Clene Inc. · NASDAQ:CLNN
Clene — Investor Presentation (December 2021)
34 slides from Clene's investor presentation, Dec 17, 2021
Slide 1: CLNN (NASDAQ) December 14, 2021
CLNN (NASDAQ) December 14, 2021
Slide 2: Forward Looking Statements
2 Forward Looking Statements This presentation contains "forward - looking statements" within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 . Clene's actual results may differ from its expectations, estimates, and projections and consequently, yo
Slide 3: Management Team
CLENE™ BOARD CHAIR CSO, FOUNDER Management Team David J. Rob Robert Mark Michael Ted Mary Anne Matlin Etherington Glanzman Mortenson Hotchkin Jeong McNeil EMM cieonsuss® — CRtreuion Drovers GED Seco — ET] Kewsmeron-— @ieuso Creating Elemental Solutions for Human Health™ en pctene
Slide 4: CLENE Overview
4 CLENE | Overview September 30, 2021 Cash and restricted cash on hand (unaudited): $60.6M ALS Registration Trial Topline data in 2H 2022 2 >270 patient years of CNM - Au8 clinical exposure CNM - Au8® a gold nanocrystal suspension, in development as the first cellular energetic catalyst to remyelina
Slide 5: Pipeline
CLENE NANOTHERAPEUTIC } ‘cNM-AUs® g CNM-ZnAg (anes) CNM.AgzNI7 Talverane es). CNM-PtAU7 Giinonegs) InDICaTION Amyotrophic ster ms apne utp Seer | ome Pipeline PHASE? RESEARCH PRECLINICAL INDFILING PHASE PHASE PHASES ANTICIPATED RESULTS
Slide 6: Neurons With High Energetic Demand Are At Risk For Neurodegenerative Disease
6 Fu, H., et al; Nature Neuroscience (2018) 21: 1350 - 1358. Zhu et al. Proc Natl Acad Sci USA . 2015 Mar 3;112(9):2876 - 81. Rone et al. J Neurosci . 2016 Apr 27;36(17):4698 - 707. Neurons With High Energetic Demand Are At Increased Risk For Neurodegenerative Disease ~0.5% NAD + /NADH unit decline
Slide 7: Catalytically Active Nanocrystal Suspension
ϳ Vertices, Edges, & Facets Key to Catalytic Activity CNM - Au8 Catalytically Active Nanocrystal Suspension Clean Surfaced, Highly Faceted Shape Enhances Catalytic Activity CNM - Au8 Nanocrystal CNM - Au8 ® | Catalytically - Active Nanocrystals Intersection of Physics and Biology Electron Sharing Dr
Slide 8: Nanocrystal Mechanistic Effects
ϴ CNM - Au8 Nanocrystal Mechanistic Effects Improved Energy Production and Utilization Promotes Neuroprotection and Remyelination CNM - Au8 | Improves Energy Production to Promote Neuroprotection and Remyelination
Slide 9: Significant Global Opportunity
9 CNM - Au8 | Significant Global Opportunity 707,158 Ϭϯ͕ϵϬϯ 828,703 596,407 256,455 1,407,701 0 400,000 800,000 1,200,000 1,600,000 U.S. Canada W. Europe Central/E. Europe Japan China Est. Diagnosed PD Patients by Region Source: Lancet Neurol. 2018 Nov;17(11):939 - 953 ; ~6.1M patients globally, dat
Slide 10: Preclinical Evidence for Energetic Improvement
10 CNM - Au8 | Preclinical Evidence for Energetic Improvement Therapeutic Activity Across Remyelination + Neuroprotection Models ƚ&ŝ͕ EŵĚŝĐŝ͕/Đ͘ZďŝƐƚ͘^ĐŝZƉ͘ ϬϬ &ď ͖ Ϭ ; Ϳ͗ ϵϯϲ ͘ MN Neuroprotection ALS SOD1, In Vitro Forebrain Neuroprotection C9ORF72 In Vitro Alzheimer’s Disease Induced Neurons In Vi
Slide 11: Neuroprotection & Remyelination Clinical Trials
11 CNM - Au8 | Neuroprotection & Remyelination Data on File, Clene Nanomedicine, Inc. Phase 2 and Phase 3 Clinical Trials ǀŝĚĐĨED Ͳ Ƶ ϴ ƚƌƚŵƚĨĨĐƚ>^ ĚŝƐƐƉƌŐƌƐƐŝ Ongoing Established brain target engagement & safety Planned : Demonstrate Disease Modification Ongoing Established brain target engagement
Slide 12: Safety Summary
12 CNM - Au8 | Safety Summary Kǀƌ ϳϬ zƌƐĨ^ƵďũĐƚ džƉƐƵƌtŝƚŚƵƚLJ ^ĨƚLJ^ŝŐƐ Patient Exposure Across PD, MS, & ALS • Long - term dosing experience up to 115 weeks All Animal Toxicology Studies Resulted in No - Adverse Effect Level (NOAEL) Findings Clean Toxicology Findings • Multiple species up to 9 - mont
Slide 13: Phase 2 Brain Energy Metabolism Study Design
13 • D ifference in brain NAD + and NADH fraction at Week 12 (End of Treatment) • Difference in bioenergetic metabolites (e.g., ATP, NAD ) concentration at Week 12 – 16 • D ifference in brain membrane markers ( PE , PC , etc.) at Week 12 – 16 Change in Brain Bioenergetic Potential (NAD + /NADH) vs.
