Austin, Texas-based specialty pharmaceutical company Savara Pharmaceuticals has released encouraging top-line results from its Phase 2 clinical trial evaluating an investigational therapy to treat methicillin-resistant Staphylococcus aureus (MRSA) lung infection in cystic fibrosis (CF) patients. The inhaled experimental treatment called AeroVanc was able to meet the study’s primary endpoint and the company is confident about further examinations.
AeroVanc therapy was assessed for MRSA lung infection in CF patients, showing it could significantly decrease the density of MRSA in sputum when compared to placebo. In addition, the trial also revealed encouraging results on its secondary endpoints including clinically significant improvements of pulmonary activity, respiratory symptoms, time between exacerbations, as well as necessity for antibiotic treatment.
“It is well known that people with CF who suffer from chronic Pseudomonas lung infection greatly benefit from inhaled antibiotics, such as TOBI,” stated the CEO of Savara, Rob Neville, in a press release. “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.”
AeroVanc therapy is based on an intravenously administered antibiotic approved by the U.S. Food and Drug Administration (FDA), called vancomycin, which has been proven effective in treating MRSA lung infections. There is, however, poor penetration of intravenous treatment in the lungs in the long term. Savara is developing AeroVanc as an inhaled dry powder form of vancomycin in a capsule-based device to be self-administered.
In addition, the delivery of the antibiotic directly to the site of infection in the lungs is expected to increase clinical efficacy as well as decrease the exposure of other parts of the body to the drug, lowering side effects associated to systemic drug delivery.
Savara assessed the safety and efficacy of AeroVanc from a randomized, double-blind, placebo-controlled phase 2 clinical study initiated in April 2013 that included 87 patients who suffer from cystic fibrosis with persistent MRSA lung infection. The patients were randomly chosen to be administered either 32 mg or 64 mg doses of the dry powder form of vancomycin inhaled two times per day during 28 days, or placebo.
“The consistency of effect across all clinically relevant endpoints in the AeroVanc study was very encouraging, as was the magnitude of the treatment effect,” added the chairman of the Department of Pediatrics of Case Western Reserve University School of Medicine, and Rainbow Babies and Children’s Hospital, Michael Konstan, MD. “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 being developed with support from Cystic Fibrosis Foundation Therapeutics Inc., which is a nonprofit drug discovery and development affiliate of the CF Foundation, which granted the company a $1.7 million research award in 2013. In addition, the National Heart, Lung, and Blood Institute of the National Institutes of Health also contributed financial support to the study.
Cystic fibrosis is a genetic and debilitating disease that affects about 32,000 Americans, with a significant percentage (30%) eventually suffering from MRSA lung infections. Even though the condition can cause a faster decline of lung function and reduce survival there are no inhaled treatments currently approved for its treatment. Therefore, the FDA already granted Savara both a Fast Track designation and a Qualified Infectious Disease Product (QIDP) status for AeroVanc in order to accelerate its developmental process.