Core Points - A shareholder class action lawsuit has been filed against Marinus Pharmaceuticals, alleging that the company made materially false and misleading statements regarding its business and operations [3] - The lawsuit specifically claims that Marinus understated the risk of failing to meet early-stopping criteria in the RAISE trial and did not disclose the potential consequences of such failure, including the possibility of stopping the Phase 3 RAISE II trial [3] - The deadline for shareholders to request to be appointed as lead plaintiff in the case is August 5, 2024 [4] Company Information - Marinus Pharmaceuticals, Inc. is publicly traded on NASDAQ under the ticker MRNS [3] - The company is currently facing legal challenges related to its disclosures and operational risks, which may impact investor confidence and stock performance [3] Legal Representation - Holzer & Holzer, LLC is representing shareholders in this litigation, emphasizing their commitment to recovering losses for investors affected by corporate misconduct [2] - The firm has a history of recovering hundreds of millions of dollars for shareholders since its founding in 2000 [2]

For more information, submit a form, email attorney Aaron Dumas, Jr., or give us a call at (800) 350-6003. The Allegations: Robbins LLP is Investigating Allegations that Marinus Pharmaceuticals, Inc. (MRNS) Misled Investors Regarding the Viability of its Drug Candidate SAN DIEGO, June 05, 2024 (GLOBE NEWSWIRE) -- Robbins LLP informs investors that a shareholder filed a class action on behalf of persons and entities that purchased or otherwise acquired Marinus Pharmaceuticals, Inc. (NASDAQ: MRNS) securities ...

NEW YORK, June 05, 2024 (GLOBE NEWSWIRE) -- WHAT TO DO NEXT: To join the Marinus class action, go to https://rosenlegal.com/submit-form/? case_id=25735 or call Phillip Kim, Esq. toll-free at 866-767-3653 or email case@rosenlegal.com for information on the class action. A class action lawsuit has already been filed. If you wish to serve as lead plaintiff, you must move the Court no later than August 5, 2024. A lead plaintiff is a representative party acting on behalf of other class members in directing the l ...

Here's Why MRNS Could Experience a Turnaround The RSI reading of 9.98 for MRNS is an indication that the heavy selling could be in the process of exhausting itself, so the stock could bounce back in a quest for reaching the old equilibrium of supply and demand. Technically, every stock oscillates between being overbought and oversold irrespective of the quality of their fundamentals. And the beauty of RSI is that it helps you quickly and easily check if a stock's price is reaching a point of reversal. So, b ...

Marinus Pharmaceuticals (MRNS) has been on a downward spiral lately with significant selling pressure. After declining 7.6% over the past four weeks, the stock looks well positioned for a trend reversal as it is now in oversold territory and there is strong agreement among Wall Street analysts that the company will report better earnings than they predicted earlier. How to Determine if a Stock is Oversold Technically, every stock oscillates between being overbought and oversold irrespective of the quality o ...

Marinus Pharmaceuticals (MRNS) came out with a quarterly loss of $0.68 per share in line with the Zacks Consensus Estimate. This compares to loss of $0.67 per share a year ago. These figures are adjusted for non-recurring items.A quarter ago, it was expected that this epilepsy drug developer would post a loss of $0.64 per share when it actually produced a loss of $0.74, delivering a surprise of -15.63%.Over the last four quarters, the company has surpassed consensus EPS estimates two times.Marinus Pharmaceu ...

PART I – FINANCIAL INFORMATION [Item 1. Consolidated Financial Statements](index=3&type=section&id=Item%201.%20Consolidated%20Financial%20Statements) The company's Q1 2024 financials show decreased assets and revenue, a wider net loss, and a shift to a stockholders' deficit [Consolidated Balance Sheets](index=3&type=section&id=Consolidated%20Balance%20Sheets) Total assets decreased to $137.4 million while total liabilities remained stable, resulting in a stockholders' deficit Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $104,253 | $120,572 | | Total current assets | $131,276 | $165,246 | | Total assets | $137,350 | $170,908 | | **Liabilities & Equity** | | | | Total current liabilities | $42,758 | $40,624 | | Total liabilities | $153,784 | $154,143 | | Total stockholders' (deficit) equity | $(16,434) | $16,765 | | Total liabilities and stockholders' (deficit) equity | $137,350 | $170,908 | [Consolidated Statements of Operations and Comprehensive Loss](index=4&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Quarterly revenue declined to $7.7 million due to lower federal contract revenue, widening the net loss to $38.7 million Consolidated Statement of Operations Highlights (in thousands, except per share data) | Account | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Product revenue, net | $7,509 | $3,332 | | Federal contract revenue | $152 | $7,048 | | Total revenue | $7,679 | $10,380 | | Research and development | $24,118 | $27,933 | | Selling, general and administrative | $18,626 | $15,204 | | Total expenses | $43,500 | $43,343 | | Loss from operations | $(35,821) | $(32,963) | | Net loss | $(38,669) | $(34,730) | | Net loss per share—basic and diluted | $(0.68) | $(0.67) | [Consolidated Statements of Cash