Insmed has announced that in a Phase 2 clinical trial of Arikayce (formerly Arikace) inhaled liposomal amikacin for nontuberculous mycobacterial (NTM) lung infections, the product failed to produce a statistically significant reduction in mycobacterial density; however, it did meet a secondary endpoint, with significantly more patients treated with Arikayce having negative cultures by the end of the study than patients receiving placebo.
According to the company, “The design of the study’s primary endpoint was not culture conversion because it was assumed that culture conversion would not be achievable in 84 days treatment, particularly given the severe, treatment-resistant patient population that was the subject of this study.”
The study involved 90 patients with NTM lung disease who had been treated according to American Thoracic Society/Infectious Disease Society of America (ATS/IDSA) guidelines for a minimum of six months without elimination of positive mycobacterial cultures.
Insmed Executive VP, Development and Chief Medical Officer Renu Gupta commented, “We are encouraged by the achievement of culture conversion in this trial, which we believe is the ultimate goal in the treatment of mycobacterial infections. The design of this trial was such that the patients who entered the trial and received drug were clearly resistant to guideline therapy, making them the most treatment-challenged NTM patients. Therefore the hurdle for showing any improvement with a therapy is extremely high.”
The company said that it “plans to incorporate these results into discussions with the regulatory agencies in the United States and Europe to determine next steps for Arikayce” and that it still plans to apply for breakthrough therapy designation.
In late 2013, Insmed announced that it had already initiated the Scientific Advice Working Party (SAWP) process with the EMA for Arikayce for the treatment of NTM lung infections, and it received orphan drug status for that indication from the EU earlier this year. The FDA granted the product orphan drug designation in March 2013.
Insmed is also developing Arikayce (which it spelled “Arikace” as recently as February 19, 2014) for the treatment of P. aeruginosa infections in cystic fibrosis patients.
Read the Insmed press release.
