Benzinga | Feb 2, 2021 06:42AM EST

BiomX Announces Results Of Phase 1a Pharmacokinetic Study For Inflammatory Bowel Disease/Primary Sclerosing Cholangitis Evaluating Delivery Of Oral BX002 Phage Therapy; Says BX002 Demonstrated Safety And Tolerability

- BX002 demonstrated safety and tolerability with successful delivery of a high concentration of viable phage to the lower gastrointestinal tract -

- First clinical study detailing pharmacokinetics of oral phage therapy under a U.S. FDA IND protocol -

- Efficacy results in reduction of target bacteria expected by mid-2022 from planned Phase 1b/2a study -

BiomX Inc. (NYSE:PHGE), a clinical stage company developing natural and engineered phage therapies that target specific pathogenic bacteria, today announced positive results of a first-in-human Phase 1a pharmacokinetic study of BX002. BX002 is BiomX's orally administered phage therapy candidate targeting Klebsiella pneumoniae (K. pneumoniae) bacteria in the gut, which have been linked to the pathogenesis of both inflammatory bowel disease (IBD) and primary sclerosing cholangitis (PSC). In the Phase 1a study, BX002 was demonstrated to be safe and well-tolerated, with no serious adverse events and no adverse events leading to discontinuation. In addition, the study met its objective of delivering high concentrations of viable phage to the gastrointestinal tract of approximately 1010 PFU (plaque forming units). This equals approximately 1,000 times more viable phage compared to the bacterial burden of K. pneumoniae in IBD and PSC patients as measured in stool.

"Successful oral delivery of phage therapy, which to our knowledge has now been demonstrated rigorously in a clinical study for the first time, has the potential to open up a broad range of oral phage therapy applications," commented Timothy K. Lu, M.D., Ph.D., BiomX scientific co-founder, Associate Professor of Biological Engineering, Electrical Engineering and Computer Science, and head of the Synthetic Biology Group in the Research Laboratory of Electronics, at the Massachusetts Institute of Technology. "The results show excellent safety, as expected, and the pharmacokinetics demonstrate the potential to orally administer phage in a quantity sufficient to address the gut bacterial burden of K. pneumoniae in IBD and PSC patients. In addition, these highly encouraging results highlight the strengths of the BiomX discovery and manufacturing platform and the extremely broad potential of phage technology for addressing challenges in human health."

Based on the Phase 1a study results, BiomX plans to advance to a Phase 1b/2a study evaluating the efficacy of BX003 for the reduction of K. pneumoniae in individuals that carry the target bacteria. In November 2020, BiomX announced the consolidation of its IBD and PSC programs to develop one product candidate with a broad host range for both indications, designated BX003. Results from the Phase 1b/2a study are expected by mid-2022.

The randomized, single-blind, multiple-dose, placebo-controlled Phase 1a pharmacokinetic study was conducted under an investigational new drug (IND) application approved by the U.S. Food and Drug Administration. The study evaluated the safety and tolerability of orally administered BX002 in 18 healthy volunteers. Subjects were randomized to receive orally either BX002 (n=14) or placebo (n=4), twice daily for three days. Subjects were monitored for safety for seven days in a clinical unit, with follow-up for safety assessments done at 14 and 28 days after completion of dosing. Viable phage were detected at high concentrations in samples from all subjects in the BX002 group, compared to no detected levels prior to treatment.