MN-166 COMBAT ALS trial down to single digit required enrollment while MN-001 hypertriglyceridemia trial in type 2 diabetes patients requires final two patientsLA JOLLA, Calif., July 24, 2025 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875) (the “Company”), today provides an update on the enrollment of two key ongoing clinical trials for the Company’s developmental compou ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED MARCH 31, 2025 MEDICINOVA, INC. (Exact name of registrant as specified in its charter) Delaware 33-0927979 (State or Other Jurisdiction of Incorporation or Organization) 4275 Executive Square, Suite 300 La Jolla, CA 92037 (Address of Principal Executive Offices) (Zip Code) ☐ TRANSITION REPORT PURSUA ...

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].