Shares in Marinus Pharmaceuticals (MRNS) surged 63% in Monday’s after-hours trading, after Marinus announced positive top-line results from its Phase 3 clinical trial of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy with refractory seizures.
In the trial, patients given ganaxolone showed a significant 32.2% median reduction in 28-day major motor seizure frequency, compared to a 4% reduction for those receiving the placebo, achieving the primary endpoint.
Ganaxolone was generally well tolerated with a safety profile consistent with previous clinical studies. The most frequent adverse event was somnolence.
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 (MAA) for ganaxolone for the treatment of CDD to the European Medicines Agency (EMA) by the end of Q3 2021.
“The Marigold Study has two important firsts. It’s the first double-blind placebo controlled study providing evidence of efficacy specific to CDD and the first Phase 3 trial to examine three times a day dosing of ganaxolone in pediatric patients,” cheered Scott Braunstein, M.D., CEO of Marinus Pharmaceuticals.
“We believe we are one step closer to providing the first treatment indicated for CDD, and plan to continue our investments in the oral ganaxolone franchise” he added.
The trial showed numerical trends favoring ganaxolone across several predefined secondary endpoints, however, ganaxolone did not meet statistical significance. Ganaxolone did meet statistical significance in exploratory secondary endpoints.
Earlier in the day, Marinus also revealed a five-year development contract with the Biomedical Advanced Research and Development Authority (BARDA), part of the U.S. Department of Health and Human Services. The news sent shares 6% higher in Monday’s trading.
The contract is to support the development of IV ganaxolone for the treatment of refractory status epilepticus (RSE), a life-threatening condition in which a significant number of patients do not respond to first- and second-line anticonvulsant drugs.
RSE can occur as a result of a variety of serious, acute medical conditions or after exposure to nerve agents.
The agreement covers a base period during which BARDA will provide subject matter expertise and $21 million to fund, on a cost share basis, the company’s planned Phase 3 clinical trial of ganaxolone for the treatment of RSE and will fund preclinical studies of ganaxolone in nerve agent exposure animal models.
Depending on favorable clinical and preclinical outcomes in the base period, the contract includes up to approximately $30 million of additional BARDA funding spanning three options in support of manufacturing, supply chain, clinical, regulatory and toxicology activities.
Under the contract, Marinus will be responsible for cost-sharing in the amount of $33 million if all development options are completed. (See MRNS stock analysis on TipRanks)
Shares in Marinus are down 3% year-to-date, but the stock scores a bullish Strong Buy Street consensus with 4 back-to-back buy ratings. Meanwhile the average analyst price target stands at $6.50 (208% upside potential).
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