Clene Inc. · NASDAQ:CLNN
Clene — Investor Presentation (November 2021)
33 slides from Clene's investor presentation, Nov 9, 2021
Slide 1: CLNN (NASDAQ) November 8, 2021
CLNN (NASDAQ) N o v e mb e r 8 , 2 0 2 1
Slide 2: Forward Looking Statements
Forward Looking Statements 2 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: Creating Elemental Solutions for Human Health
Creating Elemental Solutions for Human Health Œ 3 CL E N E Ρ | M a n a g em e n t Te a m
Slide 4: CLENE Overview
CLENE | Overview September 30, 2021 Cash and restricted cash on hand (unaudited): $60.6M ALS R e g i s t r a t i o n Trial Topline data in 2H 2022 2 >230 patient years of CNM - Au8 clinical exposure CNM - Au8® a gold nanocrystal suspension, in development as the first energetic catalyst to remyelina
Slide 5: Nanotherapeutic Pipeline
NANOTHERAPEUTIC INDICATION. «RESEARCH PRECLINICAL INDFILING PHASE? ~~» PHASE? pase3. ANTICIPATED RESULTS Amyotrophic hans Lateral sad compuereD CNM-Aug? Me See ONGOING Gola Nanocrystal ‘Suspension 1H 2023 Multiple selerosis eoiione cOMPLETED Parkinson's Disease 7a SAP RESCUEPD Phase 2 i ich in 2021)
Slide 6: Neurons With High Energetic Demand Are At Risk
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 Brain Energy Potential Declines
Slide 7: CNM-Au8 Catalytically-Active Nanocrystals
Vertices, Edges, & Facets Key to Catalytic Activity 7 CNM - Au8 Catalytically Active Nanocrystal Suspension Clean Surfaced, Highly Faceted Shape Enhances Catalytic Activity CNM - Au8 Nanocrystal CNM - A u 8 ® | C a ta l y t i c a l l y - A ct i v e Na noc r y s t a l s Intersection of Physics and Bi
Slide 8: CNM-Au8 Improves Energy Production to Promote Neuroprotection and Remyelination
CNM - Au8 Nanocrystal M e ch a ni s tic Effects Improved Energy Production and Utilization Promotes Neuroprotection and Remyelination CNM - A u 8 | Im p r o v e s E n e r g y P r o d u c t i on t o Promote Neuroprotection and Remyelination 8
Slide 9: CNM-Au8 Significant Global Opportunity
CNM - Au8 | Significant Global Opportunity 70 7 , 158 10 3,9 0 3 828,703 596,4 0 7 256,455 1,407,701 0 4 00 , 0 0 0 800,000 1 , 2 00 , 00 0 1 , 6 00 , 0 0 0 U . S . C a n a d a W. Europe Central/E. Europe J a p a n C h i n a Est. Diagnosed PD Patients by Region Source: Lancet Neurol. 2018 Nov;17(11)
Slide 10: CNM-Au8 Preclinical Evidence for Energetic Improvement
CNM - Au8 | Preclinical Evidence for Energetic Improvement Therapeutic Activity Across Remyelination + Neuroprotection Models 10 Data on File, Clene Nanomedicine, Inc. Robinson et al. Sci Rep. 2020 Feb 11;10(1):1936. MN Neuroprotection ALS SOD1, In Vitro Forebrain Neuroprotection C9ORF72 In Vitro Al
Slide 11: CNM-Au8 Phase 2 and Phase 3 Clinical Trials
11 Data on File, Clene Nanomedicine, Inc. On g o i n g Established brain target e n g a geme n t & s a f e t y Planned : Demonstrate Disease Modification Ongoing Established brain target e n g a geme n t & s a fe t y CNM - Au8 | N e u r o p r o t e c t i on & R e my e li n a t i on P h a se 2 a n d
Slide 12: CNM-Au8 Safety Summary
CNM - Au8 | Safety Summary Over 230 Years of Patient Exposure Without Any Safety Signals Patient Exposure Across PD, MS, & ALS • Long - term dosing experience up to 110 weeks All Animal Toxicology Studies Resulted in No - Adverse E ff ect Level (NOAEL) Findings Clean Toxicology Findings • Multiple sp
Slide 13: CNM-Au8 Effects on Brain Energetic Metabolites
• Difference 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 • Difference in brain membrane markers (PE, PC, etc.) at Week 12 – 16 Change in Brain Bioenergetic Potential (NAD + /NADH) vs. Baseline
Slide 14: CNM-Au8 Improves Brain Energy Metabolism
CNM - Au8 Improves Brain Energy Metabolism Increases NAD+/NADH Ratio in MS & PD Phase 2 Results 1 ƒ E n dpo i n t 2 ƒ Endpoints NAD is an essential molecule responsible for cellular energy production 14
Slide 15: Phase 2 ALS Study Design
(January 2020); FPFV 16 - Jan - 2020; 45 of 42 enrolled (Nov - 2020) Phase 2 15 1 Study was powered for MUNIX primary endpoint Neurophysiology MUNIX 1 Pulmonary Function Forced Vital Capacity F u n c t i o n & Q o L ALSFRS - R, ALSSQOL - SF Disease Progression & Survival
Slide 16: Pioneered Use of MUNIX Biomarker
16 | Pioneered Use of MUNIX Biomarker Primary Endpoint: Spinal Cord Lower Motor Neuron Protection Muscle Fibers Spinal Cord Motor Ne u r o n Bulbar Onset ALS (Brainstem) Limb Onset ALS (Spinal Cord) Primary Endpoint: Spinal Cord Lower Motor Neuron Motor Unit Index (MUNIX) Sum Bi c e p s b r a chi i
Slide 17: Results Support Phase Development
| Results Support Phase 3 Development 17 Data on File, Clene Nanomedicine, Inc.
