Enrollment complete in Phase 3 TrustTSC trial
with topline data anticipated in the first half of Q4 2024
USPTO grants additional method of use patent
for ganaxolone in TSC
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat seizure disorders, today announced completion of enrollment
in the global Phase 3 TrustTSC trial evaluating oral ganaxolone for
the treatment of seizures associated with tuberous sclerosis
complex (TSC) in children and adults.
“We are pleased to announce we have completed enrollment in our
pivotal Phase 3 trial in tuberous sclerosis complex,” said Alex
Aimetti, Ph.D., Chief Scientific Officer at Marinus
Pharmaceuticals. “The TrustTSC study enrolled highly refractory TSC
patients experiencing a significant seizure burden despite
treatment with multiple antiseizure medications, including the
majority of patients having exposure to mTOR inhibitors. We would
like to thank the TSC community for their support, trust and
participation as we strive to bring a new treatment option to TSC
patients.”
Joseph Hulihan, M.D., Chief Medical Officer of Marinus added,
“Based on the findings in Marinus’ Phase 2 TSC trial, the titration
schedule of ganaxolone was modified in the Phase 3 TrustTSC trial
with the goal of reducing the frequency of somnolence-related
adverse events and improving patients’ response to treatment. With
less than two percent of TrustTSC participants discontinuing due to
somnolence-related adverse events, this improvement in tolerability
may have the potential to yield meaningful efficacy outcomes in
this patient population.”
Topline data from the TrustTSC trial is anticipated in the first
half of the fourth quarter of 2024. Marinus is targeting submission
of a supplemental New Drug Application to the U.S. Food and Drug
Administration in April 2025 with priority review requested.
Marinus also announced that the United States Patent and
Trademark Office (USPTO) has granted a new method of use patent
(U.S. Patent No. 11,980,625) for ganaxolone in the treatment of
TSC, expiring in 2040. This is Marinus’ second method of use patent
granted for ganaxolone in TSC, further strengthening the Company’s
intellectual property position.
About the TrustTSC Trial TrustTSC (NCT05323734) is a
global Phase 3 randomized, double-blind, placebo-controlled
clinical trial of adjunctive oral ganaxolone treatment in children
and adults with TSC-related epilepsy. The trial consists of a
four-week baseline period, followed by a 16-week double-blind
treatment phase, followed by either a two-week down titration
period or a four-week crossover period for qualifying individuals
who choose to enter the long term open-label phase. The primary
endpoint is percent change in 28-day TSC-associated seizure
frequency.
TrustTSC enrolled participants at sites in the U.S., Western
Europe, Canada, Israel, Australia and China. To be eligible,
participants were required to have inadequate seizure control and
been treated with at least two prior antiseizure medications
(although there are exceptions for younger participants). It is the
first controlled trial in TSC to allow enrollment of participants
taking a full range of concomitant medications, including
everolimus and cannabidiol.
About Tuberous Sclerosis Complex Tuberous sclerosis
complex (TSC) is a rare, multisystem genetic disorder caused by
inherited mutations in the TSC1 gene or TSC2 gene. It is often
characterized by non-cancerous tumors, skin abnormalities, and
severe neurological manifestations including refractory seizures
and neurodevelopmental delays. The condition is a leading cause of
genetic epilepsy, often occurring in the first year of life as
either focal seizures or infantile spasms. While the disease
phenotype can be extremely variable, neurologic manifestations such
as epilepsy can be seen in up to 90% of TSC patients.
About Marinus Pharmaceuticals Marinus is a
commercial-stage pharmaceutical company dedicated to the
development of innovative therapeutics for seizure disorders. The
Company first introduced FDA-approved prescription medication
ZTALMY® (ganaxolone) oral suspension CV in the U.S. in 2022 and
continues to invest in the potential of ganaxolone in IV and oral
formulations to maximize therapeutic reach for adult and pediatric
patients in acute and chronic care settings. For more information,
visit www.marinuspharma.com.
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, statements regarding statements regarding our
expected clinical development plans, enrollment in our clinical
trials, regulatory communications and submissions for ganaxolone,
and the timing thereof; our expected data readouts; our
expectations and beliefs regarding the FDA and EMA with respect to
our product candidates; our expectations regarding continued
investment in IV and oral formulations of ganaxolone; and other
statements regarding the company's future operations, financial
performance, financial position, prospects, objectives and other
future event.
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, the Company’s ability to continue as a going concern;
unexpected actions by the FDA or other regulatory agencies with
respect to our products and product candidates; 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 our operating plan for
as long as anticipated; 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; our ability to develop new formulations of
ganaxolone or prodrugs; our ability to obtain, maintain, protect
and defend intellectual property for our product candidates;
delays, interruptions or failures in the manufacture and supply of
our product candidates; the company’s expectations, projections and
estimates regarding expenses, future revenue, capital requirements,
and the availability of and the need for additional financing; the
company’s ability to obtain additional funding to support its
clinical development and commercial programs; the risk that drug
product quality requirements may not support continued clinical
investigation of our product candidates and result in delays or
termination of such clinical trials and product approvals; and the
availability or potential availability of alternative products or
treatments for conditions targeted by us that could affect the
availability or commercial potential of our product candidates.
This list is not exhaustive and these and other risks are described
in our periodic reports, including the annual report 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240517339301/en/
Investors Sonya Weigle SVP, IR, HR
& Corporate Affairs Marinus Pharmaceuticals, Inc.
sweigle@marinuspharma.com
Media Molly Cameron Director,
Corporate Communications & Investor Relations Marinus
Pharmaceuticals, Inc. mcameron@marinuspharma.com
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