Enrollment complete and on track to report
topline data from the TrustTSC trial in tuberous sclerosis complex
(TSC) in the first half of Q4 with NDA filing targeted for April
2025
TrustTSC maintained a double-blind
discontinuation rate of 6.2% with 93% of patients continuing to the
open-label extension
Patients who completed Marinus’ Phase 2 TSC
trial and entered the long-term extension demonstrated a median
reduction in seizure frequency of 56% within two years (n=9)
Real-world claims data support a significant
unmet need in TSC with approximately 26% of patients having tried
and failed three or more antiseizure medications
Notice of Allowance received from the USPTO for
a patent application that claims ganaxolone oral titration regimens
for treating a broad range of epilepsies; the patent is expected to
grant shortly with a term that runs through September 2042
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat seizure disorders, today is hosting an Investor and Analyst
Day and will present an in-depth review of the Company’s
development program for ZTALMY® (ganaxolone) oral suspension CV in
tuberous sclerosis complex (TSC) ahead of the Phase 3 TrustTSC
trial topline data readout anticipated in the first half of the
fourth quarter of 2024. The event begins at 9 a.m. ET and will be
available via webcast here.
“Today, we are highlighting exciting clinical and commercial
progress as we prepare to report topline data from our global Phase
3, TrustTSC trial,” said Scott Braunstein, M.D., Chairman and Chief
Executive Officer of Marinus. “Newly reported data from our Phase 2
TSC trial patients showed durable and increasing reductions in
seizure frequency in the long-term extension follow-up period.
These data, along with a low discontinuation rate in the TrustTSC
trial and a high proportion of patients transitioning into the
open-label extension, increase our confidence that ZTALMY has the
potential to be a meaningful treatment option for TSC patients with
refractory epilepsy.”
"Patients with TSC face significant challenges due to the
complex nature of their condition, which is often characterized by
severe neurological manifestations, including refractory seizures
and neurodevelopmental delays. Despite advancements in treatment,
there remains a substantial unmet need for therapies that can
reduce the frequency of seizures in children and adults,” said Dr.
Mary Kay Koenig, M.D., Director of the Center for Treatment of
Pediatric Neurodegenerative Disease (CTPND), Director of Research
for the Division of Child & Adolescent Neurology at the
University of Texas McGovern Medical School and an Investigator in
the TrustTSC trial.
Dr. Braunstein added, “Alongside our clinical momentum, we are
poised and ready for a rapid and efficient U.S. launch in TSC,
pending FDA approval, based on our established commercial
infrastructure, insights gained from our successful launch in CDD,
and deep understanding of the market. In addition, we are excited
to share that we expect the patent office to grant new intellectual
property for ganaxolone that covers the titration regimen used in
the TrustTSC trial. With a U.S. market opportunity of approximately
$2.5 billion in CDD and TSC, we believe our differentiated approach
and ganaxolone’s unique mechanism of action, combined with expected
favorable reimbursement dynamics, can enable broader patient access
and address critical unmet needs in these challenging therapeutic
areas.”
Highlights from today’s presentations are summarized below.
ZTALMY®
- On track to achieve full year 2024 ZTALMY net product revenue
guidance of between $33 and $35 million
- Data from several proprietary market research studies show a
consistent unmet need among tuberous sclerosis complex (TSC)
patients with refractory epilepsy and indicate likely broad payer
coverage given the high disease burden and need for new
therapies
- Real-world claims data indicates that approximately 26% of
coded TSC patients have tried and failed three or more antiseizure
medications, supporting the Company’s addressable patient
population assumptions and representing an attractive commercial
opportunity
Clinical Pipeline
Tuberous Sclerosis Complex
(TSC)
Phase 3 TrustTSC Trial
- Topline data for the global Phase 3 TrustTSC trial of oral
ganaxolone in TSC remains on track for the first half of the fourth
quarter of 2024
- Enrollment (n=129) completed in May 2024, with last patient
visit in September
- The trial is designed with 90% power to detect a 25% difference
from placebo in percent seizure reduction
- The fully enrolled trial maintained a low double-blind
discontinuation rate of 6.2% with approximately 93% of patients
continuing to the open-label extension
- Targeting submission of a supplemental New Drug Application
(NDA) to the U.S. Food and Drug Administration (FDA) in April 2025
with a request for priority review
Phase 2 TSC Trial: Long-term Extension
Follow-up
- New long-term extension follow-up data showed durable and
increasing reductions in seizure frequency among patients treated
with ganaxolone over a period of up to two years
- Nine of the 23 patients enrolled in the open-label Phase 2 TSC
trial qualified for and entered the long-term extension; seven
patients completed two years of follow up with two patients
discontinuing within that time
- Patients (n=9) experienced a cumulative median reduction in
seizure frequency of 56% within two years
- Patients (n=6 with available data) experienced a median
reduction in seizure frequency of 87% during months 22-24
- Safety findings were consistent with the double-blind phase; no
new safety findings had emerged at the time of analysis
- Data accepted for presentation at the American Epilepsy Society
Annual Meeting in December 2024
Other Rare Genetic Epilepsies
- Presenting strategy to expand ZTALMY development in a range of
developmental and epileptic encephalopathies, including
Lennox-Gastaut syndrome, with clinical trials to begin in the
second half of 2025, pending the TSC topline data
- Targeting submission of an Investigational New Drug application
for a novel oral ganaxolone prodrug in the fourth quarter of
2025
General Business and Financial
Update
- Full year 2024 guidance remains unchanged with projected ZTALMY
net product revenue between $33 and $35 million and combined
selling, general and administrative and research & development
expenses in the range of approximately $135 to $140 million,
including stock-based compensation expense of approximately $20
million
- The Company expects that cash and cash equivalents of $64.7
million as of June 30, 2024, will be sufficient to fund the
Company’s operating expenses and capital expenditure requirements
into the second quarter of 2025
- The Company continues to make investments to expand ZTALMY
manufacturing capacity necessary for the global launch of the CDKL5
deficiency disorder indication and potential TSC expansion
- Targeting total Company profitability within 18 months of a
U.S. TSC launch
About Marinus Pharmaceuticals
Marinus is a commercial-stage pharmaceutical company dedicated
to the development of innovative therapeutics for seizure
disorders. The Company’s product, ZTALMY® (ganaxolone) oral
suspension CV, is an FDA-approved prescription medication
introduced in the U.S. in 2022. For more information, please visit
www.marinuspharma.com and follow us on Facebook, LinkedIn and
X.
