivalents and
marketable securities of $329.5 million, compared to $358.8 million
at December 31, 2022. The decrease in cash and marketable
securities resulted primarily from cash used in operations of $84.1
million, partially offset by cash provided by financing and option
exercise activities.
Operating expenses were $78.3 million for the three month period
ended March 31, 2023, compared to $57.6 million in the comparable
prior year period.
Research and development expenses for the three month period
ended March 31, 2023 were $62.2 million, compared to $47.9 million
in the comparable prior year period. The increase is attributable
primarily to additional activities related to the Phase 3 clinical
trial activities, including the MAESTRO-NASH Outcomes for which no
expenses were incurred in the prior year quarter, and an increase
in headcount.
General and administrative expenses for the three month period
ended March 31, 2023 were $16.2 million, compared to $9.7 million
in the comparable prior year period. The increase in general and
administrative expenses for the latest three month period is due
primarily to increases in commercial preparation activities,
including an increase in headcount.
Interest income for the three month period ended March 31, 2023
was $3.8 million, compared to $0.1 million in the comparable prior
year period. The increase in interest income was attributable
primarily to higher interest rates and a greater principal
balance.
Interest expense for the three month period ended March 31, 2023
was $2.3 million, compared to $0.0 million in the comparable prior
year period. The increase in interest expense was attributable to
the Loan Facility we entered into in May 2022.
About the Resmetirom Phase 3 Registration Program for
the Treatment of NASH
Madrigal is currently conducting four Phase 3 clinical trials to
demonstrate the safety and efficacy of resmetirom for the treatment
of NASH: MAESTRO-NASH, MAESTRO-NAFLD-1, MAESTRO-NAFLD-OLE, and
MAESTRO-NASH-OUTCOMES.
MAESTRO-NASH is a multicenter, randomized, double-blind,
placebo-controlled Phase 3 study of resmetirom in patients with
liver biopsy-confirmed NASH and was initiated in March 2019. The
subpart H portion of the study enrolled more than 1,000 patients
with biopsy-proven NASH (at least half with F3 (advanced) fibrosis,
the remainder F2 or F1B (moderate fibrosis) with a few earlier F1
patients, randomized 1:1:1 to receive once-daily resmetirom 80 mg,
resmetirom 100 mg, or placebo. After 52 weeks of treatment, a
second liver biopsy is performed. The dual primary surrogate
endpoints on biopsy were NASH resolution with ≥2-point reduction in
NAS (NAFLD Activity Score), and with no worsening of fibrosis OR a
1-point decrease in fibrosis with no worsening of NAS. Achievement
of either primary endpoint was considered a successful trial
outcome. A key secondary endpoint was lowering of LDL-C.
Patients enrolled in the MAESTRO-NASH study (approximately
1,750) continue on therapy after the initial 52-week treatment
period for up to 54 months to accrue and measure hepatic clinical
outcome events including progression to cirrhosis on biopsy (52
weeks and 54 months) and hepatic decompensation events, as well as
all-cause mortality.
MAESTRO-NAFLD-1 was initiated in December 2019 and the 52-week
multicenter, randomized, placebo-controlled Phase 3 study of
resmetirom in over 1,200 patients with NAFLD, presumed NASH, has
completed the double-blind arms and an open-label 100 mg arm. An
additional open-label active treatment arm in patients with early
(well-compensated) NASH cirrhosis is ongoing. The primary endpoint
was to evaluate the safety and tolerability of resmetirom. A
separate 52 week Phase 3 clinical trial, an open-label extension
study of MAESTRO-NAFLD-1 (MAESTRO-NAFLD-OLE), is ongoing.
Patients in the 52-week Phase 3 MAESTRO-NAFLD-1 study were
randomized 1:1:1:1 to receive once-daily resmetirom 80 mg,
resmetirom 100 mg, placebo in double-blind arms or resmetirom 100
mg in an open-label arm. MAESTRO-NAFLD-1 (unlike MAESTRO-NASH), did
not include a liver biopsy and represents a “real-life” NASH study.
Patients with 3 metabolic risk factors were documented with NASH or
NAFLD by historical liver biopsy or noninvasive techniques. Using
noninvasive measures, MAESTRO-NAFLD-1 was designed to provide
incremental safety information to support the NASH indication as
well as provide additional data regarding clinically relevant key
secondary efficacy endpoints to better characterize the potential
clinical benefits of resmetirom on cardiovascular- and
liver-related endpoints. The primary safety endpoint and several
key secondary endpoints were met, including LDL-C, apolipoprotein
B, and triglyceride lowering and reduction of liver fat as
determined by MRI-PDFF. Additional secondary and exploratory
endpoints were assessed including reduction in liver enzymes,
FibroScan, and MRE scores, and other NASH biomarkers.
