Masitinib

Overview:

  • Also known as: AB-1010
  • Pharmaceutical Company:AB Sciences
  • Route and Dose of Administration: Oral (4.5mg/kg/day or 4.5-6 mg/kg/day)
  • Type: Immunomodulator
  • Emerging Treatment for: Primary Progressive MS or Relapse-Free Secondary Progressive MS
  • Status: Phase III clinical trial
How it Works

Masitinib inhibits the function of proteins called tyrosine kinase that target mast cells and macrophages which are involved in inflammatory and immune responses. Blocking tyrosine kinases results in inhibition of the inflammatory process.

Research and Results

Phase II Trial

In a small phase II, multicenter, randomized study, 35 people with primary progressive MS and relapse-free secondary progressive MS took either masitinib (3 to 6 mg/kg/day) or placebo for at least 12 months. The primary outcome measure was a change in the Multiple Sclerosis Functional Composite (MSFC) which measures changes in leg function/walking, arm/hand function, and cognitive function and showed improvement for the masitinib-treated group compared with those in the placebo group. While the results were not statistically significant, they showed enough therapeutic benefit to support a larger clinical trial.

Phase III Trial

A phase III, multicenter, randomized, double-blind clinical trial will examine the safety and efficacy of masitinib (4.5mg/kg/day or 4.5mg/kg/day progressing to 6 mg/kg/day) compared with placebo in 656 individuals with primary progressive MS and relapse-free secondary progressive MS over 96 weeks. The primary outcome measures the effectiveness of masitinib through changes in Expanded Disability Status Scale (EDSS) which quantifies the level of disability. Other outcome measures include the MSFC and Multiple Sclerosis Quality of Life-54 (MSQOL-54) which assesses quality of life. In January 2018, the results of an interim analysis revealed no safety concerns and predicted a high success rate with the current sample size. Results of the study are expected mid-2019.

Adverse Effects Reported

The adverse effects reported from the phase II clinical trial were loss of strength and energy, rash, nausea, edema (swelling), and diarrhea. The frequency of adverse effects was similar between the masitinib and placebo group, however, participants taking masitinib experienced a higher incidence of severe and serious events.

References

Vermersch P, et al. Masitinib treatment in patients with progressive multiple sclerosis: a randomized pilot study. BMC Neurol . 2012;12:36.

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