AT the American Academy of Neurology (AAN) Annual Meeting 2025, researchers presented new data from the long-term open-label extension of the Phase II VISIONARY-MS trial, reporting imaging and physiologic findings consistent with improved neuronal function and remyelination in patients with multiple sclerosis (MS).
The study evaluated participants treated with CNM-Au8, an investigational agent that targets cellular energy metabolism. During the 144-week follow-up, patients underwent repeated brain MRI scans and multi-focal visual evoked potential (mf-VEP) assessments to track changes in neuronal integrity and signal conduction.
Findings indicated that improved mf-VEP latency and amplitude were associated with better outcomes in low contrast letter acuity (LCLA) and cognitive testing, as well as changes in MRI diffusion tensor imaging metrics—specifically magnetization transfer ratio (MTR) and axonal diffusivity (AD). These correlations suggest a link between clinical function and physiologic or anatomical changes over time.
According to the abstract, VEP and MRI data showed consistent directional improvement within individuals, aligning with earlier efficacy signals observed during the 48-week double-blind portion of the trial. Notably, this is the first Phase 2 MS study to report combined clinical, physiologic, and imaging evidence potentially indicative of remyelination.
As with all open-label research, results should be interpreted with caution. Further investigation in larger, controlled trials will be necessary to confirm these findings and determine their clinical significance.
Nonetheless, the consistency across modalities offers a potential framework for assessing remyelination in future MS studies and may inform the development of therapeutic strategies targeting neuronal repair.
Reference: Greenberg GM et al. Physiologic and Anatomical Evidence of Neuronal Repair and Remyelination from the Long-Term Open Label Extension of the Phase 2 VISIONARY-MS Trial. 2025 American Academy of Neurology (AAN) Annual Meeting, April 5-9, 2025, San Diego, California, USA.
