MediciNova Supports NIH-Funded Trial for MN166 in ALS

MediciNova, Inc., a biopharmaceutical company listed on the NASDAQ Global Market and the Tokyo Stock Exchange, has announced a significant financial boost from the National Institutes of Health (NIH) for an important clinical trial. The NIH's Neurological Disorders and Stroke (NINDS) division has granted $22 million to support an Expanded Access Protocol (EAP) aimed at evaluating the efficacy of MediciNova's investigational drug MN-166 (ibudilast) in the treatment of Amyotrophic Lateral Sclerosis (ALS). This initiative is in collaboration with an academic group.

The funding is part of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), which was enacted to provide patients with serious, life-threatening diseases access to investigational drugs that have not yet received FDA approval. The EAP trial is intended for ALS patients who are not eligible for the ongoing COMBAT-ALS trial, thus expanding treatment opportunities for individuals in advanced stages of the disease.

This EAP will assess the impact of MN-166 on neurofilament light, a biomarker associated with neuron damage, along with other clinical data in 200 ALS patients. Dr. Yuichi Iwaki, MediciNova’s President and CEO, emphasized the importance of this collaboration, noting that the NIH/NINDS grant and academic partnership mark considerable progress in the battle against ALS. Dr. Iwaki acknowledged the role of participants, their families, healthcare providers, and staff in making this trial possible, and expressed gratitude towards the NIH and NINDS for their support through ACT for ALS.

MN-166 (ibudilast) is an oral small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, including macrophage migration inhibitory factor (MIF). It is currently in late-stage clinical development for several neurodegenerative diseases, including ALS, progressive multiple sclerosis (MS), and degenerative cervical myelopathy (DCM). Additionally, MN-166 is being explored for its potential in treating glioblastoma, Long COVID, chemotherapy-induced peripheral neuropathy (CIPN), and substance use disorder. It has also been evaluated for patients at risk of developing acute respiratory distress syndrome (ARDS).

MediciNova has received Orphan Drug Designation for MN-166 in ALS from both the U.S. FDA and the European Medicines Agency (EMA). The drug has also been granted Fast Track Designation by the FDA for ALS treatment and holds Orphan Disease Designation for glioblastoma treatment.

MediciNova, Inc. is a clinical-stage biopharmaceutical company that is developing a diverse pipeline of novel small molecule therapies targeting inflammatory, fibrotic, and neurodegenerative diseases. The company's lead asset, MN-166 (ibudilast), is currently in Phase 3 trials for ALS and DCM, and is Phase 3-ready for progressive MS. MN-166 is also being tested in Phase 2 trials for Long COVID and substance dependence. Another compound in MediciNova’s pipeline, MN-001 (tipelukast), has undergone a Phase 2 trial for idiopathic pulmonary fibrosis (IPF) and is currently being evaluated in a second Phase 2 trial for non-alcoholic fatty liver disease (NAFLD).

MediciNova has a solid history of securing investigator-sponsored clinical trials funded by government grants, demonstrating its capability to advance its drug development programs through collaborative efforts and substantial financial backing.