Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage
biopharmaceutical company pursuing novel therapeutics for
nonalcoholic steatohepatitis (NASH), today provides a summary of
recent corporate accomplishments and reports second quarter 2023
financial results.
Paul Friedman, M.D., Chief Executive Officer of Madrigal,
stated, “The recent submission of the resmetirom NDA represents an
important milestone for Madrigal and the NASH community. Based on
the depth and breadth of efficacy and safety data we have generated
through the MAESTRO program, I believe we are well-positioned to
support the FDA’s review of resmetirom. In parallel with our
regulatory activities, our Commercial and Medical Affairs teams are
intently focused on preparing for a potential first-to-market
launch in the U.S.”
Becky Taub, M.D., Chief Medical Officer and President of
Research & Development of Madrigal, stated, “In addition to the
positive biopsy results supporting our regulatory filings in the
U.S. and Europe, the MAESTRO program has generated extensive
biomarker and imaging data to help advance noninvasive strategies
that may be used in real-world clinical practice to identify
appropriate patients for resmetirom and monitor treatment response.
The MAESTRO-NASH results were featured in the opening session of
the EASL Congress earlier this summer, and we look forward to
presenting additional noninvasive data from the study at future
scientific congresses.”
Remy Sukhija, Chief Commercial Officer of Madrigal, added, “As a
once-daily, oral medication that is intended to treat the
underlying causes of NASH in the liver, resmetirom has a unique
opportunity to become the foundational therapy for patients with
NASH with liver fibrosis. Our conviction in resmetirom’s potential
is grounded in extensive market research with hepatologists and
gastroenterologists. Based on the efficacy and safety data observed
in the MAESTRO-NASH trial, these liver specialist physicians report
strong intent-to-prescribe resmetirom for appropriate patients, if
approved.”
Mr. Sukhija continued, “NASH care pathways are evolving rapidly
in anticipation of an approved treatment, and the new
recommendation from the American Diabetes Association to screen for
NASH in patients with type 2 diabetes, alongside similar existing
recommendations from hepatology and gastroenterology medical
societies, should expand the population of identified patients in
need of treatment. Additionally, Madrigal’s efforts to improve
disease education – including the ‘NASH Explored’ campaign and the
new ‘Taking on Fatty Liver and NASH’ campaign – are gaining
momentum and driving engagement with healthcare providers, patients
and payers.”
Recent Corporate Highlights
- Madrigal announced submission of the NDA seeking accelerated
approval of resmetirom for the treatment of NASH with liver
fibrosis. The clinical development program for resmetirom is
comprised of 18 clinical studies supporting the NDA: twelve Phase 1
studies, two Phase 2 studies, and four Phase 3 studies.
- Madrigal presented the Phase 3 MAESTRO-NASH primary results
during the opening general session of the EASL Congress™. This
first scientific presentation of detailed MAESTRO-NASH results
confirmed achievement of the primary endpoints across multiple
patient subgroups. Noninvasive imaging and biomarker data supported
findings on liver biopsy and demonstrated broad treatment response
to resmetirom. Further analyses of the results reinforced the
resmetirom safety and tolerability profile.
- Madrigal highlighted its support for International NASH Day and
announced expanded partnerships with patient advocacy groups
focused on NASH disease education.
- Madrigal launched Taking on Fatty Liver and NASH, a new disease
education campaign and website that is designed to educate, inform,
and inspire people with NASH with liver fibrosis to find the right
care team, understand their risk, and manage their liver health. It
provides information about NASH disease progression, noninvasive
testing options, and community resources from patient advocacy
groups.
- The Institute for Clinical and Economic Review (ICER) published
a Final 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.
Financial Results for the Six Months Ended June 30,
2023
As of June 30, 2023, Madrigal had cash, cash equivalents and
marketable securities of $298.4 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
$159.4 million, partially offset by the capital raised under the
Loan Facility (“Loan Facility”) with Hercules Capital, Inc.
(“Hercules”) and our at-the-market sales agreement.
