A1 Refereed original research article in a scientific journal
Speech-based digital endpoints track ALS progression and align with standard clinical outcomes: evidence from the VRG50635 trial
Authors: Neumann, Michael; Kothare, Hardik; Bartlett, Meredith; Roesler, Oliver; Suendermann-Oeft, Christiane; Hosamath, Abhishek; Arbatti, Lakshmi; Pautler, David; Suendermann-Oeft, David; Cadavid, Diego; Scannevin, Robert H.; Hoffmann, Ines; Tarachandani, Anil; Haley, Tara; Raines, Shane; Damme, Philip Van; Tienari, Pentti; Solje, Eino; Jokela, Manu; Genge, Angela; O’Connell, Colleen; van Eijk, Ruben P. A.; van den Berg, Leonard H.; Ramanarayanan, Vikram
Publication year: 2026
Journal: Scientific Reports
eISSN: 2045-2322
DOI: https://doi.org/10.1038/s41598-026-48100-6
Publication's open availability at the time of reporting: Open Access
Publication channel's open availability : Open Access publication channel
Web address : https://doi.org/10.1038/s41598-026-48100-6
We report on the utility of speech-based digital endpoints measured during a Phase 1b study of VRG50635 in Amyotrophic Lateral Sclerosis (ALS). Fifty-four participants with ALS were enrolled and participated in an 8-week pretreatment run-in, followed by three 8-week dosing periods and an 8-week follow-up. They completed a speech assessment every two weeks in the clinic or at home. We observed moderate to high correlations between digital measures of speech timing and articulatory motor function, and the ALS Functional Rating Scale-Revised, slow vital capacity and plasma neurofilament light chain. Furthermore, speech measures can show functional decline before the ALSFRS-R does, while also capturing differences between participants with bulbar symptoms and those without. The results support the feasibility and utility of digital speech endpoints to study disease impact in ALS clinical trials.
Funding information in the publication:
This study was entirely funded by Verge Genomics.
