Core Insights - MediciNova, Inc. is nearing completion of patient randomization in its Phase 2/3 COMBAT-ALS trial, with only single-digit enrollment remaining [2][3] - The company is also seeking the final two patients for its Phase 2 trial targeting dyslipidemia and fatty liver disease in type 2 diabetes patients [2][3] - The COMBAT-ALS program has generated significant interest within the ALS community, and the company is preparing for regulatory discussions with the FDA, expecting top-line data by the end of next year [3] MediciNova's Development Programs - MN-166 (ibudilast) is in late-stage clinical development for neurodegenerative diseases, including ALS, and has received Orphan Drug Designation and Fast Track Designation from the FDA [4] - MN-001 (tipelukast) is being developed for fibrotic and metabolic disorders, showing promise in down-regulating genes that promote fibrosis and inflammation [5] - The company focuses on addressing unmet medical needs in difficult-to-treat conditions through its development activities [6] Funding and Strategic Development - MediciNova's strategy includes advancing its pipeline through various funding sources, including investigator-sponsored trials, government grants, and strategic alliances [7]

PART I. FINANCIAL INFORMATION Presents MediciNova's unaudited condensed consolidated financial statements and management's discussion for the quarter ended March 31, 2025 [ITEM 1. CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (unaudited)](index=6&type=section&id=ITEM%201.%20CONDENSED%20CONSOLIDATED%20FINANCIAL%20STATEMENTS%20(unaudited)) Details MediciNova's unaudited condensed consolidated financial statements, including balance sheets, statements of operations, equity, cash flows, and accompanying notes for Q1 2025 [Condensed Consolidated Balance Sheets](index=6&type=section&id=CONDENSED%20CONSOLIDATED%20BALANCE%20SHEETS) Provides a snapshot of MediciNova's financial position, detailing assets, liabilities, and equity as of March 31, 2025, and December 31, 2024 Condensed Consolidated Balance Sheet Highlights | Metric | March 31, 2025 ($) | December 31, 2024 ($) | | :-------------------------------- | :------------- | :---------------- | | Cash and cash equivalents | $36,571,466 | $40,359,738 | | Total current assets | $37,679,307 | $41,074,279 | | Total assets | $52,431,150 | $55,875,926 | | Total current liabilities | $2,133,684 | $2,959,123 | | Total liabilities | $2,500,674 | $3,372,375 | | Total stockholders' equity | $49,930,476 | $52,503,551 | | Accumulated deficit | $(429,615,362) | $(426,751,242) | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20OPERATIONS%20AND%20COMPREHENSIVE%20LOSS) Outlines MediciNova's financial performance, including operating expenses, interest income, and net loss for the three months ended March 31, 2025 and 2024 Condensed Consolidated Statements of Operations and Comprehensive Loss | Metric | Three months ended March 31, 2025 ($) | Three months ended March 31, 2024 ($) | | :------------------------------------ | :-------------------------------- | :-------------------------------- | | Research, development and patents | $1,839,802 | $1,781,869 | | General and administrative | $1,362,708 | $1,354,124 | | Total operating expenses | $3,202,510 | $3,135,993 | | Operating loss | $(3,202,510) | $(3,135,993) | | Interest income | $336,111 | $397,510 | | Net loss | $(2,864,120) | $(2,754,518) | | Basic and diluted net loss per common share | $(0.06) | $(0.06) | | Comprehensive loss | $(2,859,018) | $(2,763,410) | [Condensed Consolidated Statements of Stockholders' Equity](index=8&type=section&id=CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20STOCKHOLDERS'%20EQUITY) Details changes in MediciNova's stockholders' equity, including share-based compensation, net loss, and foreign currency adjustments for Q1 2025 and Q1 2024 Changes in Stockholders' Equity | Metric | Balance at Dec 31, 2024 ($) | Share-based Compensation ($) | Net Loss ($) | Foreign Currency Translation Adjustments ($) | Balance at Mar 31, 2025 ($) | | :-------------------------- | :---------------------- | :----------------------- | :------- | :------------------------------------- | :---------------------- | | Total stockholders' equity | $52,503,551 | $285,943 | $(2,864,120) | $5,102 | $49,930,476 | | Metric | Balance at Dec 31, 2023 ($) | Share-based Compensation ($) | Net Loss ($) | Foreign Currency Translation Adjustments ($) | Balance at Mar 31, 2024 ($) | | :-------------------------- | :---------------------- | :----------------------- | :------- | :------------------------------------- | :---------------------- | | Total stockholders' equity | $62,378,424 | $215,430 | $(2,754,518) | $(8,892) | $59,830,444 | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20CASH%20FLOWS) Presents MediciNova's cash inflows and outflows from