Widetrial, an integrated service and technology platform, will support an expanded access program (EAP) to provide the investigational therapy ibudilast (MN-166) to amyotrophic lateral sclerosis (ALS) patients who are not eligible for clinical trials. The program is funded by a $22 million federal grant awarded to the Mayo Clinic in Florida.
The EAP aims to enroll approximately 200 ALS patients at three Mayo Clinic locations—Minnesota, Florida, and Arizona—as well as other participating U.S. institutions. This initiative seeks to broaden the diversity of locations and patient backgrounds included in the program by increasing physician involvement.
Expanding Access to Ibudilast
Jess Rabourn, CEO of Widetrial, emphasized the company's commitment to providing access to potential therapies for all patients, regardless of their eligibility for traditional research trials. EAPs, also known as compassionate use programs, offer investigational therapies to patients with serious and life-threatening illnesses who cannot participate in clinical trials.
In addition to offering potentially life-saving treatment to a larger patient population, EAPs provide researchers with additional opportunities to gather clinical data on experimental therapies. The MN-166 program is funded under the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), signed into law in 2021. MediciNova is developing MN-166 for ALS treatment and will supply the medication for the EAP study.
Mechanism of Action and Prior Clinical Data
Ibudilast's active ingredient is designed to reduce immune cell activity in the brain, mitigating inflammatory damage believed to drive neurodegeneration in ALS. This mechanism is expected to enhance nerve cell survival and slow disease progression. In a Phase 2 trial (NCT02238626), MN-166, when added to riluzole, was associated with a higher proportion of patients experiencing stabilization or improvements in disease severity and quality of life measures compared to riluzole alone. The trial also suggested a tendency to extend survival.
The EAP study will include ALS patients ineligible for ongoing MN-166 clinical trials. The primary objective is to assess the treatment's effects on disease progression and levels of neurofilament light chain (NfL), a biomarker of nerve cell damage, over a six-month treatment period. Reductions in NfL levels are generally considered indicative of a therapy's potential to slow disease progression.
A unique aspect of this study is that patients will have the ability to monitor their NfL levels throughout the trial. Blood samples will also be stored to establish an ALS biobank for future research. MediciNova is also sponsoring a Phase 2b/3 clinical trial (COMBAT-ALS, NCT04057898) to evaluate MN-166 against a placebo in approximately 230 ALS patients.
