Through our CardioSafe 3D and LiverSafe 3D drug rescue programs, we believe we can recapture substantial value from the prior investment by pharmaceutical companies and others who have developed and established the efficacy potential of new drug candidates that have been terminated prior to FDA approval due to heart or liver safety concerns. Using our hPSC technology, together with medicinal chemistry, we plan to generate a drug rescue pipeline consisting of new, proprietary, small molecule variants (Drug Rescue Variants) of once-promising drug candidates. We believe focusing on drug rescue candidates previously optimized for efficacy may give us a valuable “head start advantage” in our efforts to produce new, proprietary Drug Rescue Variants faster and less expensively than the drug rescue candidates discovered and developed previously using only conventional live animal models and in vitro cellular assays. We believe each lead Drug Rescue Variant will have the potential to be an FDA-approved NME in our pipeline.
The initial goal of each drug rescue program will be to produce, with our medicinal chemistry collaborator, a portfolio of Drug Rescue Variants of the once-promising but discontinued drug candidate. We will then use our CardioSafe 3D and, when validated, our LiverSafe 3D, biological assay systems to identify the lead Drug Rescue Variant in the portfolio that demonstrates an improved therapeutic index compared to the original drug candidate (that is, equal or improved efficacy with reduced toxicity or metabolism issues). We will then validate that the lead Drug Rescue Variant demonstrates reduced toxicity and/or metabolism issues in both our proprietary biological assay systems and in the same in vitro and in vivo preclinical testing model(s) that the biotechnology or pharmaceutical company used to determine efficacy and safety for the original blocked drug candidate.
We believe the results of confirmatory in vitro and in vivo safety and efficacy studies to be valuable drug rescue milestones demonstrating the improvement of our lead Drug Rescue Variant compared to the discontinued drug candidate.