Insmed announced that the first patient has been dosed in a Phase 3b trial of its Arikayce amikacin liposome inhalation suspension in patients with nontuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium complex (MAC). Arikayce is delivered via the PARI Lamira nebulizer system.
The FDA approved Arikayce for the treatment of NTM lung disease caused by MAC in patients without alternative treatment options or with limited alternative options in September 2018. The company said that the new clinical trial program consisting of two Phase 3b trials, ARISE and ENCORE, is intended to support an sNDA for expanded use of Arikayce for MAC lung infections and to meet FDA post-marketing requirements for the initial approval.
The ARISE study is expected to enroll approximately 100 patients with newly diagnosed MAC lung disease and is designed to validate patient-reported outcome (PRO) endpoints that will be used in the ENCORE study. ENCORE is expected to enroll 250 patients who will receive either Arikayce or placebo for 12 months followed by 3 months of assessment after discontinuation of the therapy.
Insmed Chief Medical Officer Martina Flammer said, “We are very pleased to initiate the frontline clinical development program for Arikayce, potentially laying the groundwork for a new standard of care for patients with MAC lung disease. Arikayce has already changed the treatment landscape of refractory MAC lung disease since its FDA approval more than two years ago, and we are excited to build on that foundation with the initiation of these studies. Our hope is that this program will not only provide pivotal data for Arikayce in a frontline setting, but also will validate PRO tools for the assessment of treatment benefit in clinical trials of patients with MAC lung disease going forward.”
In October 2020, Arikayce was approved by the EC for the treatment of NTM lung infections caused by MAC in adult patients without cystic fibrosis who have limited treatment options, and in Japan, a marketing application for Arikayce for the treatment of NTM lung disease caused by MAC in patients who have not responded sufficiently to other treatment is under review.
Read the Insmed press release.
