AGS-499

AGS-499 is a synthetic small molecule activator of telomerase reverse transcriptase (TERT) developed by Neuromagen Pharma. It is designed to treat neurodegenerative and age-related diseases by increasing the expression and activity of TERT, which provides neuroprotective effects, promotes neurogenesis, and delays cellular senescence. Preclinical studies in ALS mouse models (hSOD1-G93A) have shown that AGS-499 can delay the onset and progression of the disease, increase motor neuron survival, and extend overall lifespan. The drug has received FDA Orphan Drug Designation for the treatment of ALS and is being advanced toward first-in-human clinical trials.