Marinus Shares RAISE Trial Progress and Q1 2024 Financials

Marinus Pharmaceuticals, a company devoted to innovative seizure therapeutics, has announced that an independent Data Monitoring Committee (DMC) has recommended continuing their pivotal Phase 3 RAISE trial. This trial is evaluating intravenous (IV) ganaxolone for treating refractory status epilepticus (RSE). Following an interim analysis, Marinus has opted to complete patient enrollment at around 100 participants, with results anticipated in summer 2024. These findings will help decide if development of IV ganaxolone should proceed.

Dr. Scott Braunstein, Chairman and CEO of Marinus, mentioned that although the trial did not meet early stopping criteria, the final outcome will only be discernible after full data analysis. The company is also looking into potential cost-saving strategies to solidify their financial position, notably as they near completion of enrollment in the global Phase 3 TrustTSC trial for tuberous sclerosis complex.

This RAISE trial for IV ganaxolone is bolstered partially by support from the Department of Health and Human Services, Administration for Strategic Preparedness and Response, and Biomedical Advanced Research and Development Authority (BARDA).

Marinus plans to finish enrollment of approximately 130 patients in their Phase 3 TrustTSC trial of ZTALMY® (ganaxolone) oral suspension CV by mid-May 2024, with topline results expected early in the fourth quarter of 2024. Subsequently, they aim to file a supplemental New Drug Application (sNDA) with the U.S. Food and Drug Administration in the first half of 2025, requesting priority review.

Additionally, Marinus is advancing a second-generation ganaxolone formulation, designed to enhance safety, efficacy, tolerability, and dosing frequency. They continue the commercial launch of ZTALMY® in the U.S., estimating net product revenue of $7.4 to $7.6 million for the first quarter of 2024. Their cash, cash equivalents, and short-term investments are preliminarily set at $113.3 million as of March 31, 2024. The company is reviewing cost reduction measures, expected to be put into action in Q2 2024 to extend the financial runway.

Status epilepticus (SE) is a severe, life-threatening condition marked by prolonged seizures due to mechanisms failing to terminate seizures or abnormally initiating them. SE affects roughly 150,000 patients annually in the U.S., with significant morbidity, mortality, and healthcare costs. Refractory SE (RSE) occurs when patients do not respond to first- and second-line treatments.

Ganaxolone, a neuroactive steroid, modulates synaptic and extrasynaptic GABAA receptors to enhance inhibitory signaling. IV ganaxolone's pharmacokinetic and pharmacodynamic properties make it suitable for SE treatment, demonstrating rapid and sustained seizure cessation in studies. The U.S. FDA has granted orphan drug designation to IV ganaxolone for potential SE treatment.

The RAISE (Randomized Therapy in Status Epilepticus) trial (NCT04391569) is a Phase 3 double-blind, randomized, placebo-controlled clinical study aimed at evaluating the safety and efficacy of IV ganaxolone in RSE patients. An independent DMC performs unblinded interim analysis when about two-thirds of the study's participants have completed the trial.

Marinus Pharmaceuticals focuses on developing seizure therapeutics and has introduced their FDA-approved medication ZTALMY® (ganaxolone) oral suspension CV in the U.S. Their ongoing efforts aim to enhance the therapeutic scope for both adult and pediatric patients in acute and chronic care settings.