Flows](index=5&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operations was $37.5 million, contributing to a $16.3 million net decrease in cash for the quarter Consolidated Statement of Cash Flows Highlights (in thousands) | Account | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Net cash used in operating activities | $(37,461) | $(41,478) | | Net cash provided by (used in) investing activities | $20,885 | $(51,995) | | Net cash provided by (used in) financing activities | $257 | $(174) | | Net decrease in cash and cash equivalents | $(16,319) | $(93,647) | | Cash and cash equivalents—end of period | $104,253 | $146,904 | [Notes to Consolidated Financial Statements](index=7&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) Notes disclose substantial doubt about the company's ability to continue as a going concern due to insufficient cash to fund operations - The company is a commercial-stage pharmaceutical company focused on seizure disorders, with ZTALMY® (ganaxolone) approved in the U.S. and Europe for seizures associated with CDKL5 Deficiency Disorder (CDD)[21](index=21&type=chunk) - There is **substantial doubt** about the company's ability to continue as a going concern, as cash and investments of **$113.3 million** as of March 31, 2024, are not sufficient to fund operations for the one-year period after the financial statements are issued[28](index=28&type=chunk) - The company has a **$75.0 million** senior secured term loan with Oaktree, maturing in May 2026, with an interest rate of **11.50%**[81](index=81&type=chunk)[83](index=83&type=chunk) - A revenue interest financing agreement with Sagard provides for quarterly payments based on a percentage of U.S. net sales of ZTALMY, subject to a hard cap of **190%** of the **$32.5 million** investment amount[94](index=94&type=chunk)[95](index=95&type=chunk)[96](index=96&type=chunk) - Subsequent to the quarter end, the **RAISE trial's interim analysis did not meet pre-defined stopping criteria**, and the company implemented a **20% reduction** in its workforce[137](index=137&type=chunk)[138](index=138&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=41&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses ZTALMY® commercialization, pipeline developments, and expresses substantial doubt about the company's ability to continue as a going concern [Overview and Pipeline](index=44&type=section&id=Overview%20and%20Pipeline) The company is advancing its ganaxolone pipeline for rare epilepsies, though the RSE trial faces uncertainty after its interim analysis - ZTALMY® (ganaxolone) is approved in the U.S. and Europe for seizures associated with CDD, with European commercialization partner Orion preparing for launches in select countries in 2024[147](index=147&type=chunk) - The global Phase 3 TrustTSC trial for Tuberous Sclerosis Complex is actively enrolling, with **top-line data anticipated in Q4 2024**[181](index=181&type=chunk) - The Phase 3 RAISE trial for RSE **did not meet pre-defined interim stopping criteria**, and the company will complete enrollment at 100 patients, with top-line results expected in summer 2024[186](index=186&type=chunk) - The company is involved in a patent dispute with Ovid Therapeutics regarding the use of ganaxolone for Status Epilepticus, with a final decision expected by August 2024[194](index=194&type=chunk) [Results of Operations](index=72&type=section&id=Results%20of%20Operations) Q1 2024 revenue decreased due to lower federal contract revenue, while operating expenses remained flat, resulting in a net loss of $38.7 million Revenue Comparison (in thousands) | Revenue Source | Q1 2024 | Q1 2023 | | :--- | :--- | :--- | | Product revenue, net | $7,509 | $3,332 | | Federal contract revenue | $152 | $7,048 | | Collaboration revenue | $18 | $0 | | **Total revenue** | **$7,679** | **$10,380** | Expense Comparison (in thousands) | Expense Category | Q1 2024 | Q1 2023 | | :--- | :--- | :--- | | Research and development | $24,118 | $27,933 | | Selling, general and administrative | $18,626 | $15,204 | | **Total Operating Expenses** | **$43,500** | **$43,343** | - The decrease in R&D expenses was primarily due to reduced clinical trial activity for CDD, lower costs for the TSC trial, and reduced drug development costs[243](index=243&type=chunk) - The increase in SG&A expenses was driven by a **$1.5 million rise in commercial costs**, **$1.0 million in stock-based compensation**, and **$0.6 million in personnel costs**[246](index=246&type=chunk) [Liquidity and Capital Resources](index=75&type=section&id=Liquidity%20and%20Capital%20Resources) With $113.3 million in cash, the company's runway extends only into Q1 2025, raising substantial doubt about its going concern status - The company's cash, cash equivalents, and short-term investments totaled **$113.3 million** as of March 31, 2024[253](index=253&type=chunk) - Existing cash is projected to fund operations into **Q1 2025**, which raises **substantial doubt** about the company's ability to continue as a going concern[253](index=253&type=chunk)[272](index=272&type=chunk) - The company has drawn **$75.0 million** under its credit agreement with Oaktree, with quarterly principal payments scheduled to begin on June 30, 2024[254](index=254&type=chunk)[256](index=256&type=chunk) - The BARDA contract provides up to an estimated **$51 million** for the development of IV-ganaxolone for RSE on a cost-sharing basis[263](index=263&type=chunk)[264](index=264&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=85&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, Marinus