Marinus Pharmaceuticals Announces Topline Results From Phase 3 TrustTSC Trial of Oral Ganaxolone in Tuberous Sclerosis Complex and Commences Process to Explore Strategic Alternatives
Marinus Pharmaceuticals (Nasdaq: MRNS) announced that its Phase 3 TrustTSC trial evaluating oral ganaxolone for treating tuberous sclerosis complex (TSC)-associated seizures failed to meet its primary endpoint. While the ganaxolone group showed a 19.7% median reduction in seizure frequency compared to 10.2% for placebo, the results weren't statistically significant (p=0.09). Following this outcome, Marinus is discontinuing further ganaxolone development, implementing cost reductions including workforce cuts, and exploring strategic alternatives with Barclays as advisor. The company will continue supporting ZTALMY®, its FDA-approved treatment for CDKL5 deficiency disorder, which currently treats over 200 patients.
Marinus Pharmaceuticals (Nasdaq: MRNS) ha annunciato che il suo studio di Fase 3 TrustTSC, che valuta ganaxolone per il trattamento delle crisi associate al complesso di sclerosi tuberosa (TSC), non ha raggiunto il suo obiettivo principale. Sebbene il gruppo in trattamento con ganaxolone abbia mostrato una riduzione mediana del 19,7% nella frequenza delle crisi rispetto al 10,2% del gruppo placebo, i risultati non sono stati statisticamente significativi (p=0,09). A seguito di questo esito, Marinus interromperà ulteriore sviluppo del ganaxolone, attuando riduzioni dei costi, compresi i tagli al personale, ed esplorerà alternative strategiche con Barclays come consulente. L'azienda continuerà a sostenere ZTALMY®, il suo trattamento approvato dalla FDA per il disturbo da carenza di CDKL5, che attualmente tratta oltre 200 pazienti.
Marinus Pharmaceuticals (Nasdaq: MRNS) anunció que su ensayo de Fase 3 TrustTSC, que evalúa ganaxolone para el tratamiento de las convulsiones asociadas al complejo de esclerosis tuberosa (TSC), no logró cumplir con su objetivo principal. Si bien el grupo de ganaxolone mostró una reducción media del 19,7% en la frecuencia de convulsiones en comparación con el 10,2% del placebo, los resultados no fueron estadísticamente significativos (p=0,09). Tras este resultado, Marinus está discontinuando el desarrollo adicional de ganaxolone, implementando recortes de costos, incluidos despidos, y explorando alternativas estratégicas con Barclays como asesor. La compañía continuará apoyando ZTALMY®, su tratamiento aprobado por la FDA para el trastorno por deficiencia de CDKL5, que actualmente trata a más de 200 pacientes.
Marinus Pharmaceuticals (Nasdaq: MRNS)는 경미한 증상이 있는 상피종복합체(TSC)와 관련된 발작 치료를 위한 경구용 가나졸론의 신뢰도 시험 3상(TrustTSC) 결과가 주요 목표를 달성하지 못했다고 발표했습니다. 가나졸론 그룹은 플라세보 그룹의 10.2%에 비해 발작 빈도가 19.7% 감소했으나, 결과는 통계적으로 유의미하지 않았습니다 (p=0.09). 이 결과에 따라 Marinus는 가나졸론의 추가 개발을 중단하고 인력 감축을 포함한 비용 절감 조치를 시행하며 Barclays를 고문으로 두고 전략적 대안을 탐색할 것입니다. 회사는 현재 200명 이상의 환자를 치료하고 있는 CDKL5 결핍 장애에 대한 FDA 승인 치료제 ZTALMY®에 대한 지원을 계속할 것입니다.
Marinus Pharmaceuticals (Nasdaq: MRNS) a annoncé que son essai de Phase 3 TrustTSC évaluant ganaxolone pour le traitement des crises associées au complexe de sclérose tubéreuse (TSC) n'a pas atteint son objectif principal. Bien que le groupe ganaxolone ait montré une réduction médiane de 19,7 % de la fréquence des crises contre 10,2 % pour le placebo, les résultats n'étaient pas statistiquement significatifs (p=0,09). Suite à ce résultat, Marinus mettra fin au développement ultérieur de ganaxolone, mettra en œuvre des réductions de coûts, y compris des licenciements, et explorera des alternatives stratégiques avec Barclays en tant que conseiller. L'entreprise continuera de soutenir ZTALMY®, son traitement approuvé par la FDA pour le trouble de la déficience en CDKL5, qui traite actuellement plus de 200 patients.
Marinus Pharmaceuticals (Nasdaq: MRNS) hat bekannt gegeben, dass seine Phase-3-Studie TrustTSC, die die orale Einnahme von Ganaxolon zur Behandlung von an den tuberösen Sklerose-Komplex (TSC) gebundenen Anfällen untersucht, das primäre Ziel nicht erreicht hat. Während die Ganaxolon-Gruppe eine mediane Reduktion der Anfallshäufigkeit um 19,7 % im Vergleich zu 10,2 % bei Placebo zeigte, waren die Ergebnisse statistisch nicht signifikant (p=0,09). Nach diesem Ergebnis wird Marinus die weitere Entwicklung von Ganaxolon einstellen, Kostensenkungsmaßnahmen, einschließlich Stellenabbau, umsetzen und strategische Alternativen mit Barclays als Berater prüfen. Das Unternehmen wird weiterhin ZTALMY® unterstützen, seine von der FDA zugelassene Behandlung für die CDKL5-Defizienzkrankheit, die derzeit über 200 Patienten behandelt.
