Marinus Pharmaceuticals Provides Business Update, Reports Third Quarter 2023 Financial Results

RADNOR, PA — Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) recently reported business highlights and financial results for the third quarter ended September 30, 2023.

“With strong quarter over quarter growth and robust payer coverage one year into the launch of ZTALMY, we continue to demonstrate our unique commercial capabilities in the orphan epilepsy space and are enthused by the opportunity ZTALMY and the ganaxolone franchise represent as an important long-term value driver for Marinus,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.

Dr. Braunstein continued, “We remain acutely focused on advancing our Phase 3 clinical trials in refractory status epilepticus and tuberous sclerosis complex. While we’re disappointed that we now project RAISE enrollment to conclude by the end of the first quarter, we remain confident in the benefit that IV ganaxolone could bring to critically ill RSE patients and the significant commercial opportunity. We are committed to successfully completing both the RAISE and TrustTSC trials in 2024 and continue to make the investments to prepare for these commercial launches.”

ZTALMY^®

• ZTALMY^® (ganaxolone) oral suspension CV net product revenue of $5.4 million for the third quarter of 2023
  • Continued growth in commercial patients with approximately 140 patients active on therapy at the end of the third quarter
• Increased full year 2023 expected ZTALMY net product revenues to between $18.5 and $19 million from a range of $17 to $18.5 million

CDKL5 Deficiency Disorder

• Initiated the Marinus Access Program expanding global availability of ZTALMY for eligible patients with seizures associated with CDKL5 deficiency disorder (CDD) in geographies where the product is not commercially available and as supported by local regulatory requirements
• The Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has accepted and granted priority review of a New Drug Application (NDA) for ZTALMY in CDD; the NDA was submitted in China by Tenacia Biotechnology under the terms of a collaboration agreement with Marinus
• Orion Corporation continues to prepare for commercial launches of ZTALMY in select European countries in 2024

Clinical Pipeline

Status Epilepticus

• Over 75% of patients required for an interim analysis are now enrolled in the Phase 3 RAISE trial of intravenous (IV) ganaxolone in refractory status epilepticus (RSE)
  • Enrollment for the interim analysis expected to conclude in the first quarter of 2024 with topline data now anticipated in the second quarter of 2024, assuming pre-defined stopping criteria for an interim analysis are met
• 21 patients have now been treated for super refractory status epilepticus (SRSE) under emergency investigational new drug (EIND) applications
• Phase 3 RAISE II trial in RSE (for European registration) enrollment anticipated to begin before year end 2023
• To focus additional resources on the expansion of RSE clinical programs, including further investigation of potential development opportunities in SRSE, Marinus voluntarily discontinued the Phase 2 RESET trial in established status epilepticus

Ganaxolone development in the RAISE trial is being funded in part by the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response at the U.S. Department of Health and Human Services, under contract number 75A50120C00159.

Tuberous Sclerosis Complex

• Continue to enroll patients in the global Phase 3 TrustTSC trial of oral ganaxolone in tuberous sclerosis complex with topline data anticipated mid-2024

Second Generation Product Development

• Enrollment in the multiple ascending dose (MAD) trial is well underway with preliminary data expected by year end 2023
• Planning to finalize clinical program design for Lennox-Gastaut syndrome in the first half of 2024, pending results of the MAD trial

General Business and Financial Update

• For the fiscal year 2023, the Company is updating its revenue and operating expense guidance:
  • The Company now expects ZTALMY net product revenues of between $18.5 and $19 million; this represents an increase from the previous guidance of between $17 and $18.5 million
  • The Company now expects GAAP operating expenses, inclusive of G&A and R&D, to be in the range of $158 to $162 million, of which the Company expects stock-based compensation to be approximately $16 million; this represents a decrease from the prior guidance range of $160 to $165 million
• Expect that cash, cash equivalents, and short-term investments of $176.4 million as of September 30, 2023, will be sufficient to fund the Company’s operating expenses, capital expenditure requirements, and maintain the minimum cash balance of $15 million required under the Company’s debt facility into the fourth quarter of 2024
  • During the quarter, a total of 3.7 million shares were sold through the Company’s at-the-market (ATM) facility contributing net proceeds of $25.9 million

Financial Results

• Recognized $5.4 million and $13.0 million in net product revenues for the three and nine months ended September 30, 2023, respectively, as compared to $0.6 million in each of the same periods in the prior year. Net product revenue consists of ZTALMY product sales, which was launched in the U.S. in the third quarter of 2022.
• Recognized $1.9 million and $10.8 million in Biomedical Advanced Research and Development Authority (BARDA) federal contract revenue for the three and nine months ended September 30, 2023, respectively, as compared to $1.8 million and $5.1 million for the same periods in the prior year, respectively. The increase on a year-to-date basis was primarily driven by activities associated with the startup of the API onshoring initiative.
• Research and development (R&D) expenses were $23.7 million and $73.0 million for the three and nine months ended September 30, 2023, respectively, as compared to $19.0 million and $58.5 million, respectively, for the same periods in the prior year; the increase on a year-to-date basis was due primarily to increased investment associated with the Company’s API onshoring effort, increased TSC and RSE clinical trial activity, and increased headcount.
• Selling, general and administrative (SG&A) expenses were $14.9 million and $45.8 million for the three and nine months ended September 30, 2023, respectively, as compared to $13.4 million and $42.2 million, respectively, for the same periods in the prior year; the increase on a year-to-date basis was due primarily to increased headcount associated with the U.S. launch of ZTALMY.
• The Company had net losses of $33.0 million and $99.6 million for the three and nine months ended September 30, 2023, respectively; cash used in operating activities was $91.0 million for each of the nine months ended September 30, 2023 and 2022
• At September 30, 2023, the Company had cash, cash equivalents, and short-term investments of $176.4 million, compared to $240.6 million at December 31, 2022.

Readers are referred to, and encouraged to read in its entirety, the Company’s Quarterly Report on Form 10-Q for the nine months ended September 30, 2023, to be filed with the Securities and Exchange Commission, which includes further detail on the company’s business plans, operations, financial condition, and results of operations.

Financial Results

Selected Financial Data (in thousands, except share and per share amounts)

For more information visit www.marinuspharma.com.

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