Simultaneously, Insmed continues its discussions with the U.S. Food and Drug Administration (FDA) regarding the clinical hold previously placed on the ARIKACE clinical study in CF patients with Pa lung infections.
"I am pleased to announce that we are moving forward with the ARIKACE clinical development program in NTM in the U.S., and with the European CF program," said Timothy Whitten, President and CEO of Insmed. "We look forward to continuing our dialogue with FDA regarding the CF clinical program in the U.S., and continue to believe that ARIKACE has the potential to be an important treatment option for CF and NTM patients."
The phase 2 clinical trial for ARIKACE in NTM patients will consist of a randomized, placebo-controlled study of approximately 100 adult patients with recalcitrant NTM lung disease. Patients who are NTM culture positive will continue with their antibiotic treatment regimen, and receive additionally, either ARIKACE 560 mg, delivered once daily via an optimized, investigational eFlow® Nebulizer System (PARI Pharma GmbH), or placebo once daily. The primary efficacy endpoint will be change in mycobacterial density from baseline to the end of 84 days of treatment. At the conclusion of the randomized portion of the study, eligible patients will receive ARIKACE 560 mg once daily for an additional 84 days in an open-label design, primarily to measure longer-term safety and efficacy. The clinical trial design was previously agreed upon by Insmed and FDA. The Company expects to begin enrolling patients in the phase 2 clinical trial in mid-2012.
The European study in CF patients with Pa lung infections will be a randomized, phase 3 trial comparing ARIKACE 560 mg, delivered once daily via an optimized, investigational eFlow Nebulizer System, to TOBI®(1) (inhaled tobramycin solution), which is a marketed inhaled antibiotic that is delivered twice daily. The Company anticipates that the study will be conducted in approximately 300 patients. The primary endpoint will be change in pulmonary function (FEV-1) measured after three 28 day on-treatment and three 28 day off-treatment cycles (about six months). A key secondary endpoint will be time to pulmonary exacerbation. The study design was previously agreed upon by Insmed and the European Medicines Agency. Eligible patients will have the option to participate in a longer term open-label safety study. The Company expects to begin enrolling patients in the phase 3 European clinical study in the second quarter of 2012.