Data from the 52-week first 1,000 patient portion of
MAESTRO-NASH, together with data from MAESTRO-NAFLD-1,
MAESTRO-NAFLD-OLE, Phase 2 and Phase 1 data, including safety
parameters, will form the basis for a planned subpart H submission
to FDA for accelerated approval of resmetirom for treatment of
NASH.
In August 2022, Madrigal initiated MAESTRO-NASH-OUTCOMES, a
randomized double-blind placebo-controlled study in approximately
700 patients with early NASH cirrhosis to allow for noninvasive
monitoring of progression to liver decompensation events. A
positive outcome is expected to support the full approval of
resmetirom for noncirrhotic NASH, potentially accelerating the
timeline to full approval. In addition, this study has the
potential to support an additional indication for resmetirom in
patients with well-compensated NASH cirrhosis.
About NASH
Nonalcoholic steatohepatitis (NASH) is a more advanced form of
nonalcoholic fatty liver disease (NAFLD). NAFLD is estimated to
afflict more than 20% of adults globally, about 30% in the United
States. Of that population, 20% may have NASH.
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 NASH progresses to significant liver fibrosis (stages F2
and F3) the risk of adverse liver outcomes increases dramatically.
NASH is rapidly becoming the leading cause of liver transplantation
in the U.S. There are currently no FDA-approved therapies available
for the treatment of NASH.
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, thyroid hormone receptor (THR)-β
selective agonist designed to target key underlying causes of NASH
in the liver. 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; resmetirom’s potential to be a cost-effective specialty
therapy for NASH patients with significant liver fibrosis; 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, objectives, timing
and support for making for making a Subpart H (Accelerated Approval
of New Drugs for Serious or Life-Threatening Illnesses) submission
to FDA, projections or objectives for obtaining accelerated or full
approval for resmetirom, 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 and
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.
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,”
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; risks of obtaining and
maintaining regulatory approvals, including, but not limited to,
potential regulatory delays or rejections; 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, generally and in relation to COVID-19 related
measures and individual precautionary measures that may be
implemented or continued for an uncertain period of time; 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; 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; 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 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 in the section appearing in Part I,
Item 1A of its Annual Report on Form 10-K for the year ended
December 31, 2022, filed with the SEC on February 23, 2023, as
updated from time to time by Madrigal’s other filings with the
SEC.
Investor Contact Alex Howarth, Madrigal
Pharmaceuticals, Inc., IR@madrigalpharma.com
Media ContactChristopher Frates, Madrigal
Pharmaceuticals, Inc., media@madrigalpharma.com
|
Madrigal Pharmaceuticals, Inc. |
Condensed Consolidated Statements of
Operations |
(in thousands, except share and per share
amounts) |
(unaudited) |
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
March 31, |
|
|
2023 |
|
|
2022 |
|
Revenues: |
|
|
Total revenues |
$ |
- |
|
$ |
- |
|
Operating expenses: |
|
|
Research and development |
|
62,154 |
|
|
47,929 |
|
General and administrative |
|
16,182 |
|
|
9,658 |
|
Total operating expenses |
|
78,336 |
|
|
57,587 |
|
Loss from operations |
|
(78,336 |
) |
|
(57,587 |
) |
Interest income, net |
|
3,776 |
|
|
69 |
|
Interest expense |
|
(2,336 |
) |
|
- |
|
Net loss |
$ |
(76,896 |
) |
$ |
(57,518 |
) |
|
|
|
Basic and diluted net loss per common share |
$ |
(4.23 |
) |
$ |
(3.36 |
) |
Basic and diluted weighted average number of common shares
outstanding |
|
18,187,924 |
|
|
17,103,395 |
|
|
|
|
Madrigal Pharmaceuticals, Inc. |
Condensed Consolidated Balance Sheets |
(in thousands) |
(unaudited) |
|
|
|
|
|
|
|
|
|
|
March 31, |
December 31, |
|
2023 |
2022 |
|
|
|
Assets |
|
|
Cash, cash equivalents and marketable securities |
$ |
329,477 |
$ |
358,774 |
Other current assets |
|
1,807 |
|
2,595 |
Other non-current assets |
|
1,165 |
|
1,203 |
Total assets |
$ |
332,449 |
$ |
362,572 |
|
|
|
Liabilities and
Equity |
|
|
Current liabilities |
$ |
98,348 |
$ |
115,894 |
Long-term liabilities |
|
83,965 |
|
49,289 |
Stockholders’ equity |
|
150,136 |
|
197,389 |
Total liabilities and stockholders’ equity |
$ |
332,449 |
$ |
362,572 |
|
|
|
Madrigal Pharmaceuticals (NASDAQ:MDGL)
Gráfico Histórico do Ativo
De Mar 2024 até Abr 2024
Madrigal Pharmaceuticals (NASDAQ:MDGL)
Gráfico Histórico do Ativo
De Abr 2023 até Abr 2024