MediciNova's Pivotal 2026: Key Data Looms for ALS and Metabolic Drugs

LA JOLLA, CA – January 06, 2026 – As the new year begins, biopharmaceutical company MediciNova is standing at a critical juncture, with 2026 poised to be what its CEO calls a "pivotal year." The company announced it has successfully completed patient enrollment for three late-stage clinical trials for its lead drug candidates, MN-166 (ibudilast) and MN-001 (tipelukast). The news sets the stage for a series of high-stakes data readouts that could bring new hope to patients with devastating diseases and reshape the company's future.

In a letter to shareholders, CEO Yuichi Iwaki highlighted the firm's progress despite global economic headwinds, culminating in the completion of enrollment for studies targeting amyotrophic lateral sclerosis (ALS), chemotherapy-induced peripheral neuropathy (CIPN), and a cluster of metabolic disorders. With these milestones achieved, the focus now shifts entirely to the upcoming clinical results, which will determine if years of research and development can translate into effective treatments.

The High-Stakes Race for an ALS Breakthrough

At the forefront of MediciNova's pipeline is MN-166, or ibudilast, in the Phase 2b/3 COMBAT-ALS trial. The company confirmed that enrollment of 234 patients with ALS was completed in September 2025. This randomized, double-blind, placebo-controlled study is evaluating the drug's ability to slow the relentless progression of ALS, a fatal neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to muscle paralysis. The current standard of care offers only modest benefits, and the recent failure of another company's confirmatory trial has underscored the urgent need for new, effective therapies.

The COMBAT-ALS trial, identified as NCT04057898 on ClinicalTrials.gov, will assess the drug's efficacy over a 12-month treatment period. The primary endpoint is the Combined Assessment of Function and Survival (CAFS), a comprehensive measure designed to capture both functional decline and survival benefit. Top-line results are anticipated by the end of 2026. Positive data would pave the way for regulatory submissions to the FDA, for which MediciNova says preparations are already underway.

Adding to the momentum, MediciNova initiated a large-scale Expanded Access Program (EAP) for MN-166 in March 2025, supported by a substantial $22 million research grant from the National Institutes of Health (NIH). As of mid-December 2025, the program had enrolled 87 patients across 12 U.S. sites, providing access to the investigational therapy for patients outside of the primary clinical trial. This NIH-funded initiative not only provides crucial access but also signals a high level of interest from the medical research community in the potential of ibudilast.

A Diversified Pipeline Tackles Multiple Ailments

While the ALS program captures significant attention, MediciNova's strategy extends beyond a single indication. The company is leveraging its two lead compounds across a range of inflammatory, fibrotic, and neurodegenerative conditions, creating multiple opportunities for success.

Ibudilast is also being evaluated in the OXTOX study, a Phase 2b investigator-initiated trial for chemotherapy-induced peripheral neuropathy (CIPN) in patients with metastatic colon cancer. CIPN is a painful and debilitating side effect of certain chemotherapies, like oxaliplatin, that can force doctors to reduce or stop life-saving cancer treatment. The trial, which completed enrollment of 100 patients in Australia in December 2025, aims to see if ibudilast can mitigate this neurotoxicity. While the timing for results is not yet fixed, a positive outcome could open a significant new market for the drug.

Meanwhile, MediciNova's second compound, MN-001 (tipelukast), is targeting a nexus of widespread metabolic diseases. In November 2025, the company completed enrollment for a Phase 2 trial investigating tipelukast in patients with hypertriglyceridemia, non-alcoholic fatty liver disease (NAFLD), and Type 2 diabetes. This cluster of conditions affects millions and represents a massive unmet medical need, particularly for NAFLD and its more severe form, NASH, for which there are currently no widely approved pharmacological treatments.

This Phase 2 trial, NCT05464784, is designed to measure changes in liver fat content and fasting serum triglycerides over 24 weeks. With top-line data from this study expected in the summer of 2026, it will be the first of MediciNova's key readouts this year, offering an early look at the potential of its metabolic program.

The Science of Small Molecules with Big Potential

The broad therapeutic potential of MediciNova's pipeline is rooted in the unique mechanisms of its small molecule drugs. Unlike highly targeted biologics, these compounds influence multiple biological pathways involved in disease.

MN-166 (ibudilast) is an orally administered drug that acts as a phosphodiesterase type-4 (PDE4) and macrophage migration inhibitory factor (MIF) inhibitor. This allows it to suppress pro-inflammatory cytokines and attenuate the activation of glial cells—immune cells in the brain and spinal cord that are implicated in the progression of neurodegenerative diseases. This anti-neuroinflammatory action is the scientific rationale for its use in ALS. Its potential has been previously demonstrated in a Phase 2 trial for progressive multiple sclerosis, where results published in the New England Journal of Medicine showed it significantly reduced the rate of brain atrophy, a key marker of disease progression.

MN-001 (tipelukast) also boasts a multi-faceted mechanism, acting as a leukotriene (LT) receptor antagonist, a phosphodiesterase (PDE) inhibitor, and a 5-lipoxygenase (5-LO) inhibitor. These actions give it both anti-inflammatory and anti-fibrotic properties. The rationale for its use in metabolic disease was further strengthened by a recent academic collaboration, which revealed that its primary metabolite, MN-002, significantly enhances cholesterol efflux from macrophages. This suggests the drug could help clear harmful lipid buildups, a key process in both atherosclerosis and fatty liver disease.

A Defining Year for a Clinical-Stage Biotech

For a clinical-stage company like MediciNova, which reported a net loss of $9.20 million in the first three quarters of 2025, the upcoming data readouts are a make-or-break moment. The company's stock (NASDAQ: MNOV) has seen modest gains in recent months, reflecting cautious optimism as these clinical milestones were achieved. However, the true test will come with the data.

Positive results from any of the three trials could trigger significant value inflection, attracting potential partners, facilitating regulatory filings, and validating the company's long-term scientific strategy. In his letter, Dr. Iwaki expressed his conviction for the year ahead, stating, "2026 will be a pivotal year for MediciNova—a year when we can finally reach the goals we have been striving toward." With top-line data for its metabolic program expected in the summer and the much-anticipated ALS results due by year-end, the investment and patient communities are watching closely to see if those goals will indeed be met.