Marinus Pharmaceuticals Inc. shares surged 49.1% to $1.72 per share in morning trade Monday after the company said its pediatric epilepsy drug had positive preliminary data in a four-person phase 2 study. The ganaxolone drug is intended to treat the rare genetic disorder CDKL5, which causes early-onset seizures that are hard to control. Patients are still enrolling in the mid-stage clinical trial, which is expected to enroll up to 10 patients, with additional data expected in the middle of this year. Three of the four patients already enrolled had a reduction in seizure frequency of 52% to 88%, with the fourth patient ending trial participation after four months “due to lack of efficacy,” Marinus said. Noting the small sample size, RBC Capital Markets analyst Simos Simeonidis said the results — particularly the response rate and range of reductions in seizure frequency — were nevertheless “encouraging” in a hard-to-treat group of patients. Marinus shares have risen 28.6% over the last three months, compared with a 5.6% rise in the S&P 500 .
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