Slide 1: RESCUE-ALS Phase 2 Trial Topline Results
Phase 2 Results RESCUE-ALS Phase 2 Trial Topline Results 1
Slide 2: Disclosures & Acknowledgements
Disclosures & Acknowledgements • Robert Glanzman, MD FAAN is an employee of Clene Nanomedicine, Inc. • Funding support from FightMND Australia is gratefully acknowledged • We thank ALS patients and their caregivers for participating in RESCUE-ALS • Presenting on behalf of trial investigators 2
Slide 3: Oral CNM-Au8 Improves Energy Production to Promote Neuroprotection and Remyelination
Oral CNM-Au8 | Improves Energy Production to Promote Neuroprotection and Remyelination CNM-Au8 Mechanistic Improved Energy Promotes Nanocrystal Effects Production and Neuroprotection and Utilization Remyelination 3
Slide 4: RESCUE-ALS | Design & Baseline Demographics
RESCUE-ALS | Design & Baseline Demographics 36-Week Blinded Treatment Period with Long-Term Open-Label Extension Baseline Wk12 Wk24 Wk36 Wk48 Wk60 Wk72 Wk84 Wk96 Wk108+ Screening (4-weeks) Double-Blind Period Long-Term Open Label Extension Baseline Sex Onset Site Months ALSFRS- ENCALS Value Age n, (
Slide 5: Evidence for Motor Neuron Protection
Evidence for Motor Neuron Protection Primary Endpoint (MUNIX %, LS Mean Change) All Placebo All Randomized Limited Rate of MUNIX Decline in Bulbar Onset +45% Insufficient Spinal Cord Lower Motor Neuron Progression in Early Bulbar Trial Participants 5 RESCUE-ALS: A Phase 2, randomized, double-blind,
Slide 6: Significant Impact on ALSFRS-R Decline
Significant Impact on ALSFRS-R Decline Exploratory (ALSFRS-R Responder Analysis, < 6-point decline) All Randomized All Bulbar Sensitivity Analysis All Limb 6 RESCUE-ALS: A Phase 2, randomized, double-blind, placebo-controlled study of CNM-Au8 to slow disease progression in ALS. MNDA Virtual Symposiu
Slide 7: Significant Quality of Life Improvement
Significant Quality of Life Improvement Exploratory (ALS Specific QOL-SF) All Randomized 7 RESCUE-ALS: A Phase 2, randomized, double-blind, placebo-controlled study of CNM-Au8 to slow disease progression in ALS. MNDA Virtual Symposium, 2021 Data on File, Clene Nanomedicine, Inc.
Slide 8: Significant Impact on Disease Progression
Significant Impact on Disease Progression Exploratory Endpoint (Disease Progression) All Randomized All Bulbar Sensitivity 1ALS Disease Progression defined as: • Death, or • Tracheostomy, or All • Non-invasive ventilation, or Limb • Gastrostomy tube 8 RESCUE-ALS: A Phase 2, randomized, double-blind,
Slide 9: Joint Rank Trend | Survival & ALSFRS-R
Joint Rank Trend | Survival & ALSFRS-R Exploratory Endpoint Pre-specified (Combined Assessment of Survival and Function [CAFS]) All Randomized Score participants CAFS based on relative function or time of death Survival ALSFRS-R Scoring Decline 9 Berry et al. Amyotroph Lateral Scler Frontotemporal D
Slide 10: Impact on Long-Term Survival
Impact on Long-Term Survival All Randomized All OLE Participants (CNM-Au8 Treated) 10
Slide 11: Impact on Long-Term Survival | by Randomization Group
Impact on Long-Term Survival | by Randomization Group All CNM-Au8 Randomized All Placebo Randomized 11
Slide 12: Safety Summary | Well Tolerated & No Safety Signals
Safety Summary | Well Tolerated & No Safety Signals • No CNM-Au8 related serious adverse events (SAEs) • No CNM-Au8 related drug discontinuations • No imbalances in treatment emergent adverse event (TEAEs) by system organ classification • TEAEs were predominantly mild-to-moderate and transient • Mos
Slide 13: Conclusions
Conclusions Evidence of CNM-Au8 therapeutic efficacy ü Improved survival ü Significant slowing in disease progression ü Significant reduction in functional decline ü Significant improvement in quality of life ü Preservation of lower motor neurons CNM-Au8, well tolerated and safe in ALS Larger