Insmed Incorporated (Nasdaq CM: INSM), a biopharmaceutical company focused on developing and commercializing an inhaled anti-infective to treat patients battling serious lung diseases that are often life-threatening, announces positive developments in both of its clinical development programs for ARIKACE ®, or liposomal amikacin for inhalation (LAI).
The Company reports that its Phase 3 study of once-daily ARIKACE to treat Pseudomonas aeruginosa (Pa) in cystic fibrosis (CF) patients conducted at 70 sites in Europe and Canada met its primary endpoint of non-inferiority compared with twice-daily TOBI ®* (tobramycin inhalation solution) for relative change in forced expiratory volume in one second (FEV 1), measured at the end of the third treatment cycle (24 weeks) as compared to baseline.
The Company also announces that the U.S. Food and Drug Administration (FDA) designated ARIKACE as a Qualified Infectious Disease Product (QIDP) for the treatment of Non-Tuberculous Mycobacteria (NTM) lung infections. Additionally, the FDA granted Fast Track designation to ARIKACE for the treatment of NTM.
Phase 3 Clinical Trial Design for ARIKACE to Treat Pa in CF PatientsThe Phase 3 trial was an open-label, multi-center, randomized study designed to assess the comparative safety and efficacy of ARIKACE and TOBI in CF patients with Pa. A total of 302 adult and pediatric CF patients with chronic Pa were randomized to receive 28-days of ARIKACE treatment delivered once-daily via an investigational eFlow ® Nebulizer System, or TOBI delivered twice-daily via the PARI LC Plus ® Nebulizer System over a 24-week treatment period. The primary endpoint was relative change in FEV 1 measured after three treatment cycles, with each cycle consisting of 28 days “on” treatment and 28 days “off” treatment. The study was designed to demonstrate non-inferiority to TOBI at a 5% non-inferiority margin with 80% power agreed upon by the Company and the European Medicines Agency (EMA). Secondary endpoints measured were relative changes in FEV 1 at other time points, time to and number of pulmonary exacerbations, time to antibiotic rescue treatment, change in density of Pa in sputum, respiratory hospitalizations and changes in Patient Reported Outcomes assessing Quality of Life. Primary Endpoint The Phase 3 clinical trial of ARIKACE achieved its primary endpoint of non-inferiority to TOBI for relative change in FEV 1 from baseline to end of study. Secondary Endpoints Overall, secondary endpoints showed comparability of once-daily ARIKACE compared with twice-daily TOBI consistent with the primary endpoint of the study. Complete trial data will be reviewed in greater detail and is expected to be released at a scientific conference later this year. The Company also intends to pursue publication of trial data in a peer-reviewed journal.