Marinus Pharmaceuticals, Inc., a pharmaceutical firm dedicated to developing innovative therapeutics to treat rare seizure disorders, announced positive top-line results from its registrational Phase 3 clinical trial on Monday. The trial aimed to assess the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy with refractory seizures.
According to the results, CDKL5 deficiency disorder patients placed on ganaxolone reported a median 32.2% significant decrease in 28-day seizure frequency – achieving the trial's primary endpoint. The Phase 3 trial's primary endpoint was the percentage change in the 28-day frequency of common motor seizures during the double-blind phase relative to the six-week prospective baseline period. Ganaxolone was significantly well tolerated with a safety profile consistent with previous studies.
Due to these results, Marinus aims to submit an NDA for ganaxolone in the treatment of CDKL5 deficiency disorder to FDA in mid-2021. The firm also plans to submit a Marketing Authorization Application (MAA) for ganaxolone for the treatment of CDD to the European Medicines Agency (EMA) by the end of Q3 2021.
Quoting Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals, "The Marigold Study has two important firsts. The first double-blind placebo-controlled study provides evidence of efficacy specific to CDD and the first Phase 3 trial to examine three times a day dosing of ganaxolone in pediatric patients. We believe we are one step closer to providing the first treatment indicated for CDD and plan to continue our oral ganaxolone franchise investments."
The trail indicated numerical trends favoring ganaxolone across various predefined secondary endpoints. However, ganaxolone did not meet statistical significance in exploratory secondary endpoints.
Marinus plans to present the top-line results at an upcoming scientific meeting as it continues its pre-commercial development plans, while also exploring commercialization opportunities for ganaxolone in CDD with third parties to ensure full access for CDD patients.