A PHASE IIIb trial is assessing whether amikacin liposome inhalation suspension can improve clinical and microbiologic outcomes in adults with newly diagnosed Mycobacterium avium complex lung disease, a chronic infection associated with high symptom burden, lung function decline, impaired quality of life, and substantial mortality.
Presented at ATS 2026 in Orlando, Florida, the ENCORE study was designed to evaluate the efficacy and safety of amikacin liposome inhalation suspension, also known as ALIS, when added to azithromycin and ethambutol in adults with a new occurrence of Mycobacterium avium complex lung infection who had not received antibiotics for their current infection.
Mycobacterium avium Complex Lung Disease Trial Design
ENCORE is a global, randomized, double-blind, multicenter, placebo-controlled Phase IIIb trial. Adults with noncavitary Mycobacterium avium complex lung disease were eligible if they had respiratory symptoms attributable to current infection, an average Quality of Life-Bronchiectasis Respiratory Domain score of 85 or lower, and two positive sputum cultures, including one before screening and one at screening.
Participants were randomized 1:1 to receive once-daily ALIS 590 mg or empty liposome control, each given with azithromycin 250 mg and ethambutol 15 mg/kg. Treatment continued for 12 months, followed by a 3-month off-treatment follow-up period. Randomization was stratified by region and history of MAC lung infection, including initial or subsequent occurrence.
Patient-Reported Outcomes and Culture Conversion
The primary endpoint is change from baseline to Month 13 in patient-reported respiratory symptom score, derived from the Quality of Life-Bronchiectasis Respiratory Domain. This focus places patient-reported respiratory symptoms at the center of treatment evaluation in Mycobacterium avium complex lung disease, a field where many studies have historically emphasized microbiologic endpoints.
Secondary endpoints include sputum culture conversion by Month 13, durability of culture conversion at Month 15, culture conversion by Month 12, culture conversion by Month 6, and change from baseline to Month 13 in fatigue symptoms measured using PROMIS Short Form Fatigue 7a.
Safety Data Expected From ENCORE
Treatment-emergent safety data will be summarized from the first dose through 28 days after discontinuation of ALIS or empty liposome control. According to the abstract, ENCORE is the first Phase III, double-blind, randomized controlled trial in Mycobacterium avium complex lung disease to use a fully validated patient-reported outcome tool to evaluate clinical treatment efficacy alongside microbiologic outcomes.
The last patient’s final visit has been completed, and topline data were expected in the March/April timeframe. Full primary, principal secondary, and safety endpoints are expected to clarify the role of ALIS in newly diagnosed Mycobacterium avium complex lung disease.
Reference
Charles L. Daley. Amikacin Liposome Inhalation Suspension for Newly Diagnosed Mycobacterium Avium Complex Lung Disease: Efficacy and Safety from a Phase 3b Study (ENCORE). ATS 2026, Orlando, Florida, USA, 2026.
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