Insmed Incorporated, a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, 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 (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.

"The ARISE study represents a clear and unambiguous win for the entire NTM community. We are thrilled that these results not only validate a PRO tool in NTM lung disease, but also show that patients treated with an ARIKAYCE-based regimen felt better versus patients in the comparator arm, as measured by this instrument. Coupled with extraordinary culture conversion outcomes, these findings give us great confidence that our Phase 3 registrational trial, ENCORE, is well-positioned to achieve both statistically and clinically meaningful results, leading to a sizeable increase in the number of patients who could hope to benefit from ARIKAYCE," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed.

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

"I want to thank the many patients, caregivers, and investigators who participated in ARISE and made this impressive outcome possible. We look forward to discussing these excellent results from this well-executed study in the near future with regulators," noted Kevin Mange, M.D., M.S.C.E., Chief Development Officer of Insmed.