Clene Inc. · NASDAQ:CLNN
Clene — Investor Presentation (May 2021)
32 slides from Clene's investor presentation, May 12, 2021
Slide 1: CLNN (NASDAQ)
CLNN (NASDAQ)
Slide 2: Forward Looking Statements
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, you
Slide 3: Clene Investment Highlights
3 Unmet Medical Need & Market Opportunity Lead Asset: CNM - Au8 for Neuro Repair Clinical D e v e lo p m e nt Pipeline Strong IP Portfolio Financials CNM - ZnAg for COVID - 19 CLENE | Investment Highlights • Nanocatalyst of Intracellular Biological Reactions • Robust Preclinical Remyelination & Neur
Slide 4: Management Team
Lanxide Corporation Dupont Lanxide Composites Lanxide Armor Company Lanxide Performance Materials Lanxide Electronic Components CDO CLENE | Management Team Ted Jeong , DM 4
Slide 5: Platform & Pipeline
Novel electro - chemistry platform produces catalytic Clean Surface Nanocrystal drugs designed to avoid toxicities associated with synthetic chemistry 130+ Gr a n t ed Patents CLENE | Platform & Pipeline (Anticipated Launch in 2021) Brain Imaging Biomarker Study Brain Imaging Biomarker Study 1 H 2 0
Slide 6: Evolution of Gold as a Therapeutic Modality
Chinese & Ayurvedic Gold Preparations (China, Arabia, India) Monoatomic Gold Salts for Rheumatoid Arthritis (IM Sodium Aurothiomalate; IM Aurothioglucose; Oral Auranofin) gold 3,4,5 - triacetyloxy - 6 - (acetyloxymethyl oxane - 2 - thiolate; triethylphosphanium Au ( Gold) 2020+ Catalytic Clean Surfa
Slide 7: Bioenergetic Nanocatalyst
CNM - Au8 | Bioenergetic Nanocatalyst Improved Cellular Energetic Support E n e r g y > 100 Trillion Nanocrystals per 60 mL Dose (At 30mg) Oral Suspension; Once Daily 13 nm Median Diameter (R i b o s o me = 2 0 - 30 n m ) Clean Surfaced Faceted Nanocrystal Novel mechanism of action to address a rang
Slide 8: Integrating Physics With Biology
CNM - Au8 | Integrating Physics With Biology Electron Transfer Is Fundamental to Energy Production Surface Based C a t a l y t i c A c t i v i t y Electrons (e - ) Move Freely Across Na n o c r y s t a l S u r f ace V e r t i c e s , E d g e s , & Faces Key to Catalytic Activity AuNP Catalyzed Oxida
Slide 9: Treating Bioenergetic Failure
Treating Bioenergetic Failure | Common Pathological Mechanism In Neurodegenerative Disorders (MS, ALS, PD) Respiratory In s u f f ici e ncy D y sp h a g i a / Dysarthria Cognitive Im p a i r m e nt Muscle Weakness/Atrophy ALS Neuronal Metabolic Failure MS Oligodendrocyte Remyelination Failure and Ne
Slide 10: Mechanism of Action Therapeutic Effects
CNM - Au8 | MOA Therapeutic Effects a Nicotinamide Adenine Dinucleotide a 10
Slide 11: Significant Global Opportunity
11 CNM - Au8 | Significant Global Opportunity 707,158 103,903 828,703 596,407 256,455 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 So u r c e : La n c e t N eurol . 2 0 1 8 N
Slide 12: Evidence for Bioenergetic Improvement
12 CNM - Au8 | Evidence for Bioenergetic Improvement Therapeutic Activity Across Remyelination + Neuroprotection Models 1 Robinson et al. Sci Rep. 2020, DOI: 10.1038/s41598 - 020 - 58709 - w. . 2 Ho et al. Society for Neuroscience Meeting, 2019. Ho et al. Motor Neuron Disease Associating Meeting, 20