operating, investing, and financing activities for the three months ended March 31, 2025 and 2024 Condensed Consolidated Statements of Cash Flows | Metric | Three months ended March 31, 2025 ($) | Three months ended March 31, 2024 ($) | | :------------------------------------ | :-------------------------------- | :-------------------------------- | | Net loss | $(2,864,120) | $(2,754,518) | | Non-cash stock-based compensation | $285,943 | $215,430 | | Net cash used in operating activities | $(3,783,661) | $(3,868,248) | | Net change in cash and cash equivalents | $(3,788,272) | $(3,860,643) | | Cash and cash equivalents, end of period | $36,571,466 | $47,138,799 | [Notes to Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Provides detailed explanations and disclosures for MediciNova's condensed consolidated financial statements, covering accounting policies and financial items [1. Interim Financial Information](index=10&type=section&id=1.%20Interim%20Financial%20Information) Describes MediciNova's business, operating segment, accounting policies for R&D, and evaluation of new FASB ASUs - MediciNova is a biopharmaceutical company focused on developing novel therapeutics for serious diseases with unmet medical needs, primarily in the U.S. market. Its main product candidates are MN-166 (ibudilast) for neurological disorders and MN-001 (tipelukast) for fibrotic and metabolic disorders[25](index=25&type=chunk) - The company operates as a single operating segment focused on the acquisition and development of small molecule therapeutics[29](index=29&type=chunk) - Research and development costs, including patent-related expenses, are expensed in the period incurred[32](index=32&type=chunk)[33](index=33&type=chunk) - The company is evaluating the potential impact of recently issued FASB ASUs 2023-09 (Income Taxes) and 2024-03 (Expense Disaggregation Disclosures), which will enhance income tax and expense disclosures in future periods[37](index=37&type=chunk)[38](index=38&type=chunk) [2. Revenue Recognition](index=14&type=section&id=2.%20Revenue%20Recognition) Explains MediciNova's revenue recognition policies, primarily for research and development services, based on performance obligation satisfaction - Revenues historically consist mainly of research and development services, recognized upon the satisfaction of performance obligations, either at a point in time or over time[39](index=39&type=chunk) [3. Fair Value Measurements](index=14&type=section&id=3.%20Fair%20Value%20Measurements) Details MediciNova's fair value measurement hierarchy and the valuation of mutual funds as of March 31, 2025, and December 31, 2024 - Fair value measurements are categorized into a three-tier hierarchy (Level 1, 2, 3) based on the observability of inputs[40](index=40&type=chunk)[41](index=41&type=chunk) Fair Value of Mutual Funds | | March 31, 2025 ($) | December 31, 2024 ($) | Valuation Inputs | | :---------- | :------------- | :---------------- | :--------------- | | Mutual funds | $21,728,258 | $21,501,081 | Level 1 | [4. Commitments and Contingencies](index=16&type=section&id=4.%20Commitments%20and%20Contingencies) Outlines MediciNova's operating lease liabilities, potential milestone payments, and confirms no material legal proceedings - The company has operating leases for real estate in the United States and Japan, including a new Tokyo office lease effective June 2024[43](index=43&type=chunk) Operating Lease Liabilities | Metric | March 31, 2025 ($) | December 31, 2024 ($) | | :-------------------------------- | :------------- | :---------------- | | Current operating lease liabilities | $189,860 | $193,769 | | Non-current operating lease liabilities | $165,198 | $211,460 | | Total operating lease liabilities | $355,058 | $405,229 | | Weighted-average remaining lease term | 1.76 years | 1.98 years | | Weighted-average discount rate | 9.6% | 9.6% | - Future potential milestone payments under license agreements for MN-166 (ibudilast) and MN-001 (tipelukast) are **$10 million**, and for all other products, they total **$16.5 million** as of March 31, 2025[46](index=46&type=chunk) - The company is not aware of any material legal proceedings or claims that would adversely affect its business[47](index=47&type=chunk) [5. Stock-based Compensation](index=17&type=section&id=5.