Developing superior and novel vaccines

...using immunogenic proteins in optimal conformation.

Large & Rapidly Growing Market

Annual Vaccine Sales ($Billions)


Sources:
1. WHO Projections 2014
2. Global Vx Mkt Pipeline Analysis 2014 by PNS Pharma

Our Pipeline

We carefully select our target disease areas and vaccine candidates to address areas of significant unmet medical need based on the following criteria: well-defined commercial landscape and efficient market adoption, low biological risk and established clinical pathways. The following table summarizes our current pipeline:

Infographic: PipelineOur lead vaccine candidate, VAX-24, is a preclinical, 24-valent PCV designed to provide the broad-spectrum coverage of Pneumovax 23 with an immunogenicity profile comparable to Prevnar 13. Our second PCV, known as VAX-XP, builds on the technical proof of concept established by VAX-24 and would, if approved, expand the breadth of coverage to at least 30 strains, including emerging strains responsible for IPD and antibiotics resistance, without compromising immunogenicity due to carrier suppression.

Our preclinical proof of concept studies for VAX-24 measured serotype-specific IgG antibody responses, the surrogate endpoint for pediatrics, and opsonophagocytic activity, or OPA, responses, the surrogate endpoint for adults of our vaccine candidates against Prevnar 13 and Pneumovax 23. In these studies, our vaccine candidates have shown comparable responses to the 13 common strains in Prevnar 13 and superior responses to the 23 common strains in Pneumovax 23.

We believe our PCVs could receive regulatory approval based on a demonstration of non-inferiority to the standard of care using well-defined surrogate immune endpoints, consistent with how other PCVs have obtained regulatory approval in the past, rather than requiring clinical field efficacy studies. We expect to submit an investigational new drug, or IND, application for VAX-24 to the U.S. Food and Drug Administration, or FDA, in the second half of 2021.

In addition to our PCV franchise, we are developing a novel conjugate vaccine candidate for Group A Strep. Group A Strep causes 700 million cases, the majority of which are of pharyngitis, commonly known as strep throat, worldwide each year and increases the risk for severe invasive infections, such as sepsis, necrotizing fasciitis and toxic shock syndrome. There is currently no vaccine against Group A Strep. In September 2019, we announced a grant of up to $15.1 million, awarded by CARB-X, a global non-profit partnership dedicated to accelerating antibacterial innovation to tackle the rising global threat of drug-resistant bacteria, to develop this vaccine candidate.

We are also developing a novel protein vaccine candidate targeting the keystone pathogen responsible for periodontitis, a chronic oral inflammatory disease affecting an estimated 65 million adults in the United States. Our initial goal is to develop a therapeutic vaccine to slow or stop disease progression; however, the results from clinical trials may inform the potential adoption of prophylactic immunization.

References

1. For the Polysaccharide/Alum comparator, we used 23 polysaccharides in Pneumovax 23 at an equivalent dose with the addition of strain 6A and alum as the objective of the study was to evaluate whether VAX-24 showed a conjugate-like response in all 24 strains.
2. For the Polysaccharide/Alum comparator, we used 23 polysaccharides in Pneumovax 23 and 9 additional polysaccharides with alum for comparison.