Slide 13: MOA & Remyelination Data Published
CNM - Au8 | MOA & Remyelination Data Published Robinson et al. Sci Rep. 2020 Feb 11;10(1):1936. doi: 10.1038/s41598 - 020 - 58709 - w Lysolecithin R e m y e li n a ti o n Data 13
Slide 14: Clinical Program Overview
Successful Phase 1 First - In Humans Safety Tr i al + C h r o n ic Animal Toxicity Studies P h a s e 2 B r a i n T a r g e t Engagement 31 P - Magnetic Resonance Phase 2 MS Clinical Remyelination & Neurorepair CNM - Au8 | Clinical Program Overview Phase 3 Phase 2 & 3 ALS Clinical Neurorepair Phase 2
Slide 15: Clean Toxicology Findings
CNM - Au8 | Clean Toxicology Findings All Studies Resulted in No Adverse Effect Level (NOAEL) a Standard ICH M3(R2) Toxicology Program Genotoxicity I n V itro & I n V ivo (Rodent) Safety Pharmacology CNS, CV, Renal Dos e R a n ge Finding Rodent, Minipig Single Dose To x i c o ki n e t i c s Canine M
Slide 16: Phase 1 First In Human Study Completed
• Most frequent TEAEs by System Organ Class: Nervous/GI - Nearly all of the TEAEs were Grade 1 s e v e r i t y (m i l d ) • No serious TEAEs, TEAEs leading to discontinuation of treatment, or TEAEs considered severe, life - threatening, or resulting in death • No dose responsive TEAEs observed in SA
Slide 17: Phase 2 Brain Target Engagement Study Design
17 • Difference in NAD+ and NADH fraction at Week 12 - 16 • Difference in other bioenergetic metabolites (e.g., ATP, PCr, 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 1 ι 2 ι C
Slide 18: NAD+/NADH Age Related Decline By P-MRS Imaging
NAD + /NADH | Age Related Decline By 31 P - MRS Imaging Zhu et al. Proc Natl Acad Sci USA . 2015 Mar 3;112(9):2876 - 81. 18 Closed squares = averaged data by age group: 21 – 26 yrs, 33 – 36 yrs, and 59 – 68 yrs old; Open squares= individual subject values ~0.05% NAD + /NADH unit decline per year (~0
Slide 19: Improved Brain Metabolic Markers in MS
CNM - Au8 Improved Brain Metabolic Markers Elevated NAD+/NADH & Normalized ATP Levels in MS Glanzman, R., J. Ren, A. Rynders, B. Greenberg, R.B. Dewey, K. S. Ho, and M. T. Hotchkin. “Effects of Nanocatalysis on CNS Bioenergetic Markers in Patients Treated with CNM - Au8: Interim Results from a Phase
Slide 20: Phase 2 ALS Study Design
(January 2020); FPFV 16 - Jan - 2020; 45 of 42 enrolled (Nov - 2020) Phase 2 Anticipated full unblinded data readout: 2H 2021 Exploratory Endpoints • Other Electromyography (SH i, NP i, MUSIX, MScan) • ALSFRS - R • Change in Rate of ALSFRS - R progression • QOL • Combined Joint - Rank (Survival + AL
Slide 21: Predictive Endpoints of Disease Progression
Predictive E n dp o i n t s of Disease Progression Clinical E n dp o i n t s • ALSFRS - R • Pulmonary Function (Vital Capacity) • Mortality • Loss of Motor Units M o t o r Un i t I n d e x ( MU N IX) Measuring ALS Disease Progression Electromyography Predicts Clinical Progression Historical Longitud
Slide 22: Emerging Evidence of Primary Endpoint Improvement