%20Stock-based%20Compensation) Discusses MediciNova's equity incentive plan, performance options, stock-based compensation expense, and unamortized compensation cost - The 2023 Equity Incentive Plan is the successor to the 2013 Plan, with **2,025,173 shares** remaining available for future grants as of March 31, 2025[49](index=49&type=chunk)[50](index=50&type=chunk) - As of March 31, 2025, **1,030,000 shares** underlying performance options were subject to vesting based on the achievement of corporate objectives and employee performance for 2025[51](index=51&type=chunk) Stock-based Compensation Expense | Expense Category | Three months ended March 31, 2025 ($) | Three months ended March 31, 2024 ($) | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Research, development and patents | $124,191 | $81,669 | | General and administrative | $161,752 | $133,761 | | Total stock-based compensation expense | $285,943 | $215,430 | - As of March 31, 2025, there was **$1.0 million** of unamortized compensation cost related to unvested stock option awards, expected to be recognized over a remaining weighted-average vesting period of **0.79 years**[56](index=56&type=chunk) [6. Stockholders' Equity](index=20&type=section&id=6.%20Stockholders'%20Equity) Addresses MediciNova's At-The-Market (ATM) issuance sales agreement and the absence of sales under it during the reporting period - The company has an At-The-Market (ATM) issuance sales agreement for up to **$75.0 million** in common stock sales, but no shares were sold under this agreement in the three months ended March 31, 2025, or 2024[58](index=58&type=chunk)[59](index=59&type=chunk) [7. Net Loss Per Share](index=20&type=section&id=7.%20Net%20Loss%20Per%20Share) Presents MediciNova's basic and diluted net loss per common share and the exclusion of anti-dilutive stock options Net Loss Per Common Share | Metric | Three months ended March 31, 2025 ($) | Three months ended March 31, 2024 ($) | | :------------------------------------------ | :-------------------------------- | :-------------------------------- | | Basic and diluted net loss per common share | $(0.06) | $(0.06) | | Shares used to compute basic and diluted net loss per common share | 49,046,246 shares | 49,046,246 shares | - Potentially dilutive outstanding stock options totaling **7,909,394 shares** (Q1 2025) and **8,578,644 shares** (Q1 2024) were excluded from diluted net loss per common share calculation due to their anti-dilutive effect[61](index=61&type=chunk) [ITEM 2. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS](index=21&type=section&id=ITEM%202.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) Management's analysis of MediciNova's financial performance, liquidity, and capital resources for the three months ended March 31, 2025 [Overview](index=21&type=section&id=Overview) Introduces MediciNova's biopharmaceutical focus, product development strategy, and accumulated deficit as of March 31, 2025 - MediciNova is a biopharmaceutical company focused on developing novel therapeutics for serious diseases with unmet medical needs, primarily in the U.S. market, with key product candidates MN-166 (ibudilast) and MN-001 (tipelukast)[65](index=65&type=chunk) - The company's strategy involves advancing MN-166 and MN-001 through investigator-sponsored trials, grants, and company-funded trials, while also seeking strategic partnerships[67](index=67&type=chunk) - MediciNova has an accumulated deficit of **$429.6 million** as of March 31, 2025, and expects to incur substantial net losses for the next several years[66](index=66&type=chunk) [Research, Development and Patents Expenses](index=22&type=section&id=Research,%20Development%20and%20Patents%20Expenses) Analyzes MediciNova's research, development, and patent expenses, noting stability with shifts in MN-001 and personnel costs Research, Development and Patents Expenses (in thousands) | Expense Category | Three months ended March 31, 2025 (in thousands $) | Three months ended March 31, 2024 (in thousands $) | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | External development expense: MN-166 | $1,084 | $1,064 | | External development expense: MN-001 | $219 | $94 | | R&D personnel expense | $388 | $431 | | Patent expenses | $100 | $140 | | Total research, development and patent expense | $1,840 | $1,782 | - Research, development and patents expenses remained relatively stable quarter-over-quarter, with an increase in MN-001 related expenses and stock option expense offset by decreased payroll costs from a reduction in headcount[76](index=76&type=chunk) [General and Administrative Expenses](index=22&type=section&id=General%20and%20Administrative%20Expenses) Discusses MediciNova's general and administrative expenses, noting their stability and potential for future increases with business expansion General and Administrative Expenses | Expense Category | Three months ended March 31, 2025 ($) | Three months ended March 31, 2024 ($) | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | General and administrative | $1,362,708 | $1,354,124 | - General and administrative expenses may increase in future periods if the company expands its infrastructure due to product development success, capital raising, or increased business development activities[71](index=71&type=chunk) [Critical Accounting Estimates](index=22&type=section&id=Critical%20Accounting%20Estimates) Highlights the