is not required to provide market risk disclosures - The company is a smaller reporting company and is not required to provide quantitative and qualitative disclosures about market risk[280](index=280&type=chunk) [Item 4. Controls and Procedures](index=85&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of the quarter's end, with no material changes to internal controls - Based on an evaluation as of March 31, 2024, the CEO and CFO concluded that the company's disclosure controls and procedures were **effective**[281](index=281&type=chunk) - No changes occurred during the quarter ended March 31, 2024, that have materially affected, or are reasonably likely to materially affect, the company's internal control over financial reporting[282](index=282&type=chunk) PART II – OTHER INFORMATION [Item 1. Legal Proceedings](index=87&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings - The company reports no material legal proceedings as of the filing date[285](index=285&type=chunk) [Item 1A. Risk Factors](index=87&type=section&id=Item%201A.%20Risk%20Factors) Key risks include the uncertain outcome of the Phase 3 RAISE trial and potential adverse findings from post-marketing studies for ZTALMY - A significant risk is that the **Phase 3 RAISE trial for RSE may not succeed**, as the pre-defined interim analysis stopping criteria were not met[291](index=291&type=chunk) - The company must complete several post-marketing studies for ZTALMY as required by the FDA and EMA, and adverse findings could negatively impact the product's label[287](index=287&type=chunk)[288](index=288&type=chunk)[290](index=290&type=chunk) - Unfavorable results from any single clinical trial for ganaxolone could adversely impact the development programs for other indications[295](index=295&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=91&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered sales of equity securities occurred during the reporting period - None[296](index=296&type=chunk) [Item 5. Other Information](index=92&type=section&id=Item%205.%20Other%20Information) Several directors and an officer adopted Rule 10b5-1 trading plans during the first quarter of 2024 - Four individuals, including three directors and the Chief Commercial Officer, adopted Rule 10b5-1 trading plans during the quarter[300](index=300&type=chunk)

Exhibit 99.1 Marinus Pharmaceuticals Provides Business Update and Reports First Quarter 2024 Financial Results RADNOR, Pa. – May 8, 2024 – Marinus 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 first quarter ended March 31, 2024. "Our unwavering commitment is to develop innovative treatment options for individuals with seizure disorders," said S ...

Financial Data and Key Metrics Changes - The company reported net product revenue of $19.6 million for the full year 2023, which exceeded the revised guidance range of $18.5 million to $19 million [32][67] - The projected U.S. ZTALMY net product revenues for 2024 are between $32 million and $34 million, representing nearly 70% growth compared to 2023 [33][68] - The company ended 2023 with cash, cash equivalents, and short-term investments of $150.3 million, expected to provide a cash runway into the fourth quarter of 2024 [96] Business Line Data and Key Metrics Changes - The ZTALMY product revenues for Q4 2023 were $6.6 million, compared to $2.3 million in Q4 2022, indicating strong growth [97] - The discontinuation rates for the TrustTSC trial are below 7%, showing confidence in the revised titration schedule [93] - The company has achieved over 85% enrollment in the TrustTSC trial, with a high transition rate to the open-label extension [93] Market Data and Key Metrics Changes - The addressable market for refractory status epilepticus (RSE) is estimated at approximately 35,000 patients per year in the U.S. [29] - The addressable patient population in refractory TSC is projected to be about 10,000 patients in the United States [51] - The company is targeting a significant market opportunity in the MENA region, with plans for distribution strategies to begin in the second half of the year [27] Company Strategy and Development Direction - The company plans to build leadership in the hospital market by investing in future status epilepticus research and making commercial investments to ascertain value-based pricing [29] - There is a focus on expanding the ZTALMY business into TSC, leveraging synergies with the CDD market [57] - The company aims to achieve profitability for the ZTALMY franchise within 6 to 12 months of the 2025 TSC launch [31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming Phase 3 data readouts for RSE and TSC, which are expected to drive significant growth for the ganaxolone franchise [72] - The management highlighted the importance of aligning development and execution with key milestones for the acute care business in 2024 [58] - There is optimism regarding the reimbursement dynamics for TSC, based on the success seen with ZTALMY in the CDD population [74] Other Important Information - The company plans to submit a supplemental NDA in the first half of 2025 with a priority review expected [43] - The interim results for the RAISE trial are anticipated to be reported in the second quarter of 2024 [48][89] - The company is preparing for a potential launch in 2025 for the IV formulation of ganaxolone for RSE [56] Q&A Session Summary Question: What are the reimbursement dynamics for TSC based on ZTALMY? - Management noted that over 80% of commercial plans have straightforward guidelines, with no patients denied therapy since launch, indicating strong payer recognition [74] Question: What remains to be done for the NDA package? - The company plans to gather data from approximately 100 patients for the NDA filing, with a pre-NDA meeting with the FDA expected soon after positive data [78] Question: What are the stopping criteria for the RAISE trial? - The stopping criteria are based on co-primary endpoints, including cessation within 30 minutes and lack of progression to IV anesthesia within 36 hours, with a required p-value of 0.0293 for statistical significance [111]

