05 May 2022
- 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)-- Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that it has resumed screening and recruitment for the Phase 3 RAISE (Randomized Therapy in Status Epilepticus) trial in refractory status epilepticus (RSE). The company continues to expect topline RAISE trial results in the second half of 2023.
Marinus announced in February that it was pausing recruitment in the RAISE trial after routine stability monitoring showed visible particulates of aluminum phosphate in the drug solution. During the pause, the chair of the independent Data Monitoring Committee for the trial reviewed the clinical safety data and determined no concerning safety events were identified in patients already treated with the batches of IV ganaxolone.
The trial has resumed utilizing new batches of the current IV formulation. Marinus has implemented a reduced shelf life of 12 months. In agreement with the U.S. Food and Drug Administration, ganaxolone clinical supplies will be stored under refrigerated conditions for the entire duration of clinical use. Marinus anticipates modifying the IV ganaxolone formulation with a new buffer by end of the third quarter of 2022, targeting a shelf life of at least 24 months.
The company continues to expand the number of participating clinical trial sites for the RAISE trial in the U.S. and Canada and is working closely with key investigators and site coordinators to support enrollment efficiencies with more than 50 sites now activated.
The resupply will also allow the Phase 2 RESET (Researching Established Status Epilepticus Treatment) trial to continue to advance. The trial is on track to enroll the first patient in the second half of 2022. The Phase 3 RAISE II trial to support a potential marketing authorization application for RSE remains on track to commence in the first half of 2023 and is expected to utilize the anticipated new formulation of IV ganaxolone. As previously disclosed, the clinical supply interruption did not affect ganaxolone’s oral suspension formulation.
Ganaxolone development in the RAISE trial is being 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.
Status epilepticus (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 – 150,000 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).
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-characterized target in the brain known to have anti-seizure effects. It is being developed in IV and oral dose formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. 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 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 top line data from our clinical trials; our expectations regarding the development of new formulations and the expected shelf life thereof; and other statements regarding the Company's future operations, financial performance, financial position, prospects, objectives and other future event.
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, and results of clinical trials; 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 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, maintain, protect and defend intellectual property for 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 anticipated; the company’s ability to obtain additional funding 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 conditions targeted by us 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, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.Print
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