Marinus Pharmaceuticals Reports Mixed Results from Phase 3 RAISE Trial for Refractory Status Epilepticus Treatment

Marinus Pharmaceuticals

RADNOR, PAMarinus Pharmaceuticals, Inc. (Nasdaq: MRNS) recently announced the topline results from its Phase 3 RAISE trial, which evaluated the safety and efficacy of intravenous (IV) ganaxolone in treating refractory status epilepticus (RSE). This condition involves prolonged or rapidly recurring seizures that do not respond to first- and second-line treatments, posing significant health risks.

In the double-blind, randomized, placebo-controlled study, patients with RSE who had failed at least two antiseizure medications were assigned to receive either IV ganaxolone or a placebo, alongside standard care. The trial included 96 participants, with 49 receiving IV ganaxolone and 47 receiving a placebo.

The trial revealed mixed results:

  • Primary Endpoint Success: The first primary endpoint was achieved. A significant proportion of patients experienced cessation of status epilepticus within 30 minutes of initiating IV ganaxolone compared to the placebo group (80% vs. 13%; p<0.0001).
  • Secondary Endpoint Failure: The trial did not meet the second primary endpoint. It failed to show statistical significance in the proportion of patients not progressing to IV anesthesia for 36 hours after starting IV ganaxolone compared to placebo (63% vs. 51%; p=0.162).

Both treatment arms reported similar rates of serious adverse events, with hypotension being more common in the IV ganaxolone group (19 events for IV ganaxolone vs. 18 for placebo).

Marinus remains optimistic about the potential of IV ganaxolone for treating RSE. The drug’s rapid onset of antiseizure effects was supported by continuous electroencephalogram (EEG) monitoring, which showed durable reductions in seizure burden through 36 hours. Patients receiving IV ganaxolone exhibited an 88% median reduction in seizure burden, compared to 38% for the placebo group. These findings suggest that factors other than the severity of the status may have necessitated IV anesthesia, calling into question the measure’s accuracy in assessing seizure control.

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Marinus plans to continue analyzing the full dataset from the RAISE trial and will engage with the U.S. Food and Drug Administration to discuss a potential path forward for IV ganaxolone in RSE. The company intends to present the trial data at an upcoming medical meeting.

Additionally, Marinus will continue to offer IV ganaxolone for patients with super refractory status epilepticus under emergency investigational new drug applications.

Financially, Marinus expects its cash and cash equivalents to cover operating expenses, including capital expenditures and working capital, into the second quarter of 2025. This projection accounts for cost reduction measures announced earlier this quarter and recent amendments to its credit agreement with Oaktree Fund Administration, LLC, and its Revenue Interest Financing Agreement with Sagard Healthcare Royalty Partners, LP.

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