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Business Wire 20-Nov-2023 4:01 PM
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.
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.