MediciNova has presented positive interim data from an ongoing clinical trial to evaluate the efficacy and safety of MN-166 (ibudilast) in the treatment of amyotrophic lateral sclerosis (ALS). The study, entitled “Adaptive Design Single Center Phosphodiesterase Type 4 (PDE4) Inhibitor – Ibudilast (MN-166) Phase 1b/2a Clinical Trial [NCT02238626] for Amyotrophic Lateral Sclerosis (ALS) Patients  Not Requiring Non-Invasive Ventilation (no-NIV) up to 5 years and  Requiring Non-Invasive Ventilation (NIV) up to 10 years from Disease Onset,” was presented by principal investigator Dr. Benjamin Rix Brooks at the 26th International Symposium on ALS/MND (amyotrophic lateral sclerosis/motor neurone disease).
The clinical trial (ClinicalTrials.gov Identifier: NCT02238626), which is recruiting patients, is a randomized and placebo-control study to evaluate the efficacy and safety of MN-166 (ibudilast) in the treatment of early and advanced stage ALS patients. MN-166, licensed by MediciNova from Kyorin Pharmaceutical for potential utility in relapse-remitting multiple sclerosis (RRMS), is a small molecule phosphodiesterase (PDE) -4 and -10 inhibitor and a macrophage migration inhibitory factor (MIF), whose anti-neuroinflammatory and neuroprotective properties have been proven in previous preclinical and clinical trials. The trial aims to enroll 60 ALS patients without NIV (Non-Invasive Ventilator) and an additional 60 ALS patients with NIV.
The presentation, referring to results from a total of 36 subjects, covered several efficacy points addressed by the trial, such as functional activity (ALSFRS-R), respiratory function, and muscle strength. The first results included the ALSFRS-R score from baseline to month six, a measure of functional impairment, which was below rates observed in other ALS trials. Moreover, non-invasive ventilation (NIV) usage was lower in clinical trial participants when compared to a sample from ALS Multidisciplinary Clinic patients. In the non-NIV group, slow vital capacity (SVC), a measure of respiratory function, declined by 1.50% predicted/month, a value also below rates previously observed. No significant adverse effects differences were found between the drug and placebo groups. The subjects will now proceed to the six-month, open-label extension part of the study.
Dr Yuichi Iwaki, MD, president and chief executive officer of MediciNova, said in a press release, “We are very pleased with the encouraging interim data, particularly the slow vital capacity and NIV utilization data which may indicate that MN-166 has potential to improve respiratory function in ALS patients. If the final results of the study confirm it, MN-166 would potentially offer a substantial clinical benefit in the treatment of ALS as most patients die from respiratory failure.”