RADNOR, PA — Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) has announced that its Phase 3 TrustTSC trial, which evaluated the use of oral ganaxolone for treating seizures in individuals with tuberous sclerosis complex (TSC), did not meet its primary endpoint. The trial aimed to measure the percent change in 28-day seizure frequency in both children and adults, but the results did not achieve statistical significance.
Despite the ganaxolone treatment showing a median seizure reduction of 19.7% compared to 10.2% for the placebo group, the outcome with a p-value of 0.09 indicated the results were not statistically significant. The trial, which involved 129 participants globally, allowed patients to continue their existing antiseizure medications, including mTOR inhibitors and cannabidiol, marking a significant step in combining ganaxolone with other standard care treatments.
CEO Scott Braunstein expressed disappointment over the trial’s results, stating, “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.” The safety profile of ganaxolone remained consistent with previous studies, with somnolence being the most common adverse event.
In light of these results, Marinus plans to halt further clinical development of ganaxolone for TSC. The company will also implement cost-reduction measures, including workforce downsizing. Marinus remains committed to its commercial product, ZTALMY® (ganaxolone), which is approved for treating seizures related to CDKL5 deficiency disorder.
Looking ahead, Marinus is exploring strategic alternatives to maximize shareholder value, engaging Barclays as an advisor. However, the company cautions that there are no guarantees of reaching new agreements or transactions and will provide updates only when definitive actions are taken.
The TrustTSC trial spanned multiple countries, including the U.S., Western Europe, and China, among others, and emphasized the complexity of treating TSC, a genetic disorder known for its diverse and severe neurological impacts, including epilepsy.
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