Slide 14: Improves Brain Energy Metabolism
14 CNM - Au8 Improves Brain Energy Metabolism Increases NAD+/NADH Ratio in MS & PD WŚƐ ZƐƵƚƐ 1 ° Endpoint 2 ° Endpoint NAD is an essential molecule responsible for cellular energy production Exploratory (ATP Normalization) Glanzman et al. 2021. International Parkinson and Movement Disorder Society (
Slide 15: RESCUE-ALS Phase 2 Study Design
15 (January 2020); FPFV 16 - Jan - 2020; 45 of 42 enrolled (Nov - 2020) WŚƐ 1 Study was powered for MUNIX primary endpoint Neurophysiology MUNIX 1 Pulmonary Function Forced Vital Capacity Function & QoL ALSFRS - R, ALSSQOL - SF Disease Progression & Survival Vucic et al. BMJ Open. 2021 Jan 11;11(1):
Slide 16: MUNIX Biomarker Primary Endpoint
16 ͮ WŝƌĚhƐĨDhE/yŝŵƌŬƌ Primary Endpoint: Spinal Cord Lower Motor Neuron Protection Muscle Fibers Spinal Cord Motor Neuron Bulbar Onset ALS (Brainstem) Limb Onset ALS (Spinal Cord) Primary Endpoint: Spinal Cord Lower Motor Neuron Motor Unit Index (MUNIX) Sum ŝĐƉƐ ďƌĐŚŝŝ Tibialis Anterior Abductor Pol
Slide 17: Evidence for Motor Neuron Protection
17 | Evidence for Motor Neuron Protection Primary Endpoint (MUNIX %, LS Mean Change) All Randomized All Placebo Limited Rate of MUNIX Decline in Bulbar Onset Limb Ƶďƌ Insufficient Spinal Cord Lower Motor Neuron Progression in Early Bulbar Trial Participants RESCUE - ALS: A Phase 2, randomized, doubl
Slide 18: MUNIX Biomarker Efficacy in Limb Onset
18 | MUNIX Biomarker Efficacy in Limb Onset Primary Endpoint (MUNIX %, LS Mean Change, Limb Onset Subset) Pre - specified: Limb Onset 45% Biceps brachii Tibialis Anterior Abductor Pollicis Brevis Abductor Digiti Minimi Spinal Cord Lower Motor Neuron Motor Unit Index (MUNIX) Sum + + н RESCUE - ALS: A
Slide 19: Significant Impact on ALSFRS-R Decline
19 | Significant Impact on ALSFRS - R Decline Exploratory (ALSFRS - R Responder Analysis, < 6 - point decline) All Randomized All Bulbar All Limb ^ƐŝƚŝǀŝƚLJ RESCUE - ALS: A Phase 2, randomized, double - blind, placebo - controlled study of CNM - Au8 to slow disease progression in ALS. MNDA Vi rtual S
Slide 20: Significant Quality of Life Improvement
20 | Significant Quality of Life Improvement Exploratory (ALS Specific QOL - SF) All Randomized RESCUE - ALS: A Phase 2, randomized, double - blind, placebo - controlled study of CNM - Au8 to slow disease progression in ALS. MNDA Vi rtual Symposium, 2021 Data on File, Clene Nanomedicine, Inc.
Slide 21: Significant Impact on ALS Disease Progression
21 | Significant Impact on ALS Disease Progression džƉƌƚƌLJĚƉŝƚ;ŝƐƐWƌŐƌƐƐŝͿ 1 ALS Disease Progression defined as : • Death, or • Tracheostomy, or • Non - invasive ventilation, or • Gastrostomy tube All Bulbar All Limb Sensitivity RESCUE - ALS: A Phase 2, randomized, double - blind, placebo - controlle
Slide 22: Joint Rank | Survival & Disease Progression
22 Joint Rank | Survival & Disease Progression ^ƵŵŵƚĚ^ĐƌŝŐdžŵƉ Berry et al. Amyotroph Lateral Scler Frontotemporal Degener . 2013 Apr;14(3):162 - 8.
