Financial Performance - Total current assets decreased from $51,174,380 on December 31, 2023, to $47,863,595 on March 31, 2024, a decline of approximately 6.4%[15] - Operating expenses increased to $3,135,993 for the three months ended March 31, 2024, compared to $2,963,243 for the same period in 2023, representing a rise of about 5.8%[17] - The net loss for the first quarter of 2024 was $2,754,518, a slight improvement from the net loss of $2,917,504 in Q1 2023, indicating a reduction of approximately 5.6%[17] - Cash and cash equivalents decreased from $50,999,442 at the beginning of the period to $47,138,799 at the end of March 2024, a decrease of about 7.5%[22] - The total stockholders' equity decreased from $62,378,424 on December 31, 2023, to $59,830,444 on March 31, 2024, a decline of about 4.4%[15] - The company reported interest income of $397,510 for Q1 2024, down from $509,046 in Q1 2023, reflecting a decrease of approximately 22%[17] - The company’s cash flow from operating activities for Q1 2024 was $(3,868,248), compared to $(3,177,413) in Q1 2023, indicating an increase in cash used in operations[22] - The accumulated deficit of the company reached $418.5 million as of March 31, 2024, indicating significant net losses since inception[68] Research and Development - Research and development expenses totaled $1,781,869 for Q1 2024, up from $1,476,676 in Q1 2023, marking an increase of approximately 20.6%[17] - The company plans to advance the development of MN-166 (ibudilast) through a combination of investigator-sponsored clinical trials and trials funded by government grants[70] - The company has incurred research, development, and patents expenses primarily related to clinical development, with expectations to increase these costs through the remainder of 2024[76] - Research, development, and patents expenses increased to $1.8 million for the three months ended March 31, 2024, compared to $1.5 million in the same period of 2023, primarily due to higher manufacturing costs for MN-166[85] - Future potential milestone payments for product development total $26.5 million as of March 31, 2024, with $10 million related to MN-166 and MN-001[48] Stock and Equity - The company has maintained its common stock at 49,046,246 shares issued and outstanding as of March 31, 2024, consistent with the previous quarter[19] - As of March 31, 2024, 1,355,923 shares remain available for future grants under the 2023 Equity Incentive Plan[52] - The Company adopted the 2023 Equity Incentive Plan in June 2023, succeeding the 2013 Plan, with a maximum of 9,934,567 shares potentially available for issuance[51] - As of March 31, 2024, the company had a total of 1,016,000 shares underlying performance options subject to vesting based on achievement of corporate objectives for 2024[53] - The total stock-based compensation expense for the three months ended March 31, 2024, was $215,430, a decrease of 46.5% compared to $403,263 for the same period in 2023[56] - The weighted-average fair value of each stock option granted during the three months ended March 31, 2024, was $1.00, down from $1.60 in 2023[58] - As of March 31, 2024, there were 8,578,644 potentially dilutive outstanding stock options excluded from diluted net loss per share due to their anti-dilutive effect[63] Lease and Operating Expenses - The Company reported cash paid for operating lease liabilities of $64,594 for the three months ended March 31, 2024, compared to $64,990 for the same period in 2023[45] - Total operating lease liabilities as of March 31, 2024, amounted to $569,072, down from $626,586 as of December 31, 2023[45] - The weighted-average remaining lease term is 2.60 years, with a weighted-average discount rate of 9.3%[45] - General and administrative expenses decreased to $1.4 million for the three months ended March 31, 2024, from $1.5 million in the same period of 2023, mainly due to a reduction in performance-based stock option expenses[86] Strategic Partnerships and Future Plans - The company intends to pursue strategic partnerships with leading pharmaceutical companies to support clinical development and commercialization of its products[70] - The Company is currently evaluating the potential impact of ASU 2023-07 on its consolidated financial statements and related disclosures, effective for fiscal years beginning after December 15, 2023[39] - The company has not made any milestone payments under its licensing agreements during the three months ended March 31, 2024[48] - The company believes it carries reasonably adequate insurance for product liability risks associated with its drug products[46] - The company has not experienced any material changes in its critical accounting policies during the three months ended March 31, 2024[82] Cash Position - As of March 31, 2024, the company had available cash and cash equivalents of $47.1 million and working capital of $45.4 million, sufficient to fund operations at least through the end of 2025[90] - Net cash used in operating activities was $3.9 million for the three months ended March 31, 2024, compared to $3.2 million in the same period of 2023, reflecting a $0.7 million change related to decreased net loss[88] - The company has not sold any shares of common stock under the ATM Agreement in the three months ended March 31, 2024, and 2023[61] - No shares of common stock were sold under the at-the-market issuance sales agreement in the three months ended March 31, 2024, and 2023[92]

LA JOLLA, Calif., May 07, 2024 (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), today announced that it has received an issue notification from the U.S. Patent and Trademark Office for a new patent which covers extended-release oral formulations of MN-166 (ibudilast). This new patent is expected to expire no earlier than September 2040. The allowed claims cover a formula ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 Form 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from. to Commission file number: 001-33185 MEDICINOVA, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 33-0927979 (State or Other Jurisdiction of Inc ...