Operating expenses were $86.5 million and $164.8 million for the
three month and six month periods ended June 30, 2023, compared to
$70.3 million and $127.9 million in the comparable prior year
periods.
Research and development expenses for the three and six month
periods ended June 30, 2023 were $68.6 million and $130.8 million,
compared to $58.5 million and $106.4 million in the comparable
prior year periods. The increase is attributable primarily to
additional activities related to the Phase 3 clinical trials, and
an increase in head count.
General and administrative expenses for the three and six month
periods ended June 30, 2023 were $17.8 million and $34.0 million,
compared to $11.8 million and $21.4 million in the comparable prior
year periods. The increase is due primarily to increases in
commercial preparation activities, including an increase in
headcount and an increase in non-cash stock compensation.
Interest income for the three and six month periods ended June
30, 2023 was $3.6 million and $7.3 million, compared to $0.3
million and $0.4 million in the comparable prior year periods. The
increase in interest income was due primarily to a higher average
interest rate in 2023.
Interest expense for the three and six month periods ended June
30, 2023 was $2.9 million and $5.2 million, compared to $0.8
million and $0.8 million in the comparable prior year periods. The
increase in interest expense was as a result of the Loan Facility
we entered with Hercules.
About the Resmetirom Phase 3 Registration Program for
the Treatment of NASH
Resmetirom is a once daily, oral, thyroid hormone receptor
(THR)-β selective agonist designed to target key underlying causes
of NASH in the liver.
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. The portion of the study designed to
support a subpart H approval enrolled more than 1,000 patients with
biopsy-proven NASH with fibrosis, randomized 1:1:1 to receive
once-daily resmetirom 80 mg, resmetirom 100 mg, or placebo. 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 after 52 weeks of treatment. Achievement of either
primary endpoint was considered a successful trial outcome.
In December 2022, Madrigal announced that both daily oral doses
of resmetirom achieved both MAESTRO-NASH primary liver biopsy
endpoints. Multiple secondary endpoints were also achieved,
including statistically significant reductions by resmetirom as
compared with placebo in atherogenic lipids and lipoproteins, liver
enzymes, fibrosis biomarkers, and imaging tests.
Resmetirom was generally safe and well-tolerated at both the 80
mg and 100 mg doses. Consistent with previous Phase 2 and Phase 3
data, the most common adverse event reported with greater frequency
in the resmetirom groups versus placebo was an excess of generally
mild and transient diarrhea and nausea at the beginning of
therapy.
Patients enrolled in MAESTRO-NASH (approximately 1,750 total
enrollment) 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. This portion of the study is designed to
generate confirmatory data that, if positive, will help verify
resmetirom’s clinical benefit and support full approval.
MAESTRO-NAFLD-1 was a 52-week multicenter, randomized,
placebo-controlled, double-blind Phase 3 study of resmetirom in
~1,200 patients with NAFLD, presumed NASH. MAESTRO-NAFLD-1 might be
considered a “real-world” NASH study in that diagnosis was based on
noninvasive measures rather than liver biopsy. The primary endpoint
was to evaluate the safety and tolerability of resmetirom.
Patients in the MAESTRO-NAFLD-1 study were randomized 1:1:1:1 to
receive once-daily resmetirom 80 mg, resmetirom 100 mg, or placebo
in double-blind arms or resmetirom 100 mg in an open-label arm.
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 of MAESTRO-NAFLD-1 and key secondary
endpoints were achieved: resmetirom was safe, well-tolerated and
provided statistically significant improvements in LDL-C,
apolipoprotein B, triglycerides, and liver fat as measured by
MRI-PDFF.
An additional open-label active treatment arm in 180 patients
with early (well-compensated) NASH cirrhosis was conducted.
Resmetirom was safe and well tolerated in the MAESTRO-NAFLD-1
open-label cohort of patients with well-compensated NASH cirrhosis.
