According to Insmed, the Phase 3b ENCORE study of once-daily Arikayce amikacin liposome inhalation suspension plus azithromycin and ethambutol in patients with newly diagnosed Mycobacterium avium complex (MAC) lung infection met its primary endpoint. The study demonstrated a significant improvement in change from baseline in respiratory symptom score at month 13 reported by patients receiving Arikayce compared to scores reported by patients receiving placebo. The trial also met culture conversion endpoints.
The ENCORE trial enrolled 425 patients who received either once-daily Arikayce plus multidrug therapy or placebo plus multidrug therapy for 12 months. Patients reported respiratory symptom scores using the Quality of Life – Bronchiectasis (QoL-B) questionnaire. In September 2023, the company announced that the Phase 3 ARISE study had demonstrated the effectiveness of the QOL-B questionnaire for patient-reported outcomes in order to use that questionnaire in the ENCORE study.
The company says that it now plans to submit an sNDA to the FDA and to submit trial data to the Japanese PMDA seeking label changes later this year. Arikayce has been approved in the US for the treatment of NTM lung disease caused by MAC in patients who had limited or no alternative treatment options since 2018. The inhalation suspension has also been approved in Japan for that indication since 2021. In Europe, Arikayce has been approved the treatment of NTM lung infections caused by MAC in certain patients who have limited treatment options since 2020. Arikayce is delivered by PARI‘s Lamira nebulizer.
Insmed Chief Medical Officer Martina Flammer commented, “These results are an exciting win for patients living with MAC lung disease and a powerful validation of Arikayce’s ability to deliver real clinical benefit as part of a multidrug treatment regimen. We are energized by the potential for patients with a new MAC infection to see benefit with Arikayce earlier in their treatment journey and look forward to exploring the expansion of the Arikayce indication in the US and Japan, with the ultimate goal of improving outcomes for an even greater number of patients fighting this difficult disease.”
Read the Insmed press release
