Safety concerns halt BX004 phage therapy trial for CF infection
Internal analysis showed unexpectedly high rate of adverse events
Biomx is ending a Phase 2b trial of BX004, its investigational nebulized phage therapy for Pseudomonas aeruginosa infection in people with cystic fibrosis (CF).
The move comes on the heels of a safety review of the trial (NCT06998043) conducted by an independent data monitoring committee, which recommended a revised dosing regimen. But after assessing these recommendations and conducting an internal analysis that revealed an unexpectedly high rate of adverse events, the company has decided to discontinue the trial.
In August, the Food and Drug Administration placed the U.S. portion of the trial on clinical hold due to questions about a third-party drug-delivery device.
“Our first priority is the improved treatment, health, and safety of patients in the cystic fibrosis community, including those who enrolled in this trial,” Jonathan Solomon, CEO of Biomx, said in a company press release. “The projected timelines and resources required to evaluate the issue and potentially proceed safely with an alternative dosing or treatment strategy were beyond the company’s currently available resources, leading us to make the difficult decision to discontinue the program.”
BX004 uses cocktail of viruses to help manage infections
In CF, genetic mutations cause the body to produce thick, sticky mucus that can clog organs and lead to a range of symptoms. Mucus buildup in the lungs makes breathing difficult and creates an environment for chronic lung infections.
P. aeruginosa, a common environmental bacterium, infects more than 60% of adults with CF and often becomes resistant to standard antibiotics over time.
“Patients living with cystic fibrosis continue to face significant challenges and require improved treatment options, including for Pseudomonas infections,” Solomon said.
Biomx’s BX004 uses a cocktail of bacteriophages — viruses that selectively attack and kill P. aeruginosa without harming humans — to help manage these hard-to-treat infections.
Patients living with cystic fibrosis continue to face significant challenges and require improved treatment options, including for Pseudomonas infections.
An eralier Phase 1b/2a trial (NCT05010577) of BX004 in adults with CF and chronic P. aeruginosa infection demonstrated encouraging safety and efficacy.
After 10 days of twice-daily inhaled treatment, three BX004-treated patients tested negative for P. aeruginosa, compared with none in the placebo group. Participants with reduced lung function at the study’s start experienced an average improvement of 5.67% with BX004, relative to those on a placebo. BX004 was well tolerated, with no serious side effects and no evidence of bacterial resistance.
The Phase 2b study was designed to evaluate BX004 for eight weeks in up to 63 participants, using twice-daily nebulized inhaled dosing. The main goal was to measure changes in P. aeruginosa levels, with additional assessments of lung function and treatment-related adverse events.
Subject to sufficient funding and other resources, BiomX will now focus on BX011, a phage cocktail targeting Staphylococcus aureus infections associated with diabetic foot infections.
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