Inhaled antibacterial treatment for NTM lung infections as an addition to standard (oral or i.v.) treatment. As a further treatment option it will be additional to current costs. Will be used for treatment of adults with refractory NTM lung infection caused by MAC. Will be administered via Lamira (nebuliser handset and aerosol head) used with the PARI eBase Controller. It must not be administered by any other route or using any other nebulisation system . There are currently no products indicated for treatment of NTM lung disease in the US or EU . In the US, Arikayce is the first drug to be approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs . Arikayce also was approved under the Accelerated Approval pathway and was granted Orphan Drug, Fast Track, Breakthrough Therapy, Priority Review, and Qualified Infectious Disease Product (QIDP) designations.
CONVERT study (NCT02344004) on-going with estimated completion date of July 19.
Insmed announced positive top-line results from PIII CONVERT study (n=336) of ALIS (Amikacin Liposome Inhalation Suspension) in pts with treatment-refractory NTM Lung Disease caused by MAC. The study demonstrated that the addition of ALIS to guideline-based therapy (GBT) eliminated evidence of NTM lung disease caused by MAC in sputum by Month 6 in 29% of patients, compared to 9% of patients on GBT alone (p < 0.0001). serious treatment emergent adverse events were similar between treatment arms with no distinctions between treatment arms due to hearing loss or renal impairment. Overall the rate of reported adverse events in the ALIS plus GBT arm was higher and these events were mild or moderate in nature and transient. These findings are consistent with adverse events similar to those seen in other clinical studies of inhaled antibiotics.
CONVERT study = NCT02344004 .
Insmed announces it has achieved its patient enrolment objective in the PIII CONVERT or INS-212 study, which is evaluating ARIKAYCE in treatment refractory NTM lung disease caused by MAC. The primary efficacy endpoint is the proportion of subjects who achieve culture conversion at Month 6 in the ARIKAYCE plus multi-drug regimen arm compared to the multi-drug regimen without ARIKAYCE arm .
NCT02344004, a PIII randomized, open-label, multicenter study in adults with Nontuberculous Mycobacterial (NTM) infection caused by Mycobacterium avium complex (MAC) who are refractory to treatment was initiated Jan 15 . Trial due to complete Oct 16 .
Insmed reports that in a PII study, liposomal amikacin missed its primary endpoint of reducing bacterial density (used as a measure of infection). However, in a secondary endpoint, 11 of the 44 patients taking Arikayce turned in negative cultures at the end of the study, a statistically significant improvement over the placebo arm .
Insmed remains on track to report top-line data from a PII trial in the US and Canada to treat NTM lung disease by end of 1Q 2014 .
The Company plans to start a PII randomized study of approximately 100 adult patients with recalcitrant NTM lung disease in mid-2012. Patients who are NTM culture positive will continue with their antibiotic treatment regimen, and receive additionally, either ARIKACE 560mg, delivered once daily via an optimized, investigational eFlow® Nebulizer System (PARI Pharma GmbH), or placebo once daily. The primary efficacy endpoint is change in mycobacterial density from baseline to day 84 of treatment. Eligible patients will be able to receive ARIKACE 560mg once daily for an additional 84 days in an open-label design. The clinical trial design was previously agreed with the FDA .
US FDA has lifted the clinical hold on ARIKACE® in patients with non-tuberculous mycobacteria (NTM) lung disease. Insmed continues to engage in discussions with FDA regarding the clinical hold placed on ARIKACE in Cystic Fibrosis (CF) patients with Pseudomonas lung infections. Insmed also announced that it will move ahead with the 9-month dog inhalation toxicity study of ARIKACE as previously requested by FDA to determine if the findings of the rat inhalation carcinogenicity study are observed in a non-rodent model. 
The US FDA is continuing the clinical hold placed on the Phase III trial for Arikace in patients with non-tuberculous mycobacterial (NTM) lung disease. Based on its review of the information provided to date, including the rat inhalation carcinogenicity study results, the FDA has insufficient information to assess the risks of Arikace in NTM patients in the planned Phase III trial. FDA requires a Phase II trial in adult NTM patients intended to provide proof-of-concept efficacy and safety data for Arikace in NTM before a Phase III trial can proceed. 
U.S. FDA has placed a clinical hold on Insmed´s P3 clinical trials for liposomal amikacin for inhalation in CF patients with Pseudomonas lung infections and patients with non-tuberculous mycobacterial (NTM) lung disease. A clinical hold is a notification issued by FDA to the sponsor to delay a proposed clinical trial or suspend an ongoing clinical trial. The decision was based on an initial review of the interim results of a long-term rat inhalation carcinogenicity study. In this study, rats received daily doses of liposomal amikacin by inhalation for up to two years. FDA has requested additional information and data from the rat study. Insmed anticipates being able to supply the currently requested information and data within the next 30 days. 
A pivotal PIII study in nontuberculous mycobacteria lung infections has been agreed with the FDA. Development of the clinical program has been done in partnership with the National Institute of Allergy and Infectious Diseases at the National Institutes of Health. Data from the study is expected 1H 2013 .