RADNOR, PA — Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) showcased new findings from its pivotal Phase 3 RAISE trial at the Neurocritical Care Society Annual Meeting, highlighting the potential of intravenous (IV) ganaxolone in treating refractory status epilepticus (RSE). The trial, which assessed patients whose seizures were unresponsive to at least two antiseizure medications, revealed significant results that could influence future treatment protocols.
In the RAISE trial, patients were randomized to receive either IV ganaxolone or a placebo, alongside standard care. One of the trial’s co-primary endpoints—cessation of status epilepticus (SE) within 30 minutes—was successfully met, with 80% of ganaxolone-treated patients achieving this outcome compared to only 13% in the placebo group.
The secondary endpoints presented included a remarkable reduction in median time to SE cessation, with ganaxolone achieving seizure control in 4.2 minutes versus 307.2 minutes for placebo. Additionally, 45% of patients treated with ganaxolone required no treatment escalation within 24 hours, a significant improvement over the 19% in the placebo group.
Despite falling short on the second co-primary endpoint—preventing progression to IV anesthesia within 36 hours—the trial demonstrated promising secondary outcomes. Notably, ganaxolone patients experienced a 93% reduction in EEG seizure burden, compared to a 36% reduction for those on placebo.
Dr. Brandon Foreman from the University of Cincinnati, who presented the data, noted the rapid antiseizure activity of ganaxolone, emphasizing its potential role in managing RSE. Although challenges remain, such as achieving statistical significance across all endpoints, the trial’s results provide valuable insights into the treatment’s benefits and limitations.
Joseph Hulihan, Marinus’ Chief Medical Officer, highlighted the importance of these findings in advancing RSE research, emphasizing the critical insights gained from this first placebo-controlled trial. The company plans to engage with the FDA to discuss the next steps for ganaxolone.
Supported partly by the Department of Health and Human Services and BARDA, this research marks a significant milestone in the pursuit of effective treatments for this complex disorder, offering hope for improved outcomes in patients with highly refractory seizures.
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