role of management estimates in financial statements and confirms no material changes to critical accounting policies - The preparation of consolidated financial statements requires management to make estimates and judgments that affect the reported amounts of assets and liabilities and expenses[72](index=72&type=chunk) - There have been no material changes to the critical accounting policies discussed in the Annual Report on Form 10-K during the three months ended March 31, 2025[73](index=73&type=chunk) [IPR&D and Goodwill](index=24&type=section&id=IPR%26D%20and%20Goodwill) Explains MediciNova's accounting for indefinite-lived IPR&D and goodwill, including impairment testing and potential future charges - In-process research and development (IPR&D) and goodwill are indefinite-lived intangible assets that are not amortized but are tested annually for impairment[74](index=74&type=chunk) - A qualitative impairment assessment as of December 31, 2024, indicated no impairment for goodwill and indefinite-lived intangible assets[75](index=75&type=chunk) - A sustained decline in stock price or other material changes in assumptions could result in a material goodwill and/or intangible asset impairment charge in future periods[75](index=75&type=chunk) [Results of Operations](index=24&type=section&id=Results%20of%20Operations) Compares MediciNova's financial results for the three months ended March 31, 2025, and 2024, focusing on key expense categories and interest income [Comparison of the three months ended March 31, 2025 and 2024](index=24&type=section&id=Comparison%20of%20the%20three%20months%20ended%20March%2031,%202025%20and%202024) Compares MediciNova's research, development, patent, general, and administrative expenses, and interest income for Q1 2025 versus Q1 2024 - Research, development and patents expenses were **$1.8 million** for both the three months ended March 31, 2025 and 2024, remaining relatively stable[76](index=76&type=chunk) - General and administrative expenses were **$1.4 million** for both the three months ended March 31, 2025 and 2024, also remaining relatively stable[77](index=77&type=chunk) Interest Income Comparison | Metric | Three months ended March 31, 2025 ($) | Three months ended March 31, 2024 ($) | Change ($) | | :------------- | :-------------------------------- | :-------------------------------- | :----- | | Interest income | $336,111 | $397,510 | $(61,399) | - The decrease in interest income was primarily driven by a decrease in the company's cash balance generating interest[78](index=78&type=chunk) [Liquidity and Capital Resources](index=24&type=section&id=Liquidity%20and%20Capital%20Resources) Assesses MediciNova's cash position, working capital, and funding outlook, noting net cash used in operating activities and ATM agreement status Net Cash Used in Operating Activities | Metric | Three months ended March 31, 2025 ($) | Three months ended March 31, 2024 ($) | Change ($) | | :-------------------------------- | :-------------------------------- | :-------------------------------- | :----- | | Net cash used in operating activities | $(3,783,661) | $(3,868,248) | $84,587 (decrease in usage) | Liquidity Position (as of March 31, 2025) | Metric | Amount ($) | | :------------------------ | :----------- | | Cash and cash equivalents | $36.6 million | | Working capital | $35.5 million | - The company believes it has sufficient working capital to fund operations at least through **May 2026**, but cannot provide assurance that these resources will be sufficient to conduct all planned research and development programs[80](index=80&type=chunk) - No shares of common stock were sold under the **$75.0 million** At-The-Market (ATM) issuance sales agreement in the three months ended March 31, 2025, or 2024[81](index=81&type=chunk)[82](index=82&type=chunk) [ITEM 3. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK](index=26&type=section&id=ITEM%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) States that MediciNova has no applicable disclosures regarding quantitative and qualitative market risk for the reporting period - The company has no applicable disclosures for quantitative and qualitative market risk[83](index=83&type=chunk) [ITEM 4. CONTROLS AND PROCEDURES](index=26&type=section&id=ITEM%204.