Insmed Completes Arikace Enrollment

Insmed Inc. has completed patient enrollment in its Phase II study of Arikace (liposomal amikacin for inhalation) for patients with recalcitrant nontuberculous mycobacterial (NTM) lung disease in the U.S. and Canada. Arikace has received Orphan Drug, Qualified Infectious Disease Product (QIDP) and Fast Track designations from the FDA for the treatment of NTM lung infections. Ninety patients will participate in the trial. The company has concluded that the study is sufficiently powered to achieve statistical significance with 80% power to demonstrate a one-step change in the seven-step scale measuring bacterial density.

The Phase II study is a randomized, placebo-controlled trial in adult patients with recalcitrant NTM lung disease. Eligibility for the study required patients to have been on the American Thoracic Society/Infectious Disease Society of America (ATS/IDSA) guideline therapy for at least six months prior to screening with persistently positive mycobacterial culture. All patients in the study continued with their antibiotic regimen, and received additionally, either Arikace or placebo, delivered once daily via an optimized, investigational eFlow® Nebulizer System (PARI Pharma GmbH).

The primary efficacy endpoint in the study is the semi-quantitative measurement of the change in mycobacterial density on a seven-point scale from baseline (day one) to the end of the randomized portion of the trial on day 84. Certain secondary, tertiary and exploratory endpoints being measured include, but are not limited to, time to sputum conversion, change in clinical signs and symptoms, change in patient related outcomes/quality of life and safety.

In addition, Insmed has commenced the Scientific Advice Working Party (SAWP) process with the EMA and expects to have discussions with that agency regarding Arikace for NTM lung disease during 4Q13. The company is also in the process of evaluating other international markets, such as Japan, where NTM lung infections are already a significant and growing unmet medical need.