Arcturus Therapeutics Holdings Inc. · NASDAQ:ARCT
Arcturus Therapeutics — Q2 2021 Earnings Presentation
46 slides from Arcturus Therapeutics's earnings presentation, Aug 9, 2021
Slide 1: | -ARCTURUS “THERAPEUTICS |
[*: “Se | -ARCTURUS 4 “THERAPEUTICS |
Slide 2: Forward Looking Statements
ARCTURUS THERAPEUTICS BUILDING INN FORWARD LOOKING STATEMENTS This presentation contains forward-looking statements. These statements relate to future events and involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be mat
Slide 3: Company Highlights
ARCTURUS THERAPEUTICS K Company Highlights a Arcturus is a Clinical-Stage mRNA Vaccines and Medicines Company Publicly Traded (Nasdaq: ARCT) * Headquarters: San Diego, CA * Number of Employees: 154 * Founded: 2013 Promising Vaccine and Therapeutic Candidates * LUNAR-COV19 (COVID-19 Vaccine) * LUNAR-
Slide 4: Proprietary mRNA Technologies Driving Promising Vaccine and Therapeutic Programs
ARCTURUS THERAPEUTICS Proprietary mRNA Technologies Driving Promising Vaccine and Therapeutic Programs Broad and Strong Intellectual Property Portfolio mRNA & STARR™ mRNA mRNA Chemistry Program Indication 5 ——— LUNAR-COV19 COVID-19 Vaccines mRNA Design & Modifications mRNA Manufacturing Process LUNA
Slide 5: Arcturus Pipeline of mRNA Medicines
ARCTURUS THERAPEUTICS Arcturus Pipeline of mRNA Medicines BUILDING INNOVATIVE RNA MEI Fiandiise Product ee Cell Prevalence Anticipated Name Target Worldwide ei stones LUNAR-COV19 COVvID-19 Myocytes & Initiation (ARCT-021) (ingle Shot) _(Mttamnuscular Dendritic Cells Global Bhased H2 2021 VACCINES —
Slide 6: Partnerships Maximize Platform
ARCTURUS THERAPEUTICS Partnerships Maximize Platform Program Partner Indication LUNAR-HBV Gotuenafohmen LUNAR-NASH LUNAR-GSD3 ultragenyX LUNAR-RARE ultragenyX LUNAR-COV19 Undisclosed (ARCT-021) Global Entity LUNAR-COV19 (ARCT-154) ‘Vingroup Hepatitis B Virus (HBV) Nonalcoholic Steatohepatitis (NASH)
Slide 7: LUNAR Delivery Technology
ARCTURUS THERAPEUTICS LUNAR® Delivery Technology Biodegradable, highly optimized for each cell type LUNAR® particle cellmembrane .- Packaged inside cell © 4S - ay +. mRNAtranslates mRNA release ——_into protein - ee
Slide 8: STARR mRNA Expression Superior to Conventional mRNA
ARCTURUS THERAPEUTICS kK STARR™ mRNA Expression Superior to Conventional mRNA UILDING. INNOVATIVE Self-Transcribing and Replicating mRNA (STARR™) delivered with LUNAR® provides higher mem protein expression and potentially longer-lasting duration of protein expression in mouse Protein Expression Ove
Slide 9: STARR COVID Vaccine Candidates
STARR™ COVID Vaccine Candidates ARCT-021 ARCT-154
Slide 10: STARR COVID Vaccine Candidates Have Significant Advantages
STARR™ COVID Vaccine Candidates Have Significant Advantages K STARR™ mRNA technology * Lower dose level than conventional mRNA provides manufacturing efficiencies and potential safety benefits ¢ Self-amplifying mechanism of mRNA expression provides superior T cell responses & LUNAR® Delivery Technol
Slide 11: Lyophilized Product Advantages Over Conventional Frozen Liquid
ARCTURUS THERAPEUTICS Lyophilized Product Advantages Over Conventional Frozen Liquid x BUILDING INNOVATIVE Freeze Thaw y ConventionalRNA / Vaccine Cold Chain, Frozen Liquid Requires Ultra-Low Ready for Use Temperature Storage Arcturus RNA Vaccine Cold Chain Bulk Dilue Formulation Added Lyophilized P
Slide 12: Global Manufacturing Partners
ARCTURUS THERAPEUTICS Global Manufacturing Partners x Germany Recipharm VinsrouP Global manufacturing partners include Aldevron, ARCALIS, Catalent, Polymun, Recipharm and Vingroup Manufactured GMP finished doses of lyophilized ARCT-021 (> 15 Million) for stockpiling purposes Forecast Capacity: More