%20CONTROLS%20AND%20PROCEDURES) Details the evaluation of MediciNova's disclosure controls and procedures, confirming their effectiveness and reporting no material changes in internal control [Evaluation of Disclosure Controls and Procedures](index=26&type=section&id=Evaluation%20of%20Disclosure%20Controls%20and%20Procedures) Concludes that MediciNova's disclosure controls and procedures were effective at a reasonable assurance level as of March 31, 2025 - The Chief Executive Officer and Chief Financial Officer concluded that the company's disclosure controls and procedures were effective at the reasonable assurance level as of March 31, 2025[86](index=86&type=chunk) [Changes in Internal Control over Financial Reporting](index=26&type=section&id=Changes%20in%20Internal%20Control%20over%20Financial%20Reporting) Reports no material changes in MediciNova's internal control over financial reporting during the most recent fiscal quarter - There has been no change in the company's internal control over financial reporting during the most recent fiscal quarter that has materially affected, or is reasonably likely to materially affect, its internal control over financial reporting[87](index=87&type=chunk) PART II. OTHER INFORMATION Presents additional information beyond financial statements, including legal proceedings, risk factors, and other disclosures [ITEM 1. LEGAL PROCEEDINGS](index=27&type=section&id=ITEM%201.%20LEGAL%20PROCEEDINGS) Confirms MediciNova is not involved in any material legal proceedings as of March 31, 2025 - The company is not involved in any material legal proceedings as of March 31, 2025[90](index=90&type=chunk) [ITEM 1A. RISK FACTORS](index=27&type=section&id=ITEM%201A.%20RISK%20FACTORS) Refers to previously disclosed risk factors in the Annual Report on Form 10-K, noting no material changes for the current quarter - Readers should carefully consider the risk factors discussed in Part I, Item 1A of the Annual Report on Form 10-K for the year ended December 31, 2024[91](index=91&type=chunk) - No material changes from the previously disclosed risk factors in the Annual Report on Form 10-K were identified during the quarter ended March 31, 2025[91](index=91&type=chunk) [ITEM 2. UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS](index=27&type=section&id=ITEM%202.%20UNREGISTERED%20SALES%20OF%20EQUITY%20SECURITIES%20AND%20USE%20OF%20PROCEEDS) Reports no unregistered sales of equity securities or use of proceeds for the period - None to report[92](index=92&type=chunk) [ITEM 3. DEFAULTS UPON SENIOR SECURITIES](index=27&type=section&id=ITEM%203.%20DEFAULTS%20UPON%20SENIOR%20SECURITIES) Reports no defaults upon senior securities for the period - None to report[92](index=92&type=chunk) [ITEM 4. MINE SAFETY DISCLOSURES](index=27&type=section&id=ITEM%204.%20MINE%20SAFETY%20DISCLOSURES) States that this item is not applicable to MediciNova's operations - Not applicable[92](index=92&type=chunk) [ITEM 5. OTHER INFORMATION](index=27&type=section&id=ITEM%205.%20OTHER%20INFORMATION) Reports no adoption or termination of Rule 10b5-1 or non-Rule 10b5-1 trading arrangements by officers or directors - No officers or directors informed the company of the adoption or termination of Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during the quarter ended March 31, 2025[92](index=92&type=chunk) [ITEM 6. EXHIBITS](index=28&type=section&id=ITEM%206.%20EXHIBITS) Lists the exhibits filed with the Form 10-Q, including certifications from principal executive and financial officers, and Inline XBRL documents - The report includes certifications from the Principal Executive Officer and Principal Financial Officer (Exhibits 31.1, 31.2, 32.1, 32.2) and Inline XBRL documents (Exhibits 101.INS, 101.SCH, 104)[94](index=94&type=chunk) SIGNATURES Provides the official signatures of MediciNova's President, CEO, and CFO, certifying the report - The report is signed by Yuichi Iwaki, M.D., Ph.D., President and Chief Executive Officer, and Jason Kruger, Chief Financial Officer, on behalf of MediciNova, Inc[99](index=99&type=chunk)

Core Viewpoint - MediciNova, Inc. is actively engaging with investors at the upcoming DBC Conference, highlighting its focus on innovative treatments for serious diseases with unmet medical needs, particularly in neurologic and metabologic disorders [1][2]. Company Overview - MediciNova, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for serious diseases, primarily targeting the U.S. market [2]. - The company's leading programs include MN-166 (ibudilast) for various neurological disorders and MN-001 (tipelukast) for fibrotic and metabolic disorders [2]. - Current therapeutic targets for MN-166 include amyotrophic lateral sclerosis (ALS), progressive multiple sclerosis (MS), chemotherapy-induced peripheral neuropathy, and Long COVID, among others [2]. - MN-001 is being developed for conditions such as nonalcoholic fatty liver disease (NAFLD) and hypertriglyceridemia [2]. - The company plans to advance its pipeline through a mix of investigator-sponsored clinical trials, government grants, and strategic alliances [2]. Upcoming Events - CEO Yuichi Iwaki and CBO David H. Crean will participate in investor meetings at the DBC Conference on May 14, 2025, in New York City [1]. - The conference aims to connect public and private executives with institutional investors and corporate clients across various sectors, including Healthcare & Life Sciences [1].

