Insmed Incorporated (Nasdaq CM: INSM), a biopharmaceutical company, announced positive data through six treatment cycles (72 weeks total duration) of its Phase 2 clinical trial program for ARIKACE® (liposomal amikacin for inhalation) in cystic fibrosis (CF) patients with Pseudomonas lung infections. The data were presented at the 34th European Cystic Fibrosis Conference in Hamburg, Germany, by Predrag Minic, M.D., Professor of Pediatrics and Head of Pediatrics Pulmonology Department, Mother and Child Health Institute, Belgrade, Serbia, and co-lead investigator of the study.

The open label Phase 2 study was designed to evaluate ARIKACE over multiple treatment cycles in CF patients with Pseudomonas lung infections. The study enrolled 49 patients to receive ARIKACE 560 mg once daily for 28 days of therapy, followed by a 56-day off-treatment observation period. ARIKACE was administered using an optimized, investigational eFlow® Nebulizer System (PARI Pharma GmbH).

The data demonstrated that ARIKACE, delivered once-daily for 28 consecutive days, followed by 56 days off-treatment, for a total of six cycles, resulted in statistically significant improvement in lung function that was sustained over a 72 week period. Specifically, inhalation of 560 mg of ARIKACE produced a mean increase in pulmonary function (FEV1) of 11.7% at the end of the 28 day treatment period of the sixth cycle (p<0.0001).

During the course of the study, FEV1 improvement was also sustained at the end of 56 days off-treatment in each of cycles one through six, with an estimated relative increase in FEV1 of 5.7% (95% CI +3.0%, +8.5%; p=0.0001). Overall, ARIKACE was well-tolerated during all six cycles, with adverse events reported as consistent with those expected in a population of CF patients receiving inhaled medicines.

"ARIKACE demonstrated consistent results of improved lung function throughout all six cycles of this study," said Professor Minic. "In addition, there were no unexpected adverse events associated with the longer term use of the drug. Taken as a whole, these data indicate that ARIKACE has the potential, if approved, to be an important product for CF patients with Pseudomonas lung infections."

ARIKACE also demonstrated statistically significant reduction from baseline in Pseudomonas aeruginosa density, including mucoid strains, which was sustained during the treatment and off-treatment periods of the six cycles. The estimated change from baseline in Log10 CFU over time was -0.6 log (95% CI, -0.2 to -0.9 log; p=0.0030). Mucoid strains of Pseudomonas are often difficult to suppress with antibiotics and play a significant role in progression of CF lung disease. In addition, there was no significant shift in the minimum inhibitory concentration (MIC90) of ARIKACE against Pseudomonas over the course of the six cycles, which suggests a lack of resistance development during the study period.

"These results further strengthen the clinical data package Insmed is developing for ARIKACE in the treatment of CF patients," said Renu Gupta, M.D., Executive Vice President Development & Chief Medical Officer of Insmed. "The data are also indicative of why we are so excited to begin our Phase 3 clinical program in the CF indication, which we expect to initiate in the second half of this year. For potential registration of ARIKACE in the U.S., we intend to conduct a placebo-controlled clinical trial, and for European registration, we intend to conduct a trial that compares ARIKACE to Novartis Corporation's TOBI®. Results from these studies are anticipated in the first half of 2013."

"We are pleased that the development of ARIKACE continues to proceed expeditiously," said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. "Based on the currently available data, we believe ARIKACE has the potential to address a significant medical need for CF patients suffering from Pseudomonas lung infections."

Cystic Fibrosis Foundation Therapeutics, Inc., a nonprofit affiliate of the Cystic Fibrosis Foundation, previously provided Insmed with $3.9 million to support the development of ARIKACE in CF. The Foundation is the leading organization devoted to curing and controlling CF.

Source: Insmed Incorporated