Financial Performance - Revenues for the three months ended September 30, 2023, were $1,000,000, compared to $0 for the same period in 2022, indicating a significant revenue generation[16] - The net loss applicable to common stockholders for the three months ended September 30, 2023, was $723,107, a decrease from $3,652,184 in the same period of 2022, showing an improvement of approximately 80%[16] - Revenues for the three months ended September 30, 2023, were $1.0 million, an increase from $0.0 million in the same period of 2022, due to a milestone payment under the Genzyme Agreement[83] - For the nine months ended September 30, 2023, revenues were $1.0 million, consistent with the three-month results, attributed to the Genzyme Agreement milestone payment[87] Cash Flow and Liquidity - Cash and cash equivalents increased to $51,507,361 as of September 30, 2023, up from $18,505,493 at December 31, 2022, representing a growth of approximately 178%[14] - The company reported a net cash used in operating activities of $6,931,978 for the nine months ended September 30, 2023, compared to $8,904,519 for the same period in 2022, indicating a reduction of about 22%[20] - Cash provided by investing activities was $39,907,694 for the nine months ended September 30, 2023, compared to cash used of $10,000,000 in the same period of 2022, reflecting a positive shift in cash flow[20] - As of September 30, 2023, the company had available cash and cash equivalents of $51.5 million and working capital of $50.2 million, sufficient to fund operations at least through the end of 2024[94] Operating Expenses - Total operating expenses decreased to $2,146,888 for the three months ended September 30, 2023, down from $3,897,467 in the same period of 2022, reflecting a reduction of approximately 45%[16] - Research, development, and patents expenses decreased to $0.8 million for the three months ended September 30, 2023, from $2.5 million in 2022, primarily due to reduced manufacturing expenses for MN-166[84] - Research, development, and patents expenses for the nine months ended September 30, 2023, were $4.0 million, down from $7.1 million in 2022, mainly due to decreased manufacturing expenses[88] - General and administrative expenses remained stable at $1.4 million for the three months ended September 30, 2023, compared to the same period in 2022[85] - General and administrative expenses increased to $4.4 million for the nine months ended September 30, 2023, compared to $4.3 million in the same period of 2022, reflecting a $0.1 million rise primarily due to performance-based stock option expenses[89] Assets and Equity - Total assets decreased to $68,136,561 as of September 30, 2023, from $74,154,905 at December 31, 2022, a decline of about 8%[14] - Total stockholders' equity decreased to $64,669,252 as of September 30, 2023, down from $70,242,035 at December 31, 2022, representing a decrease of approximately 8%[14] Research and Development - MediciNova's research and development costs totaled $0.7 million for Q3 2023, down from $2.4 million in Q3 2022, and $3.7 million for the nine months ended September 30, 2023, compared to $6.8 million for the same period in 2022[31] - The company is focused on developing MN-166 (ibudilast) for multiple neurological disorders and MN-001 (tipelukast) for fibrotic diseases, with plans for strategic partnerships to support product development[69] - The company has experienced slower enrollment in clinical trials due to the COVID-19 pandemic but has seen an increase in patient visits recently[70] Stock and Equity Compensation - Total stock-based compensation expense for the three months ended September 30, 2023, was $219,142, up 38.4% from $158,298 in the same period of 2022[58] - As of September 30, 2023, there were 8,304,749 stock options outstanding, with a weighted average exercise price of $5.34[56] - The estimated fair value of stock options granted during the nine months ended September 30, 2023, was $1.60, compared to $1.47 for the same period in 2022[60] - The Company has 1,629,818 shares available for future grants under the 2023 Equity Incentive Plan as of September 30, 2023[53] Other Financial Metrics - The basic and diluted net loss per common share for the three months ended September 30, 2023, was $0.01, an improvement from $0.07 in the same period of 2022[16] - Interest income increased to $0.4 million for the three months ended September 30, 2023, from $0.3 million in 2022, driven by higher interest rates[86] - Interest income rose significantly to $1.4 million for the nine months ended September 30, 2023, up from $0.4 million in 2022, marking a $1.0 million increase driven by higher interest rates[90] - The accumulated deficit was $413.6 million, with expectations of substantial net losses continuing for several years[68]

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 June 30, 2023 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission file number: 001-33185 MEDICINOVA, INC. (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Incorporation or Organi ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q Commission file number: 001-33185 MEDICINOVA, INC. (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Incorporation or Organization) 4275 Executive Square, Suite 300 La Jolla, CA 92037 (Address of Principal Executive Offices) (Zip Code) (858) 373-1500 (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, 20 ...

PART I [Forward-Looking Statements and Summary Risk Factors](index=3&type=section&id=Forward-Looking%20Statements%20and%20Summary%20Risk%20Factors) The report contains forward-looking statements subject to risks and uncertainties, including capital needs, product development, and market acceptance - Forward-looking statements carry inherent risks and uncertainties that could cause actual results to differ materially[11](index=11&type=chunk) - Key risks include the inability to raise additional capital, generate product revenues, develop and commercialize candidates, or face clinical trial and regulatory delays[12](index=12&type=chunk)[17](index=17&type=chunk) - The company has incurred significant operating losses and expects continued losses, with an accumulated deficit of **$407.1 million** as of December 31, 2022[181](index=181&type=chunk) - Success is highly dependent on MN-166 (ibudilast) and MN-001 (tipelukast) product candidates, neither of which has received regulatory approval[17](index=17&type=chunk)[189](index=189&type=chunk) [Item 1. Business](index=6&type=section&id=Item%201.