As observed in patients with noncirrhotic NASH, mild GI adverse
events were seen at the beginning of therapy. Resmetirom reduced
LDL-C, other atherogenic lipids and lipoproteins, and MRI-PDFF in
patients with NASH cirrhosis and also reduced liver and spleen
volume.
A separate 52 week Phase 3 clinical trial, an open-label active
treatment extension study of MAESTRO-NAFLD-1 (MAESTRO-NAFLD-OLE),
in about 700 patients is ongoing.
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, form the basis for Madrigal’s subpart H submission to
FDA for accelerated approval of resmetirom for treatment of NASH
with liver fibrosis.
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.
In NASH, thyroid hormone beta activity in the liver is impaired,
leading to a reduction in mitochondrial function and beta-oxidation
of fatty acids, which in turn drive inflammation and liver
fibrosis.
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 the first specialty therapy for
NASH patients with significant liver fibrosis; statements
concerning potential accelerated 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, 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, demonstrating clinical benefit to
support accelerated approval, 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; 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 amended by our Form 10-K/A
filed with the SEC on March 3, 2023, and 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 |
|
Six Months
Ended |
|
June 30, |
|
June 30, |
|
|
2023 |
|
|
2022 |
|
|
|
2023 |
|
|
2022 |
|
Revenues: |
|
|
|
|
|
Total revenues |
$ |
- |
|
$ |
- |
|
|
$ |
- |
|
$ |
- |
|
Operating
expenses: |
|
|
|
|
|
Research and development |
|
68,605 |
|
|
58,499 |
|
|
|
130,759 |
|
|
106,428 |
|
General and administrative |
|
17,845 |
|
|
11,774 |
|
|
|
34,027 |
|
|
21,432 |
|
Total operating expenses |
|
86,450 |
|
|
70,273 |
|
|
|
164,786 |
|
|
127,860 |
|
Loss from operations |
|
(86,450 |
) |
|
(70,273 |
) |
|
|
(164,786 |
) |
|
(127,860 |
) |
Interest income, net |
|
3,551 |
|
|
323 |
|
|
|
7,327 |
|
|
392 |
|
Interest expense |
|
(2,901 |
) |
|
(780 |
) |
|
|
(5,237 |
) |
|
(780 |
) |
Net loss |
$ |
(85,800 |
) |
$ |
(70,730 |
) |
|
$ |
(162,696 |
) |
$ |
(128,248 |
) |
|
|
|
|
|
|
Basic and diluted net loss per common share |
$ |
(4.69 |
) |
$ |
(4.14 |
) |
|
$ |
(8.91 |
) |
$ |
(7.50 |
) |
Basic and diluted weighted average number of common shares
outstanding |
|
18,310,952 |
|
|
17,103,395 |
|
|
|
18,249,778 |
|
|
17,103,395 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Madrigal
Pharmaceuticals, Inc. |
|
|
|
Condensed
Consolidated Balance Sheets |
|
|
|
(in
thousands) |
|
|
|
(unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
June
30, |
December
31, |
|
|
|
|
|
2023 |
|
|
2022 |
|
|
|
|
|
|
|
|
|
|
Assets |
|
|
|
|
|
Cash, cash
equivalents and marketable securities |
$ |
298,418 |
|
$ |
358,774 |
|
|
|
|
Other
current assets |
|
3,177 |
|
|
2,595 |
|
|
|
|
Other
non-current assets |
|
866 |
|
|
1,203 |
|
|
|
|
Total assets |
$ |
302,461 |
|
$ |
362,572 |
|
|
|
|
|
|
|
|
|
|
Liabilities and Equity |
|
|
|
|
|
Current
liabilities |
$ |
99,706 |
|
$ |
115,894 |
|
|
|
|
Long-term
liabilities |
|
99,249 |
|
|
49,289 |
|
|
|
|
Stockholders’ equity |
|
103,506 |
|
|
197,389 |
|
|
|
|
Total liabilities and stockholders’ equity |
$ |
302,461 |
|
$ |
362,572 |
|
|
|
|
|
|
|
|
|
|
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