Arikayce amikacin: Phase II data

The double-blind, North American Phase II TARGET NTM (TR02-112) trial in 89 evaluable patients with treatment-resistant NTM lung infection showed that once-daily 560 mg Arikayce administered with the eFlow Nebulizer System from Pari GmbH (Starnberg, Germany) plus standard of care (SOC) missed the primary endpoint of reducing mycobacterial density as measured by a 7-point scale from baseline to day 84 vs. placebo plus SOC (p=0.148). Arikayce plus SOC did meet the secondary endpoint of a greater proportion of patients with culture conversion to negative by day 84 vs. placebo plus SOC (25% vs. 7%, p=0.01).

Patients receiving Arikayce also experienced a greater number of treatment-emergent adverse events vs. patients receiving placebo (240 vs. 140 events). The most common side effect was laryngeal irritation. Following the double-blind portion of the trial, all patients had the option to receive Arikayce in an 84-day, open-label extension. Additional data will be presented at the American Thoracic Society meeting in San Diego in May. Insmed plans to meet with FDA and EMA to determine next steps for Arikayce to treat NTM lung infection. Insmed also said it plans to apply for breakthrough therapy designation in the U.S. for Arikayce in the indication based on the culture conversion data in TARGET NTM. ...