Key highlights
- Digital endpoints measured changes in mobility, breathing, sleep, and speech — functions critically affected by ALS.
- Statistically significant measures of disease progression were identified across multiple endpoints after just eight weeks of monitoring pre-treatment, with the potential to reshape ALS clinical trial design.
- Biomarkers were gathered using:
- Touchless wireless sensors (Emerald Innovations) to monitor walking speed, turning in bed, breathing variability, sleep efficiency, and time awake after sleep onset.
- Accelerometers (ActiGraph) to track total steps and time in moderate-to-vigorous activity using wearable devices.
- An internet-based, tablet-delivered speech platform (Modality.AI) to assess sustained phonation and time to read a passage.
Leadership statements
Dr. Diego Cadavid, Chief Medical Officer of Verge Genomics, said: “We are thrilled to be the first company to evaluate multiple digital clinical biomarker endpoints in an ALS therapeutic trial to gauge short-term disease progression across key functional domains.”
Dr. Dina Katabi, President and Co-Founder of Emerald Innovations, noted: “Verge’s ALS trial was designed from the outset to continuously and objectively measure changes in various key ALS patient functions in real-world settings while minimizing patient burden.”
Christine Guo, Chief Scientific Officer at ActiGraph, commented: “This study demonstrates how continuous, high-frequency digital data can close measurement gaps and pave the way for more effective evaluation of potential new ALS treatments.”
David Suendermann-Oeft, CEO of Modality.AI, added: “The innovative study design allowed us to assess ALS-related changes in participants’ speech before their first dose of VRG50635. We observed significant changes in articulation and respiration, both critical for communication and quality of life.”
Study status
Enrollment in the Phase 1b proof-of-concept study was completed in August 2024, with efficacy and safety results expected in the second half of 2025.