MediciNova Announces Update and Basic Characteristic Randomized Patients’ of Phase 2/3 Clinical Trial of MN-166 (Ibudilast) in ALS (COMBAT-ALS Clinical Trial) Presented at the 36th International Symposium on ALS/MND

Rhea-AI Summary

MediciNova (NASDAQ:MNOV) reported an update from the COMBAT-ALS Phase 2b/3 trial of MN-166 (ibudilast) presented at the 36th International Symposium on ALS/MND (Dec 5–7, 2025).

Key facts: 234 participants randomized, enrollment completed Sept 2025. Baseline metrics: mean age 60.6, mean ALSFRS-R 40.6, mean disease duration 12.5 months. Sex split: 36.8% female, 63.2% male. Racial mix: Caucasian 90.2%, Asian 5.1%.

The company noted COVID-19–related enrollment challenges, is offering continued access via FDA individual expanded access, and cited FDA Orphan Drug and Fast Track designations and European orphan designation. Top-line data are anticipated by end of 2026.

Positive

• 234 patients randomized with enrollment completed Sept 2025
• Top-line data expected by end of 2026
• Regulatory designations: FDA Orphan Drug and Fast Track; European orphan designation
• Expanded access via FDA Individual Patient program for continued MN-166 treatment

Negative

• Slow enrollment and site disruptions due to COVID-19 extended recruitment timelines
• No efficacy top-line results yet; outcome pending until end of 2026

Key Figures

Randomized participants 234 patients Phase 2b/3 COMBAT-ALS trial enrollment completed September 2025

Female participants 86 patients (36.8%) Baseline characteristics in COMBAT-ALS trial

Male participants 148 patients (63.2%) Baseline characteristics in COMBAT-ALS trial

Mean age 60.6 years Age at screening in COMBAT-ALS trial

Mean ALSFRS-R score 40.6 ALSFRS-R at screening in COMBAT-ALS trial

Disease duration 12.5 months Mean duration from first symptom in COMBAT-ALS trial

Randomized patients 234 patients Previously disclosed enrollment target for COMBAT-ALS met

Top-line data timing End of 2026 Expected COMBAT-ALS top-line data readout

Market Reality Check

$1.56 Last Close

Volume Volume 98,606 vs 20-day average 141,105 (relative volume 0.7x), indicating subdued trading ahead of this update. normal

Technical Shares at $1.56 are trading above the 200-day MA of $1.38, with the stock 34.18% below its 52-week high and 38.05% above its 52-week low.

Peers on Argus 1 Up

Biotech peers showed mixed moves: APLT -5.83%, ABVC -4.74%, ANL -18.55%, while QNTM +4.04% and ALXO +3.65% (and +7.75% on momentum scan) gained. No clear sector-wide pattern tied to this ALS update.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Dec 01	Mechanistic publication	Positive	-2.8%	Peer-reviewed MN-002 data and Phase 2 MN-001 enrollment completion update.
Nov 18	Leadership/strategy	Positive	+2.1%	Appointment of Dr. Breder as clinical and regulatory advisor.
Nov 06	Industry award	Positive	-7.3%	Biotech award recognizing MN-166 development and COMBAT-ALS progress.
Nov 04	Clinical enrollment	Positive	-5.3%	Completion of enrollment in MN-001-NATG-202 Phase 2 trial.
Oct 30	Scientific publication	Positive	+0.8%	Publication on MN-002’s cholesterol efflux mechanism in atherosclerosis.

Pattern Detected

Recent news with generally positive scientific or clinical content has often seen muted or negative next-day price moves, indicating a tendency toward selling or limited enthusiasm on good news.

Recent Company History

Over the past few months, MediciNova has highlighted multiple R&D milestones. On Jul 24, it reported strong enrollment progress in ALS and metabolic trials, supported by a $22M NIH grant. Through Aug–Sep 2025, several updates on the COMBAT-ALS and MN-001 programs detailed enrollment completion and trial design, with mixed short-term price reactions. More recently, publications and awards underscored mechanistic and translational progress. Today’s COMBAT-ALS baseline-characteristics update fits this pattern of incremental de-risking without new efficacy data.

Market Pulse Summary

This announcement details completion of randomization and baseline characteristics for 234 ALS patients in the Phase 2b/3 COMBAT-ALS trial of MN-166, with top-line data expected by end of 2026. It reinforces that demographics and disease profiles align with prior ALS studies, supporting generalizability. Historically, MediciNova has reported steady clinical progress across MN-166 and MN-001. Investors may monitor future updates on efficacy endpoints, regulatory interactions, and cash runway disclosures in periodic filings to assess program risk and funding needs.

Key Terms

amyotrophic lateral sclerosis medical

"MN-166 (ibudilast) in Amyotrophic Lateral Sclerosis (ALS), known as the COMBAT-ALS study."

