Insmed has announced that it will move ahead with a Phase 3 trial of Arikayce inhaled liposomal amikacin for the treatment of nontuberculous mycobacteria (NTM) lung infections, with results expected in 2017. According to the company, it met with the FDA regarding the study, and “the FDA acknowledged that exploration of the effectiveness of Arikayce in a broader population is appropriate, based on the results of the recently conducted Phase 2 trial, which showed statistically significant negative culture conversion in patients refractory to standard therapy.”
Negative culture conversion was a secondary endpoint of the Phase 2 study, which missed its primary endpoint when treatment with Arikayce failed to produce a statistically significant reduction in mycobacterial density.
Insmed said that it will also undertake a second Phase 3 study designed to confirm results from the Phase 2 study. A subgroup of patients in that study who had mycobacterium avium complex (MAC) NTM lung infections responded especially well to Arikayce treatment, the company said, and it will explore the use of the drug for that indication. The second study is expected to be completed by mid-2016.
The company also said that it plans to file an MAA for Arikayce for the treatment of refractory NTM lung infections and for P. aeruginosa lung infections in cystic fibrosis patients by the end of 2014.
Insmed President and CEO Will Lewis commented, “Our planned filing in Europe is a significant step forward in our goal to bring Arikayce to market to benefit the thousands of European NTM patients refractory to standard therapy, as well as cystic fibrosis patients with Pseudomonas aeruginosa lung infections. We will continue to resource our clinical, commercial and manufacturing capabilities in order to expedite our regulatory submissions and prepare for commercial launch, initially in Europe.”
He continued, “We are encouraged by the FDA’s support for our larger Phase 3 study. We have already made extensive preparations for both trials, including design of the protocols and identification of CROs (clinical research organizations). Given the encouraging results of the previous trial, clinicians are keen to work with us to enroll patients quickly. We look forward to continuing to work with the FDA on the regulatory review and to sharing interim data from these studies in a timely fashion.”
Read the Insmed press release.
