Slide 1: Long-Term Open Label Extension in Stable RMS Participants with Chronic Optic Neuropathy
Phase 2 Long-Term Open Label Extension In Stable RMS Participants with Chronic Optic Neuropathy Michael Barnett, MBBS PhD FRACP FRCP On behalf of the VISIONARY-MS Investigators
Slide 2: Disclosures
Disclosures Long-Term Open Label Extension • The University of Sydney received industry standard financial renumeration as a clinical trial site • I am a consulting research director for Sydney Neuroimaging Analysis Centre (SNAC), which was contracted to analyse blinded MRI and VEP data • I am a con
Slide 3: Treatment and Participant Disposition in the Long-Term Extension
Treatment and Participant Disposition in the Long-Term Extension Long-Term Open Label Extension CNM-Au8 30mg Oral Suspension Randomized n=73 Treatment duration for Eligible for LTE up to 3-years n=69 Clean Surfaced, Highly Faceted Nanocrystals Enrolled in LTE n=55 (80%) Cellular Energetic Original C
Slide 4: Clinical Results | Long-Term LCLA
Clinical Results | Long-Term LCLA Improvement Long-Term Open Label Extension Low Contrast Letter Acuity (Original Double-Blind Primary Endpoint) Original CNM-Au8 Ex-Placebo to CNM-Au8 LTE: LS mean difference vs. randomization baseline: # p<0.0001, *** p<0.001, ** p<0.01, *p<0.05 4
Slide 5: Multi-Focal VEP | Long-Term Amplitude
Multi-Focal VEP | Long-Term Amplitude Improvement Long-Term Open Label Extension Visual Pathway Signal Strength Original CNM-Au8 Ex-Placebo to CNM-Au8 LTE: LS mean difference vs. randomization baseline: # p<0.0001, *** p<0.001, ** p<0.01, *p<0.05 5 VEP: Visual Evoked Potential
Slide 6: Multi-Focal VEP | Long-Term Latency
Multi-Focal VEP | Long-Term Latency Improvement Long-Term Open Label Extension Visual Pathway Conduction Velocity Original CNM-Au8 Ex-Placebo to CNM-Au8 LTE: LS mean difference vs. randomization baseline: # p<0.0001, *** p<0.001, ** p<0.01, *p<0.05, ^p<0.10 6
Slide 7: OCT | Long-Term Ganglion Cell Layer Preservation
OCT | Long-Term Ganglion Cell Layer Preservation Long-Term Open Label Extension GCL Volume Change in the Most Affected Eyes at Baseline Original CNM-Au8 Ex-Placebo to CNM-Au8 LTE: LS mean difference vs. randomization baseline: # p<0.0001, *** p<0.001, ** p<0.01, *p<0.05, ^p<0.10; 7 Most affected eye
Slide 8: Clinical Results | Long-Term SDMT
Clinical Results | Long-Term SDMT Improvement Long-Term Open Label Extension Working Memory and Cognition (Original Exploratory Endpoint) Original CNM-Au8 Ex-Placebo to CNM-Au8 LTE: LS mean difference vs. randomization baseline: # p<0.0001, *** p<0.001, ** p<0.01, *p<0.05 8
Slide 9: MRI DTI | Evidence for Remyelination and Axonal Integrity
MRI DTI | Evidence for Remyelination and Axonal Integrity Long-Term Open Label Extension Diffusion Tensor Imaging | T2 Lesion Axial Diffusivity and Myelination Metrics T2 Lesion MWF T2 Lesion MTR T2 Lesion Axial Diffusivity In the Cerebrum In the Cerebrum In the Cerebrum LTE: LS mean difference vs.
Slide 10: CNM-Au8 Safety
CNM-Au8 Safety Long-Term Open Label Extension CNM-Au8 treatment was safe and well-tolerated during the LTE • Treatment emergent adverse events (TEAEs) were transient and predominantly mild-to-moderate • 6 SAEs were reported over 82.9 years of cumulative participant follow-up including: (2) nephrolit