Final Results from a Phase 2 Study of TPN-101 for the Treatment of C9orf72-Related Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia

Final Phase 2 study results of TPN-101 show excellent safety and potential disease-modifying effects in C9orf72-related ALS/FTD, with reduced neuroinflammation and neurodegeneration via LINE-1 inhibition. TPN-101 demonstrated significant benefits in respiratory function and ALSFRS-R scores, suggesting a global clinical benefit with longer treatment. Transposon plans to advance TPN-101 into a Phase 3 study for C9-ALS and other neurodegenerative disorders.