Forward-Looking Statements
To the extent that statements contained in this press release
are not descriptions of historical facts regarding Marinus, they
are forward-looking statements reflecting the current beliefs and
expectations of management made pursuant to the safe harbor
provisions of the Private Securities Litigation Reform Act of 1995.
Words such as "may", "will", "expect", "anticipate", "estimate",
"intend", "believe", and similar expressions (as well as other
words or expressions referencing future events, conditions or
circumstances) are intended to identify forward-looking statements.
Examples of forward-looking statements contained in this press
release include, among others, our expectation to report topline
data from the TrustTSC trial in TSC in the first half of Q4 and a
targeted NDA filing of April 2025; our expectation that the patent
office will grant new intellectual property for ganaxolone that
covers the titration regimen used in the TrustTSC trial; our net
product revenue guidance; our expectation that we will initiate
additional clinical trials as a result of the pending TSC topline
data; our plans to submit an Investigational New Drug application
for a novel oral ganaxolone prodrug in the fourth quarter of 2025;
our full-year 2024 guidance and related financial projections; our
expected cash runway; our expected timeline for profitability; as
well as other statements regarding our commercial and clinical
strategy, development plans and timelines and other future
events.
Forward-looking statements in this press release involve
substantial risks and uncertainties that could cause our clinical
development programs, future results, performance or achievements
to differ significantly from those expressed or implied by the
forward-looking statements. Such risks and uncertainties include,
among others, unexpected market acceptance, payor coverage or
future prescriptions and revenue generated by ZTALMY; the pricing
and reimbursement process can be time consuming and may delay
commercialization of ZTALMY in one or more European countries; our
dependence on Orion to commercialize ZTALMY in Europe pursuant to
the exclusive collaboration agreement; unexpected actions by the
FDA or other regulatory agencies with respect to our products;
competitive conditions and unexpected adverse events or patient
outcomes from being treated with ZTALMY, uncertainties and delays
relating to the design, enrollment, completion, and results of
clinical trials; unanticipated costs and expenses; the company’s
cash and cash equivalents may not be sufficient to support its
operating plan for as long as anticipated; our ability to comply
with the FDA’s requirement for additional post-marketing studies in
the required time frames; the timing of regulatory filings for our
other product candidates; clinical trial results may not support
regulatory approval or further development in a specified
indication or at all; actions or advice of the FDA or EMA may
affect the design, initiation, timing, continuation and/or progress
of clinical trials or result in the need for additional clinical
trials; the size and growth potential of the markets for the
company’s product candidates, and the company’s ability to service
those markets; the company’s expectations, projections and
estimates regarding expenses, future revenue, capital requirements,
and the availability of and the need for additional financing;
delays, interruptions or failures in the manufacture and supply of
our product candidates; the company’s ability to obtain additional
funding to support its clinical development and commercial
programs; the company’s ability to protect its intellectual
property; and the effect of the COVID-19 pandemic on our business,
the medical community, regulators and the global economy. This list
is not exhaustive and these and other risks are described in our
periodic reports, including our annual reports on Form 10-K,
quarterly reports on Form 10-Q and current reports on Form 8-K,
filed with or furnished to the Securities and Exchange Commission
and available at www.sec.gov. Any forward-looking statements that
we make in this press release speak only as of the date of this
press release. We assume no obligation to update forward-looking
statements whether as a result of new information, future events or
otherwise, after the date of this press release.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240920105991/en/
Investors Sonya Weigle Chief People
and Investor Relations Officer Marinus Pharmaceuticals, Inc.
sweigle@marinuspharma.com
Media Molly Cameron Director,
Corporate Communications & Investor Relations Marinus
Pharmaceuticals, Inc. mcameron@marinuspharma.com
Marinus Pharmaceuticals (NASDAQ:MRNS)
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Marinus Pharmaceuticals (NASDAQ:MRNS)
過去 株価チャート
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