ZTALMY® (ganaxolone) Phase 3 Marigold Trial Results

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

Marinus Pharmaceuticals, Inc., a pharmaceutical company dedicated to the development of therapeutics to treat seizure disorders, announced on 15 April 2022 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. 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. 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. Elia M. Pestana Knight, M.D., a Principal Investigator for the Marigold trial and pediatric epileptologist at the Cleveland Clinic Neurological Institute.

Dr. Pestana Knight is a member of Marinus’ Scientific Advisory Board. She joined the Board after the completion of the Marigold randomised trial.

The paper presents the results of the Phase 3 Marigold trial, a double-blind placebo-controlled trial in which 101 patients were randomised 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.

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 characterised 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 the US in July 2022 following scheduling by the US Drug Enforcement Administration.

Ganaxolone does not currently have a marketing authorisation in the UK for treating seizures caused by CDD. NICE began a Health Technology Appraisal in the UK in January 2022. Orion Corporation signed a European wide marketing and distribution agreement with Marinus Pharmaceuticals for ganaxolone in August 2021.

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.

About Marinus Pharmaceuticals

Marinus is a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders. Ganaxolone is a neuroactive steroid GABAA receptor modulator that acts on a well-characterised target in the brain known to have anti-seizure effects. It is being developed in IV and oral dose formulations intended to maximise therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. For more information visit www.marinuspharma.com.