The trial (NCT02714036) has enrolled 35 ALS patients who will be treated with 100 mg daily doses (50 mg twice a day) of MN-166. The treatment is expected to reduce the activation of a specific type of brain cells, the glial cells, which are key players in some neurological diseases.
MN-166 is an oral small molecule capable of inhibiting the action of the proteins phosphodiesterase-4 and -10 and macrophage migration inhibitory factor, leading to the suppression of inflammation, activation of neurons’ function and reduction of activated glial cells.
The compound is being tested in other neurological disorders, including multiple sclerosis (MS), ALS and substance abuse/addiction. A previous Phase 2 trial (NCT02238626) showed the potential of MN-166 to delay the progression of ALS and improving survival of patients.
This ongoing multi-center, open-label Phase 1/2 study will evaluate MN-166 in patients with ALS, by tracking an ALS biomarker, the [11C]-PBR28.
The PBR28 protein is overproduced in activated glial cells and previous studies demonstrated it can be efficiently tracked using positron emission tomography (PET) imaging and correlated to the progression of the disease. Clinical outcomes of the patients also will be evaluated.
The 35 ALS patients included in the study must have been been diagnosed with ALS, either sporadic or familial, according to established criteria. Also, they must not have taken Rilutek (riluzole) for at least 30 days, or be on a stable dose of the medicine for at least 30 days, prior to screening.
Patients will be treated for 36 weeks. At the end of this period, researchers will measure the effect of MN-166 on reducing glial cell activation via [11C]-PBR28 uptake in specific parts of the brain, the motor cortices and brain stem.
As a primary goal, the team will investigate the affect of MN-166 on several markers of neurological inflammation measured by blood biomarkers.
Secondary goals of the study involve safety and tolerability profiles and clinical outcomes, including ALSFRS-R (ALS functional rating scale), muscle strength measured by HHD (hand-held dynamometry) and SVC (slow vital capacity).
“We are very pleased that this study is now fully enrolled. We look forward to providing further updates as we expect to receive data from this study next year,” Yuichi Iwaki, PhD, president and CEO of MediciNova, said in a press release.
MN-166 is approved for the treatment of post-stroke complications and bronchial asthma in Japan and Korea since 1989.
The study is led by Nazem Atassi, MD, at Massachusetts General Hospital’s Neurological Clinical Research Institute and Harvard Medical School.