Marinus Pharmaceuticals Provides Update on the Phase 3 RAISE Trial and Reports Preliminary First Quarter 2024 Financial Results
- Marinus Pharmaceuticals plans to complete enrollment in the Phase 3 RAISE trial with topline results expected in summer 2024.
- The company will assess the future development of IV ganaxolone in refractory status epilepticus based on the final RAISE results.
- Enrollment in the TrustTSC trial is expected to be completed in mid-May, with topline data on track for the first half of Q4 2024.
- Cost reduction activities are under review to extend the cash runway, with preliminary Q1 2024 net product revenue of $7.4-$7.6 million and cash equivalents of $113.3 million as of March 31, 2024.
- The Phase 3 RAISE trial did not meet pre-defined stopping criteria at the interim analysis.
- The company's financial results are preliminary and subject to adjustment.
- Cost reduction activities may indicate financial challenges for the company.
- The future development of IV ganaxolone is contingent on the final RAISE trial results.
Insights
Marinus Pharmaceuticals' continuation of the Phase 3 RAISE trial, despite not meeting early stopping criteria, signals a commitment to the research and potential market introduction of IV ganaxolone for refractory status epilepticus. The mention of topline results expected in the summer of 2024, however, suggests a significant time before market readiness, which might concern investors looking for near-term profitability. Furthermore, the preliminary Q1 2024 financials with net product revenue between $7.4 and $7.6 million, coupled with a solid cash reserve of $113.3 million, provide a cushion for ongoing developments but also hint at the necessity for cost reduction strategies to preserve capital.
Investors should monitor the eventual realization of cost savings and their impact on the company's burn rate. Additionally, the review of the TrustTSC trial outcomes and subsequent filings for a supplemental New Drug Application will be vital in assessing the company's mid-term pipeline strength and potential revenue streams. While the current financial position seems stable, the long R&D timelines typical for the pharma industry often require sustained investment, which might weigh on the stock's short-term performance.
In the context of drug development, Marinus Pharmaceuticals' decision to carry on with the Phase 3 RAISE trial of IV ganaxolone, despite not achieving early stopping criteria, reflects a nuanced approach to clinical research. The interim analysis, although an important milestone, does not always presage the final trial results. The company's pivot towards evaluating cost-saving measures indicates a strategic balance between scientific pursuits and financial prudence, given the intensive resource requirements of late-stage clinical trials.
For stakeholders, the key takeaway involves balancing optimism for potential breakthrough treatments against the rigorous, uncertain nature of clinical trials. The phased release of trial results and regulatory submissions, such as the TrustTSC trial and the prospective supplemental New Drug Application, are critical junctions for Marinus that could herald shifts in investor sentiment and stock valuations. Such data points serve as important indicators for the company's trajectory and should be evaluated alongside broader market conditions and the competitive landscape within the pharmaceutical industry.
The pharmaceutical sector's performance, particularly that of companies like Marinus Pharmaceuticals, is often closely tied to clinical trial outcomes and product commercialization successes. Marinus' ongoing trials and its current revenue from ZTALMY suggest a company in transition, from research and development to market realization. The projected enrollment completions and topline data announcements serve as inflection points that can potentially realign investor expectations and market capitalization.
Market analysts would note the partnership with BARDA, which may provide both financial support and validation for the company's research endeavors, potentially influencing investor confidence. As Marinus navigates the path to potential approvals, its stock may experience volatility in anticipation of these critical data reports. The disclosed financial health of the company, specifically its cash reserves, provides some reassurance of operational stability but also underscores the need for strategic financial management to maintain investor trust amidst long R&D horizons.
Trial did not meet pre-defined stopping criteria at the interim analysis; Marinus has completed RAISE enrollment at approximately 100 patients with topline results expected summer 2024
Future development of IV ganaxolone in refractory status epilepticus to be assessed following review of the final RAISE results
Enrollment in the TrustTSC trial expected to complete mid-May with topline data on track for the first half of Q4 2024
Cost reduction activities to extend cash runway are under review and expected to be implemented Q2 2024
ZTALMY® (ganaxolone) Q1 2024 preliminary net product revenue of between
Marinus has decided to complete enrollment in the RAISE trial at approximately 100 patients with topline results expected in the summer of 2024. Those results will be used to determine whether to continue development of IV ganaxolone. Marinus remains blinded to the RAISE trial data.
“While we are disappointed that RAISE did not meet the early stopping criteria, we will only be able to determine the trial’s outcome once we unblind and analyze the full data set,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “We will also be evaluating potential cost-saving strategies to provide the strongest capital position as we approach enrollment completion in the global Phase 3 TrustTSC trial in tuberous sclerosis complex.”
Ganaxolone development in the RAISE trial is being supported in part by the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA) under contract number 75A50120C00159.
