RADNOR, Pa.–(BUSINESS WIRE)–Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to developing innovative therapies to treat seizure disorders, today announced that it has resumed screening and recruitment for Phase 3 RAISE ( Randomized treatment in status epilepticus) trial in refractory status epilepticus (RSE). The company continues to expect the best results from the RAISE trial in the second half of 2023.
Marinus announced in February that it was suspending enrollment in the RAISE trial after routine stability monitoring showed visible particles of aluminum phosphate in the drug solution. During the pause, the chair of the trial’s independent data monitoring committee reviewed the clinical safety data and determined that no safety events were identified in patients previously treated with the batches of Ganaxolone IV.
The trial resumed using new lots of the current IV formulation. Marinus has implemented a reduced shelf life of 12 months. In agreement with the United States Food and Drug Administration, clinical supplies of ganaxolone will be stored under refrigerated conditions for the duration of clinical use. Marinus plans to modify the ganaxolone IV formulation with a new buffer by the end of Q3 2022, aiming for a shelf life of at least 24 months.
The Company continues to increase the number of participating clinical trial sites for the RAISE trial in the United States and Canada and is working closely with key investigators and site coordinators to support recruitment efficiency with more of 50 sites now activated.
The resupply will also allow the Phase 2 RESET (Research Established Status Epilepticus Treatment) trial to continue to progress. The trial is on track to enroll the first patient in the second half of 2022. The RAISE II Phase 3 trial to support a potential marketing authorization application for RSE remains on track to begin in the first half of 2023 and is expected to use the new ganaxolone IV formulation. As previously noted, the clinical supply disruption did not affect the formulation of ganaxolone oral suspension.
The development of ganaxolone in the RAISE trial 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 status epilepticus
Status epilepticus (SE) is a condition characterized by prolonged or repetitive seizures which, if treatment is delayed or ineffective, can lead to medical or neurological complications. SE is a neurological emergency affecting between 75,000 and 150,000 patients in the United States each year. Benzodiazepines are the first line treatment for SE. If these fail to end the ES, one or more IV DEAs are given as a second line of treatment. If treatment with the first IV AED following benzodiazepines is ineffective, the patient is considered to have refractory ES (RSE).
About Marinus Pharmaceuticals
Marinus is a pharmaceutical company dedicated to the development of innovative therapies to treat seizure disorders. Ganaxolone is a neuroactive GABA steroidA receptor modulator that acts on a well-characterized target in the brain known to have anti-epileptic effects. It is being developed in IV and oral dose formulations intended to maximize therapeutic reach for adult and pediatric patient populations in acute and chronic care settings. For more information, visit www.marinuspharma.com.
To the extent that the statements in this press release are not descriptions of historical facts regarding Marinus, they are forward-looking statements reflecting management’s current beliefs and expectations made pursuant to the provisions of the Private Safe Harbor. Securities Litigation Reform Act of 1995. Words such as “may”, “will”, “expect”, “anticipate”, “estimate”, “intend”, “believe” and similar expressions (as that other words or phrases that refer to 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 expected clinical development plans and the expected timing thereof; the safety and efficacy of ganaxolone; enrollment in our clinical trials; regulatory communications and submissions; our expectations regarding the timing of early data from our clinical trials; our expectations regarding the development of new formulations and their expected shelf life; and other statements regarding the Company’s future operations, financial performance, financial condition, prospects, objectives and other future events.
The forward-looking statements contained in this press release involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements. . These risks and uncertainties include, among others, uncertainties and delays related to the design, recruitment, completion and results of clinical trials; unforeseen costs and expenses; early clinical trials may not be indicative of the results of subsequent clinical trials; clinical trial results may not support regulatory approval or further development in a specific indication or at all; the actions or advice of the FDA or EMA 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 develop new formulations of ganaxolone or prodrugs; our ability to obtain, retain, protect and defend the intellectual property of our product candidates; delays, interruptions or failures in the manufacture and supply of our product candidates; the Company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as expected; the company’s ability to obtain additional financing to support its clinical development and commercial programs; the effect of the COVID-19 pandemic on our business, the medical community, regulators and the global economy; and the availability or potential availability of alternative products or treatments for the conditions we target that could affect the availability or commercial potential of our product candidate. This list is not exhaustive and these and other risks are described in our periodic reports, including our annual reports on Form 10-K, our quarterly reports on Form 10-Q and our current reports on Form 8-K, deposited or provided in securities. and Exchange Commission and available at www.sec.gov. Any forward-looking statements we make in this press release speak only as of the date of this press release. We undertake no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release.