Insmed Reports Results From Phase 2 Trial Of ARIKAYCETM - Quick Facts

(RTTNews) - Insmed Incorporated (INSM) reported results from phase 2 clinical trial of ARIKAYCETM, or liposomal amikacin for inhalation, for the treatment of patients with treatment resistant nontuberculous mycobacterial lung infections. ARIKAYCE did not achieve statistical significance on the primary endpoint of bacterial density reduction. The results relating to the secondary endpoint of culture conversion were encouraging, as per the data.

The randomized, double-blind, placebo-controlled phase 2 clinical trial compared ARIKAYCE, added to standard of care treatment, versus standard of care treatment plus placebo, in 90 adult patients with treatment resistant nontuberculous mycobacterial lung disease.

The primary efficacy endpoint of the study was a semi-quantitative measurement of the change in mycobacterial density on a seven-point scale from baseline to the end of the randomized portion of the trial. ARIKAYCE did not meet the pre-specified level for statistical significance although there was a positive trend in favor of ARIKAYCE. ARIKAYCE did achieve statistical significance with regard to the secondary endpoint of culture conversion, with 11 out of 44 patients treated with ARIKAYCE (added to standard of care treatment) demonstrating negative cultures by day 84 of the study as compared to 3 out of 45 patients treated with placebo. Patients receiving ARIKAYCE experienced a greater number of adverse events than those receiving placebo.

The company plans to incorporate the results into discussions with the regulatory agencies in the United States and Europe to determine next steps for ARIKAYCE. The company intends to apply for Breakthrough Therapy Designation for ARIKAYCE in the United States based upon the culture conversion results.