We are so excited and hopeful to be able to officially share this news:
MARINUS PHARMACEUTICALS INITIATES CLINICAL TRIAL WITH GANAXOLONE IN PCDH19 FEMALE PEDIATRIC EPILEPSY
RADNOR, PA, February 3, 2015 (Globe Newswire) — Marinus Pharmaceuticals, Inc.
(Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative neuropsychiatric therapeutics, announced that it has initiated a Phase 2 clinical trial with ganaxolone, a synthetic analog of the endogenous neurosteroid allopregnanolone, in female children with epilepsy caused by a mutation of the protocadherin 19 gene (PCDH19). PCDH19 female pediatric epilepsy is a rare disease that is estimated to affect approximately 15,000-30,000 females in the U.S. and is characterized by onset of cluster seizures before age 5, cognitive and sensory impairment of varying degrees, and behavioral disturbances. There are currently no approved therapies for PCDH19 female pediatric epilepsy.
The multicenter, open-label clinical trial is designed to evaluate the safety and efficacy of ganaxolone as adjunctive therapy for uncontrolled seizures in PCDH19 female pediatric epilepsy. “We are excited to advance the development of ganaxolone into this rare disease that affects the female pediatric population,” commented Christopher M. Cashman, Chief Executive Officer of Marinus Pharmaceuticals. “This is an important step in our clinical development strategy to advance ganaxolone for the large epilepsy market while in parallel pursuing unmet medical needs in pediatric orphan indications, including PCDH19 female pediatric epilepsy.”
This Phase 2 study is designed to enroll approximately 10 female pediatric patients between the ages of 2 and 10 years old, with a confirmed PCDH19 genetic mutation. After establishing baseline seizure frequency, patients will be treated with ganaxolone administered as either oral liquid suspension or capsules for up to 26 weeks. The primary endpoint of the study is percent change in seizure frequency per 28 days relative to baseline. Data from the study is anticipated later this year.
Dr. Gail Farfel, Chief Clinical and Regulatory Officer of Marinus Pharmaceuticals, stated, “Scientific researchers have linked the PCDH19 mutation to low levels of allopregnanolone, a naturally occurring neurosteroid. We believe that the novel mechanism of ganaxolone, our synthetic analog of allopregnanolone, coupled with data from earlier trials conducted in pediatric patients with intractable seizures, provides sound rationale to develop ganaxolone for treatment of PCDH19 female pediatric epilepsy.”