Insmed Incorporated (NASDAQ:INSM) shares are trading higher after the company released 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 (QOL-B) respiratory domain works effectively as a patient-reported outcome (PRO) instrument in patients with MAC lung disease.
Insmed plans to propose to the FDA that the QOL-B respiratory domain PRO be the primary endpoint for the ENCORE study without any modifications.
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 (12.24 vs. 7.76, p=0.1073).
Patients in the ARIKAYCE arm also achieved nominally statistically significantly higher culture conversion rates at Month 7 versus patients in the comparator arm (78.8% vs. 47.1%, p=0.0010), and culture conversion was faster and more likely to persist through Month 7 for the ARIKAYCE arm.
In parallel, the company continues to enroll patients in ENCORE, which will use the PRO tool validated in ARISE, with 250 patients expected to be enrolled by the end of 2023.
Enrollment in ENCORE will continue into 2024, with topline data expected in 2025.
Price Action: INSM shares are up 16.80% at $26.44 on the last check Tuesday.
