Marinus Pharmaceuticals Joins the Loulou Foundation in Collaboration on CANDID, a Comprehensive Observational Study in CDKL5 Deficiency Disorder

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced it has joined forces with the Loulou Foundation, a private foundation dedicated to advancing research on the causes and treatment of CDKL5 deficiency disorder (CDD), and six other biotech and pharmaceutical organizations, to undertake a key observational CDD clinical study to better understand the natural history and the utility of various clinical assessments. Marinus and the other industry organizations will share in the funding and governance of a three-year observational study in CDD patients – the Clinical Assessment of NeuroDevelopmental measures In CDD (CANDID) study. The Loulou Foundation will serve as the study coordinator, and the study will involve CDD clinical centers worldwide.

“Seizures in CDD are often refractory to treatment, and despite available anti-seizure medications, there remains an unmet need for treatments specific to CDD. At Marinus, we are developing ganaxolone, an investigational drug for the treatment of rare epilepsies, including CDD,” said Alex Aimetti, Ph.D., Vice President, Scientific Affairs at Marinus Pharmaceuticals. “We are pleased to be collaborating on the CANDID study to gain a more complete view of how CDD affects patients and to define relevant endpoints for future clinical trials. We are privileged to be adding to the scientific knowledge about this serious disorder and to be able to support the CDD community by partnering with the Loulou Foundation on this study.”

The CANDID study aims to provide researchers with information on the suitability of clinical assessments across multiple domains. The study also aims to document the longitudinal trajectories in CDD patients across the various assessments. The results of the CANDID study will ultimately be shared with the entire scientific and medical community to aid in the design of clinical trials and to inform therapeutic development for CDD and related neurodevelopmental disorders, including Rett syndrome and Angelman syndrome.

In September, Marinus’ New Drug Application for the use of ganaxolone to treat seizures associated with CDD was accepted for filing with priority review by the U.S. Food and Drug Administration (FDA). The FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of March 20, 2022. Validation of Marinus’ marketing authorization application by the European Medicines Agency is expected by the end of October. These applications for commercialization are supported by data from the Marigold trial, a Phase 3, double-blind placebo-controlled study which enrolled 101 patients and demonstrated that patients treated with ganaxolone experienced a 30.7% median reduction in 28-day major motor seizure frequency, compared to a 6.9% reduction for those receiving placebo, achieving the trial’s primary endpoint (p=0.0036). Patients in the Marigold open label extension treated with ganaxolone for at least 12 months (n=48) experienced a median 49.6% reduction in major motor seizure frequency. In the Marigold trial and its open label extension, ganaxolone was generally well tolerated with a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence.