FDA puts US trial of CF treatment BX004 on hold, citing nebulizer
Biomx says it's given FDA data on 3rd-party device to address questions
The Food and Drug Administration (FDA) placed the U.S. portion of a Phase 2b trial of BX004, a treatment for Pseudomonas aeruginosa infection in people with cystic fibrosis (CF), on clinical hold because of questions about a third-party drug delivery device.
The treatment’s developer, Biomx, believes the FDA’s hold on the trial (NCT06998043) is temporary, according to a company press release.
“We are actively engaged with the FDA to promptly address their queries regarding the third-party nebulizer,” said Jonathan Solomon, CEO of Biomx. “Importantly, the FDA’s notification relates solely to the nebulizer, and we remain optimistic and confident that in response to the hold, we have provided the FDA with data that can satisfactorily support the use of the nebulizer and that this can be resolved promptly so that we may resume treating patients with cystic fibrosis in the U.S.”
While U.S. screening and enrollment are suspended, enrollment is continuing as planned in the European Union (EU), the company said. The nebulizer has EU certification that it meets safety, health, and environmental protection requirements.
CF treatment has orphan drug, fast track status
Biomx has FDA orphan drug and fast track designations, statuses that are intended to help expedite the development of promising therapies.
CF is caused by genetic mutations that make the body produce abnormally thick, sticky mucus. When this builds up in organs, it can lead to a variety of CF symptoms. Respiratory problems and lung infections are common in people with CF, as mucus buildup in the lungs allows bacteria to thrive and reproduce.
One such bacterium is P. aeruginosa, which is abundant in the environment. Although it doesn’t usually cause illness, P. aeruginosa occurs in more than 60% of adults with CF. Standard antibiotic treatments may be effective at first, but over time, P. aeruginosa can develop resistance to these therapies.
With BX004, Biomx aims to provide an alternative to antibiotics. The medication includes a mixture of bacteriophages, a type of virus that can infect and kill bacteria without harming humans. This cocktail includes several bacteriophages that target P. aeruginosa.
A Phase 1b/2a trial (NCT05010577) showed signs of safety and efficacy in adults with CF and chronic P. aeruginosa infection. Three patients who inhaled BX004 twice a day for 10 days tested negative for P. aeruginosa in secretions from the respiratory tract at the end of treatment, compared with none from the placebo arm. Participants who had impaired lung function at the study’s start saw an average 5.67% improvement in lung function relative to the placebo group.
The medication was generally safe and well tolerated, with no serious side effects. There were no signs that P. aeruginosa developed resistance to the medication during the treatment period.
The Phase 2b study is assessing BX004 for a longer time in a larger group of participants. Over eight weeks, up to 63 participants with CF will receive twice daily doses of either BX004 or a placebo. They will inhale these doses using the third-party nebulizer.
The study’s primary outcome metric is change in the amount of P. aeruginosa bacteria. Investigators will also examine lung function and incidence of treatment-related adverse events.
Before the FDA-imposed clinical hold, the trial was recruiting at several sites across the U.S., with its first patient dosed in July.
The company “remains committed to providing timely updates and full transparency to patients, physicians, and investors as the situation develops,” Solomon said.
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