The announcement of positive topline results in a late-stage epilepsy trial prompted a rally in Marinus Pharmaceuticals Inc. (NASDAQ: MRNS) shares on Tuesday. The results come from its registrational Phase 3 clinical trial (Marigold Study) evaluating the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy with refractory seizures.

The trial’s primary efficacy endpoint was the percentage change in 28-day frequency of major motor seizures during the double-blind phase relative to the six-week prospective baseline period. Patients given ganaxolone showed a significant 32.2% median reduction in 28-day major motor seizure frequency, compared to a 4.0% reduction for those receiving the placebo, achieving the primary endpoint. Ganaxolone generally was well tolerated, with a safety profile consistent with previous clinical studies. The most frequent adverse event was somnolence.

The trial showed numerical trends favoring ganaxolone across several predefined secondary endpoints. However, they did not meet statistical significance. Ganaxolone also did meet statistical significance in exploratory secondary endpoints.

Overall, the positive results from the trial demonstrated that ganaxolone can provide significant seizure reduction in patients with CDD, an important advance for the CDD community.

Based on these results, Marinus plans to submit an NDA for ganaxolone in the treatment of CDD to the U.S. Food and Drug Administration (FDA) in mid-2021 and a Marketing Authorization Application for ganaxolone for the treatment of CDD to the European Medicines Agency by the end of the third quarter of 2021.

Marinus Pharmaceuticals stock traded up about 76% to $3.71 on Tuesday, in a 52-week range of $1.01 to $3.87. The consensus price target is $5.44.

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