Marinus Pharmaceuticals Announces Publication in The Lancet Neurology of ZTALMY® (ganaxolone) Phase 3 Marigold Trial Results
Results demonstrate safety and efficacy of ZTALMY, first FDA-approved treatment for seizures associated with CDKL5 deficiency disorder in patients two years and older
RADNOR, Pa.–(BUSINESS WIRE)–April 14, 2022–Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that The Lancet Neurology has published results from the pivotal Phase 3 Marigold trial of ZTALMY® (ganaxolone) for the treatment of seizures associated with CDKL5 deficiency disorder (CDD). The paper, “Efficacy and safety of ganaxolone in patients with CDKL5 deficiency disorder: a randomized, double-blind, placebo-controlled, Phase 3 trial,” can be accessed on The Lancet Neurology website, here. This was the first double-blind placebo-controlled study providing evidence of efficacy in CDD-associated seizures.
“The publication of these data highlights the importance of bringing new treatments to people affected by very refractory epilepsy, including the CDKL5 deficiency disorder community, and adds to the growing body of clinical research data currently available,” said Dr. Elia M. Pestana Knight, M.D., a Principal Investigator for the Marigold trial and pediatric epileptologist at the Cleveland Clinic Neurological Institute. “The results demonstrate that ZTALMY was effective in treating seizures associated with CDKL5 deficiency disorder and provides physicians with a novel medication for the management of this difficult to treat patient population.” Dr. Pestana Knight is a member of Marinus’ Scientific Advisory Board. She joined the Board after the completion of the Marigold randomized trial.
The paper presents the results of the Phase 3 Marigold trial, a double-blind placebo-controlled trial in which 101 patients were randomized and individuals treated with ZTALMY showed a median 30.7% reduction in 28-day major motor seizure frequency, compared to a median 6.9% reduction for those receiving placebo, achieving the trial’s primary endpoint (p=0.0036). ZTALMY was generally well-tolerated and showed a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence.
“We are pleased to have results from the Marigold trial published in The Lancet Neurology, a highly ranked peer-reviewed journal, at this inflection point for Marinus,” said Alex Aimetti, Senior Vice President of Scientific Affairs, Marinus Pharmaceuticals. “These trial results exemplify our confidence in the unique mechanism of action of ganaxolone to benefit CDD patients with uncontrolled seizures, and we will continue to explore its potential across a range of seizure disorders.”
About CDKL5 Deficiency Disorder
CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a mutation of the cyclin‑dependent kinase‑like 5 (CDKL5) gene, located on the X chromosome. CDD is characterized by early‑onset, difficult‑to‑control seizures and severe neurodevelopmental impairment.
About ZTALMY® (ganaxolone) oral suspension
ZTALMY® is the first and only FDA-approved treatment indicated specifically for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older. ZTALMY, a neuroactive steroid that acts as a positive allosteric modulator of the GABAA receptor, is taken three times daily. It is expected to be available in July following scheduling by the U.S. Drug Enforcement Administration.
Indication and Usage
ZTALMY is indicated for the treatment of seizures associated with cyclin-dependent kinase like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older.