%20Business) MediciNova is a biopharmaceutical company developing novel therapeutics for serious diseases, focusing on MN-166 and MN-001, relying on third parties and strategic partnerships - MediciNova is a biopharmaceutical company developing novel therapeutics for serious diseases with unmet medical needs, focusing on the U.S. market[21](index=21&type=chunk) - Primary product candidates are MN-166 (ibudilast) for neurological disorders and MN-001 (tipelukast) for fibrotic diseases[21](index=21&type=chunk)[51](index=51&type=chunk)[89](index=89&type=chunk) - The company's strategy involves advancing MN-166 and MN-001 development through non-dilutive financing and seeking strategic partnerships for commercialization[47](index=47&type=chunk) [Overview](index=6&type=section&id=Overview) MediciNova develops novel therapeutics for unmet medical needs in the U.S., with key candidates MN-166 for neurological disorders and MN-001 for fibrotic diseases - The company focuses on developing MN-166 (ibudilast) for progressive MS, ALS, chemotherapy-induced peripheral neuropathy, and ARDS prevention[21](index=21&type=chunk) - MN-001 (tipelukast) is under development for nonalcoholic fatty liver disease (NAFLD) and idiopathic pulmonary fibrosis (IPF)[21](index=21&type=chunk) - Additional pipeline candidates include MN-221 (bedoradrine) for acute asthma and MN-029 (denibulin) for solid tumor cancers[21](index=21&type=chunk) [Our Product Candidates and Programs](index=7&type=section&id=Our%20Product%20Candidates%20and%20Programs) MediciNova develops product candidates for unmet medical needs, primarily MN-166 (anti-inflammatory/neuroprotective) and MN-001 (anti-fibrotic/inflammatory), with MN-221 license terminated - MN-166 (ibudilast) is a novel, first-in-class, oral, anti-inflammatory and neuroprotective agent, licensed from Kyorin Pharmaceuticals in 2004[51](index=51&type=chunk) - MN-166 has received FDA Fast Track designation for progressive MS, ALS, and methamphetamine dependence, and Orphan-Drug designation for ALS and glioblastoma[52](index=52&type=chunk)[53](index=53&type=chunk) - MN-001 (tipelukast) is a novel, orally bioavailable small molecule with anti-fibrotic and anti-inflammatory activity, licensed from Kyorin in 2002[89](index=89&type=chunk)[90](index=90&type=chunk) - MN-001 has received FDA Fast Track designation for NASH with fibrosis and IPF, and Orphan-Drug designation for IPF[90](index=90&type=chunk)[101](index=101&type=chunk) - The license agreement for MN-221 (bedoradrine) for acute asthma exacerbations was terminated in October 2022[46](index=46&type=chunk)[84](index=84&type=chunk) [MN-166 (ibudilast)](index=12&type=section&id=MN-166%20%28ibudilast%29) MN-166 (ibudilast) is a first-in-class anti-inflammatory and neuroprotective agent showing positive Phase 2b results in progressive MS and Phase 2 results in ALS and ARDS prevention - MN-166 (ibudilast) is a novel, first-in-class, oral, anti-inflammatory and neuroprotective agent, inhibiting MIF and PDEs, and attenuating activated glia cells[51](index=51&type=chunk) - In the SPRINT-MS Phase 2b trial for progressive MS, MN-166 demonstrated a statistically significant **48% reduction** in whole brain atrophy and a **26% reduction** in confirmed disability progression risk compared to placebo[57](index=57&type=chunk) - A Phase 2 clinical trial of MN-166 in ALS achieved its primary safety and tolerability endpoint, showing a higher responder rate in functional activity compared to placebo[62](index=62&type=chunk) - A Phase 2 clinical trial of MN-166 in alcohol use disorder reduced the odds of heavy drinking by **45%** and attenuated alcohol cue-elicited activation in the ventral striatum[70](index=70&type=chunk) - In a Phase 2 clinical trial for ARDS prevention in COVID-19 patients, MN-166 achieved statistical significance for subjects free of respiratory failure at Day 7 (**71%** vs **35%** for placebo, p=0.02) and discharged from hospital (**65%** vs **29%** for placebo, p=0.02)[80](index=80&type=chunk) [MN-221 (bedoradrine)](index=20&type=section&id=MN-221%20%28bedoradrine%29) MN-221 (bedoradrine), a beta-2-adrenergic receptor agonist for acute asthma, showed some clinical improvements despite not meeting its primary endpoint, and its license was terminated in October 2022 - MN-221 (bedoradrine) is a novel, highly selective beta-2-adrenergic receptor agonist for acute asthma exacerbations, designed for intravenous infusion[84](index=84&type=chunk)[85](index=85&type=chunk) - A Phase 2b trial did not statistically meet the primary FEV1 endpoint but showed significant improvements in Dyspnea Index scores and a reduced treatment failure rate (**74%** placebo vs **43%** MN-221) in a subgroup[87](index=87&type=chunk) - The license agreement for MN-221 was terminated in October 2022, with no further financial obligations to Kissei Pharmaceutical Co., Ltd[84](index=84&type=chunk) [MN-001 (tipelukast)](index=20&type=section&id=MN-001%20%28tipelukast%29) MN-001 (tipelukast) is an orally bioavailable small molecule with anti-fibrotic and anti-inflammatory properties, showing significant triglyceride reduction in NASH/NAFLD and positive biomarker effects in IPF - MN-001 (tipelukast) acts through leukotriene receptor antagonism, PDE inhibition, and 5-lipoxygenase inhibition, down-regulating genes promoting fibrosis and inflammation[89](index=89&type=chunk)[90](index=90&type=chunk) - In a Phase 2 clinical trial for NASH/NAFLD, MN-001 significantly reduced mean serum triglycerides by **41.3%** (p=0.02) in an interim analysis[95](index=95&type=chunk) - A subgroup analysis showed Type 2 diabetes mellitus patients treated with MN-001 experienced a greater reduction in serum triglyceride levels (**50.8%** vs **17.8%**) and a significantly greater increase in HDL (**15.8%** vs **1.0%**, p<0.0002) compared to non-T2DM subjects[97](index=97&type=chunk) - In a Phase 2 IPF trial, MN-001 demonstrated no worsening IPF events and a substantial reduction in the LOXL2 biomarker, while being safe and well-tolerated[101](index=101&type=chunk) [MN-029 (denibulin)](index=23&type=section&id=MN-029%20%28denibulin%29) MN-029 (denibulin) is a novel tubulin binding agent for solid tumors, showing anti-tumor activity in preclinical studies and well-tolerated in Phase 1 trials with some stable disease - MN-029 (denibulin) is a novel tubulin binding agent for solid tumors, licensed from Angiogene Pharmaceuticals, inhibiting tubulin polymerization and disrupting cell cytoskeleton[102](index=102&type=chunk) - Preclinical studies showed MN-029 damaged tumor blood vessels and caused vascular shutdown within tumors, in addition to direct effects on cancer cells[103](index=103&type=chunk) - Phase 1 clinical trials indicated MN-029 was well-tolerated at doses that reduced tumor blood flow, with stable disease in **12** patients in the first trial and one partial response in the second[104](index=104&type=chunk)[106](index=106&type=chunk) [Our Strategy](index=11&type=section&id=Our%20Strategy) MediciNova's strategy is to build a sustainable biopharmaceutical business by developing differentiated products for unmet medical needs, advancing MN-166 and MN-001, and seeking strategic partnerships - The company aims to build a sustainable biopharmaceutical business by developing differentiated products for serious diseases with unmet medical needs[47](index=47&type=chunk) - Strategy includes advancing MN-166 (ibudilast) and MN-001 (tipelukast) development with non-dilutive financings, government grants, and investigator-sponsored trials[47](index=47&type=chunk) - MediciNova plans to consider strategic partnerships with leading pharmaceutical companies to complete product development and commercialization[47](index=47&type=chunk) [Sales and Marketing](index=26&type=section&id=Sales%20and%20Marketing) MediciNova lacks internal marketing and sales capabilities, planning to rely on strategic partners for product commercialization - The company has no current marketing and sales capabilities[111](index=111&type=chunk) - MediciNova expects to rely on strategic partners for product commercialization[111](index=111&type=chunk) [Manufacturing](index=26&type=section&id=Manufacturing) MediciNova relies entirely on third-party manufacturers for API and finished products for all development stages and future commercial production - MediciNova relies on third parties to manufacture bulk API and finished investigational products for research, development, preclinical, and