Robert Glanzman MD FAAN, et al. “A Blinded Interim Update on RESCUE - ALS: A Randomized, Placebo - Controlled, Phase 2 Study to Determine the Effects of CNM - Au8 to Slow Disease Progression in Amyotrophic Lateral Sclerosis” Presented at ENCALS 2021 Virtual Meeting, 12 - May - 2021. Emerging Evidenc
Slide 23: Emerging Evidence of Clinical Improvement
SVC Avg. Slope Decline (% p oi n ts/mo n t h) Slope Est. (9 - mo n t h s) Empower ( − 2.73%) - 24.6% Benefit ( − 2.74%) - 24.7% PRO - ACT ( - 2.90%) - 26.1% − 1 0 . 5 − 1 9 . 6 − 6 . 7 Andrews et al. JAMA Neurol. 2018;75(1):58 - 64. Phase 2 23 Emerging Evidence of Clinical I m p rovem e n t | F or c
Slide 24: Phase 3 ALS Registration Study
Exploratory Endpoints • Combined Joint Rank (Survival + ALSFRS - R) • Voice pathology • PRO (ALSAQ) • Pharmacodynamic markers Slo w V it a l C a p a city Hand Held Dynamometry Change in AL S F R S - R Registration Study: 24 - Week Treatment Period (3:1 randomization, 120 active [30mg, 60mg]: 40 plac
Slide 25: Phase 2 MS Study Design
25 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 Relapsing MS Change in Low Contrast Letter Acuity (LCLA) At Week 24 Up to 48 - Week Placebo - Control 2:1 Randomization (Active: Placebo ) 15mg, 30mg, Pla
Slide 26: Measuring MS Functional Improvement
LCLA Phase 2 Primary: Functional Visual Improvement LCLA Correlates with clinically meaningful deficits in QOL, EDSS and MSFC, MRI, and OCT MS Functional Endpoints Phase 2 Exploratory: Neuroprotection/Remyelination Endpoints The Visual System is a Window into the Brain ( T L T V + E = 1 2 3 4 5 6 7
Slide 27: Emerging Evidence of Clinical Improvement
LCLA (Best - Corrected) Emerging Evidence of Clinical Improvement Glanzman, R., H. Beadnall, M. T. Hotchkin, A. Klistorner, M. Barnett, R. Sergott, A. Rynders, K. S. Ho, and Mark G. Mortenson. “Update to a Phase 2 clinical trial of catalytic gold nanocrystals, CNM - Au8, for the treatment of chronic
Slide 28: Strong Intellectual Property
C S N 28 ® Clean surface nanocrystal (CSN) therapeutics Trade Secrets Patent Status Issu e d & All o w e d Patents 130+ Pending Applications >30 To t a l P a t e nt s / 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) Met
Slide 29: Proprietary Nanocrystal Manufacturing
Cl e n e | P r op ri e t a r y N a n oc r y s t al Ma n u fac t u ri n g In - House ISO8 Clean Room Clinical Production in North East, MD Va l i d a t e d C M C Processes 29 Designed to be Scalable to Commercialization Patented H y d ro - el ec t r o - Crystallization P r o p r i e t a r y T r ade S
Slide 30: Anticipated Timeline & Investor Catalysts
Anticipated Timeline & Investor Catalysts 2 0 2 0 - 2 0 23 United T h e ra p e u ti c s 30
Slide 31: Clene Investment Highlights
31 Unmet Medical Need & Market Opportunity Lead Asset: CNM - Au8 for Neuro Repair Clinical D e v e lo p m e nt Pipeline Strong IP Portfolio Financials CNM - ZnAg for COVID - 19 CLENE | Investment Highlights • Nanocatalyst of Intracellular Biological Reactions • Robust Preclinical Remyelination & Neu
Slide 32: Clene Inc. Contact
Clene Inc. HQ & Clinical Development 6550 South Millrock Drive, Suite G50 Salt Lake City, UT 84121 R&D and Manufacturing 500 Principio Parkway, Suite 400 North East, MD 21901 © 2021 Clene Inc. Version: 11 - May - 2021