Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the enrollment of the first patient in a Phase 3 clinical trial of IV ganaxolone (RAISE trial) for the treatment of refractory status epilepticus (RSE), a life-threatening occurrence of continued or intermittent seizures lasting more than five minutes in duration without recovery of consciousness. RSE is considered a medical emergency that can cause permanent brain damage or death if not quickly controlled.
The RAISE trial is a randomized, double-blind, placebo-controlled trial that will evaluate the efficacy and safety of IV ganaxolone in RSE patients who have failed benzodiazepines and two or more second line intravenous antiepileptic drugs (AEDs). The study will examine the proportion of participants with RSE who experience status epilepticus cessation within 30 minutes of initiation of IV ganaxolone as well as the rate of progression to IV anesthesia. Cessation will be measured and determined by clinical and EEG findings.
Marinus also announced an agreement with Ceribell, Inc. that will place the handheld Ceribell Rapid Response EEG system in up to 80 U.S. hospitals participating in its study. The electroencephalogram (EEG) devices are designed to be compact and easy to use and will facilitate the rapid diagnosis of nonconvulsive SE, which is confirmed by EEG demonstration of ongoing ictal activity.
The Ceribell Rapid Response EEG system is intended to provide bedside clinicians the ability to quickly triage and continuously monitor patients at risk for seizures. Within the RAISE trial, it will allow clinicians to rapidly establish a diagnosis of nonconvulsive SE in the absence of immediate availability of conventional EEG. The Ceribell 20-minute certification video trains the hospital neurology team members to install the Ceribell Rapid Response EEG headband in less than seven minutes. The headband collects EEG data via a web-based portal, allowing the hospital neurology team to access the data remotely.
Ganaxolone development for RSE is funded, in part, by the Biomedical Advanced Research and Development Authority (BARDA) part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services under contract number 75A50120C00159.
About the RAISE Trial
The Phase 3 RAISE Trial is a randomized, double-blind, placebo-controlled trial in status epilepticus (SE) patients who have failed benzodiazepines and two or more second line intravenous AEDs. Approximately 80 study sites in hospitals across the U.S. will participate. The trial is designed to enroll approximately 125 patients, who will be randomized to receive ganaxolone or placebo added to standard of care. With this number of participants, the study is designed to provide over 90 percent power to detect a 30 percent efficacy difference between ganaxolone and placebo.
The co-primary endpoints for the RAISE trial are (1) proportion of patients with SE cessation within 30 minutes of treatment initiation without other medications for the treatment of SE, and (2) proportion of patients with no progression to IV anesthesia for 36 hours following treatment initiation.
About Status Epilepticus (SE)
SE is a condition characterized by prolonged or repetitive seizures which, if treatment is delayed or ineffective, may result in medical or neurologic complications. SE is a neurologic emergency affecting between 75,000 “ 150,00 patients in the U.S. each year. Benzodiazepines are the first-line treatment for SE. If these are not successful in terminating SE, one or more IV AEDs are administered as the second line of treatment. If treatment with the first IV AED following benzodiazepines is ineffective, the patient is considered to have refractory SE (RSE).
About the Ceribell Rapid Response EEG System
The Ceribell Rapid Response EEG System consists of a 10-electrode (8-channel) headband, an EEG recorder with Brain Stethoscope feature and a cloud portal for storing and reviewing EEGs. This simplified configuration of EEG enables easy access to EEG within minutes. The Ceribell EEG System received FDA 510(k) clearance in 2017 and is commercially available in the U.S.
Ceribell, Inc. (www.ceribell.com), is headquartered in Mountain View, CA. Ceribell is focused on making EEG widely available, more efficient, and more cost-effective to improve the diagnosis and treatment of patients at risk for seizures.
About Marinus Pharmaceuticals
Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders. Ganaxolone is a positive allosteric modulator of GABAA receptors that acts on a well-characterized target in the brain known to have anti-seizure, anti-depressant and anti-anxiety effects. Ganaxolone is being developed in IV and oral dose forms intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Marinus recently completed the first ever Phase 3 pivotal trial in children with CDKL5 deficiency disorder and is conducting a Phase 2 trial in tuberous sclerosis complex, as well as a Phase 2 biomarker-driven proof-of-concept trial in PCDH19-related epilepsy. The company is conducting a Phase 3 trial in status epilepticus. For more information visit www.marinuspharma.com.
To the extent that statements contained in this press release are not descriptions of historical facts regarding Marinus, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as may, will, expect, anticipate, estimate, intend, believe, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Examples of forward-looking statements contained in this press release include, among others, statements regarding our clinical development plans for ganaxolone; our expectations for clinical trial sites for our Phase 3 trial in refractory status epilepticus; the potential safety and efficacy of ganaxolone; and the therapeutic potential of ganaxolone. Forward-looking statements in this press release involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, uncertainties and delays relating to the design, enrollment, completion, results of clinical trials, and interpretation of clinical trial results; unanticipated costs and expenses; early clinical trials may not be indicative of the results in later clinical trials; clinical trial results may not support regulatory approval or further development in a specified indication or at all; actions or advice of the U.S. Food and Drug Administration may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; our ability to obtain and maintain regulatory approval for our product candidate; our ability to obtain and maintain patent protection for our product candidates; delays, interruptions or failures in the manufacture and supply of our product candidate; our ability to raise additional capital; the effect of the COVID-19 pandemic on our business, the medical community and the global economy; and the availability or potential availability of alternative products or treatments for conditions targeted by us that could affect the availability or commercial potential of our product candidate. Marinus undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the company in general, see filings Marinus has made with the Securities and Exchange Commission.
Sasha Damouni Ellis
Vice President, Investor Relations & Corporate Communications
Marinus Pharmaceuticals, Inc.