Shares in Marinus Pharmaceuticals Inc (NASDAQ: MRNS) shot up almost 40% as it posted positive clinical study findings.
The data comes from initial CDKL5 patients enrolled in an ongoing Phase 2 open-label study evaluating its ganaxolone candidate as a treatment for orphan, genetic disorders.
CDKL5 is a severe, rare genetic disorder that results in early-onset, difficult-to-control seizures.
Enrollment is continuing in the study with top-line data expected in mid-2017.
“We are encouraged by the results in these difficult-to-treat pediatric patients,” said Dr. Jaakko Lappalainen, vice president of clinical development at the company.
“Concurrent with completing this study, we will be evaluating the potential for breakthrough therapy and applying for orphan drug designation with the United States Food and Drug Administration.
"CDKL5 pediatric epilepsy may prove to be an attractive and efficient path for ganaxolone and we look forward to evaluating results from the final patients enrolled in this cohort of the study.”
Shares in New York are at US$1.61 - an increase of 38.79% at the time of writing.