MediciNova, Inc. presented an update on its Phase 2/3 clinical trial, COMBAT-ALS, evaluating MN-166 (ibudilast) in patients with Amyotrophic Lateral Sclerosis (ALS) at the 2024 Annual NEALS Meeting. Björn Oskarsson, MD, FAAN, the lead Principal Investigator, delivered the presentation, providing insights into the trial's design, objectives, and current progress.
COMBAT-ALS Trial Design and Objectives
The COMBAT-ALS trial is a Phase 2/3 study designed to assess the efficacy and safety of MN-166 (ibudilast) in ALS patients. Dr. Oskarsson's presentation covered the scientific rationale behind using ibudilast, a small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines like macrophage migration inhibitory factor (MIF). The study aims to determine if ibudilast can slow the progression of ALS, a devastating neurodegenerative disease affecting motor neurons.
The presentation also detailed the major inclusion criteria for the trial and provided an update on patient enrollment as of October 18, 2024. Specific details on the number of patients enrolled and the timeline for completion were part of the update.
NIH Funding and Expanded Access Protocol
In addition to the COMBAT-ALS trial, Dr. Oskarsson discussed a new NIH-funded Expanded Access Protocol (EAP) clinical trial for ALS patients. This EAP is supported by a $22 million grant from the National Institutes of Health (NIH) - Neurological Disorders and Stroke (NINDS) and is facilitated by the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS).
The EAP will allow individuals with ALS who do not meet the inclusion criteria for the COMBAT-ALS trial to receive treatment with MN-166 (ibudilast). The trial will evaluate neurofilament light, a biomarker for neuron damage, and clinical data in 200 ALS patients treated with MN-166.
Ibudilast's Mechanism of Action and Potential
MN-166 (ibudilast) is being developed for various neurodegenerative diseases, including progressive multiple sclerosis and degenerative cervical myelopathy, as well as for glioblastoma, Long COVID, chemotherapy-induced peripheral neuropathy (CIPN), and substance use disorder. MediciNova holds Orphan Drug Designation for MN-166 (ibudilast) in ALS by the U.S. FDA and EU EMA, and it has received Fast Track Designation by the FDA for the treatment of ALS.
Yuichi Iwaki, MediciNova’s President and CEO, stated, “The NIH/NINDS grant, and the academic collaboration are significant steps forward in the endeavor to find a solution to this devastating disease. We are honored to support this EAP trial, which will provide MN-166 (ibudilast) to more individuals in advanced stages of ALS who are not eligible to current Phase 2/3 COMBAT-ALS trial.”
