The U.S. Food and Drug Administration approved MediciNova’s plans for a pivotal Phase 2b/3 clinical trial that will explore the potential of ibudilast (MN-166) in patients with amyotrophic lateral sclerosis (ALS).
Pending the success of the trial, the company hopes the collected clinical data will support the submission of a new drug application and the approval of ibudilast for the treatment of ALS.
“We are very pleased to have successfully completed the FDA review period and look forward to initiating patient enrollment shortly,” Yuichi Iwaki, MD, PhD, president and CEO of MediciNova, said in a press release.
The randomized, placebo-controlled study is expected to enroll approximately 150 participants across several sites in the U.S.
Eligible patients must have the disease for no more than 18 months with just mild disability, determined by a score of at least 35 on the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) at screening.
Patients taking Radicava (edaravone) or Nuedexta (dextromethorphan/quinidine) may also be eligible to participate in the study if they discontinue these treatments three months prior to entering the trial.
Participants must take Rilutek (riluzole) for at least 30 days before starting a regimen of either 100 mg per day of ibudilast or a placebo for a period of nine months.
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Researchers will evaluate the ability of ibudilast to prevent ALS progression and patients’ functional disability compared with placebo by determining changes in ALSFRS-R score at the end of the treatment period.
In addition to this primary objective, they will also assess changes in the patients’ muscle strength, quality of life, and respiratory function as well as the safety and tolerability profile of ibudilast.
“While we acknowledge this was an important milestone, and we have a clear path forward, this moment is of great importance to the ALS community,” Iwaki said. “We give them our sincerest appreciation for their patience and optimism.”
“Patients will be eligible to receive study drug from MediciNova after completing the required treatment period in this trial,” he added.
Ibudilast’s benefits in ALS have been explored in a previous Phase 2 trial (NCT02238626) in combination with Rilutek.
Results showed that the ibudilast add-on treatment could significantly improve functional activity and quality of life compared with Rilutek alone. In addition, the combo therapy could also delay disease worsening and improve survival rates of ALS patients.
MediciNova is currently exploring the impact of ibudilast administered alone to ALS patients in a Phase 1/2 trial (NCT02714036). This trial has already completed patient enrollment and is still ongoing.
Ibudilast is a small molecule that inhibits the activity of the PDE4 and PDE10 enzymes and the MIF protein. This reduces the activity of immune cells in the brain and increases the production of neurotrophic factors that support the survival and growth of nerve cells that control movement.
The FDA has granted fast track status and orphan drug designation to ibudilast for the treatment of ALS. These designations are intended to expedite and support its clinical development, review, and potential approval.