Marinus Pharmaceuticals Announces Key Business Updates for Tuberous Sclerosis Complex Program
Marinus Pharmaceuticals has completed enrollment in its Phase 3 TrustTSC trial, evaluating ganaxolone for treating seizures associated with tuberous sclerosis complex (TSC) in children and adults. This trial enrolled highly refractory TSC patients, most of whom have been treated with multiple antiseizure medications. The goal is to reduce somnolence-related adverse events, and only 2% of participants discontinued due to such events. Topline data from the trial is expected in early Q4 2024, with a supplemental New Drug Application (sNDA) submission targeted for April 2025. Additionally, the USPTO has granted Marinus a new method of use patent for ganaxolone in TSC treatment, expiring in 2040.
- Enrollment completed in Phase 3 TrustTSC trial.
- Focused on highly refractory TSC patients.
- Topline data expected in early Q4 2024.
- Targeting sNDA submission to FDA in April 2025.
- USPTO granted new method of use patent, expiring in 2040.
- Only 2% discontinuation due to somnolence-related adverse events.
- Topline data won't be available until Q4 2024, delaying potential market entry.
- Dependence on clinical trial outcomes for FDA approval.
Insights
The completion of enrollment in the Phase 3 TrustTSC trial is a significant milestone for Marinus Pharmaceuticals. This phase focuses on evaluating the efficacy and safety of ganaxolone in treating seizures associated with tuberous sclerosis complex (TSC), a genetic disorder characterized by benign tumors in multiple organs and often accompanied by severe seizures. The company has adjusted the titration schedule based on earlier trial findings to reduce somnolence-related adverse events, which indicates a proactive approach in improving patient outcomes.
For investors, this means Marinus is making substantial progress in its clinical development pipeline. The anticipation of topline data in early Q4 2024 provides a clear timeline for potential catalysts. If successful, these results could significantly enhance the company's market position in an area with high unmet medical needs. Given the rarity and severity of TSC, a new viable treatment option would likely see strong demand, offering a promising revenue stream.
The potential FDA submission in April 2025 and the request for priority review further underline the ambitious yet structured approach Marinus is taking. A successful FDA approval, particularly with priority review, could expedite ganaxolone's market entry, providing early revenue opportunities and strengthening investor confidence.
The granting of a new method of use patent (U.S. Patent No. 11,980,625) for ganaxolone extends Marinus Pharmaceuticals' intellectual property protection until 2040. This is important for maintaining a competitive advantage, as it prevents other companies from marketing similar treatments using the same method. With this extended exclusivity, Marinus can focus on maximizing the commercial potential of ganaxolone without immediate threats from generic competitors.
From an investor's perspective, this new patent strengthens Marinus' strategic position in the market, ensuring long-term revenue stability and potentially higher valuation due to the sustained exclusivity.
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
“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
Marinus also announced that the United States Patent and Trademark Office (USPTO) has granted a new method of use patent (
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
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
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
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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SVP, IR, HR & Corporate Affairs
Marinus Pharmaceuticals, Inc.
sweigle@marinuspharma.com
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Marinus Pharmaceuticals, Inc.
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Source: Marinus Pharmaceuticals
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