Phase 3 Trial of Inhaled Antibiotic to Treat CF-related Lung Infections Supported by $20M in New Financing

Daniela Semedo, PhD avatar

by Daniela Semedo, PhD |

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Savara Pharmaceuticals recently announced that it has closed a $20 million Series C financing round, which will be used to advance a pivotal Phase 3 clinical trial of AeroVanc (vancomycin hydrochloride inhalation powder), the first dry powder inhaled antibiotic being developed for the treatment of methicillin-resistant Staphylococcus aureus (MRSA) lung infection in cystic fibrosis (CF) patients.

By delivering vancomycin directly to the site of infection, AeroVanc has the ability to improve its clinical efficacy and reduce adverse effects caused by broad-spectrum antibiotics. The purpose of the research trial is to evaluate the effectiveness, safety, and pharmacokinetics of AeroVanc,  Savara’s lead product, compared to placebo in subjects with CF and a chronic MRSA lung infection.

“The new financing will help support the AeroVanc Phase 3 trial, and expanding our pipeline with other new drugs for serious and life-shortening rare respiratory diseases,” said Rob Neville, chief executive officer of Savara Pharmaceuticals, in a recent press release. “Research shows that people with CF have a more rapid decline in lung function when infected with MRSA, as well as a reduction in overall survival. Our efforts will hopefully result in the FDA approval of the first inhaled antibiotic for use in MRSA infected people with CF.”

Savara is presently selecting research sites for its upcoming Phase 3 trial, with approximately 80 U.S. and Canadian sites expected to participate. The company plans to initiate trial enrollment in the final months of 2016.

CF is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe, and Australia. The condition is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein, resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to develop CF.

While the gene defect results in a myriad of medical problems for the patient, the most meddlesome clinical feature, chronic pulmonary infection with Pseudomonas aeruginosa, allows the basic pathologic process in CF to be designated an infectious disease. Ultimately, 80 percent to 95 percent of patients with CF succumb to respiratory failure brought on by chronic bacterial infection and concomitant airway inflammation.

The lungs of CF patients are often colonized or infected in childhood with organisms, such as Staphylococcus aureus and Haemophilus influenzae, that may damage the epithelial surfaces, leading to increased attachment of, and eventual replacement by, P. aeruginosa.