clinical trials[112](index=112&type=chunk) - The company expects to continue this reliance for future commercial production[112](index=112&type=chunk) - For MN-166 (ibudilast), the company has historically sourced capsules from Taisho Pharmaceutical Co., Ltd. and uses contract manufacturers[113](index=113&type=chunk) [Intellectual Property and License Agreements](index=26&type=section&id=Intellectual%20Property%20and%20License%20Agreements) MediciNova holds licenses and a robust patent portfolio, primarily method-of-use patents for MN-166 and MN-001, despite expired composition of matter patents, and faces intellectual property risks - MediciNova holds **32** issued U.S. patents and **89** issued foreign patents, with **12** pending U.S. and **31** pending foreign applications[114](index=114&type=chunk) - The company's patent portfolio for MN-166 (ibudilast) primarily covers methods of treating progressive MS, ALS, glioblastoma, and drug addiction, with expirations ranging from 2025 to 2039[121](index=121&type=chunk)[122](index=122&type=chunk)[123](index=123&type=chunk) - Composition of matter patents for MN-166 (ibudilast), MN-001 (tipelukast), and MN-221 (bedoradrine) have expired, allowing competitors to develop products with the same API if method-of-use patents are not infringed[117](index=117&type=chunk)[126](index=126&type=chunk)[137](index=137&type=chunk) - The exclusive license agreement for MN-221 (bedoradrine) with Kissei Pharmaceutical Co., Ltd. was terminated in October 2022[124](index=124&type=chunk) - MediciNova is obligated to make future milestone payments up to **$5.0 million** for MN-166 and MN-001, and up to **$16.5 million** for MN-029, plus royalties on net sales[120](index=120&type=chunk)[130](index=130&type=chunk)[133](index=133&type=chunk) [Competition](index=30&type=section&id=Competition) The biopharmaceutical industry is highly competitive, with MediciNova facing numerous entities and existing treatments that pose significant threats across its target indications - The biopharmaceutical industry is highly competitive, characterized by extensive research and rapid technological progress[139](index=139&type=chunk) - Competitors include pharmaceutical and biotechnology companies, academic institutions, and governmental agencies, many possessing greater resources[139](index=139&type=chunk)[140](index=140&type=chunk) - For progressive MS, approved drugs include Ocrevus (PPMS) and several for SPMS with relapses; other compounds are in clinical development[142](index=142&type=chunk) - For ALS, generic riluzole, Radicava, and Relyvrio are approved, with numerous other compounds in clinical development[143](index=143&type=chunk) - There are no currently approved pharmaceuticals for methamphetamine addiction or chemotherapy-induced peripheral neuropathy[144](index=144&type=chunk)[145](index=145&type=chunk) [Government Regulation](index=33&type=section&id=Government%20Regulation) MediciNova's product development is subject to extensive, lengthy, and costly government regulation by the FDA and foreign authorities, with compliance critical to avoid severe sanctions - The research, development, testing, manufacture, labeling, promotion, advertising, distribution, sampling, marketing, and import/export of pharmaceutical products are extensively regulated by government authorities[154](index=154&type=chunk) - The U.S. regulatory approval process involves preclinical tests, an effective IND application, multi-phase clinical trials, NDA submission, cGMP compliance, and FDA review and approval[160](index=160&type=chunk) - Fast Track designation, Priority Review, and Accelerated Approval programs are available to expedite development and review for serious conditions with unmet medical needs[165](index=165&type=chunk)[167](index=167&type=chunk) - Market exclusivity provisions under the FDCA include five-year exclusivity for new chemical entities and three-year exclusivity for new clinical investigations, plus potential six-month pediatric exclusivity[169](index=169&type=chunk)[170](index=170&type=chunk) - Failure to comply with applicable regulatory requirements can result in sanctions such as fines, delays, suspension or withdrawal of approvals, product recalls, and criminal prosecution[154](index=154&type=chunk)[176](index=176&type=chunk) [Human Capital Resources](index=37&type=section&id=Human%20Capital%20Resources) MediciNova has a small, experienced team of 13 full-time employees with core competencies in management, clinical development, regulatory, and corporate development - MediciNova has **13** full-time employees as of the report date[177](index=177&type=chunk) - The company's team possesses core competencies in general management, clinical development, regulatory affairs, and corporate development[177](index=177&type=chunk) - Employee relations are reported as good, with no history of work stoppages[177](index=177&type=chunk) [Company Information](index=38&type=section&id=Company%20Information) MediciNova, Inc. was incorporated in Delaware in September 2000, with principal executive offices in La Jolla, CA - MediciNova, Inc. was incorporated in Delaware in September 2000[179](index=179&type=chunk) - Principal executive offices are located at 4275 Executive Square, Suite 300, La Jolla, CA 92037[179](index=179&type=chunk) [Item 1A. Risk Factors](index=38&type=section&id=Item%201A.%20Risk%20Factors) This section details significant risks, including operating losses, funding needs, dependence on MN-166 and MN-001, regulatory uncertainties, competition, third-party reliance, and intellectual property issues - MediciNova has incurred significant operating losses since inception and expects continued losses, with an accumulated deficit of **$407.1 million** as of December 31, 2022[181](index=181&type=chunk) - The company will need additional funding to complete product development and commercialization, with current working capital projected to fund operations only through the end of 2024[185](index=185&type=chunk)[186](index=186&type=chunk)[325](index=325&type=chunk) - Future success is highly dependent on the regulatory approval and commercialization of MN-166 (ibudilast) and MN-001 (tipelukast), neither having completed clinical development[189](index=189&type=chunk) - The drug development process is lengthy, costly, and uncertain, with high risks of clinical trial failure, delays, or denial of regulatory approval[195](index=195&type=chunk)[196](index=196&type=chunk) - The company relies heavily on third parties for clinical trials and manufacturing, exposing it to risks of delays, increased costs, and lack of control[229](index=229&type=chunk)[233](index=233&type=chunk) - The expiration of composition of matter patents for key product candidates (MN-166, MN-001, MN-221) means competitors could offer products with the same active pharmaceutical ingredient if method-of-use patents are not infringed[267](index=267&type=chunk)[137](index=137&type=chunk) [Item 1B. Unresolved Staff Comments](index=62&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) MediciNova has no unresolved staff comments from the SEC as of the report date [Item 2. Properties](index=62&type=section&id=Item%202.