Savara Pharmaceuticals, an Austin-based emerging specialty pharmaceutical company developing innovative pulmonary drugs for the treatment of serious and life-threatening conditions, has announced that it has received a $1.7 million research award from Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit drug discovery and development affiliate of the CF Foundation.
The award will help advance development of Savara’s AeroVanc, the first inhaled antibiotic being developed to treat the increasing number of people with cystic fibrosis (CF) who have persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection. The announcement was made on the opening day of the 27th annual North American Cystic Fibrosis Conference, sponsored by the CF Foundation, where doctors, scientists and clinicians present the latest advancements in CF research, care and drug development.
Cystic fibrosis is a life-shortening genetic disease characterized by thick, sticky mucus in the lungs and chronic lung infections resulting in gradual loss of lung function. There are approximately 30,000 people living with cystic fibrosis in the U.S. The most prevalent lung pathogen in cystic fibrosis patients is Pseudomonas aeruginosa, which is commonly treated using inhaled antibiotics. In recent years, methicillin-resistant Staphylococcus aureus (MRSA), a bacterium that is resistant to conventional antibiotics has become increasingly common, with a prevalence of almost 30% of the U.S. cystic fibrosis patient population. Persistent MRSA infections have been found to cause a decline in lung function and are associated with a significantly shortened life expectancy. Currently there is no approved inhaled treatment for MRSA infections in cystic fibrosis patients.
MRSA is difficult to eradicate or manage using oral or IV antibiotics, and there is no standard of care to manage the infection in CF patients. Intravenous vancomycin is the antibiotic of choice for MRSA-related bronchopneumonia in CF patients, but the burden of IV administration, poor penetration into the lungs and systemic toxicities limit its use in a chronic setting.
In contrast to the established treatment of Pseudomonas aeruginosa lung infection with inhaled antibiotics, there is currently no FDA-approved inhaled antibiotic treatment available for MRSA lung infection. However, Savara Pharmaceuticals announced in April that the first patient had started study drug treatment in a Phase 2 clinical trial evaluating the safety and efficacy of its lead product candidate, AeroVanc (vancomycin hydrochloride inhalation powder) — a proprietary inhaled dry powder form of vancomycin in a capsule-based device designed for convenient self-administration for the treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in cystic fibrosis (CF) patients.
AeroVanc is the first inhaled antibiotic being developed to address the growing population of MRSA lung-infected CF patients. 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 was recently granted orphan drug status by the U.S. FDA.
“An inhaled dry powder form of vancomycin for MRSA infection will be a logical addition to this and other treatment options available for CF patients,” says Rob Neville, CEO of Savara Pharmaceuticals. “We are further encouraged by the strong interest and support of key opinion leaders, and the feedback that AeroVanc is exactly what the CF care community has been waiting for.”
“The support of the CF Foundation is yet another acknowledgment that AeroVanc addresses an unmet need for people with CF suffering from MRSA infection,” Mr. Neville notes. “The $6 million in support that Savara has received from the CF Foundation and the National Institutes of Health not only provides critical funding to advance the AeroVanc program, but is also a strong endorsement of the quality and promise of our program.”
Savara is now enrolling patients for its Phase II trial of AeroVanc. It is a randomized, double-blind, placebo-controlled study in 80 CF patients with persistent MRSA lung infection, and is being conducted at 26 CF centers nationwide. The primary objective of the study is to evaluate the efficacy of AeroVanc in reducing the quantity of MRSA colony forming units in sputum cultures. The secondary objectives include evaluation of the safety of repeat dosing with AeroVanc and the efficacy of AeroVanc in improving lung function, reducing respiratory symptoms, and prolonging the time to pulmonary exacerbations and the need for other antibiotics.mIn Phase I studies of AeroVanc in healthy volunteers and cystic fibrosis patients, AeroVanc was well tolerated and demonstrated an excellent pharmacokinetic profile.
Physicians have been using nebulized antibiotics to treat MRSA infections and have sought new options for treatment in the chronic care setting. The first successful reformulation of an antibiotic into dry powder inhaled therapy is the recently FDA-approved TOBI Podhaler (tobramycin inhalation powder) for Pseudomonas aeruginosa infections in CF patients.
“Savara’s AeroVanc is a much needed investigational product, as none of the current inhaled antibiotics address the growing problem of MRSA infection,” says Dr. Bonnie W. Ramsey, Director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute, and one of the pioneers and lead investigators of TOBI, an inhaled form of tobramycin used to treat chronic Pseudomonas aeruginosa infection in CF. “TOBI has become the cornerstone of treatment in CF, and AeroVanc has the potential to become analogous to TOBI for MRSA.”
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for cystic fibrosis. The Foundation funds more cystic fibrosis 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. The CF Foundation is a donor-supported nonprofit organization. For more information, visit http://www.cff.org
Established in 2000, Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) is the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation. CFFT supports and governs activities related to cystic fibrosis (CF) drug discovery through the many stages of drug development and clinical evaluation.
ClinicalTrials.gov service of the U.S. National Institutes of Health
Cystic Fibrosis Foundation