Marinus Pharmaceuticals Showcases ZTALMY® Progress Ahead of Launch

Marinus Pharmaceuticals Discusses ZTALMY® Developments

Marinus Pharmaceuticals, Inc. (NASDAQ: MRNS), a pioneering pharmaceutical company focused on innovative treatments for seizure disorders, is set to present significant updates regarding its development program for ZTALMY® (ganaxolone) oral suspension for Tuberous Sclerosis Complex (TSC) at an upcoming Investor and Analyst Day. This event will provide an in-depth analysis ahead of the eagerly awaited Phase 3 TrustTSC trial topline data readout, expected in the near future.

Key Insights from the Investor Event

During the event, which is scheduled to start at 9 a.m. ET, attendees can expect to learn about the promising outcomes from Marinus's ongoing clinical studies and the company's preparations for a potential U.S. launch of ZTALMY, contingent on FDA approval. This drug aims to address the urgent treatment needs of TSC patients, particularly those experiencing refractory epilepsy.

The Chief Executive Officer, Scott Braunstein, M.D., expressed optimism about the results from the Phase 2 trial, noting that patients demonstrated significant reductions in seizure frequency over extended follow-ups. These findings bolster the company's confidence that ZTALMY may provide relief for patients who have not benefited from existing treatments.

Understanding Tuberous Sclerosis Complex (TSC)

Patients diagnosed with TSC encounter numerous challenges due to the disorder's severe neurological implications, including persistent seizures and developmental delays. Despite significant strides in treatment options, there remains an evident lack of effective therapies tailored for TSC patients. Dr. Mary Kay Koenig, a key investigator in the TrustTSC trial, emphasized the critical need for effective seizure control in this vulnerable population.

Commercial Readiness for ZTALMY

Marinus Pharmaceuticals is simultaneously gearing up for a seamless U.S. launch of ZTALMY, backed by a comprehensive commercial framework and insights gained from previous successful product launches. The company anticipates new intellectual property protection for ganaxolone, particularly concerning the titration regimen utilized in the TrustTSC trial. With a market opportunity estimated at approximately $2.5 billion encompassing both CDKL5 deficiency disorder and TSC, Marinus believes that ZTALMY’s unique mechanism of action will facilitate broad reimbursement access and significantly address unmet clinical needs.

Overview of Clinical and Commercial Progress

Projected Revenue and Market Insights

Marinus expects to meet its revenue guidance for ZTALMY, anticipating net product revenue between $33 million and $35 million for the year 2024. Research analyses indicate a persistent unmet need among TSC patients, paving the way for potential extensive payer coverage. Current data reveal that around 26% of TSC patients have attempted and not succeeded with three or more antiseizure medications, supporting Marinus's ambitious commercial targets.

Clinical Trials and Future Directions

The Phase 3 TrustTSC trial, assessing oral ganaxolone in TSC, is scheduled to finish enrollment. The trial holds significant promise, with a well-designed structure aiming to prove its efficacy through robust patient engagement. Following enrollment completion, the low discontinuation rate during the trial encourages confidence in the upcoming data releases. Furthermore, planning is underway to submit a Supplemental New Drug Application to the FDA by early 2025, with hopes for priority review.

Preliminary findings from the long-term Phase 2 TSC trial indicate substantial improvements in seizure frequency among participants. Following two years of treatment, patients experienced noteworthy reductions, and findings from these trials are slated for discussion at the American Epilepsy Society Annual Meeting.

Marinus’s Commitment to Expanded Research

The company is directing efforts to broaden ZTALMY’s use in other rare genetic epilepsies, such as Lennox-Gastaut syndrome, with clinical trials anticipated to initiate following the TSC topline outcomes. Moreover, Marinus aims to submit an Investigational New Drug application for a novel oral ganaxolone prodrug, indicating the company's proactive approach in pursuing diverse therapeutic avenues.

Financial Stability and Growth Outlook

For 2024, Marinus maintains consistent guidance, predicting revenues that reflect the anticipated demand for ZTALMY. The company’s solid financial foundation, bolstered by cash reserves, is expected to adequately support operations through early 2025. Additionally, investments to enhance ZTALMY’s manufacturing capabilities and prepare for a successful launch are progressing.

About Marinus Pharmaceuticals

Marinus is committed to transforming the landscape of seizure disorder treatments through innovative therapies. ZTALMY® (ganaxolone), introduced in the U.S. in 2022, reflects the company’s mission to meet the needs of patients struggling with seizure disorders.

Frequently Asked Questions

What is ZTALMY® and its intended use?

ZTALMY® (ganaxolone) is an FDA-approved medication designed to treat seizures associated with Tuberous Sclerosis Complex.

When will the results from the TrustTSC trial be announced?

The topline results are anticipated in the first half of the fourth quarter of the year.

What are the anticipated revenue figures for Marinus Pharmaceuticals in 2024?

The company expects net product revenue between $33 million and $35 million for ZTALMY in 2024.

Is Marinus involved in any other clinical trials?

Yes, Marinus is planning to initiate trials in other rare genetic epilepsy conditions alongside ongoing studies for TSC.

How does Marinus Pharmaceuticals ensure patient access to ZTALMY?

The company is working to secure broad payer coverage and understands the high need for effective treatments among patients with refractory epilepsy.

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