- ZTALMY® continues to serve over 200 patients for CDKL5 deficiency disorder
- Ganaxolone showed numerically better seizure reduction (19.7%) versus placebo (10.2%)
- Safety profile remained consistent with previous clinical trials
- Phase 3 TrustTSC trial failed to meet primary endpoint
- Company discontinuing further ganaxolone clinical development
- Implementing workforce reductions and cost-cutting measures
- Strategic alternatives exploration suggests potential company sale or restructuring
Insights
The failure of the Phase 3 TrustTSC trial represents a significant setback for Marinus Pharmaceuticals. The trial's inability to meet its primary endpoint (
The company will maintain focus on ZTALMY®, their FDA-approved treatment for CDKL5 deficiency disorder, which currently serves over 200 patients. However, the strategic review announcement and engagement of Barclays signals serious concerns about the company's standalone future. The trial results, while showing some efficacy trends, weren't strong enough to support a supplemental New Drug Application (sNDA), significantly limiting growth potential.
This announcement represents a critical inflection point for Marinus, with severe implications for its business model and market position. The strategic alternatives exploration, coupled with cost-cutting measures, suggests potential scenarios ranging from a sale of the company to asset divestitures. With a market cap of only
The workforce reduction will help extend the cash runway, but investors should closely monitor cash burn and quarterly updates on the strategic review process. The stock will likely face significant pressure as the market digests both the trial failure and uncertain corporate future. The engagement of Barclays indicates a serious commitment to exploring all options, though there's no guarantee of a favorable outcome.
Trial did not meet the primary endpoint of percent change in 28-day TSC-associated seizure frequency (p=0.09); results showed numerically higher response rates for ganaxolone than placebo
Ganaxolone was generally well-tolerated in TrustTSC with a safety profile consistent with previous clinical trials
Cost reduction activities are underway
Marinus has engaged Barclays as an advisor to assist with evaluating strategic alternatives
“As the first controlled trial in TSC that allowed enrollment of patients taking a range of concomitant antiseizure medications, which included mTOR inhibitors and cannabidiol, these data represent a significant advancement in our understanding of the use of ganaxolone with other standard of care treatments,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “We are disappointed that the results of the TrustTSC trial are not likely to be sufficient for an sNDA filing. We are deeply grateful for the contributions of the clinical trial investigators, patients, and caregivers whose support and participation made the study possible.”
TrustTSC (NCT05323734) was a global Phase 3 randomized, double-blind, placebo-controlled clinical trial of adjunctive oral ganaxolone treatment in children and adults with TSC-related epilepsy (n=129).
Topline data demonstrated that:
-
The trial did not achieve statistical significance on its primary endpoint of percent reduction in 28-day frequency of TSC-associated seizures: median reduction was
19.7% for ganaxolone compared with10.2% for placebo (p=0.09). -
Ganaxolone was generally well-tolerated with a safety profile consistent with previous clinical trials. The most frequent adverse event was somnolence:
28.1% for ganaxolone compared with16.9% for placebo.
Marinus will continue to support and invest in the commercial growth of ZTALMY® (ganaxolone) oral suspension CV, which is FDA-approved for the treatment of seizures associated with CDKL5 deficiency disorder in patients two years of age and older, where more than 200 patients are receiving treatment.
As a result of the TrustTSC outcome, Marinus is discontinuing further ganaxolone clinical development and is taking additional steps to reduce costs, including a reduction in its workforce. The Company has also commenced a process to explore strategic alternatives with the goal of maximizing value for its stockholders. Marinus has engaged Barclays as an advisor to assist in reviewing its strategic alternatives. There can be no assurance that the exploration of strategic alternatives will result in any agreements or transactions, or as to the timing of any such agreements or transactions. Marinus does not intend to discuss or disclose further developments regarding the exploration of strategic alternatives unless and until its board of directors has approved a definitive action or otherwise determined that further disclosure is appropriate or required by law.
About the TrustTSC Trial
TrustTSC (NCT05323734) was 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 consisted of a four-week baseline period, followed by a 16-week double-blind treatment phase, which included a 28-day titration period followed by a 12-week maintenance period. Participants then had the opportunity to enter the ongoing long-term open-label extension. The primary endpoint was 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 ZTALMY® (ganaxolone) oral suspension
ZTALMY (ganaxolone) is a neuroactive steroid GABAA receptor modulator that acts on a well-characterized target in the brain known to have anti-seizure effects. It is a prescription medicine that has been approved by the
European Union Summary of Product Characteristics for ZTALMY.
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
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 expectations regarding the review and exploration of strategic alternatives and their potential impact on stockholder value; our plans to reduce costs to extend our resources; our expectations regarding the future of the company's operations, including our development and commercialization activities; and other statements regarding future events.
Forward-looking statements in this press release involve substantial risks and uncertainties that could cause our actual 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 risk that exploration of strategic alternatives may not result in any definitive transaction or enhance stockholder value and may create a distraction or uncertainty that may adversely affect our operating results, business, or investor perceptions; uncertainties regarding future costs and expenses; Marinus’ ability to continue as a going concern; Marinus’ ability to maintain compliance with its debt covenants and risks and uncertainties regarding the ability to do; 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
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Company Contacts
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Sonya Weigle
Chief People & Investor Relations Officer
Marinus Pharmaceuticals, Inc.
sweigle@marinuspharma.com
Media
Molly Cameron
Director, Corporate Communications & Investor Relations
Marinus Pharmaceuticals, Inc.
mcameron@marinuspharma.com
Source: Marinus Pharmaceuticals
FAQ
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