Core Viewpoint - MediciNova, Inc. is actively engaging with investors at the upcoming DBC Conference, highlighting its focus on innovative treatments for serious neurological and metabolic disorders [1][2]. Company Overview - MediciNova, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for serious diseases with unmet medical needs, primarily targeting the U.S. market [2]. - The company's leading programs include MN-166 (ibudilast) for various neurological disorders such as amyotrophic lateral sclerosis (ALS) and progressive multiple sclerosis (MS), and MN-001 (tipelukast) for fibrotic and metabolic disorders like nonalcoholic fatty liver disease (NAFLD) [2]. Strategic Focus - MediciNova plans to advance its pipeline through a combination of investigator-sponsored clinical trials, government grants, self-funded trials, and strategic alliances to support the clinical development of its lead programs [2].

Core Insights - MediciNova, Inc. has initiated the enrollment of the first patient in the NIH-funded Expanded Access Program (EAP) trial for MN-166 (ibudilast) aimed at patients with Amyotrophic Lateral Sclerosis (ALS) [1][2] - The EAP trial will assess the safety and efficacy of MN-166 in approximately 200 ALS patients who are not eligible for the ongoing Phase 2/3 COMBAT-ALS trial [2][3] - MN-166 (ibudilast) is a small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, currently in late-stage clinical development for various neurodegenerative diseases [4] Company Overview - MediciNova is a clinical-stage biopharmaceutical company focused on developing novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases, with a pipeline that includes 11 clinical programs [5] - The lead asset, MN-166 (ibudilast), is in Phase 3 trials for ALS and degenerative cervical myelopathy (DCM) and is ready for Phase 3 for progressive multiple sclerosis (MS) [5] - MediciNova has received Orphan Drug Designation for MN-166 in ALS from the U.S. FDA and EU EMA, and it has also been granted Fast Track Designation by the FDA for ALS treatment [4][5]

Financial Performance - The company reported significant operating losses and does not expect to generate any revenues from product sales for the foreseeable future [20]. - The company has no products approved for commercial sale, which limits its revenue generation capabilities [20]. - The company anticipates fluctuations in its quarterly or annual operating results, which could impact investor confidence [19]. Product Development and Regulatory Challenges - The company relies heavily on the success of its product candidates MN-166 (ibudilast) and MN-001 (tipelukast) for future growth [20]. - The complexity and high costs associated with obtaining regulatory approval for product candidates are significant challenges [22]. - The company faces uncertainties in completing clinical trials and obtaining regulatory approvals in a timely manner [15]. - The potential for undesirable side effects during clinical trials could delay or prevent regulatory approval [22]. - Future development and regulatory difficulties may arise even if the company successfully receives regulatory approval for its product candidates [22]. Capital and Operational Risks - There are known risks related to the inability to raise additional capital if needed, which could impact business operations [15]. - The company is dependent on third parties for conducting clinical trials and manufacturing product candidates [15].