%20Properties) MediciNova maintains its San Diego headquarters and Tokyo office space under leases, relying on third parties for laboratory, research, and manufacturing facilities - The San Diego headquarters sublease was extended for an additional **5** years through January 31, 2027[293](index=293&type=chunk) - Office space in Tokyo, Japan, was renewed in May 2021 for a two-year term with an auto-renewal option[294](index=294&type=chunk) - MediciNova has no laboratory, research, or manufacturing facilities, relying on third-party service providers[294](index=294&type=chunk) [Item 3. Legal Proceedings](index=62&type=section&id=Item%203.%20Legal%20Proceedings) MediciNova is not currently involved in any material legal proceedings, with no expected material adverse effects from ordinary course disputes - MediciNova is not involved in any material legal proceedings as of the report date[295](index=295&type=chunk) - The company may become involved in disputes in the ordinary course of business, but none are currently expected to have a material adverse effect[295](index=295&type=chunk) [Item 4. Mine Safety Disclosures](index=62&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) MediciNova has no disclosures related to mine safety PART II [Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=63&type=section&id=Item%205.%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) MediciNova's common stock is listed on NASDAQ and TSE, with approximately 11 record holders, no cash dividends paid, and a **$20 million** private placement completed in January 2021 - Common stock is listed on NASDAQ Global Market (MNOV) and Tokyo Stock Exchange (4875)[298](index=298&type=chunk) - As of February 13, 2023, there were approximately **11** holders of record of common stock[298](index=298&type=chunk) - The company has never declared or paid cash dividends and does not anticipate paying any in the foreseeable future[299](index=299&type=chunk) - On January 29, 2021, MediciNova sold **3,656,307** shares of common stock in a private placement for approximately **$20 million** in cash proceeds[301](index=301&type=chunk) [Item 6. [Reserved]](index=63&type=section&id=Item%206.%20%5BReserved%5D) This item is reserved and contains no information [Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=64&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) MediciNova incurred a **$14.1 million** net loss in 2022, increasing its accumulated deficit to **$407.1 million**, with decreased revenues, increased R&D, and liquidity projected to fund operations through 2024 Net Loss and Accumulated Deficit | Metric | 2022 (Millions) | 2021 (Millions) | | :------------------- | :-------------- | :-------------- | | Net Loss | $(14.1) | $(10.1) | | Accumulated Deficit | $(407.1) | $(393.1) | Key Financial Highlights (Years Ended December 31) | Metric | 2022 (Millions) | 2021 (Millions) | Change (YoY) (Millions) | | :-------------------------------- | :-------------- | :-------------- | :---------------------- | | Revenues | $0.0 | $4.0 | $(4.0) | | Research, Development and Patents | $9.1 | $8.5 | $0.6 | | General and Administrative | $5.5 | $5.7 | $(0.2) | | Other Expense, net | $0.247 | $0.059 | $0.188 | | Interest Income | $0.8 | $0.1 | $0.7 | Cash Flow Summary (Years Ended December 31) | Cash Flow Activity | 2022 (Thousands) | 2021 (Thousands) | | :----------------- | :--------------- | :--------------- | | Operating activities | $(12,912) | $(9,382) | | Investing activities | $(40,005) | $(29) | | Financing activities | $8 | $20,778 | | Total | $(52,909) | $11,367 | - As of December 31, 2022, the company had **$18.5 million** in cash and cash equivalents and **$40.0 million** in investments, with working capital of **$55.8 million**[325](index=325&type=chunk) - MediciNova believes it has sufficient working capital to fund operations at least through the end of 2024, with expected operating cash needs of approximately **$21.2 million** for 2023[325](index=325&type=chunk) - A shelf registration statement allows the company to offer up to **$200.0 million** of various securities, including common stock, for future capital raising[327](index=327&type=chunk)[328](index=328&type=chunk) [Item 7A. Quantitative and Qualitative Disclosures About Market Risk](index=68&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) MediciNova has no material quantitative or qualitative disclosures about market risk [Item 8. Financial Statements and Supplementary Data](index=69&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This section presents MediciNova's audited consolidated financial statements for 2022 and 2021, reflecting a **$14.1 million** net loss in 2022, an accumulated deficit of **$407.1 million**, and detailed accounting policies Consolidated Balance Sheet Highlights (as of December 31) | Metric | 2022 (Thousands) | 2021 (Thousands) | | :-------------------------------- | :--------------- | :--------------- | | Cash and cash equivalents | $18,505 | $71,431 | | Investments | $39,982 | $0 | | Total current assets | $58,987 | $72,009 | | Total assets | $74,155 | $87,406 | | Total current liabilities | $3,187 | $2,833 | | Total liabilities | $3,913 | $3,729 | | Total stockholders' equity | $70,242 | $83,677 | Consolidated Statements of Operations and Comprehensive Loss (Years Ended December 31) | Metric | 2022 (Thousands) | 2021 (Thousands) | | :-------------------------------- | :--------------- | :--------------- | | Revenues | $0 | $4,038 | | Research, development and patents | $9,144 | $8,538 | | General and administrative | $5,485 | $5,715 | | Operating loss | $(14,629) | $(10,216) | | Net loss applicable to common stockholders | $(14,069) | $(10,134) | | Basic and diluted net loss per common share | $(0.29) | $(0.21) | Consolidated Statements of Cash Flows (Years Ended December 31) | Cash Flow Activity | 2022 (Thousands) | 2021 (Thousands) | | :----------------- | :--------------- | :--------------- | | Net cash used in operating activities | $(12,912) | $(9,382) | | Net cash used in investing activities | $(40,005) | $(29) | | Net cash provided by financing activities | $8 | $20,778 | | Net change in cash and cash equivalents | $(52,925) | $11,394 | | Cash and cash equivalents, end of year | $18,505 | $71,431 | - The company's accumulated deficit reached **$407.1 million** as of December 31, 2022[351](index=351&type=chunk) - Research, development, and patent expenses increased to **$9.1 million** in 2022 from **$8.5 million** in 2021[354](index=354&type=chunk) [1. Organization and Summary of Significant Accounting Policies](index=77&type=section&id=1.%20Organization%20and%20Summary%20of%20Significant%20Accounting%20Policies) This note outlines MediciNova's organizational structure, business focus on small molecule therapeutics, key accounting policies, and critical accounting estimates, including the impact of COVID-19 - MediciNova operates in a single operating segment: acquisition and development of small molecule therapeutics[366](index=366&type=chunk) - The COVID-19 pandemic caused slower patient enrollment in clinical trials but also created opportunities for new clinical development, such as MN-166 for ARDS and Long COVID[367](index=367&type=chunk)[368](index=368&type=chunk) - Research, development, and patent expenses are expensed as incurred, totaling **$9.1 million** in 2022 and **$8.5 million** in 2021[316](index=316&type=chunk)[379](index=379&type=chunk) - Clinical trial accruals are estimated based on vendor progress, patient enrollment, and site activations, with **$1.5 million** accrued as of December 31, 2022[382](index=382&type=chunk)[346](index=346&type=chunk) - The company uses the Black-Scholes model to estimate the fair value of stock options and recognizes share-based compensation expense over the vesting period[384](index=384&type=chunk) [2. Revenue Recognition](index=81&type=section&id=2.