Marinus’ Ganaxolone gets Rare Pediatric Disease Designation for CDD

In advance of topline data from that study, Marinus has begun preparations for an Expanded Access Program (EAP) in CDD that will allow the company, on positive data, to offer ganaxolone to patients who were unable to participate in the Phase 3 study. The global, double blind, placebo-controlled Phase 3 Marigold Study evaluating the use of oral ganaxolone in children and young adults has enrolled 101 patients between the ages of 2 and 21 with a confirmed disease related CDKL5 gene variant.

Ganaxolone, a positive allosteric modulator of GABAA receptors, is being developed in intravenous and oral formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Unlike benzodiazepines, ganaxolone exhibits anti-seizure and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors. Ganaxolone has been studied in more than 1,600 subjects, both pediatric and adult, at therapeutically relevant dose levels and treatment regimens for up to four years.

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. It predominantly affects females and is characterized by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment. The CDKL5 gene encodes proteins essential for normal brain function. Most children affected by CDD cannot walk normally, talk, or care for themselves. Many also suffer from scoliosis, visual impairment, gastrointestinal difficulties and sleep disorders. There are no treatments approved specifically for CDD. Genetic testing is available to determine if a patient has a mutation in the CDKL5 gene. No previous late-stage clinical trials have been conducted in this patient population.

Marinus Pharmaceuticals Business Update and 1Q-2020 Financial Results

Marinus Pharmaceuticals, Inc. () today provided an update on its clinical development activities and reported its financial results for the first quarter ended March 31, 2020. […]
“We remain confident that our key milestones are still on track, including the topline data from our pivotal Phase 3 clinical trial in CDKL5 deficiency disorder in Q3 2020. With positive topline data, we intend to continue preparations for both our first NDA submission and the commercial launch of ganaxolone,” said Scott Braunstein, M.D., Chief Executive Officer of Marinus.

On March 31, 2020, the Company had cash and cash equivalents and investments totaling $77.8 million compared to $91.7 million on December 31, 2019. Marinus believes that its cash, cash equivalents and investments as of March 31, 2020 will enable the Company to fund operating expenses and capital expenditure requirements into the third quarter of 2021.

Research and development expenses increased to $15.0 million for the three months ended March 31, 2020, as compared to $8.9 million for the three months ended March 31, 2019. The primary drivers for the increase to our research and development expenditures are clinical and manufacturing activities in support of our Phase 3 trial in CDD and preparations for a Phase 3 trial in SE, partially offset by decreased costs for non-seizure disorder indications.

General and administrative expenses were $3.9 million for the three months ended March 31, 2020 as compared to $3.7 million in the prior year. The primary drivers of the increase were professional fees and other costs associated with an increased scale of operations.

The Company reported a net loss of $18.7 million for the three months ended March 31, 2020, compared to $12.5 million for the three months ended March 31, 2019. Cash used in operating activities increased to $14.0 million for the three months ended March 31, 2020 compared to $11.7 million for the three months ended March 31, 2019.

  • Constructive End of Phase 2 Meeting with FDA for Status Epilepticus Completed in March; Pivotal Phase 3 Clinical Trial Expected to Begin Next Quarter
  • Topline Data on Track for Q3 2020 from Pivotal Phase 3 Marigold Study in CDKL5 Deficiency Disorder and Preparations Continue for Potential NDA Filing
  • First Patients to be Screened this Quarter for Phase 2 Tuberous Sclerosis Complex Clinical Trial