Clene Inc. · NASDAQ:CLNN
Clene — Investor Presentation (October 2021)
32 slides from Clene's investor presentation, Oct 7, 2021
Slide 1: CLNN (NASDAQ) October 6, 2021
CLNN (NASDAQ) October 6, 2021
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: Management Team
Lanxide Corporation Dupont Lanxide Composites Lanxide Armor Company Lanxide Performance Materials Lanxide Electronic Components CDO CLENE | Management Team 3
Slide 4: Clene Nanomedicine
4 Clene Nanomedicine As of June 30, 2021 Cash on hand (unaudited): $63M Topline data from ALS Registration Trial 1 by mid - 2022 and 3 Phase 2 Trials 2 by end of 2021 >200 patient years of CNM - Au8 clinical exposure CNM - Au8® a gold nanocrystal suspension, in development as the first energetic cat
Slide 5: Platform & Pipeline
5 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 1 CLENE | Platform & Pipeline (Anticipated Launch in 2021) Brain Imaging Biomarker Study Brain Imaging Biomarker Study Pha
Slide 6: Catalytically-Active Nanotherapeutic
CNM - Au8® | Catalytically - Active Nanotherapeutic Improved Cellular Energy Production & Utilization 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 mechanis
Slide 7: Integrating Physics With Biology
CNM - Au8 | Integrating Physics With Biology Electron transfer (to - and - from) CNM - Au8 nanocrystals drives catalytic activity and cellular 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
Slide 8: Treating Energetic Failure in Neurodegenerative Disorders
Treating Energetic 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 Neuro
Slide 9: Mechanism of Action and Therapeutic Effects
9 CNM - Au8 | MOA Therapeutic Effects a Nicotinamide Adenine Dinucleotide a Robinson et al. Sci Rep. 2020 Feb 11;10(1):1936. doi: 10.1038/s41598 - 020 - 58709 - w.
Slide 10: Significant Global Opportunity
10 CNM - Au8 | Significant Global Opportunity 707,158 103,903 828,703 596,407 256,455 1,407,701 0 4 0 0 , 0 0 0 8 0 0 , 00 0 1,200,000 1,600,000 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):939 - 953;
Slide 11: Preclinical Evidence for Energetic Improvement
11 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, 2019. Data on File, Clene Nanomedicine, Inc. 1 2 CNM - Au8 | Preclinical Evidence for Energetic Improvement Therapeutic
Slide 12: 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 CNM - Au8 | MOA & Remyelination Data Published 12
Slide 13: 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 14: Clean Toxicology Findings
Standard ICH M3(R2) Toxicology Program Genotoxicity I n V itro & I n V ivo (Rodent) Safety Pharmacology CNS, CV, Renal Dose Range Finding Rodent, Minipig Single Dose To x i c o ki ne t i c s Canine Multi - Dose To x i c o ki ne t i c s Canine (7 - Day) MTD Toxicokinetics Canine (4 - Wk) Max Feasible
Slide 15: Well Tolerated; No Dose-Limiting Safety Issues To Date
• 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 16: Phase 2 Exploratory Study in Stable Relapsing MS (REPAIR)
• Difference in brain NAD+ and NADH fraction at Week 12 - 16 • Difference in 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
Slide 17: Age Related Decline of Brain Energy Metabolism
17 NAD + /NADH | Age Related Decline of Brain Energy Metabolism (By 31 P - MRS Imaging) Zhu et al. Proc Natl Acad Sci USA . 2015 Mar 3;112(9):2876 - 81. Closed squares = averaged data by age group: 21 – 26 yrs, 33 – 36 yrs, and 59 – 68 yrs old; Open squares= individual subject values ~0 . 5 % NAD +
Slide 18: Improved Brain Energy Metabolism: Increased NAD+/NADH Ratio
CNM - Au8 | Improved Brain Energy Metabolism Increased NAD+/NADH Ratio in MS & PD Patients Phase 2 Results 1 ƒ E n dpo i n t 2 ƒ Endpoints NAD is an essential molecule responsible for cellular energy production 18
Slide 19: Treatment Normalized Key Markers of Brain Metabolism in PD
β - ATP is used by the cell to maintain cellular metabolism and normal function Phosphorylation potential is the amount of available phosphorous that can be used to make ATP in times of stress or high metabolic activity CNM - Au8 | Treatment Normalized Key Markers of Brain Metabolism in PD Patients
Slide 20: Phase 2 RESCUE-ALS Trial 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: MUNIX: Sensitive Biomarker of ALS Disease Progression
MUNIX Decline Precedes ALSFRS - R Decline MUNIX | Sensitive Biomarker of ALS Disease Progression M o t o r U n i t Index ( M U NIX) Sensitive method of detecting motor neuron loss What is MUNIX? Why MUNIX? Early Indicator o f AL S d is e ase progression MUNIX is a method of estimating the number of
Slide 22: Phase 2 Blinded MUNIX Improvement
Phase 2 Improvement Emerging Blinded Evidence Predictive of Clinical Efficacy | MUNIX(4) Sum Change 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 Amyotr
Slide 23: Blinded Rate of Vital Capacity Loss Is Less Than Comparable Datasets
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 Bli n d ed Rate of Vital C a p a c ity Los s I s Les s Than
Slide 24: Phase 3 ALS Registration Study
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 Change in AL S F R S - R Registration Study: 24 - Week Treatment Period (3:1 randomization, 120 active [30mg, 60mg]: 40 pl
Slide 25: Phase 2 Chronic Optic Neuropathy in Relapsing MS
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 ) 15 m g , 3 0 m
Slide 26: LCLA Primary: Functional Visual Improvement
26 LCLA Phase 2 Primary: Functional Visual Improvement LCLA 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 ( T L T V + E = 1 2 3 4 5
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 ® Clean surface nanocrystal (CSN) therapeutics 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 (Prev
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 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 Secr
Slide 30: Anticipated Timeline & Investor Catalysts
Anticipated Timeline & Investor Catalysts 2 0 2 0 - 2 0 23 Facility Venture Debt $20M 30
Slide 31: 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 • Energy enhancing nanotherapeutic • Robust Preclinical Remyelination & Neuroprotection Data
Slide 32: Clene Inc. Addresses
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: 6 - October - 2021