The Novel Biomarker Clinical Endpoints Identified Measure Changes in key functions affected by ALS such as mobility, breathing, sleep and speech

Verge Genomics, a clinical-stage, tech-enabled biotechnology company pioneering the use of artificial intelligence (AI) and human data to transform drug discovery and development, today announced new findings demonstrating that multiple digital clinical biomarker endpoints can capture measurable disease progression during the eight-week, pre-treatment run-in period of the Phase 1b proof-of-concept study of VRG50635, an investigational PIKfyve inhibitor for sporadic and familial amyotrophic lateral sclerosis (ALS).

Key Highlights

• Digital endpoints measured changes in mobility, breathing, sleep, and speech, functions that are critically affected by ALS.
• Statistically significant measures of disease progression were identified across multiple endpoints after just 8 weeks of monitoring pre-treatment, which has the potential to revolutionize 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. The Emerald system uses AI/ML-enabled, touchless wireless sensors in participants’ homes to track real-world functional changes.
  • Accelerometers (ActiGraph) to track total steps and time in moderate-to-vigorous activity using wearable devices. The Actigraphy system can measure activity, mobility, and sleep patterns.
  • Internet-based, tablet-delivered speech platform (Modality.AI) to assess sustained phonation and time to read a passage using an AI-enabled, web-based speech assessment platform.

Dr. Diego Cadavid, Chief Medical Officer of Verge Genomics, will present these data in a platform session at the 77th Annual Meeting of the American Academy of Neurology in San Diego on Tuesday, April 8, 2025. “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,” said Dr. Cadavid. “Given the high unmet need and complexity of ALS, it is critical to leverage innovative technologies early in clinical development to assess potential treatment benefits.”

“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,” said Dr. Dina Katabi, President and Co-Founder of Emerald Innovations.

“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,” added Christine Guo, Chief Scientific Officer at ActiGraph.

“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,” said David “DSO” Suendermann-Oeft, CEO of Modality.AI.

Enrollment in the Phase 1b proof-of-concept Study of VRG50635 for treatment of ALS was completed in August of 2024, and the study is on track to deliver efficacy and safety results in the second half of this year. More information about this ongoing ALS Study is available at: https://clinicaltrials.gov/study/NCT06215755

About Verge Genomics

Verge is focused on developing therapeutics for complex human diseases with high unmet need, using genomics and machine learning. Verge has created a proprietary all-in-human platform, CONVERGE®, featuring one of the field’s largest and most comprehensive databases of multi-omic patient data. The company is led by experienced computational biologists and drug developers who are successfully advancing therapeutic programs in ALS and obesity toward the clinic. For additional information, please visit www.vergegenomics.com. Follow us on LinkedIn and X.

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