Marinus Pharmaceuticals Receives Positive Response from FDA on Sufficiency of One Phase 3 Clinical Trial for Filing of New Drug Application (NDA) for the Use of Ganaxolone in CDKL5 Deficiency Disorder (CDD)

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it has received a positive response from the U.S. Food and Drug Administration (FDA) that the efficacy and safety data resulting from the company’s pivotal Phase 3 Marigold Study on the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD) appear sufficient to support the filing of a New Drug Application (NDA). Adequacy of these data to support an approval of ganaxolone for the proposed indication will be a matter of future FDA review.

CDD is a rare, genetic epilepsy with refractory seizures. Marinus provided the data to the FDA in a briefing document meant to support a Type C meeting, and the FDA provided written preliminary comments in response. Based on this feedback, Marinus is targeting a pre-NDA meeting by the end of Q1 to support submission.

“We are pleased with the FDA’s assessment of the sufficiency of the efficacy and safety data from one Phase 3 clinical trial to support an NDA filing,” said Scott Braunstein, M.D., Chief Executive Officer of Marinus. “We remain on track to submit the NDA for use of ganaxolone in CDD for FDA review by mid-2021 and look forward to continued advancement of ganaxolone as the first potential FDA approved treatment specifically indicated in patients diagnosed with CDD.”

In September, Marinus reported positive topline data from the Phase 3 Marigold Study, the first double-blind placebo-controlled trial to provide evidence of efficacy in CDD and the first Phase 3 trial to examine three times a day dosing of ganaxolone in pediatric patients.

In the Phase 3 Marigold trial, patients treated with 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 trial’s primary endpoint (p=0.002). In this trial, ganaxolone was generally well tolerated with a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence.

“We see continued progress in our other development programs,” continued Dr. Braunstein. “Marinus continues to initiate new sites to participate in the Phase 3 clinical trial of IV ganaxolone for the treatment of Refractory Status Epilepticus (RSE) or RAISE trial. While the COVID-19 pandemic is lengthening the time for new trial sites to open for enrollment, we do not anticipate this having major implications on the ultimate timing of trial results.”