Insmed announced that the Phase 3 ARISE study of Arikayce amikacin liposome inhalation suspension demonstrated the effectiveness of the QOL-B questionnaire for patient-reported outcomes. The ARISE study of Arikayce in patients with nontuberculous mycobacterial (NTM) lung infection caused by Mycobacterium avium complex (MAC) was initiated in December 2020 with the goal of determining patient-reported endpoints for a second Phase 3 trial called ENCORE. The company says it will now seek FDA permission to use QOL-B as the primary endpoint for ENCORE.
The ARISE study enrolled 99 patients who received either nebulized Arikayce or placebo over 6 months. Both groups were also treated with macrolides. According to Insmed, the study not only validated the QOL-B as an endpoint, it also demonstrated that patients receiving Arikayce “achieved nominally statistically significantly higher culture conversion” one month following the end of treatment, as well as faster and more persistent culture conversion in the Arikayce arm.
The ARISE and ENCORE trials are part of a program designed meet FDA post-marketing requirements for Arikayce and to support expanded use of the inhalation suspension for MAC lung infections. In September 2018, the FDA approved Arikayce for the treatment of NTM lung disease caused by MAC in patients who had limited or no alternative treatment options. In October 2020, Arikayce was approved by the EC for the treatment of NTM lung infections caused by MAC in certain patients who have limited treatment options.
Insmed now says that it will “explore with global regulators accelerating the filing for approval of Arikayce in newly infected patients with MAC lung disease” while enrolling patients in the ENCORE study. Topline data from that study are expected in 2025.
Insmed Chief Medical Officer Martina Flammer commented, “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 Arikcayce.”
Read the Insmed press release.
