CFF Aids Development of NO-based Therapy, BIOC51, for Resistant Bacteria, Vast Therapeutics Says

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by Santiago Gisler |

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Vast Therapeutics announced that it received funding from the Cystic Fibrosis Foundation (CFF) to support further development of BIOC51, the company’s investigative treatment for antibiotic-resistant bacteria, including Pseudomonas aeruginosa, that is often the cause of lung infections in cystic fibrosis (CF) patients.

P. aeruginosa infections set in when mucus accumulation clogs the airways, and the bacteria subsequently form extracellular biofilms (a matrix of layers of bacteria sticking to each other or to surfaces). These features make P. aeruginosa hard to treat, causing long-lasting chronic infections in CF patients.

The synthetically derived BIOC51 is designed to release controlled doses of nitric oxide (NO), a natural gas that can penetrate the mucus layers in the airways to eradicate P. aeruginosa and other bacteria. The treatment can be inhaled as a dry powder or solution, and evidence suggests it can eliminate both free-floating and biofilm-protected bacteria.

“For Vast Therapeutics to receive the support of the CF Foundation is substantial,” Michael Knowles, MD, professor of pulmonary and critical care medicine at University of North Carolina at Chapel Hill and chairman of the company’s Scientific Advisory Board, said in a press release. The award’s amount was not released.

“Given the challenges of antimicrobial resistance, we are eager to see the potential of BIOC51 materialize; the development of this innovative technology to treat CF-related respiratory infections is exciting and creates hope for better treatment outcomes,” Knowles added.

BIOC51 was able to eradicate all tested bacteria in in vitro experiments in the laboratory setting, Vast Therapeutics reported, adding that it plans to bring this treatment into clinical trials in 2019.

Tested bacteria included “over 20 species of both Gram-positive and Gram-negative bacteria, 29 distinct strains of Pseudomonas aeruginosa, and 35 antibiotic-resistant strains deemed superbugs by the Center of Disease Control and World Health Organization because of their severe threat to public health,” said Mark Schoenfisch, PhD, the company’s president and CSO, and an inventor of the BIOC51 technology.

“BIOC51 has proven effective in reducing the bacterial load of NTM [nontuberculous mycobacteria] in a murine infection model,” Schoenfisch added. “It is a testament to the Vast team and the progress we’ve made in moving this drug candidate towards clinical trials.”

BIOC51 was developed at the University of North Carolina at Chapel Hill, and received a U.S. patent in January.

The U.S. Food and Drug Administration (FDA) designated BIOC51 a Qualified Infectious Disease Product (QIDP) in November 2017. QIDP status conveys incentives to developers to advance potential antibacterial or antifungal therapies targeting serious or life-threatening infections caused by antibiotic-resistant pathogens.

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