MONMOUTH JUNCTION, N.J., Sept. 14 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.
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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.
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"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."
Bronchiectasis is characterized by localized, irreversible enlargement of the bronchial tubes. Involved bronchi are dilated, inflamed, and easily collapsible, resulting in airflow obstruction and impaired clearance of secretions. The accumulation of mucus in the bronchi leads to frequent infections, which further reduce lung function in these patients. One of the most frequent pathogens infecting bronchiectasis patients is Pseudomonas aeruginosa which is associated with increased sputum production, more extensive bronchiectasis, more hospitalizations, and reduced quality of life.
The disease is often misdiagnosed and mistaken for asthma or pneumonia. There is currently no drug specifically approved for the treatment of bronchiectasis or the associated lung infections in the U.S. or EU.
About ARIKACE(TM)
ARIKACE is a form of the antibiotic amikacin, which is enclosed in nanocapsules of lipid called liposomes. This advanced pulmonary liposome technology prolongs the release of amikacin in the lungs while minimizing systemic exposure. The treatment uses biocompatible lipids endogenous to the lung that are formulated into small (0.3 micron), neutrally-charged liposomes that enable penetration of the biofilm and are highly efficient, with a very low lipid-to-drug ratio (0.65). ARIKACE can be delivered through nebulization, which enables the small aerosol droplet size (1 to 5 microns) to facilitate more effective distribution in the lungs. In addition to this clinical study in non-CF bronchiectasis patients with Pseudomonas lung infections, clinical development is underway in CF patients with Pseudomonas lung infections, with positive Phase II results reported in June 2008. ARIKACE has been granted orphan drug status in the United States by the FDA, and has received an orphan drug designation in Europe by the European Medicines Agency for the treatment of Pseudomonas infections in patients with CF. ARIKACE has also been granted orphan drug status by the FDA for the treatment of bronchiectasis in patients with Pseudomonas or other susceptible pathogens.
About PARI Pharma and the eFlow(R) Electronic Nebulizer
ARIKACE is delivered by an eFlow Nebulizer System developed by PARI Pharma GmbH. The eFlow Nebulizer System uses eFlow Technology to enable highly efficient aerosolization of medication including liposomal formulations via a vibrating, perforated membrane that includes thousands of laser drilled holes. Compared to other nebulization technologies, eFlow Technology produces aerosols with a very high density of active drug, a precisely defined droplet size, and a high proportion of respirable droplets delivered in the shortest possible period of time. Combined with its silent mode of operation, small size (it fits in the palm of the hand), light weight, and battery use, products incorporating eFlow Technology reduce the burden of taking daily, inhaled treatments. The eFlow Nebulizer System and eFlow Technology are proprietary to PARI Pharma and can be optimized to specific drug formulations.
About Transave, Inc.
Transave, Inc., is a biopharmaceutical company focused on the development of innovative inhaled pharmaceuticals for the site-specific treatment of chronic lung diseases. The company's major focus is on developing antibiotic therapy delivered via proprietary advanced pulmonary liposome technology in areas of high unmet need in lung diseases. The Transave team is dedicated to leveraging its development and commercialization expertise, along with its intellectual property, to bring life-extending and life-enhancing medicines to patients. For more information about Transave's technology and development programs, visit http://www.transaveinc.com/.
SOURCE Transave, Inc.
