RAISE trial delayed due to impacts from COVID-19 variant and recent interruption in clinical supply material
RADNOR, Pa.–(BUSINESS WIRE)–$MRNS #MarinusPharma—Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that due to the impact of the COVID-19 Omicron variant on hospital resources and an unexpected interruption of clinical supply material associated with IV ganaxolone, the RAISE, Phase 3, double-blind placebo-controlled trial for the treatment of status epilepticus, is now expected to be completed in the second half of 2023.
Marinus has temporarily paused the RAISE trial after routine monitoring of stability batches of clinical supply material indicated that it became necessary to reduce the shelf life to less than the anticipated 24-months to meet product stability testing specifications. The Company is targeting resupply of clinical trial material by the end of the second quarter 2022. Separately, Marinus is implementing improvements in the manufacturing process with the goal of achieving a 24-month or greater product shelf life for IV ganaxolone.
COVID-19, and most recently the Omicron variant, have continued to impact hospital resources and have put a significant strain on hospital staff, particularly in intensive care units, where the RAISE trial is being conducted. This has led to slower than anticipated enrollment. Despite these issues, interest in the trial remains strong with 48 sites now activated and additional sites expected, including potential expansion into Canada.
As a result of these impacts, Marinus is revising its target for announcing top line data from the RAISE trial to the second half of 2023. Timing for the Phase 2 RESET trial of adjuvant use of ganaxolone in established status epilepticus and the Phase 3 RAISE II trial (for European registration) in status epilepticus, are both expected to incur a six-month initiation delay.
The clinical supply interruption affects the IV formulation of ganaxolone and the IV clinical programs. This supply issue does not affect ganaxolone’s oral suspension formulation or the Company’s New Drug Application (NDA), which was submitted in July to the U.S. Food and Drug Administration, for the treatment of seizures associated with CDKL5 deficiency disorder, a rare, genetic epilepsy. The Prescription Drug User Fee Act action date for the NDA is March 20, 2022.
As previously announced, Marinus will hold a Fourth Quarter Business Update on March 21, 2022, with a conference call at 8am Eastern Time. For additional information about the conference call or about Marinus, please visit www.marinuspharma.com.
About Marinus Pharmaceuticals
Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of innovative therapeutics to treat 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, antidepressant and anti-anxiety effects. Ganaxolone 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.
Forward-Looking Statements
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, enrollment in our clinical trials, regulatory communications and submissions and product launches for ganaxolone, and the timing thereof; our expectations regarding resupply of clinical trial material and the timing thereof; our plans to potentially achieve a 24-month or greater product shelf life for IV ganaxolone; our expectations regarding the activation of additional clinical trial sites; our expectations and beliefs regarding the FDA and EMA with respect to our product candidates; our expectations regarding the Orion Corporation collaboration; our expectation regarding the impact of the COVID-19 pandemic on our business and clinical development plans; our financial projections; and the potential safety and efficacy of ganaxolone, as well as its therapeutic potential in a number of indications.
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, the risk that the FDA will require additional clinical trials or data; any delays in review of the NDA submission by the FDA for any reason, including the COVID-19 pandemic; the timing of regulatory filings for our product candidates; the potential that regulatory authorities, including the FDA and EMA, may not grant or may delay approval for our product candidate; 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 obtain and maintain regulatory approval for our product candidate; our ability to obtain, maintain, protect and defend intellectual property for our product candidates; the potential negative impact of third party patents on our or our collaborators’ ability to commercialize ganaxolone; delays, interruptions or failures in the manufacture and supply of our product candidate; the size and growth potential of the markets for the company’s product candidates, and the company’s ability to service those markets; the company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; the company’s expectations, projections and estimates regarding expenses, future revenue, capital requirements, and the availability of and the need for additional financing; the company’s ability to obtain additional funding to support its clinical development programs; the company’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators; the rate and degree of market acceptance of the company’s product candidates; the potential for Orion to breach the collaboration or terminate the agreement in accordance with its terms; the potential for Orion to recoup a percentage of the upfront fee depending on the additional pre-clinical testing; the effect of the COVID-19 pandemic on our business, the medical community, regulators and the global economy; our ability to obtain adequate supply; 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 the annual report 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.
Contacts
Sasha Damouni Ellis
Vice President, Corporate Affairs & Investor Relations
Marinus Pharmaceuticals, Inc.
484-253-6792
sdamouni@marinuspharma.com