Core Insights - MediciNova, Inc. announced the interim analysis results from the COMBAT-ALS Phase 2b/3 clinical trial of MN-166 (ibudilast) for Amyotrophic Lateral Sclerosis (ALS), which will be presented at the 35th International Symposium on ALS/MND [1][2] Study Update - As of November 15, 2024, 217 participants have been enrolled in the trial, with 183 assigned to either the MN-166 or placebo group [2] - The interim analysis showed positive correlations between 6-month and 12-month data for various scores, including CAFS score (0.71), modified CAFS score (0.70), and ALSFRS-R (0.69) [2] - The Data Safety Monitoring Board (DSMB) reviewed the interim results and recommended the continuation of the trial as per the protocol [2][3] Future Expectations - The company expects to complete patient assignments by June 2025, with trial results anticipated in 2026 [3] - An NIH-funded large-scale Expanded Access Program trial is set to begin next year, aiming to provide access to MN-166 for more ALS patients [3] Product Information - MN-166 (ibudilast) is a small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, currently in late-stage clinical development for ALS and other neurodegenerative diseases [4] - The compound is also being evaluated for conditions such as progressive multiple sclerosis, glioblastoma, Long COVID, and substance use disorder [4] Company Overview - MediciNova, Inc. is a clinical-stage biopharmaceutical company with a late-stage pipeline of novel small molecule therapies targeting inflammatory, fibrotic, and neurodegenerative diseases [5] - The company has 11 programs in clinical development, with MN-166 being the lead asset currently in Phase 3 for ALS and DCM [5]

Core Viewpoint - MediciNova, Inc. has received a Notice of Allowance from the U.S. Patent and Trademark Office for a patent application related to MN-001 (Tipelukast), which covers a method to decrease triglyceride synthesis in the liver, particularly beneficial for patients with insulin resistance, pre-diabetes, or diabetes. The patent is expected to expire no earlier than May 26, 2042 [1][2]. Group 1: Patent and Methodology - The allowed patent claims cover a method to decrease triglyceride synthesis in the liver through the administration of MN-001 (Tipelukast) or its metabolites [1]. - MN-001 can be administered in various formulations and dosages, providing flexibility in treatment [1]. - The patent represents a significant advancement in developing treatments for metabolic disorders, emphasizing the company's commitment to innovative therapies [2]. Group 2: About MN-001 - MN-001 (Tipelukast) is a novel, orally bioavailable small molecule that exhibits anti-inflammatory and anti-fibrotic activity through multiple mechanisms, including leukotriene receptor antagonism and inhibition of phosphodiesterases [3]. - The compound has shown efficacy in down-regulating genes associated with fibrosis and inflammation, and it inhibits triglyceride synthesis in hepatocytes [3]. Group 3: Context of Metabolic Disorders - Non-alcoholic fatty liver disease (NAFLD) is linked to metabolic syndrome, with studies indicating that 50% of patients with metabolic syndrome also have NAFLD [4]. - NAFLD is associated with increased risks of cardiovascular disease, Type 2 Diabetes Mellitus (T2DM), chronic kidney disease, and malignancy, with dyslipidemia present in 20-80% of NAFLD cases [4]. Group 4: Company Overview - MediciNova, Inc. is a clinical-stage biopharmaceutical company with a late-stage pipeline focused on novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases [5]. - The company has 11 programs in clinical development, with its lead asset, MN-166 (Ibudilast), currently in Phase 3 trials for amyotrophic lateral sclerosis and degenerative cervical myelopathy [5].

Financial Performance - The net loss for the three months ended September 30, 2024, was $2,852,179, compared to a net loss of $723,107 for the same period in 2023, indicating a significant increase in losses[8] - Net loss for the nine months ended September 30, 2024, was $8,234,897, compared to a net loss of $6,512,431 for the same period in 2023, representing an increase of approximately 26.4%[13] - The basic and diluted net loss per common share for the three months ended September 30, 2024, was $(0.06), compared to $(0.01) for the same period in 2023[8] - The company reported no revenues for the three months ended September 30, 2024, consistent with the same period in 2023, maintaining a revenue stagnation[8] Assets and Liabilities - Total current assets decreased from $51,174,380 on December 31, 2023, to $43,265,478 on September 30, 2024, representing a decline of approximately 15.5%[7] - Cash and cash equivalents decreased from $50,999,442 on December 31, 2023, to $42,280,618 on September 30, 2024, a reduction of approximately 17.1%[7] - The total liabilities decreased from $3,891,553 on December 31, 2023, to $3,053,860 on September 30, 2024, a decline of about 21.4%[7] - The total stockholders' equity decreased from $62,378,424 on