Part I [Item 1. Business](index=12&type=section&id=Item%201.%20Business.) Marinus Pharmaceuticals is a commercial-stage company developing treatments for seizure disorders, led by its primary product ZTALMY® (ganaxolone) [Overview](index=12&type=section&id=Overview) The company's main product, ZTALMY® (ganaxolone), is approved for CDD-related seizures and is in development for other rare epilepsies - ZTALMY® (ganaxolone) was approved by the U.S. FDA on March 18, 2022, for treating seizures associated with Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in patients two years of age and older[31](index=31&type=chunk) - The European Commission (EC) granted marketing authorization for ZTALMY for CDD on July 28, 2023, with **Orion Corporation handling European commercialization**[31](index=31&type=chunk) - The company is also developing ganaxolone for other rare genetic epilepsies, including **Tuberous Sclerosis Complex (TSC)** and **Refractory Status Epilepticus (RSE)**[32](index=32&type=chunk) - Ganaxolone is being developed in both intravenous (IV) and oral formulations and is a synthetic analog of allopregnanolone that targets GABAA receptors[33](index=33&type=chunk) [Our Strategy](index=12&type=section&id=Our%20Strategy) The company's strategy focuses on maximizing ganaxolone's value by targeting rare seizure disorders and expanding its product pipeline - Pursue orphan, genetic epilepsy indications for ganaxolone, such as CDD, TSC, and LGS, leveraging efficient development and commercialization paths[34](index=34&type=chunk) - Develop ganaxolone for hospital-based orphan indications, including a pivotal **Phase 3 trial (RAISE)** in Refractory Status Epilepticus (RSE)[39](index=39&type=chunk) - Advance a second-generation ganaxolone formulation and a prodrug to improve pharmacokinetic profiles, reduce dosing frequency, and generate new intellectual property[39](index=39&type=chunk) - Expand and diversify the product pipeline by acquiring additional drug candidates that align with the business strategy[39](index=39&type=chunk) [Our Products and Product Candidates](index=14&type=section&id=Our%20Products%20and%20Product%20Candidates) ZTALMY® (ganaxolone) generated $19.6 million in 2023 net revenue and is supported by a comprehensive U.S. commercial strategy ZTALMY Net Product Revenue | Period | Net Product Revenue | | :--- | :--- | | Year ended Dec 31, 2023 | $19.6 million | - The U.S. commercial strategy utilizes Orsini Pharmaceutical Services as the specialty pharmacy and ASD Specialty Healthcare as the specialty distributor[53](index=53&type=chunk)[54](index=54&type=chunk) - The U.S. Drug Enforcement Agency (DEA) has classified ganaxolone as a **Schedule V controlled substance**, subjecting it to specific regulations for manufacturing, distribution, and prescription[57](index=57&type=chunk)[59](index=59&type=chunk) - The company has several post-marketing requirements from the FDA and post-authorization measures from the EC, including carcinogenicity and toxicity studies[60](index=60&type=chunk)[63](index=63&type=chunk) [Our Pipeline](index=22&type=section&id=Our%20Pipeline) The pipeline is advancing ganaxolone through late-stage trials for RSE and TSC, with key data readouts expected in 2024 - The Phase 3 RAISE trial in Refractory Status Epilepticus (RSE) reached its interim analysis enrollment target in Q1 2024, with **top-line data expected in Q2 2024**[66](index=66&type=chunk)[71](index=71&type=chunk) - A global Phase 3 trial (TrustTSC) in Tuberous Sclerosis Complex (TSC) is actively enrolling, with **top-line data anticipated in Q4 2024**[84](index=84&type=chunk) - A second-generation formulation of ganaxolone is being developed for Lennox-Gastaut Syndrome (LGS), with a Phase 2 clinical trial expected to start in 2025[86](index=86&type=chunk)[87](index=87&type=chunk) - The company has filed a petition for post-grant review (PGR) with the USPTO to challenge Ovid Therapeutics' U.S. Patent No. 11,395,817, which claims to cover the use of ganaxolone for treating Status Epilepticus (SE)[75](index=75&type=chunk) [Safety Overview](index=32&type=section&id=Safety%20Overview) Ganaxolone has been generally well-tolerated in over 2,200 individuals, with somnolence being the most common side effect - As of October 2023, **over 2,200 individuals** have received ganaxolone, with the most common side effects with oral ganaxolone being sedation or somnolence[97](index=97&type=chunk) - In the pivotal Phase 3 Marigold Trial for CDD, the most common adverse reactions were **somnolence, pyrexia, salivary hypersecretion, and seasonal allergy**[98](index=98&type=chunk) - In a Phase 2 trial for RSE, IV ganaxolone had an acceptable safety profile, with the most common treatment-related adverse events being somnolence, mild hypotension, and sedation[103](index=103&type=chunk)[104](index=104&type=chunk) [Intellectual Property](index=36&type=section&id=Intellectual%20Property) The