Slide 18: Evidence for Motor Neuron Protection
| Evidence for Motor Neuron Protection Primary Endpoint (MUNIX %, LS Mean Change) All Randomized All Placebo Limited Rate of MUNIX Decline in Bulbar Onset 18 Data on File, Clene Nanomedicine, Inc. L i m b Bu l b a r Insufficient Spinal Cord Lower Motor Neuron Progression in Early Bulbar Trial Particip
Slide 19: MUNIX Biomarker Efficacy in Limb Onset
| MUNIX Biomarker Efficacy in Limb Onset Bi c e p s b r a chi i + Abduc t o r Pollicis Brevis + Abduc t o r Digiti Minimi + Tibialis An t e ri o r Primary Endpoint (MUNIX %, LS Mean Change, Limb Onset Subset) Pre - specified: Limb Onset Spinal Cord Lower Motor Neuron Motor Unit Index (MUNIX) Sum 45%
Slide 20: Significant Impact on ALSFRS-R Decline
| Significant Impact on ALSFRS - R Decline Exploratory (ALSFRS - R Responder Analysis) All Randomized 20 Data on File, Clene Nanomedicine, Inc.
Slide 21: Significant Quality of Life Improvement
| Significant Quality of Life Improvement Exploratory (ALS Specific QOL - SF) All Randomized 21 Data on File, Clene Nanomedicine, Inc.
Slide 22: Significant Impact on ALS Disease Progression
| Significant Impact on ALS Disease Progression Exploratory Endpoint (Disease Progression) ALS Disease Progression defined as: • D e a t h , o r • Tracheostomy, or • Non - invasive ventilation, or • Gastrostomy tube All B ul b ar All L i m b Sensitivity 22 Data on File, Clene Nanomedicine, Inc.
Slide 23: Potential Survival Signal
| Potential Survival Signal Exploratory Endpoint (Observed Survival vs. Predicted) ENCALS Predicted 23 Data on File, Clene Nanomedicine, Inc. Observed Survival Start of O p e n L a b el Extension (Wk36+)
Slide 24: Well Tolerated & No Safety Signals
| Well Tolerated & No Safety Signals Safety Summary • No CNM - Au8 related serious adverse events (SAEs) • No CNM - Au8 related drug discontinuations • No imbalances in treatment emergent adverse event (TEAEs) • TEAEs were predominantly mild - to - moderate and transient • Most common TEAEs associat
Slide 25: Phase 3 ALS Registration Study Design
25 Exploratory Endpoints • Combined Joint Rank (S u r v i v al + AL S F R S - R) • Voice pathology • PRO (ALSAQ) • Pharmacodynamic markers Slow Vital Capacity Hand - Held Dynamometry Survival Change in AL S F R S - R Registration Study: 24 - Week Treatment Period (3:1 randomization, 120 active [30mg
Slide 26: Phase 2 VISIONARY-MS Study Design
26 Exploratory Endpoints Treatment of Vis ual Pathway Deficits I n Chronic O ptic N eu r op a t h y f o r A s se s s m e n t o f R e m y el i n a t i o n i n N o n - A c t i v e Relapsing MS Change in Low Contrast Letter Acuity (LCLA) At Week 24 Up to 48 - Week Placebo - Control 2:1 Randomization (A
Slide 27: LCLA Functional Visual Improvement Endpoint
27 LCLA Phase 2 Primary: Functional Visual Improvement LCLA Correlates with clinically meaningful deficits in QOL, EDSS a n d M S F C , MR I , a n d O C T 1 MS Functional Endpoints Phase 2 Exploratory: Neuroprotection/Remyelination Endpoints The Visual System is a Window into the Brain ( T L T V + E
Slide 28: Significant Clinical Improvement Over 48 Weeks
28 Significant Clinical Improvement Across Study Population Over 48 - Weeks Primary Endpoint (LCLA, Best - Corrected) Phase 2 Low Contrast Letter Acuity (2 . 5 % G r e y - S c a l e )
Slide 29: Strong Intellectual Property
Trade Secrets Patent Status b Issued & Allowed Patents 130+ Pending Applications >30 Total Patents/ Applications >160 Patent Description Process And Me t h o d / D e v i c e (Clean Surface; Gold CSN) State of Matter (CNM - Au8) Method of Use (Prevent Demyelination & MoA) Method of Use (Bi - Metallic
Slide 30: Proprietary Nanocrystal Manufacturing
Clene | Proprietary Nanocrystal Manufacturing In - House ISO8 Clean Room Clinical Production in Maryland Va l i d a t e d C M C Processes Designed to be Scalable to Commercialization Patented H y d r o - el ec t r o - Crystallization P r o p r i e t a r y T r ade Secrets 30
Slide 31: Anticipated Timeline & Upcoming Milestones 2020-2023
Anticipated Timeline & Upcoming Milestones 2020 - 2023 31
Slide 32: Clene Company Highlights
Na n o t h e r ap e u t i cs Platform • 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: C N M - A u 8 f o r Neurorepair • CNM - Au8 improves
Slide 33: Clene Inc. Locations
Clene Inc. HQ & Clinical Development 6 55 0 S o u th Mill r o c k D ri v e , S u i te G 5 0 S al t La k e C i t y , U T 8 4 1 2 1 R&D and Manufacturing 5 00 P ri n c i p i o P ar k w a y , S u i te 4 00 North East, MD 21901 © 2 0 21 C l e n e In c . Version: 8 - November - 2021