Panel recommends revised dosing for trial of CF treatment BX004
Company working with FDA to resolve issues that led to clinical hold
An independent data monitoring committee, following a safety review, recommended that a Phase 2 trial of BX004, Biomx’s experimental treatment for Pseudomonas aeruginosa infection in people with cystic fibrosis (CF), continue with revised dosing.
Although Europe’s part of the study is advancing, the U.S. Food and Drug Administration (FDA) placed the U.S. portion of the Phase 2b trial (NCT06998043) on hold due to concerns about the third-party nebulizer used to administer the treatment.
The company said it is updating the trial protocol while working with the device manufacturer to address FDA information requests to lift the clinical hold. Biomx said it has been in talks with the FDA to resolve outstanding questions, which it considers “readily addressable.”
“We are encouraged by the DMC’s conclusion that the BX004 study may continue once the adjusted dosing regimen has been implemented,” Jonathan Solomon, Biomx’s CEO, said in a company press release. “We are working closely with our device manufacturer to provide the FDA with the remaining clarifications and remain confident in the path forward for BX004.”
Results expected late next year
Pending the resolution of the FDA’s clinical hold, the trial is expected to enroll about 60 CF patients with chronic P. aeruginosa lung infections. Participants will be randomly assigned to receive either BX004 or a placebo twice daily for eight weeks.
The main goal is to assess changes in bacterial burden in sputum (phlegm), with secondary goals including changes in lung function and measures of safety and quality of life up to six months after the last BX004 dose.
Top-line results are expected in the second quarter of 2026, depending on funding and other factors, Biomx said.
CF is caused by mutations in the CFTR gene that result in the loss or dysfunction of a protein of the same name. This leads to the production of thick, sticky mucus that builds up in the lungs and other organs, creating an ideal environment for infectious bacteria.
BX004 targets P. aeruginosa, a type of bacteria that often causes problematic lung infections in CF patients. The treatment uses a mixture of bacteriophages (phages), viruses that can infect and kill bacteria.
In a Phase 1b/2a trial (NCT05010577) involving adults with CF and chronic P. aeruginosa infections, BX004 improved lung function in patients who had impaired lung function at the study’s start and significantly reduced P. aeruginosa load after 10 days relative to a placebo.
The phage therapy was generally safe and well tolerated, with no serious side effects or signs of bacterial resistance to BX004.
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