company's intellectual property portfolio protects ganaxolone formulations, manufacturing processes, and methods of use through 2044 - The company owns patent families covering nanoparticle formulations of ganaxolone, with terms running through 2026 and a **potential five-year extension to 2031** pending[112](index=112&type=chunk) - Patents covering the synthetic process for manufacturing ganaxolone have terms running through **2030**[114](index=114&type=chunk) - Multiple patent families are directed to IV ganaxolone formulations and therapeutic regimens for treating Status Epilepticus (SE), with patent terms extending through **2036 to 2044**[115](index=115&type=chunk)[117](index=117&type=chunk) - The company has licensed patents from Ovid Therapeutics covering certain therapeutic uses of ganaxolone for CDD, with terms running through 2037[119](index=119&type=chunk) [Licenses and Collaborations](index=40&type=section&id=Licenses%20and%20Collaborations) Marinus has established key strategic collaborations with Orion, Tenacia, and others to support the global commercialization of ganaxolone - **Orion:** Exclusive rights for commercialization in Europe, with Marinus receiving a **€25 million upfront fee** and eligibility for up to €97 million in milestones plus royalties[125](index=125&type=chunk) - **Tenacia:** Exclusive rights for development and commercialization in Greater China, with Marinus receiving a **$10 million upfront payment** and eligibility for up to $256 million in milestones plus royalties[126](index=126&type=chunk) - **Purdue:** A settlement was reached in February 2024 regarding the 2022 sale of the Priority Review Voucher (PRV), with Marinus agreeing to pay Purdue **$4 million**[135](index=135&type=chunk) - **Ovid:** An exclusive patent license agreement for certain patents related to the treatment of CDD, with Marinus issuing 123,255 shares of its common stock to Ovid[136](index=136&type=chunk)[138](index=138&type=chunk) [Competition](index=44&type=section&id=Competition) The company faces significant competition from established therapies and large pharmaceutical companies across its targeted indications - **Status Epilepticus (SE):** No treatments are specifically indicated for RSE, with standard care involving benzodiazepines and second-line IV AEDs[143](index=143&type=chunk) - **CDD and TSC:** ZTALMY is the only approved drug for CDD, while competitors for TSC include Novartis's Afinitor DISPERZ® and Jazz Pharmaceuticals' EPIDIOLEX®[145](index=145&type=chunk) - **LGS:** Approved branded drugs for LGS include SYMPAZAN®, EPIDIOLEX®, and FINTEPLA®[146](index=146&type=chunk) [Manufacturing](index=46&type=section&id=Manufacturing) The company relies entirely on third-party contract manufacturing organizations for the supply of ganaxolone API and finished drug products - The company relies on contract manufacturing organizations (CMOs) for all clinical and commercial supplies of ganaxolone API and finished products[147](index=147&type=chunk) - A master supply agreement has been executed with a second API supplier in the U.S. to support **onshoring of manufacturing**[147](index=147&type=chunk) - The company's patented nanoparticulate formulation is designed to address the challenges of ganaxolone being a high-dose, water-insoluble compound[148](index=148&type=chunk) [Government Regulation](index=48&type=section&id=Government%20Regulation) The company is subject to extensive regulation by the FDA and other global authorities, covering all aspects of its product lifecycle - The company's operations are subject to extensive regulation by the FDA and other agencies under the Federal Food, Drug, and Cosmetic Act (FDC Act)[153](index=153&type=chunk) - ZTALMY is scheduled as a controlled substance under the Controlled Substances Act, subjecting it to stringent regulation by the DEA[173](index=173&type=chunk)[175](index=175&type=chunk) - The company has received **orphan drug designation** for ganaxolone in multiple indications, providing benefits like market exclusivity post-approval[199](index=199&type=chunk)[91](index=91&type=chunk) - Healthcare reform measures, including the **Inflation Reduction Act (IRA)**, impact pricing and reimbursement, creating downward pressure on drug prices[226](index=226&type=chunk)[232](index=232&type=chunk)[441](index=441&type=chunk) [Human Capital](index=86&type=section&id=Human%20Capital) As of year-end 2023, the company employed 165 full-time staff and anticipates further headcount growth to support its operations - As of December 31, 2023, the company had **165 full-time employees** and one part-time employee[273](index=273&type=chunk) [Item 1A. Risk Factors](index=86&type=section&id=Item%201A.