Advancing NUZ-001 in ALS: Targeting TDP-43 Biology Through Adaptive Clinical Development
- The evolving understanding of TDP-43 pathology in ALS and how insights into protein aggregation and neuronal stress informed the development rationale for NUZ-001
- Preclinical and early translational findings indicating that NUZ-001 functions as a stress-adaptive modulator, supporting cellular protein quality-control processes, including proteasomal function and autophagy, to reduce neuronal stress associated with aggregated proteins such as TDP-43
- Clinical development of NUZ-001 within the HEALEY ALS Platform Trial, with discussion of the trial structure, rationale for the adaptive platform design, and how such approaches support efficient evaluation of investigational therapies in ALS
- Interpreting preliminary clinical results to date and the role of the open-label extension in strengthening the evidence base for therapeutic effectiveness
- Upcoming development milestones and data readouts, and how trial design considerations, biomarker strategy, and data maturity shape confidence in signal detection for diseasemodifying ALS therapies