Marinus Pharmaceuticals Inc (NASDAQ:MRNS) has unveiled new data suggesting its drug ganaxolone is effective over the long-run in treating certain forms of pediatric epilepsy.
Data from the company’s Phase 2 trial which evaluated the impact of ganaxolone in children with CDKL5 deficiency disorder (CDD), a refractory form of pediatric epilepsy, showed the drug demonstrated “sustained, long-term” effectiveness. This is the first time a treatment has proved its worth in reducing seizure frequency in this type of epilepsy.
In its second advance, the data from a second cohort of the Phase 2 trial, which evaluated the effect of the drug when taken by children with PCDH19 epilepsy, showed a significant treatment effect in patients who were positive for a predictive clinical biomarker. But a negative point was that the frequency of epileptic seizures tended to increase for the children taking part who were negative for the biomarker.
“Having a clinical biomarker that could identify beforehand those patients with PCDH19 epilepsy that would respond to treatment with ganaxolone could be a game-changer in this refractory form of pediatric epilepsy,” concluded Christopher Cashman, the CEO of Marinus.
“Marinus Pharmaceuticals is evaluating the next steps needed to follow up on these encouraging results.”
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The results of Marinus’s recent research were presented at the American Epilepsy Society Annual Meeting in New Orleans on December 2 to the 3rd.
CDKL5 Deficiency disorder is a rare genetic disorder that is characterized by early-onset seizures as well as severe neuro-developmental impairment. Many children affected by CDD can’t walk, talk or feed themselves and are confined to wheel chairs.
Protocadherin 19 (PCDH19)-related epilepsy, meanwhile, is a type of epilepsy characterized by early-onset cluster seizures that vary in severity as well as cognitive and behavioral disturbances with or without intellectual disability.
“Both CDD and PCDH19 epilepsy are serious genetic conditions that cause early onset and difficult-to-control seizures in young children, particularly girls,” said Cashman. “Our hope in sharing this research is to advance the understanding and treatment of rare pediatric genetic epilepsies. Current anti-epilepsy drugs show limited or temporary relief in controlling the frequency or duration of seizures.”
Marinus’s investigational drug, ganaxolone, is designed to provide anti-seizure activity by calming the brain and restoring its electrical balance.
Marinus shares slipped 4.9% to hit $4.68 in Tuesday’s morning trading session.
Contact Ellen Kelleher at ellen@proactiveinvestors.com