Insmed has presented additional positive results from the open label portion of a Phase 2 trial of Arikayce inhaled liposomal amikacin for the treatment-resistant nontuberculous mycobacterial (NTM) lung infections at the American Thoracic Society annual meeting. Previously announced results from the trial were mixed.
The 84-day open label phase involved 78 of the 80 patients who participated in the initial 84-day double blind phase. In the open label phase, 5 patients who had received Arikayce and 6 who had received placebo in the double blind portion of the study became culture negative by day 168. An additional 10 patients who were culture negative at the beginning of the open label phase remained culture negative, for a total of 21 patients out of 68 who completed all 168 days.
Aradigm President and CEO Will Lewis commented, “We are encouraged by these additional and durable culture conversions which we believe is the ultimate goal in the treatment of NTM lung infections. The patients screened for admission to this trial are recalcitrant to treatment. While the entry criteria for this trial required a minimum of 6 months on standard of care therapy, over 75% of patients entering this trial were treated with standard of care therapy for more than a year, yet remained culture positive. In addition, a majority of these patients suffer from at least one additional pulmonary co-morbidity, such as bronchiectasis or cystic fibrosis, making the hurdle quite high for showing any improvement and making these results that much more encouraging for patients suffering from this disease. We now look forward to the regulatory discussions in the United States and Europe that will guide our path forward.”
According to the company, it has used the new culture conversion results to support an application for Breakthrough Therapy Designation for the product in the US, which it filed in April 2014. Arikayce for NTM already has orphan drug, QIDP and Fast Track designations in the US and orphan drug designation in Europe.
Read the Insmed press release.
