BETHESDA, Md. -- Today, the Cystic Fibrosis Foundation announced a new research award of up to $5 million to Armata Pharmaceuticals to conduct a Phase 1b/2 clinical trial to test the safety and tolerability of bacteriophage (phage therapy) in Pseudomonas aeruginosa infections in people living with cystic fibrosis (CF).
The randomized, controlled clinical trial marks the first time in the U.S. that phage will be studied in cystic fibrosis outside of the Food and Drug Administration (FDA) emergency Investigational New Drug (IND) process, which allows the use of experimental therapies for life-threatening conditions.
“Despite the approval of treatments for the underlying cause of CF, many people with CF struggle with difficult-to-treat infections. Developing new approaches to treat drug-resistant pathogens is critical,” said Michael P. Boyle, MD, president and chief executive officer of the Cystic Fibrosis Foundation. “Following the promising results reported through several individual cases, we are pursuing rigorous studies to evaluate the safety and efficacy of this emerging area of science. Better understanding of phage therapy has potential to significantly benefit people with CF as well as millions of others worldwide who are impacted by antibiotic-resistant infections.”
Pseudomonas infections, which are the second most common type of lung infection in people with CF, are very difficult to eliminate once established in the airways. Approximately 44 percent of the nearly 31,000 people with CF in the Cystic Fibrosis Foundation Patient Registry had Pseudomonas infections in 2018, and nearly 17 percent of those infected had multi-drug-resistant Pseudomonas infections.
Bacteriophages are specialized viruses that kill very specific bacterial strains. They are found abundantly in the environment and are the predators of bacteria in nature. “Phage therapy” refers to the use of these bacteriophages to treat an infection in a person. Phage has been used previously on a case-by-case basis. Rigorous clinical studies are needed to assess the safety and efficacy of phage for potential wider use in people with CF.
Armata Pharmaceuticals has developed AP-PA02, a mix of multiple different types of phages that is intended to be inhaled. In lab tests conducted by the company, it was able to kill more than 80 percent of Pseudomonas strains from people with CF.
The CF Foundation's Commitment to Infection Research
People with CF who have chronic infections are at greater risk for worsening lung disease and death, and infection remains a top concern of both patients and clinicians. Many individuals also suffer severe side effects from long-term antibiotic use, such as hearing loss, and are at increased risk of developing antibiotic-resistant infections.
In 2018, the CF Foundation dedicated at least $100 million to its Infection Research Initiative as part of a sweeping effort to advance infection research. The Infection Research Initiative is a comprehensive approach to improve outcomes associated with infections through enhanced detection, diagnosis, prevention, and treatment. Currently, the CF Foundation is funding more than 10 industry programs to develop treatments for CF-related infections and is in contact with additional companies as well as academic researchers considering the use of developing phage therapies for CF.
The CF Foundation is focused on advancing the next generation of transformative therapies to address complications, treat the underlying cause of CF for every person, and find a cure. Innovators who are interested in pursuing programs in cystic fibrosis can learn about our specific funding opportunities here, including the Infection Research Initiative, Path to a Cure, and Therapeutics Development Awards.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF 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 cff.org.