- Nano cap Marinus Pharmaceuticals (NASDAQ:MRNS) eases 5% premarket on robust volume in response to its announcement of top-line results from a small exploratory open-label study of GABA modulator ganaxolone in females with PCDH19 pediatric epilepsy, an Orphan Drug designation by the FDA.
- 64% (n=7/11) of patients experienced a reduction in seizures from baseline with 54% of the responders (n=4/7) showing a reduction greater than 50%.
- 73% (n=8/11) experienced an increase in seizure-free days.
- The trial evaluated ganaxolone as adjunctive treatment, administered as oral liquid suspension or capsules, for 26 weeks after establishing baseline seizure frequency up to 12 weeks.
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Ganaxolone is a small molecule inhibitor of the neurotransmitter GABA, which helps control epileptic seizures.