EX-99.3 4 clnn-ex99_3.htm EX-99.3

Steve Vucic PhD, DSc, FRACP, FAHMS1, Parvathi Menon PhD, FRACP1, William Huynh PhD, FRACP2, Colin Mahoney, PhD, MB, MRCPI2, Karen S. Ho, PhD MSc3, Austin Rynders, RN3, Jacob Evan3, Jeremy Evan, PA-C3, Robert Glanzman, MD FAAN3, Michael T. Hotchkin3, Matthew C. Kiernan PhD, DSc, MBBS, FRACP, FAHMS 1Concord Repatriation General Hospital, University of Sydney, Australia; 2Brain and Mind Centre, University of Sydney, Australia; 3Clene Nanomedicine, Salt Lake City, UT, USA Preliminary Biomarker Findings from the RESCUE-ALS Trial | A Phase 2 Double-Blind, Placebo-Controlled Study of CNM-Au8 to Slow Disease Progression in ALS Baseline Value mean (sd) Age (yrs) Sex n, (%) Male | Female Onset Site n, (%) Limb | Bulbar Months from Onset FVC (% pred.) ALSFRS-R Score ENCALS Risk Profile1 MUNIX Sum All (n=45) 59.1 (12.3) M: 26 (58%) F: 19 (42%) L: 33 (73%) B: 12 (27%) 15.8 (9.3) 81.5 (16.7) 38.7 (6.0) -4.4 (1.8) 378.2 (175.3) CNM-Au8 30mg (n=23) 57.0 (13.3) M: 13 (57%) F: 10 (43%) L: 16 (70%) B: 7 (30%) 15.5 (7.6) 84.5 (18.3) 38.6 (6.6) -4.6 (1.7) 380.2 (198.0) Placebo (n=22) 61.3 (10.9) M: 13 (59%) F: 9 (41%) L: 17 (77%) B: 5 (23%) 16.1 (10.9) 78.2 (14.5) 38.8 (5.4) -4.2 (1.8) 376.2 (152.7) Baseline Demographics 1Urinary p75ECD Results: CNM-Au8 treatment demonstrated trend for decreased p75ECD (ELISA, M. Rodgers, U. Flinders) Early symptomatic ALS Randomized (1:1, CNM-Au8 30 mg or placebo) 36-week treatment period with open label extension 1st EP: MUNIX(4) summed %change of ADM, APB, BB, & TA Exploratory pharmacodynamic markers: p75ECD, NfL, UCHL1, GFAP, untargeted proteomics, untargeted metabolomics Design Summary CONCLUSION: CNM-Au8 treatment decreased urinary p75 and plasma UCHL1 levels during the double-blind period; plasma NfL levels were predominantly stable in this early ALS population Acknowledgements: We thank the ALS study patients and their families for their support and willingness to engage in clinical research. We thank the site investigators for their research excellence and dedication to patients. We thank FightMND of Australia for substantially funding the RESCUE-ALS trial. 1. Sheapheard et al. 2020 Neurology 2017, 88 (12), 1137–1143. 2. Li et al. 2020 Ann. Clin. Transl. Neurol. 2020, 7 (8), 1420–1428. Plasma NfL Results: Levels were predominantly stable across both treatment groups (Quanterix, Simoa Assay) 2Plasma UCHL1 Results: CNM-Au8 treatment blunted increased UCHL1 vs. placebo (Quanterix, Simoa Assay) (n=21) (n=19) (n=22) (n=19) (n=23) (n=23) (n=22) (n=22) (n=23) (n=23) (n=22) (n=22) Exhibit 99.3