Marinus Pharmaceuticals Provides Update on the Phase 3 RAISE Trial and Reports Preliminary First Quarter 2024 Financial Results

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that an independent Data Monitoring Committee (DMC) has recommended continuing the pivotal Phase 3 RAISE trial evaluating intravenous (IV) ganaxolone for the treatment of refractory status epilepticus (RSE) following an interim analysis.

Marinus has decided to complete enrollment in the RAISE trial at approximately 100 patients with topline results expected in the summer of 2024. Those results will be used to determine whether to continue development of IV ganaxolone. Marinus remains blinded to the RAISE trial data.

“While we are disappointed that RAISE did not meet the early stopping criteria, we will only be able to determine the trial’s outcome once we unblind and analyze the full data set,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “We will also be evaluating potential cost-saving strategies to provide the strongest capital position as we approach enrollment completion in the global Phase 3 TrustTSC trial in tuberous sclerosis complex.”

Ganaxolone development in the RAISE trial is being supported in part by the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA) under contract number 75A50120C00159.

General Business and Financial Update

Marinus expects to complete enrollment in the Phase 3 TrustTSC trial of ZTALMY (ganaxolone) oral suspension CV with approximately 130 patients in mid-May 2024. The Company expects topline results early in the fourth quarter of 2024 and anticipates filing a supplemental New Drug Application to the U.S. Food and Drug Administration in the first half of 2025 with a request for priority review.

Marinus also continues to develop a second-generation ganaxolone formulation intended to provide improved pharmacodynamic and pharmacokinetic profiles that could improve safety, efficacy and tolerability and enable less frequent dosing.

The Company continues the successful U.S. commercial launch of ZTALMY resulting in preliminary unaudited net product revenue of between $7.4 and $7.6 million for the first quarter of 2024. Marinus estimates preliminary unaudited cash, cash equivalents, and short-term investments of $113.3 million as of March 31, 2024. Cost reduction activities to extend the cash runway beyond the fourth quarter of 2024 are under review and are expected to be implemented in the current quarter.