Marinus Pharmaceuticals has reported results from the pivotal Phase III RAISE trial for intravenous (IV) ganaxolone in treating refractory status epilepticus (RSE).
The trial, which met one of its two co-primary endpoints, showed significant effects on seizure cessation in patients with RSE.
The company presented the data at the Neurocritical Care Society (NCS) Annual Meeting.
Patients in the RAISE trial, who had RSE unresponsive to at least two antiseizure medications, were randomised to receive either IV ganaxolone or a placebo.
The trial’s co-primary endpoints focused on SE cessation within 30 minutes and prevention of progression to IV anesthesia within 36 hours.
Secondary endpoint results revealed at the meeting included a median time to SE cessation of 4.2 minutes for ganaxolone-treated patients versus 307.2 minutes for those on placebo.
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By GlobalDataAdditionally, 45% of ganaxolone patients avoided treatment escalation within 24 hours, compared to 19% for placebo.
Previously reported topline results highlighted that 80% of patients on IV ganaxolone achieved SE cessation within 30 minutes, significantly more than the 13% on placebo.
However, the trial could not achieve significance in the proportion of subjects not progressing to IV anesthesia over 36 hours.
It demonstrated a median reduction in EEG seizure burden of 93% in the ganaxolone group, compared to 36% for placebo.
Serious adverse events were comparable between the two groups, though hypotension was more common in the ganaxolone arm.
Marinus chief medical officer Joseph Hulihan said: “The findings we shared today underscore the potential of IV ganaxolone in the management of RSE, while also highlighting the challenges inherent in advancing research in this highly variable, complex disorder.
“We have completed the first placebo-controlled trial in RSE, which has yielded important insights into the benefit ganaxolone could provide to these critically ill patients. We look forward to reviewing our data package with the FDA and discussing next steps for IV ganaxolone.”
