Thiogenesis Therapeutics is a clinical-stage biopharmaceutical company that is developing new chemical entities (‘NCE’) that are prodrugs that act as precursors to thiol-active compounds. Thiols or thiol derivatives are any organosulfur compound that has a R-SH functional group, where the functional group is responsible for chemical reactions independent of the overall compound. Highly reactive sulfur makes thiols very active in chemistry and creates several promising mechanisms of action that have been studied for decades and have potential as therapeutics.

Thiogenesis’ lead compound TTI-0102 is a disulfide, made up of two thiols that lead to independent cysteamine molecules; it has been developed to address the important obstacles for thiol-based drugs: their short half-life, strong GI side-effects and dosing limitations. As a prodrug, TTI-0102 is metabolized into cysteamine, the metabolic process acts as a gating mechanism that eliminates the spike in cysteamine, linked to side-effects and also makes it possible to administer TTI-0102 once daily. A Phase 1 safety and dose escalation study, in Australia, was successfully completed and announced in Q2-2022.

Thiogenesis is planning submissions to FDA and/or EMA for human efficacy trials in COVID-19, mitochondrial disease (‘MELAS’), Rett syndrome and non-alcoholic fatty liver disease (‘NAFLD/NASH’). As a prodrug, TTI-0102 is eligible to use the accelerated 505 (b)(2) regulatory pathway with FDA and its equivalent with EMA; which would allow the use of third-party safety data, saving time and cost in advancing to human efficacy trials.