GTX-11m

GTX-11m is a small molecule and the active metabolite of GTX-11, an orally available drug candidate developed by GAT Therapeutics for the treatment of fibrotic diseases, including idiopathic pulmonary fibrosis (IPF) and metabolic dysfunction-associated steatohepatitis (MASH). GTX-11m acts by modulating the transforming growth factor-beta (TGF-β) signaling pathway, preventing the activation of downstream mediators SMAD2 and SMAD3. This dual antifibrotic and immunomodulatory mechanism deactivates hepatic stellate cells, reduces fibroblast-to-myofibroblast transition, and attenuates pro-inflammatory and profibrotic gene expression in macrophages. Preclinical studies have demonstrated that GTX-11 reduces lung and liver fibrosis, inflammation, and vascular remodeling. GTX-11 has successfully completed a Phase I clinical trial in healthy volunteers.