Epilepsia publishes two-year open-label
extension Marigold trial data of ZTALMY® (ganaxolone) oral
suspension CV in patients with CDKL5 deficiency disorder (CDD)
In preliminary results from a Phase 1 multiple
ascending dose (MAD) study, the second generation ganaxolone
formulation demonstrated linear kinetics through a wide dose range;
further formulation development to continue using modified-release
technology
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat seizure disorders, today announced the acceptance of seven
abstracts for presentation at the upcoming American Epilepsy
Society (AES) 2023 Annual Meeting, taking place in Orlando, Florida
December 1-5, 2023. Marinus will also host a scientific exhibit
showcasing a series of posters highlighting the potential of
ganaxolone in the treatment of refractory seizure disorders.
“The breadth of ganaxolone data and real-world evidence being
presented at this year’s AES Annual Meeting underscore our
commitment to advancing research and redefining the future of
epilepsy care,” said Joseph Hulihan, M.D., Chief Medical Officer at
Marinus. “In addition, the publication of data from the long-term
open-label extension of the Phase 3 Marigold trial in CDD and
continued efforts to develop a second generation ganaxolone
formulation reinforce our dedication to advancing innovative
treatment options that can have a significant impact on patients’
lives.”
The Epilepsia publication titled, “Long-term treatment with
ganaxolone for seizures associated with cyclin-dependent
kinase-like 5 deficiency disorder: 2-year open-label extension
follow-up,” can be accessed here. As previously reported, at two
years in the open-label extension phase of the Marigold trial,
patients (n=50) continuing treatment with ZTALMY experienced a
median 48.2% reduction in major motor seizure frequency, providing
supportive evidence for the maintenance of effect in seizures
associated with CDD. These results are being presented at AES 2023,
as outlined below.
Also announced today, preliminary data from a Phase 1 MAD study
of a second generation ganaxolone formulation demonstrated linear
kinetics through a wide dose range that could allow
individualization of treatment in patients with refractory
epilepsy, a key goal for the second generation formulation. Based
on these results, Marinus expects to apply extended-release
technologies to the formulation targeting consistent exposure to
achieve once or twice daily dosing while allowing physicians to
dose titrate to higher serum concentrations of ganaxolone. The
Company now anticipates initiating a clinical trial in
Lennox-Gastaut syndrome with a second generation formulation in
2025. The MAD study data will be submitted for presentation at an
upcoming medical meeting. IND-enabling studies for a ganaxolone
prodrug are expected to be completed by year end 2024.
While development of the second generation formulation proceeds,
Marinus intends to explore new clinical programs for ZTALMY oral
suspension in other refractory epilepsies in the second half of
2024.
Marinus AES Scientific Exhibit
Advancing Ganaxolone Research in Rare Seizure Disorders:
Updates from Marinus Pharmaceuticals Location: Room W315B
Date/Time: Monday, December 4, 2023, 8:00 - 11:00 a.m. ET
AES Presentation Details
A Phase 1 Single Ascending Dose, Open-Label Crossover
Comparative Bioavailability Study of a Second-Generation Ganaxolone
Oral Formulation Authors: J. Hulihan, M. Gasior, I. Miller, H.