A progressive disease in which nerve cells that control voluntary muscles gradually fail, leading to loss of movement, speech and eventually breathing — like an electrical wiring system in the body slowly shorting out. It matters to investors because there are few effective treatments, so clinical trial results, regulatory approvals, new therapies or diagnostics can rapidly change patient care, market opportunity and company valuations.

orphan drug designation regulatory

"MN-166 was granted Orphan Drug Designation and Fast Track Designation from FDA"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

fast track designation regulatory

"Orphan Drug Designation and Fast Track Designation from FDA as well as Orphan"

A "fast track designation" is a process that speeds up the review and approval of a product or project, allowing it to reach the market or be completed more quickly than usual. For investors, it can signal that a product may become available sooner, potentially leading to earlier revenue or benefits, and indicating a priority status that might influence company performance and market opportunities.

expanded access program regulatory

"through the FDA’s Individual Patient Expanded Access Program, and we remain committed"

A program that allows patients with serious or life‑threatening conditions to receive an experimental drug or therapy before it is fully approved by regulators, when they cannot join clinical trials. Investors care because expanded access can change a treatment’s market perception, create early real‑world safety or demand signals, and affect regulatory timelines and potential revenue — like a pre‑order system that also reveals how the product performs outside controlled testing.

open-label extension medical

"6-month open-label extension. The primary endpoint focuses on the Combined"

An open-label extension is a continuation of a clinical trial where all participants and researchers know which treatment is being given, often after an initial blinded phase. It allows further study of a drug's long-term safety and effectiveness. For investors, it can indicate ongoing interest and confidence in a product's potential, influencing perceptions of its future value.

randomized, double-blind, placebo-controlled medical

"Phase 2 MN-001-NATG-202 randomized, double-blind, placebo-controlled trial in"

A "randomized, double-blind, placebo-controlled" process is a method used to test the effectiveness of a new treatment or intervention. Participants are randomly assigned to different groups, with one receiving the real treatment and the other a fake version, called a placebo. Neither the participants nor the researchers know who is receiving which, which helps ensure unbiased results. For investors, this rigorous approach increases confidence that the findings are accurate and not influenced by guesswork or bias.

AI-generated analysis. Not financial advice.

LA JOLLA, Calif., Dec. 08, 2025 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company listed on the NASDAQ Global Market (NASDAQ: MNOV) and the Standard Market of the Tokyo Stock Exchange (Code: 4875), today announced an update and the patients’ basic characteristics data from its Phase 2b/3 clinical trial of MN-166 (ibudilast) in Amyotrophic Lateral Sclerosis (ALS), known as the COMBAT-ALS study. These results were presented at the 36th International Symposium on ALS/MND, held December 5–7, 2025, in San Diego, California, USA.

The poster presentation, titled “COMBAT-ALS Phase 2b/3 Trial of MN-166 (Ibudilast) in ALS: Trial Update and Basic Characteristics” (Reference # CTL-21), highlighted the following:

• Overview of the COMBAT-ALS Clinical Trial: Purpose, scientific rationale for MN-166 in ALS treatment, study design, and key eligibility criteria.
• Study Update: A total of 234 participants were randomized, and enrollment was completed in September 2025.
• Baseline Characteristics:
  • Total randomized patients: 234 (Female: 86 [36.8%], Male: 148 [63.2%])
  • Mean age at screening: 60.6 years
  • Racial distribution: Caucasian (90.2%), Asian (5.1%), African American (1.3%), Native Hawaiian or Other Pacific Islander (0.4%), American Indian or Alaskan Native (0.4%), Other (2.6%)
  • ALS onset type: Upper limb (46.2%), Lower limb (32.5%), Bulbar (20.9%), Unknown (0.4%)
  • Mean ALSFRS-R score at screening: 40.6
  • Mean disease duration from first symptom: 12.5 months
  • These demographics and clinical profiles are consistent with other Phase 2 and Phase 3 ALS trials, supporting the generalizability of the study findings.

Dr. Kazuko Matsuda, Chief Medical Officer of MediciNova, commented:
“We would like to express our heartfelt gratitude to the patients, caregivers, and families who made this study possible. Achieving our randomization goal was not without challenges—particularly during the COVID-19 pandemic, which brought unprecedented disruptions to clinical research. Slow enrollment, site restrictions, and uncertainty tested our resilience, but thanks to the dedication of investigators and study teams, we overcame these obstacles together. We anticipate top-line data by the end of 2026 and remain hopeful that MN-166 will represent a meaningful therapeutic advance for patients living with ALS.”

Dr. Yuichi Iwaki, President and Chief Executive Officer of MediciNova, added:
“We have been actively supporting patients who wish to continue MN-166 treatment following the COMBAT-ALS study through the FDA’s Individual Patient Expanded Access Program, and we remain committed to providing this support going forward. MN-166 was granted Orphan Drug Designation and Fast Track Designation from FDA as well as Orphan Designation by the European Commission for the treatment of ALS. Our clinical development team is working closely with experienced regulatory experts to prepare for the next steps in advancing this program.”

