Verge Genomics Reports Digital Biomarkers May Track Early ALS Progression in Pre-Treatment Trial Phase

New findings from Verge Genomics suggest that digital clinical biomarkers can detect short-term ALS progression even before treatment begins, offering a potential shift in how early-stage efficacy is measured in neurodegenerative trials.

In an eight-week pre-treatment run-in phase of its Phase 1b proof-of-concept trial for VRG50635—a PIKfyve inhibitor being investigated for both sporadic and familial ALS—Verge and its collaborators observed statistically significant changes across multiple patient functions. These included mobility, breathing, sleep, and speech, all critical domains impacted by ALS.

Data were captured using a combination of AI-enabled technologies:

• Touchless wireless sensors from Emerald Innovations monitored gait, sleep efficiency, and respiratory variability in real-world home settings.

• Wearable accelerometers from ActiGraph tracked step counts and physical activity intensity.

• A web-based speech platform from Modality.AI assessed phonation and reading performance to detect articulation and respiratory changes.

The findings will be presented by Verge's Chief Medical Officer, Dr. Diego Cadavid, during a platform session at the American Academy of Neurology’s 77th Annual Meeting in San Diego on April 8.

According to the company, integrating high-frequency, real-world digital endpoints into early clinical development may improve sensitivity in detecting therapeutic effects while reducing patient burden. The Phase 1b study completed enrollment in August 2024 and remains on track to report safety and efficacy results in the second half of 2025.

Verge Genomics develops therapeutics for complex diseases using its CONVERGE platform, which combines large-scale multi-omic human datasets with machine learning. Its clinical programs currently include ALS and obesity.

Topics: Clinical Trials