Abstract Details

CNM-Au8 is a neuroprotective investigational drug that catalytically enhances cellular metabolic energy. Results from the double-blind period demonstrated that CNM-Au8 treatment improved low contrast letter acuity (LCLA, 1° endpoint) and global neurological function assessed by the modified MS Functional Composite (2° endpoint) compared to placebo in stable RMS patients receiving approved disease-modifying immunomodulatory therapy (DMTs) as standard of care. Here we present analyses from the long-term open-label extension (LTE) study of participants from VISIONARY-MS LTE.

VISIONARY-MS LTE trial assessed the long-term safety, efficacy, tolerability, and pharmacokinetics of CNM-Au8 30mg on participants with stable relapsing MS (RMS) over 96 weeks following completion of the 48-week double-blind period.

The VISIONARY-LTE participants received daily doses of CNM-Au8 30mg. In addition to safety, functional endpoints were assessed at clinical visits every 12 weeks: LCLA for each eye by 2.5% Sloan letter chart, modified MSFC score that including the Timed 25-Foot Walk (T25FWT), Symbol Digit Modality Test (SDMT), 9-Hole Peg Test (9HPT, dominant and non-dominant hands), and LCLA (both eyes).

55 of 69 eligible participants (80%) originally randomized were eligible and participated in the LTE. The LS-mean difference (SE) at week 144 for: (i) LCLA change across both eyes vs. the original randomization baseline of participants assigned to CNM-Au8 was +8.7 letters (1.88), 95% CI: 5.0-12.4, p<0.0001 (ii) SDMT change vs. the original randomization baseline of participants assigned to CNM-Au8 was +8.03 (1.52), 95% CI: 5.01-11.0, p<0.0001. Improvements during the double-blind period were maintained in the LTE for T25FWT and 9PHT. Individual treatment responses showed consistent improvement with substantial gains in low contrast vision and cognition. Observed LCLA change from the end of the double-blind period ranged up to 38 letters across both eyes.

These results provide long-term evidence for sustained improvements in neurological functions in stable RMS patients treated with CNM-Au8 adjunctively to DMTs.