%20Risk%20Factors.) The company faces substantial risks including going concern doubts, commercial dependency on ZTALMY, and clinical development uncertainties [Risks Related to our Financial Position and Need for Additional Capital](index=87&type=section&id=Risks%20Related%20to%20our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) The company faces a going concern warning due to significant losses, with cash reserves sufficient only into Q4 2024 - The company has identified conditions and events that raise **substantial doubt about its ability to continue as a going concern**[276](index=276&type=chunk)[278](index=278&type=chunk) - As of December 31, 2023, existing cash, cash equivalents, and short-term investments are expected to fund operations only **into the fourth quarter of 2024**[285](index=285&type=chunk) - The company has a history of limited revenue and significant net losses, with an **accumulated deficit of $571.9 million** as of December 31, 2023[276](index=276&type=chunk)[279](index=279&type=chunk) - Debt agreements with Oaktree and Sagard contain restrictive covenants, and failure to comply could result in default and acceleration of repayment obligations[289](index=289&type=chunk)[303](index=303&type=chunk)[307](index=307&type=chunk) [Risks Related to the Commercialization of ZTALMY and Other Future Product Candidates](index=102&type=section&id=Risks%20Related%20to%20the%20Commercialization%20of%20ZTALMY%20and%20Other%20Future%20Product%20Candidates) The commercial success of ZTALMY is uncertain and depends on market acceptance, reimbursement, and navigating a competitive landscape - Even with U.S. and EU approval, ZTALMY faces extensive post-marketing regulatory requirements, including costly studies, which could impact its marketing[319](index=319&type=chunk)[321](index=321&type=chunk) - Commercial success depends on achieving significant market acceptance and obtaining **sufficient reimbursement** from government and private payers[330](index=330&type=chunk)[344](index=344&type=chunk) - The company faces substantial competition from well-established AEDs and companies with significantly greater financial and commercial resources[334](index=334&type=chunk)[336](index=336&type=chunk)[338](index=338&type=chunk) - As the company's first commercial product, there is a risk that its sales and marketing capabilities may not be effective enough to generate meaningful revenue[343](index=343&type=chunk) [Risks Related to Clinical Development and Regulatory Approval of Our Product Candidates](index=116&type=section&id=Risks%20Related%20to%20Clinical%20Development%20and%20Regulatory%20Approval%20of%20Our%20Product%20Candidates) Future success depends on successful clinical development and regulatory approval of ganaxolone for new indications, an uncertain process - Future success depends on the successful clinical development and regulatory approval of ganaxolone for additional indications, which requires significant capital and time[361](index=361&type=chunk) - Ganaxolone may cause undesirable side effects that could delay or prevent regulatory approval or limit its commercial profile[372](index=372&type=chunk)[373](index=373&type=chunk) - The company is required to conduct numerous post-marketing studies for ZTALMY, and adverse findings could materially impact the product's label[365](index=365&type=chunk)[366](index=366&type=chunk) - Clinical development is a lengthy and expensive process with an uncertain outcome, and the company may experience delays in its ongoing or future clinical trials[384](index=384&type=chunk)[385](index=385&type=chunk) [Risks Related to Our Dependence on Third Parties](index=133&type=section&id=Risks%20Related%20to%20Our%20Dependence%20on%20Third%20Parties) Marinus is heavily reliant on third parties for manufacturing, clinical trials, and international commercialization, posing significant operational risks - The company relies on third-party CROs to conduct clinical trials, and if these parties fail to perform, development plans could be adversely affected[403](index=403&type=chunk)[404](index=404&type=chunk) - With no manufacturing facilities, the company is **entirely dependent on third-party manufacturers (CMOs)**, creating risks of supply disruption or compliance failures[412](index=412&type=chunk)[413](index=413&type=chunk) - A product supply interruption for IV ganaxolone occurred in February 2022 due to particulates found in clinical supply, highlighting manufacturing risks[406](index=406&type=chunk)[411](index=411&type=chunk) - The company depends on collaboration partners like Orion and Tenacia for commercialization outside the U.S[418](index=418&type=chunk) [Risks Related to Regulatory Compliance](index=147&type=section&id=Risks%20Related%20to%20Regulatory%20Compliance) The company operates in a highly regulated environment where pricing pressures and complex healthcare laws pose significant compliance risks - Enacted and future legislation, including the **Inflation Reduction Act (IRA)**, may adversely affect drug pricing and reimbursement[441](index=441&type=chunk)[448](index=448&type=chunk) - Failure to comply with governmental pricing programs like the **Medicaid Drug Rebate Program** could result in significant penalties[454](index=454&type=chunk)[455](index=455&type=chunk) - The company is subject to anti-kickback, fraud and abuse, and other healthcare laws, which could expose it to criminal sanctions and civil penalties[477](index=477&type=chunk) - Compliance with complex and evolving data privacy regulations like **HIPAA and GDPR** is costly and poses a risk of significant penalties for non-compliance[482](index=482&type=chunk)[489](index=489&type=chunk) [Risks Related to Intellectual Property](index=165&type=section&id=Risks%20Related%20to%20Intellectual%20Property) The company's competitive position depends on protecting its intellectual property, which is challenged by an off-patent compound and patent disputes - The ganaxolone compound is off-patent; therefore, the company's competitive position relies on patents for formulations, manufacturing processes, and methods of use[502](index=502&type=chunk) - **Ovid Therapeutics has been issued patents** that encompass Marinus's product candidate for the treatment of Status Epilepticus (SE) and LGS, creating a risk of infringement litigation[509](index=509&type=chunk)[512](index=512&type=chunk)[513](index=513&type=chunk) - The company has filed a petition for post-grant review (PGR) of Ovid's SE patent, but the outcome is uncertain and could be costly[509](index=509&type=chunk) - Patent terms may be inadequate to protect the company's products for a sufficient amount of time, and obtaining patent term extensions is not guaranteed[518](index=518&type=chunk)[519](index=519&type=chunk) [Item 1B. Unresolved Staff Comments](index=187&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments.) The company reports no unresolved staff comments - The company reports no unresolved staff comments[557](index=557&type=chunk) [Item 1C. Cybersecurity](index=187&type=section&id=Item%201C.