Pharmaceutical

Marinus Pharmaceuticals (NASDAQ:MRNS) has announced it is initiating a Phase 3 clinical study evaluating oral ganaxolone in children with PCDH19-related epilepsy, which is a rare genetic epilepsy. As quoted in the press release: If successful, the Violet Study is intended to support the regulatory filings for approval of ganaxolone in this underserved and refractory patient …

Marinus Pharmaceuticals (NASDAQ:MRNS) has announced it is initiating a Phase 3 clinical study evaluating oral ganaxolone in children with PCDH19-related epilepsy, which is a rare genetic epilepsy.

As quoted in the press release:

If successful, the Violet Study is intended to support the regulatory filings for approval of ganaxolone in this underserved and refractory patient population.

The Violet Study is a global, double-blind, randomized, placebo-controlled pivotal Phase 3 clinical study evaluating ganaxolone in children with PCDH19-RE. The study will enroll up to 70 patients between the age of 1 and 17 with a confirmed PCDH19 mutation. All patients that meet eligibility will be stratified into one of two biomarker groups and randomized (ganaxolone or placebo) within each stratum. The trial will consist of an 8-week prospective baseline period to collect seizure data, followed by a 17-week double-blind treatment phase. Patients randomized to ganaxolone will titrate over four weeks to a dose of up to 600 mg of ganaxolone oral liquid suspension three times a day and maintain that dose for the following 13-weeks. After the double-blind period, all patients who meet certain eligibility requirements will have the opportunity to receive ganaxolone in an open label phase of the study. The Company expects to begin screening patients for enrollment into the study in the second quarter of 2019 and data from the study are estimated to be available in 2021.

“Following our End-of-Phase 2 meeting with the FDA and Scientific Advice from the EMA, we are extremely excited at the prospect of incorporating a potentially clinically useful and predictive neurosteroid biomarker into our clinical trial,” said Dr. Lorianne Masuoka, Chief Medical Officer of Marinus. “We believe this could be the beginning of a targeted, personalized treatment for patients suffering from rare genetic epilepsies. A neurosteroid biomarker that can predict response may have potential applications in epileptic conditions beyond PCDH19. This new pivotal study in PCDH19 along with our on-going Phase 3 trial in CDKL5 Deficiency Disorder (Marigold Study) strengthens Marinus’ commitment to rare forms of refractory pediatric epilepsy with no approved therapies.”

Click here to read the full press release.

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