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 U.S., Western Europe, Canada, Israel, Australia and China. To be eligible, participants were required to have inadequate seizure control and have been treated with at least two prior antiseizure medications.
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 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 U.S. Food and Drug Administration and the European Commission for appropriate patients with CDKL5 deficiency disorder.
U.S. Prescribing Information for ZTALMY® (ganaxolone) oral suspension CV.
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 U.S. in 2022.