%20Cybersecurity) The company maintains a cybersecurity program based on the NIST framework, overseen by the Audit Committee, with no material incidents to date - The company maintains a cybersecurity program that leverages industry frameworks, including the **National Institute of Standards and Technology (NIST) Cybersecurity Framework**[561](index=561&type=chunk) - Oversight is provided by the Audit Committee of the Board of Directors, with day-to-day management by the Head of IT[565](index=565&type=chunk)[566](index=566&type=chunk) - To date, the company has **not experienced any material cybersecurity incidents** or threats[569](index=569&type=chunk) [Item 2. Properties](index=189&type=section&id=Item%202.%20Properties.) The company leases approximately 22,500 square feet of office space in Radnor, Pennsylvania, under a lease expiring in 2025 - The company's principal offices are in a leased space of approximately **22,500 square feet** in Radnor, Pennsylvania, with the lease expiring in 2025[570](index=570&type=chunk) [Item 3. Legal Proceedings](index=191&type=section&id=Item%203.%20Legal%20Proceedings.) The company is not currently a party to any material legal proceedings - The company reports that it is not currently a party to any material legal proceedings[573](index=573&type=chunk) [Item 4. Mine Safety Disclosures](index=191&type=section&id=Item%204.%20Mine%20Safety%20Disclosures.) This item is not applicable to the company - This item is not applicable to the company[574](index=574&type=chunk) Part II [Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=100&type=section&id=Item%205.%20Market%20for%20Registrant's%20Common%20Equity,%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities.) The company's common stock is listed on the Nasdaq Global Market under the symbol "MRNS" - The company's common stock trades on the Nasdaq Global Market under the symbol **"MRNS"**[577](index=577&type=chunk) [Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=101&type=section&id=Item%207.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations.) The company reported a $141.4 million net loss in 2023 on $31.0 million in revenue, with cash sufficient only into Q4 2024 [Results of Operations](index=107&type=section&id=Results%20of%20Operations) Total 2023 revenue was $31.0 million, while a net loss of $141.4 million was driven by increased R&D and SG&A expenses Comparison of Operations (Years Ended December 31) | (In thousands) | 2023 | 2022 | | :--- | :--- | :--- | | **Total revenue** | **$30,989** | **$25,478** | | Product revenue, net | $19,561 | $2,872 | | Federal contract revenue | $11,374 | $6,935 | | Collaboration revenue | $54 | $15,671 | | **Total expenses** | **$162,474** | **$138,266** | | Research and development | $99,388 | $79,912 | | Selling, general and administrative | $61,152 | $56,845 | | **Loss from operations** | **($131,485)** | **($112,788)** | | (Loss) gain from sale of PRV, net | ($4,000) | $107,375 | | **Net loss** | **($141,405)** | **($19,816)** | - R&D expenses increased to **$99.4 million in 2023** from $79.9 million in 2022, mainly due to increased activity in Phase 3 trials and API supplier validation costs[629](index=629&type=chunk)[637](index=637&type=chunk) - SG&A expenses rose to **$61.2 million in 2023** from $56.8 million in 2022, driven by higher personnel, legal, and professional costs[632](index=632&type=chunk) [Liquidity and Capital Resources](index=110&type=section&id=Liquidity%20and%20Capital%20Resources) The company's $150.3 million in cash is insufficient to fund operations for one year, raising substantial doubt about its going concern status - As of December 31, 2023, the company held **$150.3 million** in cash, cash equivalents, and short-term investments[646](index=646&type=chunk) - Existing cash is only sufficient to fund operations into Q4 2024, which raises **substantial doubt about the company's ability to continue as a going concern**[646](index=646&type=chunk)[672](index=672&type=chunk) - In September 2023, the company raised net proceeds of approximately **$25.8 million** from the sale of 3.7 million shares through its at-the-market (ATM) facility[645](index=645&type=chunk)[663](index=663&type=chunk) - The company has outstanding debt of **$75.0 million** under a credit agreement with Oaktree, which matures in May 2026[649](index=649&type=chunk)[651](index=651&type=chunk)[826](index=826&type=chunk) [Item 7A. Quantitative and Qualitative Disclosures About Market Risk](index=116&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk.) As a smaller reporting company, Marinus Pharmaceuticals is not required to provide this information - The company is a smaller reporting company and is not required to provide this information[684](index=684&type=chunk) [Item 