We are a biotechnology company with expertise applying human pluripotent stem cell (hPSC) technology for drug rescue, including predictive toxicology and drug metabolism screening. Drug rescue involves the combination of hPSC technology with medicinal chemistry to generate new, proprietary, chemical variants (Drug Rescue Variants) of once-promising small molecule drug candidates discovered, developed and discontinued by biotechnology or pharmaceutical companies prior to market approval due to unexpected heart or liver safety concerns. We believe our hPSC technology platform, Human Clinical Trials in a Test TubeTM, allows us to assess the heart and liver safety profile of Drug Rescue Variants and other new drug candidates with greater speed and precision than conventional nonclinical testing and technologies used in drug development. Our core goal is to generate a pipeline of Drug Rescue Variants and license or sell them to biotechnology and pharmaceutical companies for further development and commercialization.
A major challenge in the drug development process is that conventional animal models, altered animal cell lines and cell culture systems using human cell lines not derived from hPSCs can, at best, only approximate human biology necessary to support key drug development decisions. A biotechnology or pharmaceutical company can spend nearly a decade and tens of millions of dollars to discover, optimize and validate the potential efficacy of a promising lead drug candidate and advance it in development, only to see it be discontinued due to unexpected heart or liver safety concerns. As a result, the company’s significant prior investment may be lost. Our drug rescue model is designed to leverage both substantial prior third-party investment in discovery and development of once-promising drug candidates and the clinically predictive drug development capabilities of our Human Clinical Trials in a Test TubeTM platform to generate and assess key components of the heart and liver safety profiles of Drug Rescue Variants.
With mature human heart cells produced from hPSCs, we have developed CardioSafe 3D™, a novel, three-dimensional (3D) bioassay system for predicting the in vivo cardiac effects, both toxic and non-toxic, of Drug Rescue Variants and other small molecule drug candidates before they are tested in animals and humans. We are expanding our drug rescue capabilities by developing LiverSafe 3D™, a novel bioassay system using hPSC-derived human liver cells to assess potential liver toxicity and adverse drug-drug interactions in Drug Rescue Variants and other new drug candidates early in development, before animal and human testing.
In parallel with our drug rescue activities, we plan to explore pilot nonclinical development opportunities relating to regenerative cell therapy, with emphasis on blood, heart, liver and pancreas cells derived from hPSCs.
With grant funding from the U.S. National Institutes of Health (NIH), we have successfully completed Phase 1 development of AV-101. AV-101 is an orally available small molecule prodrug candidate aimed at the multi-billion dollar neurological disease and disorders market, including neuropathic pain, a serious and chronic condition causing pain after an injury or disease of the peripheral or central nervous system, and depression. We were awarded over $8.8 million of grant funding from the NIH to support our nonclinical and Phase I clinical development of AV-101.