Insmed Initiates ARIKAYCE Trial for MAC Lung Disease

Insmed announced the first patient was dosed in the frontline clinical trial program of ARIKAYCE® (amikacin liposome inhalation suspension) in patients with nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC). The program consists of ARISE, an interventional study designed to validate cross-sectional and longitudinal characteristics of patient-reported outcome (PRO) tools in MAC lung disease, and ENCORE, a pivotal trial designed to establish, using the PRO tools validated in the ARISE trial, the clinical benefits and evaluate the safety of ARIKAYCE in patients with newly diagnosed MAC lung disease.

ARIKAYCE was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in September of 2018 for the treatment of MAC lung disease as part of a combination antibacterial drug regimen for adult patients who have limited or no alternative treatment options. It is the first and only therapy approved in the U.S. for the treatment of MAC lung disease. The frontline clinical trial program is intended to fulfill the FDA's post-marketing requirement to allow for full approval of ARIKAYCE in the U.S. and to support a supplemental new drug application (sNDA) for the use of ARIKAYCE as a frontline treatment for patients with MAC lung disease.

"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," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. "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."

ARISE is a randomized, double-blind, placebo-controlled Phase 3b study in adult patients with newly diagnosed MAC lung disease that aims to generate evidence demonstrating the domain specification, reliability, validity, and responsiveness of PRO-based scores. Patients will be randomized 1:1 to receive ARIKAYCE plus background regimen or placebo plus background regimen once daily for six months. Patients will then discontinue all study treatments and remain in the trial for one month for the continued assessment of PRO endpoints. The study is expected to enroll approximately 100 patients.

ENCORE is a randomized, double-blind, placebo-controlled phase 3b study to evaluate the efficacy and safety of an ARIKAYCE-based regimen in patients with newly diagnosed MAC lung disease. Patients will be randomized 1:1 to receive ARIKAYCE plus background regimen or placebo plus background regimen once daily for 12 months. Patients will then discontinue all study treatments and remain in the trial for three months for the assessment of durability of culture conversion. The primary endpoint is change from Baseline to Month 13 in respiratory symptom score. The key secondary endpoint is the proportion of subjects achieving durable culture conversion at Month 15. ENCORE is expected to enroll approximately 250 patients.

Approximately 150 sites are expected to be initiated globally for the ARISE and ENCORE clinical trials.