8. Financial Statements and Supplementary Data](index=116&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data.) The company's financial statements and related notes are included in the report starting on page F-1 - The company's financial statements and related notes are included starting on page F-1 of the report[685](index=685&type=chunk) [Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosure](index=116&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20with%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure.) The company reports no changes in or disagreements with its accountants - The company reports no changes in or disagreements with its accountants on accounting and financial disclosure[686](index=686&type=chunk) [Item 9A. Controls and Procedures](index=117&type=section&id=Item%209A.%20Controls%20and%20Procedures.) Management concluded that the company's disclosure controls and internal control over financial reporting were effective as of year-end 2023 - Management concluded that the company's disclosure controls and procedures were **effective** as of December 31, 2023[687](index=687&type=chunk) - Based on the COSO framework, management concluded that internal control over financial reporting was **effective** as of December 31, 2023[690](index=690&type=chunk) Part III [Item 10. Directors, Executive Officers and Corporate Governance](index=118&type=section&id=Item%2010.%20Directors,%20Executive%20Officers%20and%20Corporate%20Governance.) Information for this item is incorporated by reference from the company's 2024 definitive proxy statement - Information for this item is incorporated by reference from the 2024 definitive proxy statement[696](index=696&type=chunk) [Item 11. Executive Compensation](index=118&type=section&id=Item%2011.%20Executive%20Compensation.) Information for this item is incorporated by reference from the company's 2024 definitive proxy statement - Information for this item is incorporated by reference from the 2024 definitive proxy statement[698](index=698&type=chunk) [Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=118&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters.) Information for this item is incorporated by reference from the company's 2024 definitive proxy statement - Information for this item is incorporated by reference from the 2024 definitive proxy statement[699](index=699&type=chunk) [Item 13. Certain Relationships and Related Transactions, and Director Independence](index=118&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions,%20and%20Director%20Independence.) Information for this item is incorporated by reference from the company's 2024 definitive proxy statement - Information for this item is incorporated by reference from the 2024 definitive proxy statement[700](index=700&type=chunk) [Item 14. Principal Accountants Fees and Services](index=118&type=section&id=Item%2014.%20Principal%20Accountants%20Fees%20and%20Services.) Information for this item is incorporated by reference from the company's 2024 definitive proxy statement - Information for this item is incorporated by reference from the 2024 definitive proxy statement[701](index=701&type=chunk) Part IV [Item 15. Exhibits, Financial Statement Schedules](index=118&type=section&id=Item%2015.%20Exhibits,%20Financial%20Statement%20Schedules.) This section lists the financial statements and exhibits filed with the report - This item lists the financial statements and exhibits filed with the report[703](index=703&type=chunk) [Item 16. Form 10-K Summary](index=122&type=section&id=Item%2016.%20Form%2010-K%20Summary) The company did not provide a summary for its Form 10-K - The company did not provide a summary for its Form 10-K[711](index=711&type=chunk) Financial Statements [Report of Independent Registered Public Accounting Firm](index=237&type=section&id=Report%20of%20Independent%20Registered%20Public%20Accounting%20Firm) The auditor's report expresses substantial doubt about the company's ability to continue as a going concern due to recurring losses - The auditor's report includes a paragraph expressing **substantial doubt about the company's ability to continue as a going concern** due to recurring losses from operations[721](index=721&type=chunk) - Critical Audit Matters identified were: 1) **Clinical Trial Prepaid and Accrued Expenses** and 2) **Revenue Interest Financing Payable**, both due to complex estimation uncertainty[725](index=725&type=chunk)[727](index=727&type=chunk)[731](index=731&type=chunk) [Consolidated Financial Statements](index=240&type=section&id=Consolidated%20Financial%20Statements) The company reported a net loss of $141.4 million for 2023, with an accumulated deficit of $571.9 million Key Financial Statement Data (Year Ended Dec 31, 2023) | Metric | Amount (in thousands) | | :--- | :--- | | **Statement of Operations:** | | | Total Revenue | $30,989 | | Total Expenses | $162,474 | | Net Loss | ($141,405) | | Net Loss Per Share | ($2.63) | | **Balance Sheet (End of Period):** | | | Cash and cash equivalents | $120,572 | | Total Assets | $170,908 | | Total Liabilities | $154,143 | | Accumulated Deficit | ($571,926) | | Total Stockholders' Equity | $16,765 | Consolidated Cash Flows (Year Ended Dec 31, 2023) | (In thousands) | Amount | | :--- | :--- | | Net cash used in operating activities | ($118,001) | | Net cash used in investing activities | ($28,605) | | Net cash provided by financing activities | $26,627 | | **Net decrease in cash and cash equivalents** | **($119,979)** |