Vast Therapeutics’ Therapy Candidate BIOC51 Fares Well in Mouse Studies

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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Vast Therapeutics

Vast Therapeutics, formerly known as Novoclem Therapeutics, recently announced that the company’s first controlled nitric oxide (NO) release powder-based therapy candidate, BIOC51, can substantially reduce the bacterial load of nontuberculous mycobacteria (NTM) in the lungs of animal models of cystic fibrosis.

Vast Therapeutics is a preclinical stage pharmaceutical company exploring the power of NO in a synthetic powder-based form for the treatment of severe respiratory infections in patients with CF and other respiratory diseases.

BIOC51 is its proprietary water-soluble, NO-releasing biopolymer that has been modified with a specific type of NO to facilitate spontaneous and controlled NO release. (“Spontaneous” means it discards the need of additional enzymes.)

The level of NO release from the BIOC51 has shown positive results in eradicating planktonic and biofilm-based bacteria, and can be delivered to the lungs as a dry powder or solution, for instance, via nebulization.

Now, BIOC51 was found to eradicate several multi-drug resistant bacteria tested in vitro, and to achieve a 99 percent reduction in NTM numbers in mouse models.

“We are excited to have BIOC51 perform at the levels proved in our study. Achieving a 99% reduction in bacterial count and exceeding the performance of standard of care antibiotics validates our in vitro testing results.  Furthermore, the study confirms that our drug was delivered safely without observable adverse side effects,” Mark Schoenfisch, president and chief scientific officer of Vast Therapeutics, said in a press release.

In in vitro tests, BIOC51 has eradicated every bacteria pathogen to date, including more than 19 families of both Gram-positive and Gram-negative bacteria, 24 distinct strains of Pseudomonas aeruginosa, and 35 antibiotic-resistant strains — also known as superbugs, because of the severe threat they pose to public health.

“Our timed released drug candidate is more effective than conventional antibiotics and gas-based NO therapies, both of which have efficacy and ease-of-use limitations. In particular, today’s drugs are susceptible to antibiotic resistance because they generally function via a single mode of action that bacteria mutate to overcome. We believe our water-soluble powder-based drug candidate avoids this mode of resistance due to the innate characteristics of NO itself and its multiple modes of action,” Schoenfisch said.

Neal Hunter, Vast Therapeutics’ CEO, added: “The coupling of in vivo results with apparently being the first in the world to achieve this breadth of in vitro eradication exceeds our expectations. It heightens our responsibility to move our druggable nitric oxide into the clinic. We look forward to making a difference in reducing the bacterial burden in patients with lung infections, particularly for those in critical need, like patients with cystic fibrosis.”

The results from this study, conducted in partnership with Colorado State University, drove the name change from Novoclem to Vast Therapeutics.

“Our original name was related to a specific goal in cystic fibrosis. While we remain dedicated to CF as our initial indication, our results point toward a much broader capability in the respiratory and general anti-infective spaces,” Hunter said.

Novoclem was behind important advances in the field of CF research.

In November 2017, BIOC51 was given qualified infectious disease product (QIDP) status by the U.S. Food and Drug Administration as a potential treatment for recurrent lung infections caused by bacteria.

In January, BIOC51 was granted a U.S. patent as a possible inhalable treatment for antibiotic-resistant bacterial infections in people with cystic fibrosis due to P. aeruginosa.