US-based pharmaceutical company Marinus Pharmaceuticals has reported topline results from the Phase III RAISE clinical trial of intravenous (IV) ganaxolone for treating RSE.
The double-blind, randomised, placebo-controlled trial assessed the safety and efficacy of IV ganaxolone.
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It enrolled 96 patients with RSE who had not responded to at least two antiseizure medications.
These patients were randomly assigned to receive either IV ganaxolone or a placebo alongside standard care.
The study met its first co-primary endpoint with 80% of patients in the IV ganaxolone group experiencing cessation of status epilepticus within 30 minutes, compared with 13% in the placebo group.
Its second co-primary endpoint was not met, with 63% of the treatment group not progressing to IV anaesthesia over 36 hours versus 51% for placebo, lacking statistical significance.
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By GlobalDataBoth groups saw similar occurrence of SAEs, with hypotension being the most common SAE among those treated with IV ganaxolone.
Marinus said it remains confident in IV ganaxolone’s potential for treating RSE, citing its rapid antiseizure effects and control of status epilepticus as supported by additional continuous electroencephalogram (EEG) monitoring analyses.
Preliminary EEG data suggest that patients treated with IV ganaxolone experience a sustained reduction in seizure activity for up to 36 hours, with a median reduction of 88%, against 38% for the placebo group.
Marinus intends to discuss the potential pathways for IV ganaxolone in RSE treatment with the US Food and Drug Administration.
Marinus Pharmaceuticals chairman and CEO Scott Braunstein said: “Although the RAISE trial did not achieve statistical significance on one of its co-primary endpoints, these findings provide valuable insights that will guide our ongoing research and development in our mission to bring innovative and effective treatment options to those in need.
“We would like to thank the patients, families, investigators and their clinical trial sites for their contributions to this important research.”
