Insmed announces results from its Phase 3 ARISE study of ARIKAYCE

Insmed announced positive topline results from its Phase 3 ARISE study of ARIKAYCE in patients with newly diagnosed or recurrent nontuberculous mycobacterial, NTM, lung infection caused by Mycobacterium avium complex, MAC, who had not started antibiotics. The study met its primary objective of demonstrating that the Quality of Life – Bronchiectasis respiratory domain works effectively as a patient-reported outcome instrument in patients with MAC lung disease. Based on these results, Insmed plans to propose to the U.S. Food and Drug Administration that the QOL-B respiratory domain PRO be the primary endpoint for the ENCORE study without any modifications. Patients in ARISE were randomized 1:1 to treatment with ARIKAYCE plus macrolide-based background regimen or placebo plus macrolide-based background regimen for six months, followed by one month off treatment. ARIKAYCE-treated patients performed better than those in the comparator arm as measured by the QOL-B instrument, with 43.8% of patients achieving an improvement in QOL-B respiratory score above the estimated meaningful within-subject score difference of 14.8, compared with 33.3% of patients in the comparator arm. While the study was not powered to show a statistically significant difference between treatment arms, a strong trend toward significance was observed for improvement from baseline at Month 7. Patients in the ARIKAYCE arm also achieved nominally statistically significantly higher culture conversion rates at Month 7 versus patients in the comparator arm, and culture conversion was faster and more likely to persist through Month 7 for the ARIKAYCE arm. Based on the results of ARISE, Insmed plans to explore with global regulators accelerating the filing for approval of ARIKAYCE in newly infected patients with MAC lung disease. In parallel, the Company continues to enroll patients in ENCORE, which will use the PRO tool that has been validated in ARISE, with 250 patients expected to be enrolled by the end of 2023. Enrollment in ENCORE is expected to continue into 2024 to ensure a high degree of statistical powering and Insmed anticipates reporting topline data from ENCORE in 2025.