Trial also demonstrates strong trends in secondary clinical endpoints
AUSTIN, Texas, Feb. 23, 2015 /PRNewswire/ -- Savara Pharmaceuticals announced today positive topline results from its Phase 2 clinical trial of AeroVanc, the first inhaled antibiotic being developed to address the growing problem of methicillin-resistant Staphylococcus aureus (MRSA) lung infection in people with cystic fibrosis (CF). AeroVanc met the primary endpoint in the modified intent to treat population with a statistically significant reduction in MRSA density in sputum as compared to placebo. AeroVanc also showed encouraging trends in the trial's secondary endpoints, including clinically meaningful improvements in pulmonary function, in respiratory symptoms, and in the time to exacerbation and need of other antibiotics, as compared to placebo.
"It is well known that people with CF who suffer from chronic Pseudomonas lung infection greatly benefit from inhaled antibiotics, such as TOBI," said Rob Neville, Chief Executive Officer of Savara Pharmaceuticals. He continued, "MRSA prevalence has increased dramatically over the past decade, but there is no inhaled antibiotic available for chronic treatment. Based on the observed clinical benefits in our study, we believe that AeroVanc can be equally successful for treatment of MRSA as the current inhaled antibiotics have been for Pseudomonas."
Vancomycin is an FDA-approved intravenously administered antibiotic with proven efficacy in the treatment of MRSA infections. However, poor penetration into the lungs and systemic toxicities limit its use as a chronic treatment. AeroVanc is an investigational, proprietary inhaled dry powder form of vancomycin in a capsule-based device designed for convenient self-administration.
The AeroVanc Phase 2 trial was a randomized, double-blind, placebo-controlled study carried out in 87 people with CF and persistent MRSA lung infection to evaluate the safety and efficacy of either 32 mg or 64 mg doses of the dry powder form of vancomycin inhaled twice daily for 28 days.
The most frequently reported adverse event was cough, with no difference between the two dose levels or between the active and placebo groups. Symptoms consistent with bronchoconstriction were observed in higher frequency in subjects treated with AeroVanc at the 64 mg dose. All strains of MRSA were susceptible to vancomycin, and no change in the minimum inhibitory concentration distribution was observed during or after the AeroVanc treatment.
"The consistency of effect across all clinically relevant endpoints in the AeroVanc study was very encouraging, as was the magnitude of the treatment effect," said Michael Konstan, M.D., Chairman of the Department of Pediatrics of Case Western Reserve University School of Medicine, and Rainbow Babies and Children's Hospital, Cleveland, OH. "The unmet need in people with CF and persistent MRSA is substantial, and this study gives much hope for an important new therapeutic option."
AeroVanc is currently being developed as a treatment for persistent MRSA lung infection in people with CF. By delivering vancomycin directly to the lungs, higher vancomycin concentrations are achieved at the site of infection, which is expected to lead to improved clinical efficacy. In addition, direct delivery of the drug into the lungs reduces exposure to the drug elsewhere in the body, and is thereby expected to reduce the risk of systemic drug-related side effects. AeroVanc has received from the FDA Fast Track and Orphan Drug designations as well as Qualified Infectious Disease Product (QIDP) status providing 12 years of market exclusivity.
About Cystic Fibrosis
Cystic fibrosis is a life-shortening genetic disease characterized by thick, sticky mucus in the lungs and frequent lung infections, which result in loss of lung function. As the disease progresses, the lungs of people with CF are typically infected with bacteria that are difficult to eradicate. Physicians have been using nebulized antibiotics to treat these infections and have sought new options for treatment in the chronic care setting. Infection by MRSA has become increasingly common with a prevalence of almost 30 percent of the estimated 32,000 people with CF in the U.S. Persistent MRSA infection is associated with faster decline in lung function and reduced survival. Currently there is no approved inhaled therapy for MRSA infection in people with CF.
About Savara Pharmaceuticals
Savara Pharmaceuticals is a specialty pharmaceutical company focusing on innovative drugs for the treatment of serious and life-threatening rare respiratory diseases. The company's lead product, AeroVanc, is the first dry powder inhaled antibiotic being developed for the treatment of MRSA infection in people with cystic fibrosis. For more information, please see Savara's website at www.savarapharma.com, or www.aerovanc.com, or find us on Twitter, Facebook and LinkedIn.
Research reported in this release was supported by the Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation, as well as the National Heart, Lung, and Blood Institute of the National Institutes of Health (award number R44HL112393). The content is solely the responsibility of Savara and may not represent the official views of either organization.
Robert Neville, CEO
SOURCE Savara Pharmaceuticals