%20Revenue%20Recognition) MediciNova recognizes revenue upon satisfying performance obligations, including **$4.0 million** in 2021 from Genzyme milestone payments, while the MN-221 collaboration was terminated in October 2022 - Revenue is recognized upon the satisfaction of performance obligations, either at a point in time or over time[391](index=391&type=chunk) - In March 2021, MediciNova recognized **$4.0 million** in revenue from two clinical development milestone payments from Genzyme Corporation, related to a gene therapy product[394](index=394&type=chunk) - The collaboration agreement with Kissei Pharmaceutical Co., Ltd. for MN-221 was terminated in October 2022, with no revenue recognized from it since 2013[392](index=392&type=chunk) [3. Fair Value Measurements](index=82&type=section&id=3.%20Fair%20Value%20Measurements) MediciNova measures financial instruments at fair value using a three-tier hierarchy, with cash equivalents at Level 1 and investments at Level 2 as of December 31, 2022 - Fair value is determined using a three-tier hierarchy: Level 1 (quoted prices in active markets), Level 2 (quoted prices for similar items or non-active markets), and Level 3 (unobservable inputs)[396](index=396&type=chunk) Fair Value of Financial Instruments (as of December 31, 2022) | Instrument | Carrying Amount (Dollars) | Fair Value (Dollars) | Valuation Inputs | | :----------------------- | :------------------------ | :------------------- | :--------------- | | Money market funds | $704,882 | $704,882 | Level 1 | | Bank certificates of deposit | $39,982,213 | $39,982,213 | Level 2 | [4. Balance Sheet Details](index=82&type=section&id=4.%20Balance%20Sheet%20Details) This note details MediciNova's property and equipment, net, totaling **$45,269**, and accrued liabilities of **$2,605,308** as of December 31, 2022 Property and Equipment, Net (as of December 31) | Category | 2022 (Dollars) | 2021 (Dollars) | | :-------------------------------- | :------------- | :------------- | | Leasehold improvements | $13,532 | $15,409 | | Furniture and equipment | $121,909 | $124,731 | | Software | $342,628 | $342,628 | | Less accumulated depreciation and amortization | $(432,800) | $(425,203) | | **Property and equipment, net** | **$45,269** | **$57,565** | Accrued Liabilities and Other Current Liabilities (as of December 31) | Category | 2022 (Dollars) | 2021 (Dollars) | | :-------------------------------- | :------------- | :------------- | | Accrued compensation | $920,166 | $636,481 | | Clinical trial accruals | $1,462,354 | $1,076,411 | | Professional services fees | $38,688 | $38,041 | | Other | $184,100 | $547,270 | | **Total accrued liabilities and other current liabilities** | **$2,605,308** | **$2,298,203** | [5. Commitments and Contingencies](index=83&type=section&id=5.%20Commitments%20and%20Contingencies) MediciNova's commitments include **$681,124** in operating lease liabilities and **$26.5 million** in potential milestone payments, with adequate product liability insurance and no material legal proceedings Operating Lease Liabilities (as of December 31, 2022) | Year | Amount (Dollars) | | :--- | :--------------- | | 2023 | $216,154 | | 2024 | $189,170 | | 2025 | $197,585 | | 2026 | $206,483 | | 2027 | $17,269 | | Total minimum payments | $826,661 | | Less imputed interest | $(145,537) | | **Total lease liabilities** | **$681,124** | - Weighted-average remaining lease term is **3.90 years**, with a weighted-average discount rate of **9.8%**[401](index=401&type=chunk) - Future potential milestone payments under license agreements aggregate to **$26.5 million** (**$10 million** for MN-166/MN-001 and **$16.5 million** for other products)[404](index=404&type=chunk) - The company believes it carries reasonably adequate product liability insurance[402](index=402&type=chunk) [6. Stock-based Compensation](index=84&type=section&id=6.%20Stock-based%20Compensation) MediciNova grants stock options under its 2013 Equity Incentive Plan, with **1,949,317** shares available, and reported **$642,522** in stock-based compensation expense for 2022 - As of December 31, 2022, **1,949,317** shares remain available for future grant under the 2013 Equity Incentive Plan[406](index=406&type=chunk) Stock Option Activity (Years Ended December 31) | Metric | 2022 (Shares) | 2021 (Shares) | | :-------------------------------- | :------------ | :------------ | | Outstanding at beginning of year | 7,974,250 | 7,401,387 | | Granted | 591,700 | 1,315,000 | | Exercised | (3,000) | (360,955) | | Cancelled | (577,700) | (381,182) | | Outstanding at end of year | 7,985,250 | 7,974,250 | | Weighted Average Exercise Price (Outstanding) | $5.55 (Dollars) | $5.81 (Dollars) | | Exercisable at end of year | 7,394,381 | N/A | | Weighted Average Exercise Price (Exercisable) | $5.80 (Dollars) | N/A | Stock-Based Compensation Expense (Years Ended December 31) | Category | 2022 (Dollars) | 2021 (Dollars) | | :-------------------------------- | :------------- | :------------- | | Research, development and patents | $237,071 | $640,340 | | General and administrative | $405,451 | $1,077,173 | | **Total stock-based compensation expense** | **$642,522** | **$1,717,513** | - As of December 31, 2022, **$0.2 million** of unamortized compensation cost remains, expected to be recognized over a weighted-average vesting period of **0.20 years**[415](index=415&type=chunk) [7. Stockholders' Equity](index=87&type=section&id=7.%20Stockholders%27%20Equity) MediciNova has an ATM agreement for up to **$75.0 million** in common stock sales, completed a **$20 million** private placement in January 2021, and reserved **9,934,567** shares for future equity issuance - The ATM Agreement allows for sales of common stock up to an aggregate offering price of **$75.0 million**[416](index=416&type=chunk) - No shares were sold under the ATM Agreement in 2022 or 2021[417](index=417&type=chunk) - In January 2021, **3,656,307** shares of common stock were issued in a private placement for approximately **$20 million**[418](index=418&type=chunk) Common Stock Reserved for Future Issuance (as of December 31, 2022) | Category | Number of Shares | | :-------------------------------- | :--------------- | | For exercise of outstanding options | 7,985,250 | | For future equity awards | 1,949,317 | | **Total** | **9,934,567** | [8. Income Taxes](index=88&type=section&id=8.%20Income%20Taxes) MediciNova reported a **$14.1 million** loss before income taxes in 2022, with significant federal and California NOL carryforwards and research tax credits, offset by a full valuation allowance Loss Before Income Taxes (Years Ended December 31) | Location | 2022 (Thousands) | 2021 (Thousands) | | :--------- | :--------------- | :--------------- | | United States | $(14,084) | $(10,146) | | Foreign | $18 | $14 | | **Total** | **$(14,066)** | **$(10,132)** | Deferred Tax Assets and Liabilities (as of December 31) | Category | 2022 (Thousands) | 2021 (Thousands) | | :-------------------------------- | :--------------- | :--------------- | | Net operating loss carryforwards | $70,142 | $68,485 | | Research tax credits | $9,630 | $9,262 | | Total deferred tax assets | $83,667 | $79,999 | | Total deferred tax liabilities | $(1,510) | $(1,539) | | Net deferred tax assets | $82,157 | $78,460 | | Valuation allowance | $(82,359) | $(78,662) | | **Net deferred tax liability** | **$(202)** | **$(202)** | - Federal NOL carryforwards are approximately **$276.6 million** (**$44.3 million** carried forward indefinitely, **$232.3 million** began expiring in 2022)[421](index=421&type=chunk) - California NOL carryforwards are approximately **$182.9 million**, beginning to expire in 2028[421](index=421&type=chunk) - A valuation allowance of **$82.4 million** has been established against net deferred tax assets due to uncertainty of realization, with a **$3.7 million** increase in 2022[420](index=420&type=chunk) [9. Employee Savings Plan](index=90&type=section&id=9.