The Cystic Fibrosis Foundation Therapeutics (CFFT) has given an award worth up to $5 million to Savara Pharmaceuticals to support the continued development of AeroVanc, an inhaled antibiotic intended to treat persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in individuals with cystic fibrosis (CF).
CFFT is the non-profit drug discovery and development arm of the Cystic Fibrosis Foundation (CFF).
Specifically, the award will support testing of AeroVanc (vancomycin hydrochloride inhalation powder)’s clinical effectiveness in a pivotal Phase 3 (NCT03181932) trial called AVAIL.The study intends to recruit up to 200 CF patients, ages 6 and older, at some 80 sites across the U.S. and Canada.
AeroVanc is the first inhaled antibiotic aiming to treat MRSA lung infections in CF patients. In a Phase 2 study in CF patients with persistent MRSA infection (NCT01746095), AeroVanc was found to reduce MRSA density in sputum and showed encouraging trends of improvement in lung function. AeroVanc also seemed to prolong the time of use of other antibiotics in young adults.
“We are delighted to have the CF Foundation’s continued support and we believe this award serves as strong validation of our AeroVanc program,” Rob Neville, chief executive officer of Savara, said in a press release.
“Our AVAIL study was planned in consultation with the CF Foundation’s Therapeutic Development Network as well as key opinion leaders (KOL’s) across the country, and we are excited to have the study started and enrolling,” Neville added.
AVAIL’s primary outcome will be the mean absolute change from baseline, or study start, in the volume of air a person can forcibly exhale after a deep breath in one second (forced expiratory volume percent predicted or FEV1, a measure of lung health) at week four and week 20 of treatment. It will be measured specifically in patients ages 6 to 21 for effectiveness, the release states; in the Phase 2 trial, this group also had the best response to AeroVanc treatment.
The study will be split into two parts. In the first part, patients will be randomly assigned to receive either 30 mg of AeroVanc or placebo, twice daily, for 24 weeks or three dosing cycles (each cycle comprises 28 days of treatment and 28 days of observation).
In the second part, AeroVanc (30 mg) will be given open-label to all, twice daily, for an another 24 weeks (three dosing cycles) to evaluate its the long-term safety.
“Cystic fibrosis has been a primary focus of my research interests for many years. Inhaled antibiotics have become the standard of care to treat Pseudomonas aeruginosa lung infection and we are now on the verge of demonstrating that a similar approach should be used for MRSA” said Patrick Flume, MD, the study’s coordinating investigator, and director of the Cystic Fibrosis Program at the Medical University of South Carolina, in Charleston.
“If the results from the AVAIL study are positive, I believe AeroVanc could become the new standard for inhaled antibiotic treatment of MRSA in CF,” Flume added.
For more information about the trial and how to participate, please visit this link. It is expected to end in December 2019.
The U.S. Food and Drug Administration (FDA) designated AeroVanc an Orphan Drug and a Qualified Infectious Disease Product (QDIP). The two regulatory designations also support AeroVanc’s development by giving it a potential 12 years of market exclusivity if approved.