Whalen Poster Session #: 2.258 Date/Time: Sunday, December 3, 2023,
12:00 - 2:00 p.m. ET
Sustained Seizure Control With 2 Years of Ganaxolone
Treatment for Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency
Disorder (CDD) in the Marigold Study Open-Label Extension
Authors: E. Pestana-Knight, S. Amin, N. Bahi-Buisson, O. Devinsky,
E. Marsh, R. Rajaraman, A. Aimetti, E. Rybak, F. Kong, I. Miller,
J. Hulihan, S. Demarest Poster Session #: 2.263 Date/Time: Sunday,
December 3, 2023, 12:00 - 2:00 p.m. ET
Analysis of Real-World Characteristics of Patients with CDKL5
Deficiency Disorder Enrolled for Ganaxolone Treatment Authors:
J. Flatt, A. Aimetti, I. Miller Poster Session #: 2.302 Date/Time:
Sunday, December 3, 2023, 12:00 - 2:00 p.m. ET
Patient Journey with Status Epilepticus: Understanding
Treatment Pathways, Outcomes, and Healthcare Burden Using Patient
Level Real-World Hospital Data Authors: M. Barra, S. Saikumar,
S. Shah, D. Katariya, A. Lovink, H. Vaitkevicius, E. Rybak Poster
Session #: 2.399 Date/Time: Sunday, December 3, 2023, 12:00 - 2:00
p.m. ET
Intravenous Ganaxolone Attenuates Sarin Nerve Agent Induced
Seizures Authors: P. Anantharam, K. Burenheide, A. Hunzinger,
J. Moore, P. Beske, M. Metea, M. Saporito Poster Session #: 3.065
Date/Time: Monday, December 4, 2023, 12:00 - 1:45 p.m. ET
Ganaxolone Administration via G-tube: Subgroup Analysis of
the Phase 3 Marigold Study in CDKL5 Deficiency Disorder (CDD)
Authors: M. Gasior, A. Aimetti, J. Hulihan, L. Alvarez Poster
Session #: 3.264 Date/Time: Monday, December 4, 2023, 12:00 - 1:45
p.m. ET
Effect of Concomitant Antiseizure Medications on the Safety
and Efficacy of Ganaxolone for the Treatment of Seizures Associated
with CDKL5 Deficiency Disorder: Findings from the Phase 3 MARIGOLD
Study Authors: R. Rajaraman, J. Flatt, A. Aimetti, J. Hulihan,
S. Perry Poster Session #: 3.272 Date/Time: Monday, December 4,
2023, 12:00 - 1:45 p.m. ET
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 about Marinus 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 the significant
impact that ganaxolone may have on patients’ lives; the statement
regarding the maintenance effect of ganaxolone in seizures
associated with CDD; the expectation that applying extended-release
technologies to the second generation ganaxolone will be able to
achieve more consistent exposure, once or twice daily dosing or
individualization of dosing; the expectation that further
formulation development will be able to optimize efficacy and
tolerability in the continued development of ganaxolone; the
ability of the Company to initiate a Lennox-Gastaut syndrome
clinical trial with second generation ganaxolone in 2025; the
ability of the Company to complete IND-enabling studies for a
ganaxolone prodrug by year end 2024; the intent to explore new
clinical programs for ZTALMY in other refractory epilepsies, and
the timing thereof; the potential benefits ZTALMY will provide for
physicians and patients; statements regarding our expected clinical
development plans, enrollment in our clinical trials, regulatory
communications and submissions for ganaxolone, and the timing
thereof; the safety and efficacy of ganaxolone, as well as its
therapeutic potential in a number of indications; 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, unexpected results or delays in the commercialization
of ZTALMY; unexpected market acceptance, payor coverage or future
prescriptions and revenue generated by ZTALMY; 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 varying
interpretation of clinical data; 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; the potential that regulatory
authorities, including the FDA and EMA, may not grant or may delay
approval for our product candidates; early clinical trials may not
be indicative of the results in later clinical trials; 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; our ability to obtain and
maintain regulatory approval for our product candidate; our ability
to develop new formulations of ganaxolone or prodrugs; our ability
to obtain, maintain, protect and defend intellectual property for
our product candidates; the potential negative impact of third
party patents on our or our collaborators’ ability to commercialize
ganaxolone; delays, interruptions or failures in the manufacture
and supply of our product candidate; 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 cash and
cash equivalents may not be sufficient to support its operating
plan for as long as anticipated; 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 potential for our ex-US partners to breach their
obligations under their respective agreements with us or terminate
such agreements in accordance with their respective terms; 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 studies 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 candidate. 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/20231120723811/en/
Company Contacts
Investors Jim DeNike Senior
Director, Investor Relations Marinus Pharmaceuticals, Inc.
jdenike@marinuspharma.com
Media Molly Cameron Director,
Corporate Communications & Investor Relations Marinus
Pharmaceuticals, Inc. mcameron@marinuspharma.com
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