RADNOR, PA — Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) recently announced its decision to proceed with the Phase 3 RAISE trial of intravenous (IV) ganaxolone for treating refractory status epilepticus (RSE), a severe form of epilepsy, following an interim analysis by an independent Data Monitoring Committee (DMC). The trial did not meet the early stopping criteria but will move forward to complete enrollment of approximately 100 patients, with results anticipated in summer 2024.
The company’s CEO, Scott Braunstein, M.D., expressed disappointment over not meeting the early stopping criteria but remains hopeful pending a full data analysis. Additionally, Marinus is exploring cost-saving measures to maintain a strong financial position as it completes enrollment for another global Phase 3 trial, TrustTSC, targeting tuberous sclerosis complex.
The RAISE trial’s development is partially funded by a contract from the Department of Health and Human Services’ Biomedical Advanced Research and Development Authority (BARDA), reflecting the government’s interest in advancing treatments for critical health conditions like RSE.
In parallel, Marinus is nearing completion of enrollment for its Phase 3 TrustTSC trial of ZTALMY® (ganaxolone) oral suspension CV, with top-line results expected in the fourth quarter of 2024. A supplemental New Drug Application submission to the U.S. Food and Drug Administration is planned for the first half of 2025, seeking priority review.
Moreover, Marinus is developing a second-generation ganaxolone formulation aimed at improving safety, efficacy, and tolerability while allowing for less frequent dosing. This initiative underscores the company’s commitment to enhancing treatment options for epilepsy and other neurological conditions.
On the commercial front, Marinus has reported preliminary unaudited net product revenue of $7.4 to $7.6 million for the first quarter of 2024 from its successful U.S. launch of ZTALMY. The company also reported preliminary unaudited cash reserves of $113.3 million as of March 31, 2024. Plans are underway to implement cost reduction activities within the current quarter to extend the company’s financial runway beyond the fourth quarter of 2024.
The continuation of the RAISE trial and the progress of the TrustTSC trial could potentially offer new hope for patients with severe neurological conditions. However, the company’s strategic cost management and the success of its commercial product, ZTALMY, will be crucial in sustaining its research and development efforts in the face of financial pressures.
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