GTX-196 targets the same clinically validated pathway as 5-fluorouracil (5-FU) but retains efficacy while avoiding systemic toxicity. Unlik 5-FU, GTX-196 does not cause DNA/RNA incorporation-based toxicity nor DPD-related toxicity. Moreover, GRTX-196 selectively targets de DHODH-dependent pyrimidine biosynthesis pathway, which is the preferred source of pyrimidines for cancer cells but is not used by most normal cells.
We assembled a strong and experienced team with deep expertise in oncology drug development to execute on our mission to exploit metabolic vulnerabilities in oncogene addicted cancers. The team previously navigated the development of multiple oncology drugs from the preclinical stage to the market.
Genase Therapeutics develops small molecule inhibitors of dihydroorotate dehydrogenase (DHODH), a critical enzyme in de novo pyrimidine biosynthesis and electron transport. DHODH links mitochondrial bioenergetics, cell proliferation, ROS production, and apoptosis. Building on the discovery of a novel DHODH inhibitor by prof. Sonia Lain and colleagues of the Karolinska Institute, the team has generated a unique and highly potent development candidate ready to enter IND-enabling studies.