BETHESDA, Md. -- The Cystic Fibrosis Foundation announced today that it has awarded up to $5 million to BiomX Inc. to conduct a Phase 1b/2a clinical trial to test the safety and tolerability of bacteriophage (phage therapy) in Pseudomonas aeruginosa infections in people living with cystic fibrosis.
The Foundation has supported several phage studies to explore the feasibility of phage therapy as a treatment for drug-resistant infections in CF. Currently, people with CF can only access phage treatment in the U.S. by using the Food and Drug Administration emergency Investigational New Drug process, which allows the use of experimental therapies for life-threatening conditions.
“Developing new approaches to treat drug-resistant bacteria is critical as many people with CF struggle with difficult-to-treat infections,” said JP Clancy, MD, vice president of clinical research for the Cystic Fibrosis Foundation. “Although promising results have been reported in several individual cases, studies like this one will help us evaluate the safety and efficacy of phage as a potential therapy.”
BiomX has developed BX004, a cocktail of multiple different types of phages that targets a broad range of Pseudomonas strains. The clinical trial is composed of two parts. The first part will enroll eight people with CF and test for safety, clinical activity, and drug metabolism. Results are expected in the second quarter of 2022. The second part of the trial will evaluate the safety and efficacy of BX004 in 24 people with CF. Results from the second part are expected by the third quarter of 2022.
Pseudomonas infections, which are the third most common type of lung infection in people with CF, are very difficult to eliminate once established in the airways. Approximately 32% of the nearly 31,000 people with CF in the Cystic Fibrosis Foundation Patient Registry had Pseudomonas infections in 2020, and more than 13% 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 therapy has been used previously on a case-by-case basis to treat people with CF. Rigorous clinical studies are needed to assess the safety and efficacy of phage for potential wider use in 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 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 15 industry programs to develop treatments for CF-related infections.
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.