About MN-166 (ibudilast)

MN-166 (ibudilast) is a small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, including macrophage migration inhibitory factor (MIF). It is in late-stage clinical development for the treatment of neurodegenerative diseases such as ALS (amyotrophic lateral sclerosis), progressive MS (multiple sclerosis), and DCM (degenerative cervical myelopathy); and is also in development for glioblastoma, Long COVID, CIPN (chemotherapy-induced peripheral neuropathy), and substance use disorder. In addition, MN-166 (ibudilast) was evaluated in patients that are at risk for developing acute respiratory distress syndrome (ARDS). For the ALS indication, MediciNova has Orphan Drug Designation& Fast Track Status from the US FDA and Orphan Drug Designation from the EMA.

About MediciNova

MediciNova, Inc. is a clinical-stage biopharmaceutical company developing a broad late-stage pipeline of novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases. Based on two compounds, MN-166 (ibudilast) and MN-001 (tipelukast), with multiple mechanisms of action and strong safety profiles, MediciNova has numerous programs in clinical development. MediciNova’s lead asset, MN-166 (ibudilast), is currently in Phase 3 for amyotrophic lateral sclerosis (ALS) and degenerative cervical myelopathy (DCM) and is Phase 3-ready for progressive multiple sclerosis (MS). MN-001 (tipelukast) is in a Phase 2 trial treating hypertriglycedemia in type 2 diabetic patients. MediciNova has a strong track record of securing investigator-sponsored clinical trials funded through government grants.

Forward-Looking Statements
Statements in this press release that are not historical in nature constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, without limitation, statements regarding the future development and efficacy of MN-166 and MN-001. These forward-looking statements may be preceded by, followed by, or otherwise include the words "believes," "expects," "anticipates," "intends," "estimates," "projects," "can," "could," "may," "will," "would," “considering,” “planning” or similar expressions. These forward-looking statements involve a number of risks and uncertainties that may cause actual results or events to differ materially from those expressed or implied by such forward-looking statements. Factors that may cause actual results or events to differ materially from those expressed or implied by these forward-looking statements include, but are not limited to, risks of obtaining future partner or grant funding for development of MN-166 and MN-001, and risks of raising sufficient capital when needed to fund MediciNova's operations and contribution to clinical development, risks and uncertainties inherent in clinical trials, including the potential cost, expected timing and risks associated with clinical trials designed to meet FDA guidance and the viability of further development considering these factors, product development and commercialization risks, the uncertainty of whether the results of clinical trials will be predictive of results in later stages of product development, the risk of delays or failure to obtain or maintain regulatory approval, risks associated with the reliance on third parties to sponsor and fund clinical trials, risks regarding intellectual property rights in product candidates and the ability to defend and enforce such intellectual property rights, the risk of failure of the third parties upon whom MediciNova relies to conduct its clinical trials and manufacture its product candidates to perform as expected, the risk of increased cost and delays due to delays in the commencement, enrollment, completion or analysis of clinical trials or significant issues regarding the adequacy of clinical trial designs or the execution of clinical trials, and the timing of expected filings with the regulatory authorities, MediciNova's collaborations with third parties, the availability of funds to complete product development plans and MediciNova's ability to obtain third party funding for programs and raise sufficient capital when needed, and the other risks and uncertainties described in MediciNova's filings with the Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2024 and its subsequent periodic reports on Form 10-Q and current reports on Form 8-K. Undue reliance should not be placed on these forward-looking statements, which speak only as of the date hereof. MediciNova disclaims any intent or obligation to revise or update these forward-looking statements.

INVESTOR CONTACT:

David H. Crean, Ph.D.
Chief Business Officer
MediciNova, Inc
info@medicinova.com

FAQ

How many participants were randomized in the COMBAT-ALS Phase 2b/3 trial (MNOV) and when was enrollment completed?

A total of 234 participants were randomized and enrollment was completed in September 2025.

What baseline measures did MediciNova report for MN-166 (ibudilast) in COMBAT-ALS (MNOV)?

Baseline: mean age 60.6, mean ALSFRS-R score 40.6, mean disease duration 12.5 months, 36.8% female.

When does MediciNova expect to release COMBAT-ALS top-line results for MN-166 (NASDAQ:MNOV)?

The company anticipates reporting top-line data by the end of 2026.

What regulatory designations does MN-166 have for ALS (MNOV)?

MN-166 has received FDA Orphan Drug and Fast Track designations and an European Commission orphan designation for ALS.

Is MediciNova offering continued access to MN-166 after COMBAT-ALS (MNOV)?

Yes. MediciNova supports continued MN-166 treatment through the FDA Individual Patient Expanded Access Program.