Insmed Completes Enrollment of Phase 2 Clinical Trial of ARIKACE to Treat Nontuberculous Mycobacteria Lung Disease in U.S. and Canada

MONMOUTH JUNCTION, NJ--(Marketwired - Oct 15, 2013) - Insmed Incorporated (NASDAQ: INSM), a biopharmaceutical company focused on developing an inhaled anti-infective to treat patients battling serious lung diseases in orphan indications that are often life-threatening, today announced that the Company has completed patient enrollment in its Phase 2 clinical study of ARIKACE^®, or 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 U.S. Food and Drug Administration (FDA) for the treatment of NTM lung infections. 

Separately, Insmed announced that it commenced the Scientific Advice Working Party (SAWP) process with the European Medicines Agency (EMA) and expects to have discussions with the EMA regarding ARIKACE for NTM lung disease during the fourth quarter of 2013. In addition, the Company is in the process of evaluating other international markets, such as Japan, where NTM lung infections are already a significant and growing unmet medical need. Insmed is actively developing a strategy for more broadly commercializing ARIKACE in this globally uncontested orphan disease market.

The Phase 2 clinical study is a randomized, placebo-controlled study of ARIKACE 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. Mycobacterial density is a measurement currently used in clinical practice to assess the progress or decline of those patients with recalcitrant NTM. Following the randomized portion of the study, all eligible patients may choose to receive ARIKACE once daily for an additional 84 days in an open-label design.