%20Employee%20Savings%20Plan) MediciNova offers an employee savings plan with discretionary company contributions totaling **$75,859** in 2022 and **$72,330** in 2021 Company Contributions to Employee Savings Plan (Years Ended December 31) | Year | Contribution Amount (Dollars) | | :--- | :---------------------------- | | 2022 | $75,859 | | 2021 | $72,330 | [Item 9. Changes in and Disagreements With Accountants on Accounting and Financial Disclosure](index=90&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20With%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) MediciNova reports no changes in or disagreements with its accountants on accounting and financial disclosure matters [Item 9A. Controls and Procedures](index=90&type=section&id=Item%209A.%20Controls%20and%20Procedures) MediciNova's management concluded that disclosure controls and internal control over financial reporting were effective as of December 31, 2022, with no material changes in the fourth quarter - Disclosure controls and procedures were deemed effective as of December 31, 2022[428](index=428&type=chunk) - Internal control over financial reporting was effective as of December 31, 2022, based on the COSO 2013 framework[433](index=433&type=chunk) - No material changes in internal control over financial reporting occurred during the fourth fiscal quarter[430](index=430&type=chunk) [Item 9B. Other Information](index=91&type=section&id=Item%209B.%20Other%20Information) MediciNova terminated its exclusive license agreement with Kissei Pharmaceutical Co., Ltd. for MN-221 (bedoradrine) on October 24, 2022, with no further financial obligations except for API purchase - MediciNova terminated its exclusive license agreement with Kissei Pharmaceutical Co., Ltd. for MN-221 (bedoradrine) on October 24, 2022[434](index=434&type=chunk) - The termination resulted in no further financial obligations to Kissei, except for the purchase of **7.15kg** of active pharmaceutical ingredient for **JPY114,400,000** in December 2022[434](index=434&type=chunk) [Item 9C. Disclosure Regarding Foreign Jurisdiction the Prevent Inspections](index=91&type=section&id=Item%209C.%20Disclosure%20Regarding%20Foreign%20Jurisdiction%20the%20Prevent%20Inspections) MediciNova has no disclosures regarding foreign jurisdictions that prevent inspections PART III [Item 10. Directors, Executive Officers and Corporate Governance](index=92&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) Information on directors, executive officers, and corporate governance is incorporated by reference from the 2023 Annual Meeting of Stockholders Proxy Statement - Information on directors, executive officers, and corporate governance is incorporated by reference from the 2023 Proxy Statement[439](index=439&type=chunk) - MediciNova has adopted a Code of Ethics for Senior Officers and a Code of Business Conduct, available on its website[440](index=440&type=chunk) [Item 11. Executive Compensation](index=92&type=section&id=Item%2011.%20Executive%20Compensation) Executive compensation information is incorporated by reference from MediciNova's 2023 Annual Meeting of Stockholders Proxy Statement - Executive compensation information is incorporated by reference from the 2023 Proxy Statement[441](index=441&type=chunk) [Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=92&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Security ownership information is incorporated by reference from the 2023 Proxy Statement, with **7,985,250** shares reserved for outstanding options and **1,949,317** for future equity awards - Security ownership information is incorporated by reference from the 2023 Proxy Statement[442](index=442&type=chunk) Equity Compensation Plan Information (as of December 31, 2022) | Category | Number of Securities to be Issued Upon Exercise of Outstanding Options and Rights | Weighted Average Exercise Price of Outstanding Options and Rights (Dollars) | Number of Securities Remaining Available for Future Issuance Under Equity Compensation Plans | | :-------------------------------- | :---------------------------------------------------------------- | :-------------------------------------------------------------- | :------------------------------------------------------------------------------------------- | | Equity Compensation Plans Approved by Stockholders | 7,985,250 | $5.55 | 1,949,317 | [Item 13. Certain Relationships and Related Transactions, and Director Independence](index=93&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions%2C%20and%20Director%20Independence) Information on certain relationships, related transactions, and director independence is incorporated by reference from MediciNova's 2023 Annual Meeting of Stockholders Proxy Statement - Information on certain relationships, related transactions, and director independence is incorporated by reference from the 2023 Proxy Statement[446](index=446&type=chunk) [Item 14. Principal Accountant Fees and Services](index=93&type=section&id=Item%2014.%20Principal%20Accountant%20Fees%20and%20Services) Information on principal accountant fees and services is incorporated by reference from MediciNova's 2023 Annual Meeting of Stockholders Proxy Statement - Information on principal accountant fees and services is incorporated by reference from the 2023 Proxy Statement[447](index=447&type=chunk) PART IV [Item 15. Exhibits and Financial Statement Schedules](index=94&type=section&id=Item%2015.%20Exhibits%20and%20Financial%20Statement%20Schedules) This section lists documents filed as part of the 10-K report, including consolidated financial statements and a comprehensive list of exhibits - The report includes the audited consolidated financial statements and the Report of Independent Registered Public Accounting Firm[448](index=448&type=chunk) - A detailed list of exhibits, including organizational documents, license agreements, employment agreements, and sales agreements, is provided[450](index=450&type=chunk)[451](index=451&type=chunk) [SIGNATURES](index=97&type=section&id=SIGNATURES) This section contains the required signatures for the Annual Report on Form 10-K, affirming the filing on February 16, 2023 - The Annual Report on Form 10-K was signed by the President & Chief Executive Officer, Chief Financial Officer, and Board of Directors on February 16, 2023[455](index=455&type=chunk)[458](index=458&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission file number: 001-33185 MEDICINOVA, INC. (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Incorporation or Organization) Delaware 33-0927979 (I.R.S. Employer Identification No.) La Jolla, CA 92037 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) ...

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, 2022 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission file number: 001-33185 MEDICINOVA, INC. (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Incorporation or Organ ...

