FDA Approves Pivotal Phase 2b/3 Trial for Ibudilast in ALS Patients

FDA Approves Pivotal Phase 2b/3 Trial for Ibudilast in ALS Patients

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.

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.

18 comments

  1. David Doane says:

    This is great news, I look forward to possible participation as I have been discussing with my Neurologist the possibility of purchasing Ibudilast overseas… this would be a much better option.

  2. Dave Reckonin says:

    Progress….thank Science Almighty. Great news.
    Fast Track is still pretty much slow-track in relative terms but there’s more hope here than there is looking for something spiritual to influence things.

  3. Bill DeMay says:

    If phase 2 clinical trials showed safety and effectiveness in slowing progression, why another 9 month test? To me it’s inhumane. Seems unlikely small chance of harm is less than big certainty of continued disease progression.
    These 18 month disease symptom exclusions make no sense to me either. I’m very slow progression but saw symptoms 3-4 years ago. Are we going to have to wait for more tests to see if works for us?

  4. Alex says:

    Its possible to order this drug from Japan via internet – it costs around 100$ with delivery. Just google “buy Ibudilast” or “buy ketas” (its second name). You will find a couple of japan shops which sell it all over the world. Its up to you whether to wait for some official approval or get it this way and try yourself
    Not an advertizing. I am in the same boat unforthunatly searching for the cure.

      • Alex says:

        I didn’t try this drug before. But I made an order from a Japan internet shop once I read this article on April 18 and they already sent it to me via EMS (I have a tracking number which shows delivery progress) so I hope to get it in 14 days or so. I cannot say for 100% yet, but it looks like a valid purchase according to all details I see by now (I am an IT guy so it’s not that easy to make me pay to some shady people via internet – I see the difference between fake and real internet shops)
        I am in Russia but it should work for US and almost any other country in the same way.

        • Theresa says:

          Hi can you share the website or seller details for this drug. My father is an ALS patient and it takes quite long before it is approved for used for the local ministry here. We just want to slow down his deterioration so that he at least live a little longer

  5. I too have had ALS 2 years slow progression . I would be so thankful and overjoyed if this could halt progression . My family would be so happy to have me survive. Can you make available very soon.

  6. P. Colon says:

    I have ALS for almost 3 years now, it would be nice if they could make it available to us & let us decide. In any event it is cruel to let us wait.

  7. I have bulbar ALS was diagnosed 4 months back and since then I have been in Riluzol and some tonics .Am I a suitable candidate to add this medication ? and since it is already there to treat bronchial asthma , why should not we starte now or order it on line ?

  8. Paul DiCarlo says:

    All very good points above. I was diagnosed in May 2018 and have seen a significant decline in the past 12 months. Everyday is about survival and not living. A year ago I could shower and dress myself with ease now it is a real challenge and my wife has to assist. These trials seem to take forever and 99% of them I am excluded because of my SVC%. I really don’t think I have another year to wait. Why can’t we try these drugs outside of a trial? I would sign a waiver. I am particularly interested in the drug Levosimendan (ODM-109) which early trials were favorable to improve lung function. Wish I could purchase this outside of the phase 3 trial going on now.

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