Advancing ATH-1105 in ALS: Strategic Trial Design, Biomarker Integration & Adaptive Methodologies
- How refinements to the ALS program, including ATH-1105’s mechanism as a CNS-penetrant positive modulator of the neurotrophic HGF system, are shaping a larger, more globally inclusive Phase 2-ready design
- Integrating established biomarkers such as neurofilament light (NfL) and exploratory endpoints into trial architecture to align with the biological effects anticipated from ATH-1105
- Leveraging AI-supported models and external control arms to enhance signal detection and improve statistical power in a non-gene therapy small molecule ALS study
- Designing trials that reflect the heterogeneity of sporadic and familial ALS while aligning clinical outcome assessments with ATH-1105’s neuroprotective profile and translational rationale