Marinus Pharmaceuticals(MRNS) - 2024 Q2 - Quarterly Report

Revenue and Sales Performance - ZTALMY (ganaxolone) net product revenue reached $8.0 million and $15.5 million for the three and six months ended June 30, 2024, respectively, compared to $4.2 million and $7.6 million for the same periods in 2023, indicating a year-over-year increase of 90.5% and 103.9%[116] - ZTALMY net sales were $8.0 million and $15.5 million for the three and six months ended June 30, 2024, respectively, compared to $4.2 million and $7.6 million for the same periods in 2023, representing a year-over-year increase of 90.5% and 103.9%[154] - The addressable patient population for ZTALMY in the U.S. is estimated to be approximately 2,000 pediatric patients[125] - Approximately 50% of the CDD patient population is expected to access primary coverage through Fee-for-Service or Managed Medicaid, with the other 50% through commercial payers[126] Clinical Trials and Approvals - The FDA approved ganaxolone for the treatment of seizures associated with CDKL5 Deficiency Disorder (CDD) in patients aged two years and older on March 18, 2022, with commercial sales beginning in Q3 2022[111] - The European Commission granted marketing authorization for ZTALMY for the adjunctive treatment of epileptic seizures associated with CDD in patients aged two to 17 years on July 28, 2023, with expected commercial launches in select European countries in the second half of 2024[111] - The top-line results from the RAISE trial showed that 80% of patients achieved status epilepticus cessation within 30 minutes of initiating IV ganaxolone compared to 13% for placebo (p < 0.0001)[113] - The company anticipates top-line results from the Phase 3 TSC clinical trial in the first half of Q4 2024[112] - In the Phase 3 Marigold Trial, patients treated with ZTALMY showed a median 30.7% reduction in major motor seizure frequency compared to a 6.9% reduction in the placebo group, achieving statistical significance (p=0.0036)[120] - At two years in the open-label extension phase of the Marigold Trial, patients experienced a median 48.2% reduction in major motor seizure frequency[120] - In an open-label Phase 2 trial for TSC-associated seizures, a median 16.6% reduction in seizure frequency was observed, with 30.4% of patients achieving at least a 50% reduction[137] - The TrustTSC global Phase 3 trial enrolled 129 patients, with a primary endpoint of percent change in 28-day frequency of TSC-associated seizures, and top-line data is expected in Q4 2024[138] Financial Performance and Funding - The company incurred net losses of $35.8 million and $74.5 million for the three and six months ended June 30, 2024, compared to net losses of $31.9 million and $66.7 million for the same periods in 2023, indicating an increase in losses of 12.2% and 11.5%[154] - As of June 30, 2024, the company had cash and cash equivalents of $64.7 million, which is expected to fund operations into the second quarter of 2025[156] - The company has incurred an accumulated deficit of $646.4 million as of June 30, 2024[154] - The company plans to continue incurring substantial losses as it conducts multiple later-stage clinical trials and expands its manufacturing capabilities[155] - The BARDA Contract provides for funding of up to approximately $51 million for the development of IV-administered ganaxolone, with $21 million already recorded as of December 31, 2023[160][162] - The company has entered into collaboration agreements with Orion and Tenacia, receiving an upfront payment of €25 million ($29.6 million) and $10 million, respectively, with potential milestone payments totaling up to €97 million and $256 million[165][169] - The company expects milestone and royalty payments from collaborations for the commercialization of ganaxolone in Europe, Mainland China, Hong Kong, Macau, Taiwan, and the Middle East and North Africa[222] Legal and Regulatory Matters - The company is currently not a party to any material legal proceedings, except for a securities class action lawsuit filed on June 5, 2024, alleging violations related to clinical trials[231] - The company intends to vigorously defend against the securities class action lawsuit and plans to move to dismiss the complaint once a schedule is set[231] - Ovid Therapeutics has filed lawsuits alleging patent infringement, which could divert significant resources and impact commercialization of ganaxolone for RSE[150] - The company is evaluating the implications of Ovid's recent patents and ongoing legal challenges, which may affect the commercialization strategy for ganaxolone[149] Research and Development - Research and development expenses for the three months ended June 30, 2024, were $20.9 million, a decrease from $21.4 million in the same period of 2023, primarily due to decreased clinical trial activity[188] - Total research and development expenses for the six months ended June 30, 2024, were $45.0 million, down from $49.3 million in the same period of 2023, reflecting decreased clinical trial activity[188] - The company is exploring new potential formulations of ganaxolone and expanding its product pipeline through acquisitions that align with its business strategy[108] - A clinical trial for oral ganaxolone targeting LGS and other rare epilepsies is planned to begin in H1 2025, pending results from the TrustTSC trial[139] Operational and Administrative Expenses - Selling, general and administrative expenses increased to $16.7 million for the three months ended June 30, 2024, compared to $15.7 million in the same period of 2023, driven by higher professional fees and stock-based compensation[194] - Interest income decreased to $1.1 million for the three months ended June 30, 2024, from $2.1 million in the same period of 2023, attributed to a decrease in cash and cash equivalents[196] - Interest expense increased to $4.6 million for the three months ended June 30, 2024, compared to $4.2 million in the same period of 2023, reflecting higher debt-related costs[197] - Restructuring costs were approximately $2.0 million for the three and six months ended June 30, 2024, with no comparable costs in 2023, primarily related to severance payments and contract termination costs[195] Corporate Governance and Internal Controls - There were no significant changes to critical accounting policies during the three months ended June 30, 2024, compared to the previous year[225] - Disclosure controls and procedures were evaluated as effective at the reasonable assurance level as of June 30, 2024[227] - There were no changes in internal control over financial reporting that materially affected the company during the quarter ended June 30, 2024[228]