General Business and Financial Update
Marinus expects to complete enrollment in the Phase 3 TrustTSC trial of ZTALMY® (ganaxolone) oral suspension CV with approximately 130 patients in mid-May 2024. The Company expects topline results early in the fourth quarter of 2024 and anticipates filing a supplemental New Drug Application to the
Marinus also continues to develop a second-generation ganaxolone formulation intended to provide improved pharmacodynamic and pharmacokinetic profiles that could improve safety, efficacy and tolerability and enable less frequent dosing.
The Company continues the successful
The preliminary first quarter 2024 net product revenue results and cash, cash equivalents, and short-term investments included in this release were calculated prior to the completion of a review by the Company’s independent registered public accounting firm and are therefore subject to adjustment.
About Status Epilepticus
Status epilepticus (SE) is a life-threatening condition resulting from either the failure of the mechanisms responsible for seizure termination or from the initiation of mechanisms which lead to abnormally prolonged seizures.1 SE is the one of the most common neurological emergencies in the
About Intravenous (IV) Ganaxolone
Ganaxolone is a neuroactive steroid that works by modulating both synaptic and extrasynaptic GABAA receptors via a unique binding site to potentiate two types of inhibitory signaling.6 IV ganaxolone has pharmacokinetic and pharmacodynamic properties well-suited for the treatment of status epilepticus, with rapid and sustained SE cessation observed in pre-clinical and clinical studies.7,8,9 IV ganaxolone has received orphan drug designation from the
About the RAISE Trial
The RAISE (Randomized Therapy in Status Epilepticus) trial (NCT04391569) is a Phase 3 double-blind, randomized, placebo-controlled clinical trial to evaluate the safety and efficacy of IV ganaxolone in patients with refractory status epilepticus. The RAISE protocol provides for an independent data monitoring committee to conduct an unblinded interim analysis when two-thirds of participants, or approximately 82 patients, have completed the trial.
About Marinus Pharmaceuticals
Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The Company first introduced FDA-approved prescription medication ZTALMY® (ganaxolone) oral suspension CV in the
1 Trinka E, et al. Epilepsia. 2015;56(10):1515-1523.
2 Betjemann JP, Lowenstein DH. Lancet Neurol. 2015;14(6):615-24.
3 Guterman EL et al. JAMA Neurol. 2021;78(5):588-95.
4 Glauser T et al. Epilepsy Curr. 2016;16:48-61.
5 Brophy GM et al. Neurocrit Care. 2012;17:3-23.
6 Reddy, D. S. Front Cell Neurosci 7, 115 (2013).
7 Zolkowska D et al. Epilepsia. 59(suppl 2):220-227.
8 Gasior M et al. Clin Pharmacol Drug Dev 13: 248-25.
9 Vaitkevicius H et al. Epilepsia 2022; 63(9): 2381-2391.
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 the expectation that the results from the RAISE trial will be used to determine whether to continue development of IV ganaxolone; our plans to evaluate potential cost-saving strategies to provide the strongest capital position and extend cash runway, and the implementation and related timing thereof; our belief that a second-generation ganaxolone formulation may provide improved pharmacodynamic and pharmacokinetic profiles that could improve safety, efficacy and tolerability and enable less frequent dosing; the intent to explore new clinical programs for ZTALMY in other refractory epilepsies, and the timing thereof; the potential benefits ZTALMY will provide for physicians and patients; statements regarding our expected clinical development plans, enrollment in our clinical trials, results from our clinical trials, regulatory communications and submissions for ganaxolone, and the timing thereof; the safety and efficacy of ganaxolone, as well as its therapeutic potential in a number of indications; and other statements regarding our future operations, financial performance, financial position, prospects, objectives and other future events.
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, unexpected results or delays in the commercialization of ZTALMY; unexpected market acceptance, payor coverage or future prescriptions and revenue generated by ZTALMY; unexpected actions by the FDA or other regulatory agencies with respect to our products; competitive conditions and unexpected adverse events or patient outcomes from being treated with ZTALMY, uncertainties and delays relating to the design, enrollment, completion, and results of clinical trials; unanticipated costs and expenses; the varying interpretation of clinical data; our ability to comply with the FDA’s requirement for additional post-marketing studies in the required time frames; the timing of regulatory filings for our other product candidates; the potential that regulatory authorities, including the FDA and EMA, may not grant or may delay approval for our product candidates; 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 develop new formulations of ganaxolone or prodrugs; 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 our product candidates, and our ability to service those markets; our ability to continue as a going concern; our cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; our expectations, projections and estimates regarding expenses, future revenue, capital requirements, and the availability of and the need for additional financing; our ability to obtain additional funding to support its clinical development and commercial programs; the potential for our ex-
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Investors
Jim DeNike
Senior Director, Investor Relations
Marinus Pharmaceuticals, Inc.
jdenike@marinuspharma.com
Media
Molly Cameron
Director, Corporate Communications & Investor Relations
Marinus Pharmaceuticals, Inc.
mcameron@marinuspharma.com
Source: Marinus Pharmaceuticals
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