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Robert Glanzman, MD FAAN For the VISIONARY - MS Investigators R . G l a n z m a n , M D F AA N 1 , H . Bea d n a l l MBBS F R A C P 2 , A . Klistorner , M. Barnett, MBBS FRACP 2 , R. Sergott MD 3 , A. Rynders 1 , K. Ho, PhD 1 , M. Mortenson MS 1, M. Hotchkin 1 1 Holladay, UT/United States, 2 Camperdown, NSW/Australia, 3 Philadelphia, PA/United States of America P05 0 Update to a Phase 2 clinical trial of catalytic gold nanocrystals, CNM - Au8, for the treatment of chronic optic neuropathy 1
Disclosures I a m a n e m p l o y e e o f C l en e N a n o m e d i c i n e , I n c . and receive salary and stock options Acknowledgements Dr. Heidi Beadnall MBBS, FRACP Dr. Steve Vucic MBBS FRACP, PhD Prof Neurology Dr. Jeanette Lerchner - Scott MD, PhD, FRACP Dr. Stefan Blum MD, FRACP, PhD Dr. Bruce Taylor B m e d sc i , M B B S , MD , F R A CP Dr. Deborah Field MBBS, FRACP Dr. Richard Macdonell MD , F R A CP , F A F RM 2 Dr. Anneke Van der Walt MBChB, PhD, FRACP A/Prof Neurology
C N M - Au8 | M o A : N a n o c a t a l y t i c E l e c t r o n T r a n s f e r 3
Background DMT Treatment & Demographics 1 3 - J a nu a r y - 2021 B li n d e d D a ta U pd a te 4 B as e li ne Values Sub j e ct s n (%) Age [ y r s . ] mean (SD) EDSS m ea n (SD) Time from MS Onset [yrs.] m ea n ( SD ) ON History [%] All 52 38.0 1.8 6.0 76% (100%) (8.9) (1.5) (3.8) Female 37 37.7 1.7 6.4 73% (71%) (8.3) (1.6) (3.8) Male 15 40.1 2.0 5.1 80% (29%) (9.7) (1.4) (3.8) Mo n oclo n al Antibody 1 n (%) Oral Ther a py 2 n (%) Injectable ( Gla t iram er acetate) n (%) No DMT n (%) 27 (52%) 19 (36.5%) 2 (4%) 4 (8%) 19 (51%) 14 (38%) 2 (5%) 2 (5%) 8 (53%) 5 (33%) 0 (0%) 2 (13%) 1 Monoclonal antibody includes alemtuzumab, natalizumab, rituximab, ocrelizumab. 2 Oral includes fingolimod, dimethyl fumarate, teriflunomide, thyroxine, and oral combinations. DMT Background Treatment Baseline Demographics
5 Treatment of Vis ual Pathway Deficits I n Chronic O ptic N europathy for A ssessment of R em y elination in Non - Active Relapsing MS Change in Low Contrast Letter Acuity (LCLA) At Week 24 Up to 48 - Week Placebo - Control 2:1 Randomization (Active: Placebo ) 1 5 m g , 3 0 m g , P l a c e b o (n = 1 5 0) Exploratory Endpoints Optical Coherence Tomography (OCT) Multi - focal VEP Amplitude & Latency Full field - VEP Amplitude & Latency MRI Endpoints Visual Function (High Contrast) PRO / QOL / EDSS 1 2 Ch a ng e Co mp o s i t e Clinical Response 9HPT / SDMT / T25FW / LCLA / EDSS - 42 to - 1 24 - Week Blinded Fixed Treatment Period Up to 24 - Week Blinded Extension Period (Until LPLV 24Wk Visit) LPLV *Subject to ongoing COVID - 19 related site research restrictions generally implemented to protect MS patients taking standard - of - care immunosuppressive therapies
( T L T V + E = 1 2 3 4 5 6 7 8 9 KEY SDMT Z - Score Change Change in Enrolled Study Population Compared to Baseline (BL) Mild' EDSS (<=1.5) 6
9HPT (Non - Dominant) Z - Score Change Change in Enrolled Study Population Compared to Baseline (BL) Mild' EDSS (<=1.5) 7
T25FW Z - Score Change Change in Enrolled Study Population Compared to Baseline (BL) Mild' EDSS (<=1.5) 8
LCLA (Best - Corrected) Z - Score Change Change in Enrolled Study Population Compared to Baseline (BL) Mild' EDSS (<=1.5) 9
(m)MSFC Mean Z - Score Change 6 - component (SDMT, LCLA [L/R], 9HPT [D/ND], T25FW) ( T L T V + E = 1 2 3 4 5 6 7 8 9 KEY 10
Conclusions These interim, blinded data support the potential for CNM - Au8 therapy to demonstrate meaningful neurological improvement in patients with MS VISIONARY - MS is an innovative Phase 2 study examining the potential for CNM - Au8 to promote neurological improvement in a stable RMS population with chronic visual impairment, as an adjunct to approved DMTs CNM - Au8 is a novel, nanocatalytic therapy shown to promote remyelination and neuroprotection via increasing bioenergetic capacity, enhancing protein homeostasis, and reducing harmful ROS 11