Masitinib Phase 3 Trial Data Show Slowed Amyotrophic Lateral Sclerosis Progression, AB Science Says

Masitinib Phase 3 Trial Data Show Slowed Amyotrophic Lateral Sclerosis Progression, AB Science Says

French biotech company AB Science presented the results of the Phase 3 clinical trial for its lead compound masitinib, which showed effectiveness in improving the functioning of patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

The randomized, double-blind clinical trial (NCT02588677) compared the efficacy and safety of combining masitinib with riluzole (Rilutek or Teglutik), also an ALS drug, with a combination of a placebo and riluzole. A total of 394 ALS patients were randomly assigned to receive either a treatment combination of 4.5 mg/kg/day of masitinib and riluzole, a combination of 3 mg/kg/day of masitinib and riluzole, or a combination of a placebo and riluzole for up to 48 weeks.

The study’s primary goal was to observe changes in participating patients’ revised ALS Functional Rating Scale (ALSFRS-R), used to monitor patients’ functional change over time. A secondary goal was to study progression free survival (PFS), which trial investigators defined as either a patient’s death or a deterioration of more than nine points in a patient’s ALSFRS-R score.

The results showed that masitinib administered at 4.5 mg/kg/day along with riluzole in ALS patients generated a therapeutic benefit when compared to placebo treatment. This masitinib dosage slowed patients’ loss of function, improved their PFS, and reduced the decline in quality of life.

Patients who received 3 mg/kg/day of masitinib also improved their ALSFRS, PFS (through delayed disease progression), and quality of life scores compared to patients receiving a placebo. The study found masitinib, which is also being tested for use in a wide range of other conditions, to be safe without causing major adverse effects.

Study results were presented at the 2017 European Network for the Cure of ALS annual meeting in Ljubljana, Slovenia, on May 18-20.

At the meeting, Jesús S. Mora, international coordinator of the AB10015 masitinib trial and an ALS neurologist, delivered a discussion titled, “Masitinib as an add-on therapy to riluzole is beneficial in the treatment of amyotrophic lateral sclerosis (ALS) with acceptable tolerability: Results from a randomized controlled phase 3 trial.”

“These final data confirm findings from the study’s interim analysis, showing that masitinib is effective in slowing down ALS disease progression,” Mora said, according to a news release. “This benefit is evident from the observed effect on a clinical assessment of daily functioning (Var. ALSFRS-R) and a significant improvement in terms of clinically relevant measures of quality-of-life and respiratory function.”

At the same meeting, Professor Luis Barbeito, head of the Neurodegeneration Laboratory at Institut Pasteur in Montevideo, Uruguay, presented, “Masitinib for the treatment of amyotrophic lateral sclerosis (ALS): Preclinical overview and future clinical development.

“These Phase 3 clinical findings are strongly supported by equally compelling preclinical data, showing masitinib to generate a neuroprotective effect through targeting aberrant microglial cells and regulating neuroinflammation,” Barbeito said, according to the release.

“These data prove that masitinib is capable of slowing progression in ALS and effectively opens a new paradigm in the treatment of neurodegenerative diseases, added Professor Olivier Hermine, president of AB Science’s Scientific Committee.

Masitinib is an orally administered therapy that targets mast cells and macrophages, which are important cells for immunity, by inhibiting a limited number of kinases. Because of its activity on mast cells and the subsequent inhibition of the inflammation process, masitinib can be effective against symptoms of inflammatory and CNS-associated diseases.

 The European Medicines Agency (EMA) accepted a conditional marketing authorization application for masitinib to treat ALS, in October 2016. EMA’s decision to register masitinib for ALS is expected to be made public in coming months. The U.S. Food and Drug Administration granted masitinib orphan drug designation.

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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  1. Karen says:

    Since this has been approved compassionate use how can United States get it?
    I also see that it will be approved for general use in 2017 does anyone know when?

    • Peter says:

      I take Masitinib ( as Masivet vet drug) for GIST since Jan 2017 without side effects.
      No progression so far. I can not comment on its effectiveness on ALS, all I can say that it works better than Sutent for me and that as a vet drug its safe. Maybe its worth trying.

        • Peter says:

          I took Sutent for 12 months but had to stop due to heavy side effects. I then took Masitinib for 9 months 8,5mg/kg. Initialy my tumor got smaller by 20%, then stayed the same size. My last CT, 2 weeks ago, showed progression by 10% so I increased my dose to12mg/kg of body weight. So far no side effects.

  2. Michelle says:

    This is very encouraging. Will there be a New a Drug Application submitted to the FDA based on these trial results and if so when? Since FDA gave it orphan drug status, will that speed the application process?

  3. Tulsi says:

    Since the results are Phase 3 Clinical trial, does that mean the drug is ready for approval? When will it be available for treatment for those suffering with ALS? Where one can buy it if one does not live in Europe?

    • Tim Bossie says:

      This drug is not yet ready for approval. We will be keeping a watch over the news and provide updates as we get them.

    • don ng says:

      nomally FDA will see all the phases result then decide whther they are safe for the public but i think so far this drugs masitinib have been proven to be good so the question here is FDA when are they going to approve this drugs and also i wonder whether did AB sience has submitted to FDA for approval that all tks

  4. Dina says:

    Hi there my dad is diagnosed with als and I would like to know more about the masitinib. Is it available in and can I buy it ?

    • Tim Bossie says:

      This medication is still in trials and not available for prescription yet. We will be keeping a close watch over it and the progress of the trials to approval in order to make sure that people are informed when this happens.

  5. Robin Fox says:

    From the positive trial results thus far, I hope there are plans to expedite the manufacturing and distribution of this drug.

  6. Bernard Graziani says:

    Do you know by when the trial will end? And if fully approved, how long will it take for being prescripteur?

  7. Aaron Knobloch says:

    What stages of ALS were looked at for trial? What about us PALs with “seniority”? i’ve had ALS for ±8 years. i’m basically a quadriplegic, on a NIV ventilator 23/7, receive all of my nutrition via a RIG (a type of PEG), my ALSFRS is 8. i can still speak, but my speech is beginning to show signs of deterioration. so i’m wondering when people state that the trials indicate that the drugs slow progression, would that be true for PALs in advanced stages?

  8. Rita Diane Cooper says:

    Karen….you are right …….big Pharma is blocking it and there is to much money to lose.
    BIG PHARMA……..I hope and pray you are reading these comments and if so…..HELP! Wait until one of your family members are afflicted with this horrific disease.

  9. teri jackson says:

    I agree, last month I was diagnoised with ALS AND i HAVE 10 nd 14 yr. old daughters and its just us. I work in a drs. office which is no help including myself no one knows anything about ALS except your gonna die. I hear about trials but have no idea how to join? I live in Fresno,CA. which has nothing to offer for ALS IF ANYONE HAS ANY INFO TO HELP PLEASE LET ME KNOW. [email protected] Thanks

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