Slide 23: Impact on Joint Rank Score to Week 36
23 | Impact on Joint Rank Score to Wk36 Post Hoc (Combined Assessment of ( i ) Survival, (ii) King’s Clinical Stage 4, (iii) ALSFRS - R) Data on File, Clene Nanomedicine, Inc. ALSFRS - R Decline Survival E Ͳ /ǀƐŝǀ sƚŝƚŝ Gastrostomy Tube King’s Clinical Stage 4 By Average of Summated Scores
Slide 24: Potential Impact on Survival
24 | Potential Impact on Survival Exploratory Endpoint (Observed Survival vs. Median Predicted) ENCALS Predicted Median Survival KďƐƌǀĚ^Ƶƌǀŝǀ Data on File, Clene Nanomedicine, Inc.
Slide 25: Well Tolerated & No Safety Signals
25 | Well Tolerated & No Safety Signals Data on File, Clene Nanomedicine, Inc. • 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 class • TEAEs were predominantly mild - to - mo
Slide 26: Phase 3 ALS Registration Study Design
26 Exploratory Endpoints ‡ ŵďŝĚ:ŝƚZŬ ;^Ƶƌǀŝǀн>^&Z^ Ͳ ZͿ ‡ sŝĐƉƚŚŐLJ ‡ WZK ;>^YͿ ‡ WŚƌŵĐĚLJŵŝĐŵƌŬƌƐ Slow Vital Capacity Hand - Held Dynamometry Survival Change in ALSFRS - R Registration Study: 24 - Week Treatment Period (3:1 randomization, 120 active [30mg, 60mg]: 40 placebo) 1 ι 2 ι Phase 3 Anticipat
Slide 27: VISIONARY-MS Phase 2 Study Design
ϳ Exploratory Endpoints Treatment of Vis ual Pathway Deficits I n Chronic O ptic N europathy for A ssessment of R em y elination in Non - Active R elapsing MS Change in Low Contrast Letter Acuity (LCLA) At Week 24 Up to 48 - Week Placebo - Control 2:1 Randomization (Active: Placebo ) 15mg, 30mg , Pl
Slide 28: LCLA Phase 2 Primary: Functional Visual Improvement
ϴ LC L A Phase 2 Primary: Functional Visual Improvement LC LA Correlates with clinically meaningful deficits in QOL, EDSS and MSFC, MRI, and OCT 1 MS Functional Endpoints Phase 2 Exploratory: Neuroprotection/Remyelination Endpoints The Visual System is a Window into the Brain 9 - Hole Peg Test Symbo
Slide 29: Significant Clinical Improvement Across Study Population Over 48 Weeks
29 Significant Clinical Improvement Across Study Population Over 48 - Weeks Primary Endpoint: LCLA (Best - Corrected) & Secondary Endpoint: (m)MSFC Phase 2 2 ° - (m)MSFC 1 ° - LCLA Glanzman et al. VISIONARY - MS: Update to a Phase 2 clinical trial of catalytic gold nanocrystals, CNM - Au8; ECTRIMS 2
Slide 30: Strong Intellectual Property
30 dƌĚ^ĐƌƚƐ Patent Status b Patent Description Issued & Allowed Patents 150+ Pending Applicat ions ~20 Total Patents / Applications > 170 Process And Method/Device (Clean Surface; Gold CSN) State of Matter (CNM - Au8) DƚŚĚĨhƐ ;WƌǀƚŵLJŝƚŝΘ D Ϳ Method of Use (Bi - Metallic Au/Pt; Antimicrobial) Plasma
Slide 31: Proprietary Nanocrystal Manufacturing
31 Clene | Proprietary Nanocrystal Manufacturing In - House ISO8 Clean Room Clinical Production in Maryland Designed to be Scalable to Commercialization Validated CMC Processes Patented Hydro - electro - Crystallization Proprietary Trade Secrets
Slide 32: Anticipated Timeline & Upcoming Milestones 2020-2023
32 Anticipated Timeline & Upcoming Milestones 2020 - 2023
Slide 33: Company Highlights
33 EƚŚƌƉƵƚŝĐƐWƚĨƌŵ • Potential first - in - class nanotherapeutic with high catalytic activity to drive energy production and utilization in stressed CNS cells • Applications across neurology, infectious disease, and oncology Lead Asset: CNM - Au8 for Neurorepair • CNM - Au8 improves cellular energy
Slide 34: Version: - December - 2021 /Đ͘ ,YΘŝŝĐǀƉŵƚ ϲϱϱϬ ^ƵƚŚ DŝƌĐŬ ƌŝǀ͕^Ƶŝƚ' ϱϬ ^ƚ>ŬŝƚLJ͕hd ϴϰ
© 2021 Clene Inc. Version: 14 - December - 2021 /Đ͘ ,YΘŝŝĐǀƉŵƚ ϲϱϱϬ ^ƵƚŚ DŝƌĐŬ ƌŝǀ͕^Ƶŝƚ' ϱϬ ^ƚ>ŬŝƚLJ͕hd ϴϰ ZΘĚDƵĨĐƚƵƌŝŐ ϱϬϬ WƌŝĐŝƉŝWƌŬǁLJ͕^Ƶŝƚ ϰϬϬ EƌƚŚƐƚ͕D ϵϬ