Drug Development and Clinical Trials - The company is focused on developing MN-166 (ibudilast) for various neurological disorders, including progressive multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS) [18] - In the SPRINT-MS Phase 2b clinical trial, MN-166 (ibudilast) demonstrated a statistically significant 48% reduction in the rate of progression of whole brain atrophy compared to placebo (p=0.04) [21] - The same trial showed a 26% reduction in the risk of confirmed disability progression compared to placebo (hazard ratio = 0.74) [21] - The company has initiated a Phase 2b/3 clinical trial of MN-166 (ibudilast) in ALS, following positive results from earlier trials [24] - The company is conducting ongoing clinical trials for MN-166 (ibudilast) in chemotherapy-induced peripheral neuropathy and degenerative cervical myelopathy [30][31] - Positive preclinical findings for MN-166 (ibudilast) as an adjunctive treatment for glioblastoma were published, with ongoing clinical trials [32] - MN-166 (ibudilast) achieved the primary endpoint of safety and tolerability in a clinical trial for ALS, with a higher rate of responders on the ALSFRS-R total score compared to placebo [56] - The trial evaluating MN-166 (ibudilast) for degenerative cervical myelopathy is funded by a grant from the National Institute for Health Research (NIHR) in the UK [67] - The Phase 2 clinical trial for MN-166 (ibudilast) for ARDS caused by COVID-19 has completed 75% of planned enrollment as of August 2021 [70] - The ongoing clinical trial for MN-166 (ibudilast) for ARDS has received FDA acceptance and is currently in progress [70] - The company is aware of multiple competitors in the clinical development space for both NASH and IPF, indicating a competitive market [143][144] - The clinical development program may not lead to commercial products due to various risks, including failure to demonstrate safety and efficacy in clinical trials [186] Regulatory Designations and Approvals - The FDA has granted Fast Track designation for MN-166 (ibudilast) for both progressive MS and ALS, providing expedited development and review [22][25] - MN-001 (tipelukast) has received Fast Track designation from the FDA for the treatment of nonalcoholic steatohepatitis (NASH) with fibrosis [37] - MN-166 (ibudilast) has received Fast Track designation from the FDA for three indications: progressive MS, ALS, and methamphetamine dependence, facilitating expedited development [46] - The FDA granted Orphan-Drug designation to MN-166 (ibudilast) for ALS, providing seven years of marketing exclusivity if approved [58] - MN-001 (tipelukast) received Orphan-Drug designation from the FDA for Idiopathic Pulmonary Fibrosis (IPF), providing seven years of marketing exclusivity upon approval [38] - The FDA granted orphan-drug designation to MN-166 (ibudilast) as adjunctive therapy to temozolomide for the treatment of glioblastoma [69] - The FDA has granted Fast Track designation to MN-001 (tipelukast) for the treatment of patients with NASH with fibrosis [85] - The company has obtained regulatory authorization to conduct clinical trials for its product development programs, with INDs approved by the FDA [191] Financial Performance and Projections - For the year ended December 31, 2021, the company reported a net loss of $10.1 million, with an accumulated deficit of $393.1 million since inception [176] - As of December 31, 2021, the company had available cash and cash equivalents of $71.4 million and working capital of $69.2 million [176] - The company expects to incur significant operating losses for the foreseeable future as it continues to develop its product candidates MN-166 (ibudilast) and MN-001 (tipelukast) [179] - The company anticipates an increase in research and development expenses in 2022 compared to 2021 due to ongoing development activities [179] - The company may apply for patent term restoration for its currently owned or licensed patents, potentially extending patent life beyond current expiration dates [160] - The company anticipates that out-licensing upfront and milestone payments will be its primary source of revenue prior to commercialization [183] Market Opportunities and Competition - The economic burden of methamphetamine use in the United States is approximately $23.4 billion [60] - There are approximately 1.5 million people aged 12 or older with methamphetamine use disorder in the United States [60] - The prevalence of chemotherapy-induced peripheral neuropathy (CIPN) was 68% in the first month after chemotherapy, 60% at 3 months, and 30% at 6 months or more [66] - Approximately 28.3 million people aged 12 or older in the United States have alcohol use disorder [64] - There are no approved pharmaceuticals for chemotherapy-induced peripheral neuropathy or degenerative cervical myelopathy, highlighting a market opportunity for MN-166 [137][138] - Current treatments for opioid dependence include generic methadone and Suboxone Film, with additional products like Vivitrol and Zubsolv approved [135] - Competitors may develop more effective products, posing a risk to the company's market opportunities and potentially rendering its product candidates obsolete [214] - The company faces competition from pharmaceutical and biotechnology companies with greater resources and advanced development products targeting the same diseases [131] Safety and Efficacy Findings - An interim analysis of a clinical trial for MN-001 (tipelukast) showed a significant reduction in mean serum triglycerides, a primary endpoint [37] - In a Phase 2 clinical trial for MN-001 (tipelukast), there were no worsening IPF events in the treatment group compared to one event in the placebo group, indicating a favorable safety profile [38] - MN-001 (tipelukast) significantly reduced mean serum triglycerides by 135.7 mg/dL, resulting in a 41.3% reduction (p=0.02) in a Phase 2 clinical trial for NASH [85] - MN-001 (tipelukast) demonstrated significant anti-fibrotic effects in preclinical studies, reducing fibrosis area compared to placebo (p<0.01) and improving NAFLD activity score (p<0.01) [83] - MN-166 (ibudilast) significantly decreased basal, daily alcohol craving over the course of a study (p<0.05) [64] - In a study, 33% of subjects with recurrent glioblastoma were progression-free at six months after treatment with MN-166 (ibudilast) in combination with temozolomide [69] Strategic Partnerships and Collaborations - The company has partnered with BARDA to develop MN-166 (ibudilast) as a potential medical countermeasure against chlorine gas-induced lung injury [34] - The company is pursuing strategic partnerships with leading pharmaceutical companies to support the development and commercialization of its product candidates [42] - The company relies on strategic collaborations and third-party manufacturers for the development and commercialization of its product candidates [176] - The company relies on third-party manufacturers for the production of active pharmaceutical ingredients and finished products for clinical trials and future commercial needs [101] Risks and Challenges - The company faces risks related to regulatory compliance, including potential fines and withdrawal of approvals if foreign regulatory requirements are not met [168] - The company currently has no products approved for commercial sale and does not expect to generate any revenues from product sales in the foreseeable future [183] - The company is largely dependent on the success of its MN-166 (ibudilast) and MN-001 (tipelukast) product candidates, which have not completed the clinical development process [185] - Delays in clinical trials could result in increased costs and limit the ability to obtain regulatory approval for product candidates [205] - The inability to secure adequate funding could require the company to delay or scale back clinical or regulatory activities [182] - The company may face significant setbacks in advanced clinical trials, even after earlier trials show promising results [188] - The loss of rights to develop and market product candidates could significantly harm the company's business [209]