Positive results from Transave's Phase II clinical trial of ARIKACE in non-CF bronchiectasis patients

Transave, Inc., today reported positive results from a Phase II clinical trial in non-cystic fibrosis (CF) bronchiectasis patients for its lead investigational drug, ARIKACE(TM) (liposomal amikacin for inhalation). The Phase II data indicated that ARIKACE, delivered once daily for 28 consecutive days, demonstrated superior clinical benefit compared to placebo as measured by patient and physician reported outcomes and reduction in Pseudomonas aeruginosa density. In addition, ARIKACE was well-tolerated with overall adverse events comparable to placebo. Results were presented today at the European Respiratory Society Meeting in Vienna, Austria by Anne E. O'Donnell, MD, Professor of Medicine and Chief of the Division of Pulmonary, Critical Care and Sleep Medicine, Georgetown University Hospital, and co-lead investigator of the study.

The double-blind, placebo-controlled study was designed to evaluate ARIKACE in non-CF patients who have bronchiectasis with Pseudomonas lung infections. Sixty-four adult patients were randomized to receive ARIKACE - either 280 mg or 560 mg daily dosages - or placebo. Patients received 28 days of therapy followed by a 28-day off-treatment observation period. ARIKACE and placebo were administered once daily using an eFlow (R) Nebulizer System (PARI Pharma GmbH), a novel, highly efficient, portable aerosol delivery system. Sixteen clinical sites throughout Europe and India participated in the study.

"The positive efficacy and tolerability profile demonstrated by ARIKACE in this study is encouraging for non-CF bronchiectasis patients that have pseudomonas lung infections and for physicians who treat them," said Dr. O'Donnell. "These successful results with ARIKACE are especially important since there is nothing currently approved to treat bronchiectasis patients with these types of infections."

The study also revealed that ARIKACE -- administered at both the 280 and 560 mg doses -- was well-tolerated. Adverse events were consistent with underlying chronic lung disease in bronchiectasis patients. Patients in the 560 mg cohort had a slightly higher frequency of dry cough than those in the 280 mg cohort, but the cough was of short duration, was self-limiting, and did not result in any physician choosing to discontinue a patient from the trial.

Patients in the placebo arm of the study had a greater frequency of both hospitalizations and pulmonary exacerbations.

"This Phase II study provides important evidence of the efficacy and tolerability of ARIKACE in the treatment of Pseudomonas aeruginosa lung infections in non-CF bronchiectasis patients. We believe these data warrant confirmation of ARIKACE efficacy and safety in a Phase III study," said Renu Gupta, M.D., Transave's Executive Vice President for Development and Chief Medical Officer. "These results, in addition to previously announced positive data from a Phase II study of ARIKACE in CF patients with Pseudomonas lung infections, support the potential benefits to patients of this advanced pulmonary formulation of liposomal amikacin designed for sustained release and penetration of mucus and biofilm in the lungs."