Monday, Marinus Pharmaceuticals Inc ( MRNS ) 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. 

The results will be used to determine whether to continue the 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, 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.”

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 FDA in the first half of 2025 with a request for priority review.

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

The company continues the 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.

Price Action: MRNS shares are down 77.30% at $1.71 on the last check Monday.