ARIKAYCE was studied in the Phase 3 CONVERT trial1,2

Safety and efficacy were evaluated as part of a combination therapy in adult patients with refractory MAC lung disease1,3

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The efficacy and safety of ARIKAYCE were evaluated in an ongoing, open-label, randomized (2:1), multicenter, global, Phase 3 trial of 336 adult patients (ITT population) with refractory Mycobacterium avium complex (MAC) lung disease as confirmed by at least 2 sputum culture results.1-3

Patients were considered to have refractory MAC lung disease if they did not achieve negative sputum cultures after a minimum duration of 6 consecutive months of background regimen therapy that was either ongoing or stopped no more than 12 months before the screening visit.1

CONVERT clinical trial1,3*

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CONVERT clinical trial

*The CONVERT trial is referred to as "Trial 1" in the ARIKAYCE full Prescribing Information.

The CONVERT trial is ongoing.2

A converter was defined as a patient who had 3 consecutive monthly MAC-negative sputum cultures at any time within the first 6 months of the study. All converters without relapse or recurrence remained in the study for 12 months, starting from the first negative culture that defines culture conversion. “Relapse or recurrence” was defined as having MAC-positive sputum cultures in liquid broth media (agar negative) for 3 or more consecutive months, or having at least 1 MAC-positive sputum culture on solid media (agar positive) after achieving culture conversion.3

§The confirmatory endpoint (sustained sputum culture conversion at 3 months after cessation of therapy) has not yet been established and will be evaluated along with all other study endpoints at the conclusion of the trial.3

Culture conversion defined icon

Culture conversion defined

Culture conversion was defined as 3 consecutive monthly negative sputum cultures. In order to meet the primary endpoint at Month 6 of the study, patients would need to achieve their first negative sputum culture by Month 4.1,2

Background regimen defined

The background regimen was composed of an antimycobacterial regimen of at least 2 antibiotics based on the 2007 ATS/IDSA Statement or respective local guidelines. These drugs may have included, but were not limited to, azithromycin, clarithromycin, clofazimine, ethambutol, ethionamide, rifabutin, and rifampicin.3

At baseline, the background regimen included a macrolide (91.9%), a rifamycin (85.7%), or ethambutol (80.3%). Overall, 54.9% of patients were receiving a triple background regimen of a macrolide, a rifamycin, and ethambutol. An additional 11.3% of patients were on an existing background regimen of a macrolide, a rifamycin, ethambutol, plus a fourth antibiotic.1,2

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Key inclusion criteria included adult patients who2:

  • Had MAC lung disease documented by at least 2 positive cultures (at least 1 positive culture obtained within 6 months prior to screening and 1 positive culture at screening with cultures obtained at least 1 month apart)
  • Did not respond to active therapy for ≥6 months. Treatment was either ongoing or had been stopped no more than 12 months before screening
  • Had evidence of MAC lung disease with underlying lung disease, such as nodular bronchiectasis and/or fibrocavitary disease by chest radiography or chest CT
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Key exclusion criteria included patients who2:

  • Had cystic fibrosis
  • Had MAC lung disease that was resistant to amikacin (as identified by minimum inhibitory concentration susceptibility >64 μg/mL)

CONVERT trial baseline characteristics (ITT population)2,3

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table-CONVERT-trial-baseline

IIBased on safety population (n=223).3

Based on safety population (N=335).3

#COPD was derived from the medical history data.3

CONVERT trial demographic characteristics (ITT population)2,3

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table-CONVERT-trial-demographic

The CONVERT trial evaluated patients who were refractory to treatment. Patients were considered to have refractory MAC lung disease if they did not achieve negative sputum cultures after a minimum duration of 6 consecutive months of background regimen therapy that was either ongoing or stopped no more than 12 months before the screening visit.1

ATS=American Thoracic Society; CT=computed tomography; IDSA=Infectious Diseases Society of America; ITT=intent to treat; IV=intravenous; SD=standard deviation.