MediciNova, Inc. recently announced that the U.S. Food and Drug Administration (FDA) has approved the protocol of a novel clinical trial in patients with amyotrophic lateral sclerosis (ALS) to assess MN-166 (ibudilast) on a biomarker of ALS.
The Phase 2a trial, titled “A Single-Center, Open-Label Biomarker Study to Evaluate MN-166 (ibudilast) in Subjects with Amyotrophic Lateral Sclerosis (ALS),” is a cooperative effort between MediciNova and Drs. Haruhiko Banno and Nazem Atassi, principal investigators at Harvard Medical School and the Massachusetts General Hospital’s Neurological Clinical Research Institute, to study MN-166’s effect on reducing brain microglial activation in about 15 ALS patients. The trial is expected to begin soon.
“We are very pleased that this important regulatory step is now completed as we can continue clinical evaluation of MN-166 in the treatment of ALS. The objective of this trial is to evaluate MN-166 by measuring its effects using a novel imaging technology that will complement the conventional assays typically utilized in ALS trials,” Yuichi Iwaki, MD, PhD, president and CEO of MediciNova, said in a press release.
The ALS Biomarker Trial is an open-label, proof-of-mechanism, clinical trial of MN-166 (ibudilast), with participants being treated with ibudilast 50 mg twice a day for 36 weeks. Its primary endpoint is to assess the effects of ibudilast in the reduction of brain microglial activation as measured by [  C]-PBR28-PET, a biomarker. The trial’s secondary endpoints are its tolerability, safety, and clinical outcomes — ALS functional rating scale [ALSFRS-R], slow vital capacity [SVC], and muscle strength measured by HHD, or hand-held dynamometry — over the nine months.
MN-166 (ibudilast) is a first-in-class, orally bioavailable, small molecule phosphodiesterase (PDE) 4 and 10 inhibitor and a macrophage migration inhibitory factor that suppresses pro-inflammatory cytokines IL-1ß, TNF-a, and IL-6, and upregulates the anti-inflammatory cytokine IL-10. The drug has also been shown in clinical studies to be a toll-like receptor 4 (TLR4) functional antagonist that may contribute to the attenuation of neuroinflammation. Because of its possible neuroprotective potential, researchers want to test its therapeutic capacity in neurodegenerative diseases like ALS.