Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2022.
“2022 was a year of significant growth and execution for Marinus, underscored by the successful U.S. launch of ZTALMY,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “With a strong balance sheet and continued progress advancing our second generation formulation and Phase 3 trials in status epilepticus and tuberous sclerosis complex, we entered 2023 with increased confidence in our ability to expand the value proposition of ganaxolone in rare epilepsies.”
- Continued to execute U.S. commercial launch of ZTALMY® (ganaxolone) oral suspension CV, resulting in net product revenue of $2.3 million for the fourth quarter of 2022 and $2.9 million for the fiscal year ended December 31, 2022
- Consistent growth in commercial patients with over 90 total completed CDKL5 deficiency disorder (CDD) prescription enrollment forms received from over 60 unique accounts for the fiscal year ended December 31, 2022
- Full year 2023 expected ZTALMY net product revenues of $15 million to $17 million
- As of February 28, 2023, total coverage for ZTALMY increased to approximately 220 million lives, including both commercial and government programs
- ZTALMY received favorable coverage determinations representing approximately 125 million commercial lives, or 79% of commercial plans
- Medicaid access in all U.S. states, Washington D.C. and Puerto Rico, representing approximately 95 million lives
- Protocol amendment for the Phase 3 RAISE trial of intravenous (IV) ganaxolone in refractory status epilepticus (RSE) has been broadly adopted and topline results continue to be expected in the second half of 2023
- Phase 3 RAISE II trial in RSE (for European registration) enrollment anticipated to begin in the second half of 2023
- Phase 2 RESET trial in established status epilepticus (ESE) site activations underway with first cohort data anticipated before year end 2023
- Successfully manufactured modified IV formulation of ganaxolone with new buffer, targeting a shelf life of at least 24 months, and expect to incorporate in RAISE trial in Q2 2023
- Registration batches of modified formulation to be placed on stability by Q2 2023
- Received Notice of Allowance from the U.S. Patent and Trademark Office for a second patent with claims related to the Company’s clinical therapeutic regimen for the treatment of status epilepticus using IV ganaxolone
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.
Second Generation Product Development
- A Phase 1 single ascending dose study of a second generation oral formulation was conducted
- Preliminary Phase 1 data showed linear dose response up to 1200 mg and the potential for BID dosing with less peak / trough variability
- Second generation ganaxolone was generally well tolerated with no new safety signals
- Multiple ascending dose (MAD) study expected to initiate in the second quarter of 2023
- Planning to finalize clinical program design for Lennox-Gastaut syndrome in second half of 2023
- Prodrug development continues to advance with lead oral candidate selected; Phase 1 data targeted for 2024
Tuberous Sclerosis Complex (TSC)
- Actively screening and enrolling patients in Phase 3 TrustTSC trial of oral ganaxolone at sites in the U.S. and Europe
- Targeting approximately 90 clinical sites, including additional activations in Canada, Israel, Italy, Belgium, Australia, and China
- Topline data continues to be anticipated in the first quarter of 2024
CDKL5 Deficiency Disorder (CDD) Marketing Authorization Application (MAA)
- Marinus received the Day 180 report from the European Medicines Agency (EMA) in January 2023 containing outstanding major objections, including the choice of regulatory starting material
- The Committee for Medicinal Products for Human Use (CHMP) is expected to present its opinion on the MAA in the second quarter of 2023
- Results of Phase 2 placebo-controlled clinical trial of oral ganaxolone in PCDH19-clustering epilepsy recently published in Epilepsy Research
General Business and Financial Update
- Steven Pfanstiel promoted to Chief Operating Officer in addition to current role as Chief Financial Officer.
- Expect that cash and cash equivalents of $240.6 million as of December 31, 2022 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 second half of 2024.
- In Q4 2022, Marinus completed a follow-on equity offering, a revenue interest financing agreement, and a development and commercialization agreement with Tenacia Biotechnology for the Chinese market. Combined, these three deals brought in net funding of over $100 million within the quarter.
Financial Results (Preliminary)
- Recognized $2.3 million and $2.9 million in net product revenues for the three and twelve months ended December 31, 2022, respectively. Net product revenue consists of ZTALMY product sales in the U.S. market, and the 4th quarter of 2022 represents the first full quarter of sales based on the Company’s July 2022 launch of ZTALMY.
- Recognized $1.8 million and $6.9 million in Biomedical Advanced Research and Development Authority (BARDA) federal contract revenue for the three and twelve months ended December 31, 2022, respectively, as compared to $1.5 million and $6.4 million for the three and twelve months ended December 31, 2021, respectively.
- Recognized collaboration revenue of $3.0 million in the fourth quarter of 2022 related to the upfront payment associated with the Company’s development and commercialization agreement with Tenacia.
- Research and development (R&D) expenses were $21.4 million and $79.9 million for the three and twelve months ended December 31, 2022, respectively, as compared to $18.0 million and $73.5 million, respectively, for the same periods in the prior year; the increase was due primarily to increased R&D headcount and clinical trial activity including the ongoing RSE, TSC, and ESE trials.
- Selling, general and administrative (SG&A) expenses were $14.7 million and $56.8 million for the three and twelve months ended December 31, 2022, respectively, as compared to $10.6 million and $37.3 million, respectively, for the same periods in the prior year; the primary drivers of the change were increased headcount and commercial support for the U.S. launch of ZTALMY.
- The Company had net losses of $34.3 million and $19.8 million for the three and twelve months ended December 31, 2022, respectively; cash used in operating activities increased to $112.9 million for the twelve months ended December 31, 2022, compared to $55.5 million for the same period a year ago.
- At December 31, 2022, the Company had cash and cash equivalents of $240.6 million, compared to $122.9 million at December 31, 2021.
- Readers are referred to, and encouraged to read in its entirety, the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2022, to be filed with the Securities and Exchange Commission, which includes further details on the Company’s business plans, operations, financial condition, and results of operations.
- For the fiscal year 2023, the Company expects ZTALMY U.S. net product revenues of between $15 million and $17 million, BARDA revenues in the range of $8 million to $11 million and total GAAP operating expenses, inclusive of SG&A and R&D, to be in the range of $165 to $175 million, of which the company expects stock-based compensation to be approximately $16 million.
About Marinus Pharmaceuticals
Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The Company’s commercial product, ZTALMY® (ganaxolone) oral suspension CV, has been approved by the U.S. FDA for the treatment of seizures associated with CDKL5 deficiency disorder in patients two years of age and older. The potential of ganaxolone is also being studied in other seizure disorders, including in Phase 3 trials in tuberous sclerosis complex and refractory status epilepticus. 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 formulations to maximize therapeutic reach for adult and pediatric patients in acute and chronic care settings. For more information visit www.marinuspharma.com.