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First-in-class tyrosine kinase inhibitor shows promise in severe asthma

Presented by
Prof. Pascal Chanez, Aix-Marseille Université, France
ERS 2020
A recent phase 3 study suggests that masitinib could be a novel treatment for patients with severe asthma as it demonstrated a positive benefit/risk ratio regardless of initial eosinophils level [1].

The multicentre, prospective, randomised, placebo-controlled phase 3 trial assessed masitinib 6 mg/kg/day in adult patients with severe asthma and insufficient disease control under oral corticosteroids (OCS) at >5 mg/day. The small molecule masitinib is a selective tyrosine kinase inhibitor that targets mast cell activity and exerts inhibition on platelet-derived growth factor receptor signalling associated with airway remodelling.

The study included 355 participants for the primary analysis and 404 participants for the safety evaluation. They were randomised 2:1 to receive masitinib or placebo. All participants had a baseline FEV1 value ≥35% and <80% and at least 2 exacerbations in the previous year as well as signs of uncontrolled asthma 2 weeks before the study. The primary endpoint was defined as reduction of annualised severe asthma exacerbation rate (SAER) that included a deterioration demanding dose escalation of OCS for at least 3 days or hospitalisation. After a placebo run-in interval of 2 weeks, participants received treatment over 36 weeks, which could be followed by a blinded extension until at least week 96. This led to an average exposure time of about 60 weeks.

Compared with placebo, the rate of SAER was significantly reduced by 35% in patients on masitinib (P=0.0103; rate ratio 0.65; 95% CI 0.47-0.90). The result was supported by a sensitivity analysis of the full analysis set population. A pre-specified subgroup analysis of patients with an eosinophil level of ≥150 cells/µL showed a decrease of 38% (P=0.0156; rate ratio 0.62; 95% CI 0.42-0.91). In terms of safety, at least 1 adverse event occurred in 83.4% of the masitinib and 82% of the placebo groups. Corresponding rates for severe and serious adverse events were 48.0% vs 45.9% and 17.7% vs 16.5%, respectively. In conclusion, the investigators saw a possible new treatment option with masitinib for patients with severe asthma who are not eligible for biologic treatment.


    1. Chanez P, et al. Masitinib significantly decreases the rate of asthma exacerbations in patients with severe asthma uncontrolled by oral corticosteroids: A phase 3 multicenter study. LBA B93, ATS 2020 Virtual, 5-10 Aug.

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