HX127

HX127 is a first-in-class small molecule developed by HuntX Pharma for the treatment of Huntington's Disease (HD). The compound is designed to address axonal transport defects (ATD), a key pathological feature of neurodegenerative diseases, by specifically rescuing the axonal transport of brain-derived neurotrophic factor (BDNF). In preclinical in vivo models of Huntington's Disease, HX127 has demonstrated neuroprotective effects and the ability to reverse the characteristic triad of symptoms, including psychiatric, cognitive, and motor deficits. As of late 2024, the program is in the preclinical stage, with the company focusing on finalizing preclinical studies and preparing for regulatory safety trials following a €1.7M funding round.