The first patient has been enrolled in a National Institutes of Health (NIH)-funded Expanded Access Program (EAP) trial evaluating MN-166 (ibudilast) in patients with Amyotrophic Lateral Sclerosis (ALS), MediciNova announced today. This milestone initiates a critical study designed to provide access to the investigational therapy for approximately 200 ALS patients who are ineligible for the company's ongoing Phase 2/3 COMBAT-ALS trial.
The EAP trial, funded by the NIH's National Institute of Neurological Disorders and Stroke (NINDS) through the Act for ALS, will assess both safety and efficacy of MN-166 in patients with more advanced stages of the disease. This represents a significant opportunity for patients who would otherwise be unable to access the experimental treatment.
"We are honored to support this EAP trial, which will provide MN-166 to more individuals in advanced stages of ALS who are not eligible to our current Phase 2/3 COMBAT-ALS trial," said Dr. Yuichi Iwaki, President and CEO of MediciNova. "We extend our sincere gratitude to the NIH and NINDS for their support through the Act for ALS. Additionally, we deeply appreciate the participants and their families, as well as the healthcare providers and staff at the sites, whose efforts will make this EAP possible."
MN-166: Mechanism and Development Status
MN-166 (ibudilast) is an orally administered small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, including macrophage migration inhibitory factor (MIF). The drug has shown promise in neurodegenerative conditions and is currently in late-stage clinical development for multiple indications.
For ALS specifically, MN-166 has received significant regulatory support, including Orphan Drug Designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA has also granted Fast Track Designation for MN-166 in ALS treatment, acknowledging the urgent need for effective therapies for this devastating condition.
Expanded Access and the ALS Treatment Landscape
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness, paralysis, and eventually respiratory failure. The disease typically progresses rapidly, with an average survival of 2-5 years after diagnosis. Currently, there are limited treatment options available that significantly alter disease progression.
Expanded Access Programs, sometimes called compassionate use programs, provide a pathway for patients with serious or life-threatening conditions to access investigational treatments outside of clinical trials when no comparable alternatives exist. The NIH funding for this particular EAP reflects the implementation of the Accelerating Access to Critical Therapies for ALS Act (Act for ALS), which was signed into law in December 2021.
MediciNova's Broader Pipeline
Beyond ALS, MediciNova is developing MN-166 for multiple other indications, including progressive multiple sclerosis, degenerative cervical myelopathy, glioblastoma, Long COVID, chemotherapy-induced peripheral neuropathy, and substance use disorder.
The company's pipeline also includes MN-001 (tipelukast), which is being evaluated for idiopathic pulmonary fibrosis and non-alcoholic fatty liver disease. MediciNova has established a track record of securing investigator-sponsored clinical trials funded through government grants, as evidenced by this NIH-supported EAP.
Clinical Trial Access and Patient Impact
For ALS patients and their families, access to promising investigational therapies can provide hope in the face of a devastating diagnosis. The EAP trial represents an important opportunity for patients who might otherwise have no options for experimental treatment due to advanced disease progression or other factors that would exclude them from the more stringent eligibility criteria of the Phase 2/3 COMBAT-ALS trial.
The initiation of this EAP trial also highlights the importance of the Act for ALS in facilitating access to potential treatments. The legislation aims to accelerate the development of therapies for ALS and improve access to investigational drugs for patients who are not eligible for clinical trials.
As the trial progresses, it will provide valuable data on MN-166's safety and efficacy in a broader ALS patient population, potentially informing future development and regulatory decisions.
