Actions of Marinus Pharmaceutical (NASDAQ: MRNS) soared on Tuesday morning, trading 80% as of 11:35 a.m. EDT. The huge jump came after the company announced the positive results of a pivotal, advanced study evaluating its drug candidate ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD), a rare type epilepsy.
All of Marinus’ chips rely on ganaxolone, its only pipeline candidate. This big bet now seems to have paid off.
Ganaxolone met the primary endpoint of the Phase 3 study by reducing the median frequency of major motor seizures over 28 days by 32.2%, compared to just 4% for placebo. It also appeared to be generally well tolerated with a dropout rate in the group of patients receiving the drug below 5%.
These results greatly increase Marinus’ chances of obtaining FDA approval for ganaxolone as a treatment for CDD. There is currently no approved therapy specifically for this rare genetic disease. Other anti-epileptic drugs are used to treat it, but they are not very effective for most people.
Marinus Pharmaceuticals Medical Director Joe Hulihan also noted that the study’s promising results suggest potential in other indications. “Today’s success with ganaxolone in CDD will pave the way for us to accelerate our clinical studies in tuberous sclerosis Bourneville and possibly other rare pediatric epilepsies,” he said.
Marinus plans to present the results of ganaxolone at an upcoming scientific meeting. He also intends to launch an expanded access program in the fourth trimester that will allow CDD patients who were not in the clinical trial to start receiving the drug.
It may take some time for the biotechnology company gets all decisions from regulators about the drug, however. Marinus plans to file an application for FDA approval for ganaxolone for the treatment of CDD in mid-2021 and European Union approval by the end of the third quarter of next year. The company said it “will continue with its pre-commercial development plans, while simultaneously exploring opportunities to commercialize ganaxolone in CDD with third parties to maximize access for CDD patients.”
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