December 31, 2023, to $55,065,561 on September 30, 2024, reflecting a decline of approximately 11.7%[9] Operating Expenses - Operating expenses for the three months ended September 30, 2024, were $3,309,128, an increase of 54% compared to $2,146,888 for the same period in 2023[8] - Research, development, and patents expenses rose to $1,859,400 for the three months ended September 30, 2024, compared to $794,706 for the same period in 2023, reflecting a 134% increase[8] - Research and development costs for the three months ended September 30, 2024, totaled $1.8 million, up from $0.7 million in 2023, indicating a significant increase of approximately 157.1%[22] - Research and development costs for the nine months ended September 30, 2024, were $5.0 million, compared to $3.7 million in 2023, marking an increase of approximately 35.1%[22] Cash Flow - The company reported a net cash used in operating activities of $8,711,232 for the nine months ended September 30, 2024, compared to $6,931,978 for the same period in 2023, an increase of approximately 25.6%[13] - Cash and cash equivalents at the end of the period were $42,280,618, down from $51,507,361 at the end of the same period in 2023, reflecting a decrease of approximately 17.5%[13] - The company’s cash flow from investing activities was negative $698 for the nine months ended September 30, 2024, compared to a positive cash flow of $39,907,694 in 2023[13] Stock Options and Compensation - As of September 30, 2024, the total outstanding stock options increased to 8,359,844, with a weighted average exercise price of $5.18[48] - Stock-based compensation expense for the three months ended September 30, 2024, was $504,862, compared to $219,142 for the same period in 2023, reflecting a 130% increase[49] - For the nine months ended September 30, 2024, total stock-based compensation expense was $927,747, slightly down from $948,115 in 2023[49] - The weighted-average fair value of stock options granted during the nine months ended September 30, 2024, was $0.99 per option, compared to $1.60 per option in 2023[51] Lease Commitments - The Company has a lease commitment for its headquarters in San Diego with a term ending January 31, 2027, and a new lease in Tokyo effective June 2024 with an initial term of 12 months[35] - Total operating lease liabilities as of September 30, 2024, amounted to $457,126, down from $626,586 as of December 31, 2023, indicating a reduction of approximately 27%[37] - The Company’s operating lease costs for the nine months ended September 30, 2024, were $206,228, compared to $190,240 for the same period in 2023, representing an increase of approximately 8.4%[37] Future Prospects - The company continues to rely on the success of its product candidates, MN-166 and MN-001, for future revenue generation and market positioning[3] - Future potential milestone payments for product development of MN-166 and MN-001 are estimated at $10 million as of September 30, 2024, with additional potential milestone payments totaling $16.5 million for other products[41] - The Company has not made any milestone payments under its licensing agreements during the nine months ended September 30, 2024, and 2023[41] Regulatory and Compliance - The Company is currently evaluating the potential impact of ASU 2023-07 and ASU 2023-09 on its consolidated financial statements and related disclosures[30][31] - The Company is entitled to receive a portion of monetary damages from Genzyme if any are recovered as a result of the settled litigation with Sanofi[56]

Core Viewpoint - MediciNova, Inc. has been notified of a settlement in the Sanofi/Novartis litigation, which entitles the company to receive monetary damages, validating its intellectual property and providing non-dilutive funding for ongoing clinical development programs [1][2]. Group 1: Litigation Details - The Sanofi/Novartis litigation involved a claim for infringement of U.S. Patent No. 9,051,542, filed by Genzyme Corporation against Novartis, alleging unauthorized use of recombinant adeno-associated virus vectors for the gene therapy drug Zolgensma [3]. - MediciNova is entitled to a portion of any monetary damages recovered by Genzyme as per the terms of the assignment agreement dated December 19, 2005 [3]. Group 2: Zolgensma Overview - Zolgensma, approved by the FDA on May 24, 2019, is a gene therapy for spinal muscular atrophy (SMA) in children under two years old, priced at $2.1 million per infusion, making it one of the most expensive medicines [4]. - In 2023, Zolgensma generated over $1.3 billion in annual sales, with expectations to earn between $1.5 billion and $2 billion annually [4]. Group 3: Company Overview - MediciNova is a clinical-stage biopharmaceutical company with a late-stage pipeline focused on novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases [5]. - The company has 11 clinical development programs, with its lead asset, MN-166 (ibudilast), currently in Phase 3 trials for amyotrophic lateral sclerosis (ALS) and degenerative cervical myelopathy (DCM) [5].