MONMOUTH JUNCTION, N.J., June 17 /PRNewswire/ -- Transave, Inc., today reported interim results from a multi-cycle Phase II open label clinical trial in cystic fibrosis (CF) patients on its lead investigational drug, ARIKACE™ (liposomal amikacin for inhalation). The data indicated that ARIKACE, delivered once daily for 28 consecutive days followed by 56 days off-treatment for four cycles demonstrated statistically significant improvement in lung function that was sustained during the 56 days off study drug. ARIKACE was well-tolerated during the four cycles. Results were presented today at the 33rd European Cystic Fibrosis Society (ECFS) Conference in Valencia, Spain, by Predrag Minic, MD, 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 study is an extension of a previously reported randomized, placebo controlled Phase II study, and was designed to evaluate ARIKACE over multiple treatment cycles in CF patients with Pseudomonas aeruginosa lung infections. Forty nine patients were enrolled to receive ARIKACE 560 mg daily for 28 days of therapy followed by a 56-day off-treatment observation period. ARIKACE was administered once daily using an eFlow® Nebulizer System (PARI Pharma GmbH), a novel, highly efficient and portable aerosol delivery system.
"The sustained improvement in lung function with significant reduction in bacterial density over multiple treatment cycles with ARIKACE is encouraging and indicative of benefit for cystic fibrosis patients who have chronic Pseudomonas lung infections," said Renu Gupta, MD, Transave's Executive Vice President for Development and Chief Medical Officer. Dr. Gupta indicated that preparations are underway to launch Phase III studies to confirm efficacy of ARIKACE. "These results support the potential value of delivering amikacin through Transave's advanced pulmonary liposome technology designed for sustained release and penetration of the mucus and bacterial biofilm in the lungs."
Pulmonary function (FEV1) increased significantly among patients receiving 560 mg of ARIKACE, with an estimated relative change from baseline in FEV1 of 9.2% (95% CI +5.0%, +13.4%; p less than or equal to 0.0001) at the end of treatment during cycles one to four. The improvement in lung function was sustained at the end of the 56-day off-treatment period during the four cycles with an estimated relative change from baseline in FEV1 of 4.7% (95% CI +1.0%, +8.5%; p=0.015).
ARIKACE demonstrated statistically significant reduction in Pseudomonas density, including mucoid strains, which was sustained over the treatment period of four cycles for 12 months. Mucoid strains of Pseudomonas are often difficult to suppress with antibiotics and play a greater role in progression of CF lung disease.
ARIKACE was well-tolerated for four cycles of treatment over 12 months, and demonstrated adverse effects that are consistent with those expected in a population of CF patients receiving inhalation medicines.
"ARIKACE development continues to show promise in improving lung function for CF patients," said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. "We are pleased to support Transave's work in potentially bringing an important new treatment option to CF patients."
Cystic Fibrosis Foundation Therapeutics, Inc., a nonprofit affiliate of the Cystic Fibrosis Foundation, provided $3.9 million to support the development of ARIKACE. The Foundation is the leading organization devoted to curing and controlling cystic fibrosis.
"A once-daily drug that may offer advantages in maintaining improvements in lung function during off-treatment periods over multiple cycles would be an important advance in the treatment of cystic fibrosis patients," said Dr. Minic. "These results show significant improvement in lung function with ARIKACE and are especially important since these patients are living in a state of chronic infection often requiring continuous treatment cycles over time.
The data presented today at the 33rd European Cystic Fibrosis Society Conference are currently available on the company's website: (http://www.transaveinc.com/NewsEvents.aspx?category=Articles&archive=false).
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), neutral liposomes that enable penetration of the biofilm.
Positive results were announced in October 2009 from pooled results of two Phase II clinical trials in the treatment of CF patients with Pseudomonas lung infections. The company also previously announced positive Phase II results in September 2009 in the treatment of non-CF bronchiectasis patients who have Pseudomonas lung infections.
Transave and the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), will collaborate on the planning, design and implementation of a clinical trial beginning later this year to evaluate ARIKACE in patients with nontuberculous mycobacteria (NTM) lung disease who have failed to respond to standard, guideline-based treatment regimens. Current treatment requires lengthy multi-drug regimens that are often poorly tolerated and not very effective. No new drugs have been assessed in clinical trials for this disease in many years.
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 eFlow® Technology and PARI Pharma
ARIKACE is delivered by an eFlow® Nebulizer System developed by PARI Pharma and optimized specifically for ARIKACE. 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. eFlow Technology is not an ultrasonic nebulizer technology. Combined with its quiet mode of operation, small size (it fits in the palm of the patient's hand), light weight, and battery use, eFlow Technology reduces the burden of taking daily, inhaled treatments. PARI Pharma focuses on the development of aerosol delivery devices and comprehensive inhalation drug development to advance aerosol therapies where drug and device can be optimized together. Online at www.paripharma.com.
About The Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. For more information, please visit www.cff.org.
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 www.transaveinc.com.