Press release
INSMED ANNOUNCES POSITIVE TOPLINE RESULTS FROM PHASE 3 ARISE STUDY OF ARIKAYCE® (AMIKACIN LIPOSOME INHALATION SUSPENSION) IN PATIENTS WITH NTM LUNG DISEASE CAUSED BY MAC
—QOL-B Respiratory Domain Shown to Work Effectively as Patient-Reported Outcome Tool in Patients with MAC Lung Disease; to be Proposed to FDA as Primary
About this update from Insmed Incorporated
[{"type":"text","content":"—QOL-B Respiratory Domain Shown to Work Effectively as Patient-Reported Outcome Tool in Patients with MAC Lung Disease; to be Proposed to FDA as Primary Endpoint in ENCORE with No Modifications—\n—Patients Treated with ARIKAYCE Plus Macrolide-Based Background Regimen Had Meaningfully Larger Improvements in QOL-B Respiratory Score with Strong Trend Toward Significance vs. Macrolide-Based Background Regimen Alone— \n—ARIKAYCE-Treated Patients Had Nominally Statistically Significantly Higher Culture Conversion Rates at Month 7 vs. Comparator (78.8% vs. 47.1%, p=0.0010); Culture Conversion Began Earlier and Was More Likely to Persist Through Month 7 with ARIKAYCE Regimen—\n—No New or Unexpected Safety Signals Observed—\n—Insmed to Explore Accelerating Filing for ARIKAYCE in Newly Infected MAC Lung Disease Patients with Global Regulators on Basis of ARISE Data—\n—Insmed to Host Investor Call at 8:30 a.m. ET on Tuesday, September 5—\nBRIDGEWATER, N.J., Sept. 5, 2023 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today 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.\nThe 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. Based on these results, Insmed plans to propose to the U.S. Food and Drug Administration (FDA) that the QOL-B respiratory domain PRO be the primary endpoint for the ENCORE study without any modifications.\nPatients in ARISE (N=99) were randomized 1:1 to treatment with ARIKAYCE plus macrolide-based background regimen (ARIKAYCE arm) or placebo plus macrolide-based background regimen (comparator arm) 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 t...