Thursday, 12. June 2014

Insmed Announces Positive Open Label Data from Phase 2 Clinical Trial of ARIKAYCE

for Treatment Resistant Nontuberculous Mycobacterial Lung Infections - Poster Presentation at the American Thoracic Society Annual Meeting 2014

MONMOUTH JUNCTION, N.J.--(BUSINESS WIRE)-- Insmed Incorporated (Nasdaq:INSM) today announced additional results from the Company's phase 2 clinical trial of ARIKAYCETM, or liposomal amikacin for inhalation, for the treatment of patients with treatment resistant nontuberculous mycobacterial (NTM) lung infections. The results are highlighted in a poster entitled, "A Randomized, Double-Blind, Placebo-Controlled Study of Liposomal Amikacin for Inhalation (ARIKAYCE) in Patients with Recalcitrant Nontuberculous Mycobacterial Lung Disease," that will be presented today at the American Thoracic Society's Annual Meeting 2014 in San Diego, California by Kenneth N. Olivier, M.D., M.Ph., National Institute of Allergy Allergy
An overreaction of the immune system to a previously ordinarily harmless substance.
and Infectious Diseases and a co-Principal Investigator of the study. The poster is available on the Company's website at

At the conclusion of the 84-day double blind phase of the trial, 78 of the 80 patients agreed to receive once-daily ARIKAYCE plus standard of care treatment for an additional 84 days. Data from 68 of these patients who completed the visits during the additional open label phase were available for inclusion in the poster. These results collected from the open label phase show that 21 of these patients were culture negative for NTM at Day 168. This data reflects 10 patients who were culture negative at Day 84 as well as 5 additional patients from the ARIKAYCE arm and 6 additional patients who were on placebo, switching to ARIKAYCE during the open-label phase. The number of patients with negative cultures increased from the double blind phase of the trial in which 11 out of 44 patients treated with ARIKAYCE (added to standard of care treatment) demonstrated negative cultures by day 84 of the study as compared to 3 out of 45 patients treated with standard of care plus placebo.

"We are encouraged by these additional and durable culture conversions which we believe is the ultimate goal in the treatment of NTM lung infections," said Will Lewis, President and Chief Executive Officer of Insmed. "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 bronchiectasis
Bronchiectasis is an abnormal widening of one or more airways. Extra mucus is made in the abnormal airways which is prone to infection.
or cystic fibrosis cystic fibrosis
Genetic disorder that affects most critically the lungs, leading to thick, viscous secretions.
, 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."

In the next several months, the Company plans to incorporate the trial results into discussions with the regulatory agencies in the United States and Europe to determine next steps for ARIKAYCE in the treatment of NTM lung infections. Based upon the culture conversion results, in April 2014 the Company applied for Breakthrough Therapy Designation for ARIKAYCE in the United States. ARIKAYCE has already 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 and recently received Orphan Drug Designation from the European Medicines Agency (EMA).

A full .pdf version of this press release is available for review via the link icon below: