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Marinus did not meet early stopping criteria for status epilepticus trial

Marinus Pharmaceuticals announced that its Phase III RAISE study (NCT04391569) of IV ganaxolone for status epilepticus (SE) didn't meet early stopping criteria. They plan to complete the trial to decide on further development. An independent data monitoring committee (DMC) recommended continuing the Phase III study based on interim analysis. Topline results are expected to be released in summer 2024.

Upon completion with 100 patients, Marinus will review the trial's unblinded results to determine the drug's future development. Ganaxolone, a GABAA receptor modulator, aims to inhibit abnormal neuronal activity. While intended for IV use in SE, it's marketed orally as Ztalmy for cyclin-dependent kinase-like 5 (CDLK5) deficiency disorder (CDD).

SE is life-threatening, marked by prolonged or repetitive seizures without regaining consciousness. Ganaxolone gained FDA approval for CDD in March 2022 and UK MHRA approval in March 2024. Marinus anticipates Ztalmy to generate $263 million in global sales by 2029.