Slide 13: ARCT-021
ARCT-021 Single Shot COVID STARR™ mRNA Vaccine
Slide 14: Preclinical Data: Robust Immune Response
ARCTURUS THERAPEUTICS Preclinical Data: Robust Immune Response Humoral Immunity Cellular Immunity BUILDING INNOVATIV RNA MEDICINES STARR™ induces more robust titers compared to conventional mRNA 7 Days Post Single Dose ' 1 1 1 Single Dose. os Single Dose ‘Single Dose 1 CD8* IFN-y sR 02g § sme Dye —
Slide 15: ARCT-021 Significantly Effective in Challenge Models
ARCTURUS THERAPEUTICS ARCT-021 Significantly Effective in Challenge Models K BUILDING INNOVATIVE RNA MEDICINES Mouse Model (transgenic human ACE2) Primate Model (macaque) 1007 10° + **1 pb oois9 7 Days After SARS-CoV-2 Virus Challenge g saa e * Lung viral titers exceeded 13,100 (median) 2 so 2 404 i
Slide 16: ARCT-021 Clinical Trial Update
ARCT-021 Clinical Trial Update kK Phase 1/2 Clinical Trial BUILDING INNOVATIVE RNA MEDICINES Completed dosing all subjects (n = 106), including older adults High seroconversion rates for IgG binding antibodies, and Th1 dominant CD4+ immune responses, neutralizing antibodies (PRNT50) Geometric Mean T
Slide 17: ARCT-154
ARCT-154 STARR™ mRNA COVID Vaccine Targeting Variants of Concern
Slide 18: ARCT-154 Preclinical Data and Clinical Trial Update
ARCTURUS THERAPEUTICS kX ARCT-154 Preclinical Data and Clinical Trial Update 7 Preclinical Data ARCT-154 Neutralizing Antibody Titers in Non-Human Primates One Month Post 2nd Dose Phase 1/2/3 Clinical Trial in Vietnam; CTA Approved to Proceed * Phases 1/2/3 sponsored & funded by Vinbiocare { t * Ran
Slide 19: LUNAR-OTC (ARCT-810)
LUNAR-OTC (ARCT-810) Ornithine Transcarbamylase (OTC) Deficiency
Slide 20: Ornithine Transcarbamylase (OTC) Deficiency
ARCTURUS THERAPEUTICS kK Ornithine Transcarbamylayse (OTC) Deficiency ARCT-810 Market Opportunity BUILDING INNOVATIVE RNA MEDICINES S) The most common urea cycle disorder The urea cycle converts neurotoxicammonia to water-soluble urea that can be excreted in urine Deficiency in OTC causes elevated b
Slide 21: LUNAR-OTC Disease Normalization in OTC Mouse Model
ARCTURUS THERAPEUTICS LUNAR-OTC Disease Normalization Following Single and Repeat Dosing in OTC Mouse Model 100 > : : : 1 me/kg —— LUNAR-OTC 1 mg/kg, n=10 Surviving Animals (%) 50 > LUNAR-OTC 0.3 mg/kg, n=10 40 — High Protein Diet (Control), n=10 30 20 10 0 7 #14 21 28 35 42 49 56 Time (Days) ft + +
Slide 22: LUNAR-OTC Exceeds Target of 10% Enzyme Replacement
ARCTURUS THERAPEUTICS LUNAR-OTC Exceeds Target of 10% Enzyme Replacement at all Doses in OTC-Deficient Mouse Model Periportal Expression in the Liver of OTC Protein OTCD impacts ureagenesis (ammonia detoxification) 16 2 90 * The main site of ureagenesis is 3B 80 the periportal region of the liver* 2
Slide 23: ARCT-810 Clinical Update
ARCTURUS THERAPEUTICS ARCT-810 Clinical Update Phase 1 Clinical Trial in Healthy Volunteers Completed Phase 1b Clinical Trial in OTC-Deficiency Adults Ongoing Phase 2 Clinical Trial in OTC-Deficient Adolescents and Adults Approved to Proceed * Randomized, double-blind, placebo-controlled, nested sin
Slide 24: LUNAR-CF (ARCT-032)
LUNAR-CF (ARCT-032) Cystic Fibrosis
Slide 25: Cystic Fibrosis
ARCTURUS THERAPEUTICS Cystic Fibrosis K ARCT-032 Market Opportunity BUILDING INNOVATIVE RNA MEDICINES ®) Cystic Fibrosis: The most common rare disease in the United States Caused by genetic mutations in the CFTR gene, resulting in aberrant anion transport in epithelial cells, causing thick mucus bui
Slide 26: Delivery of LUNAR-mRNA to Rodent Airways
ARCTURUS THERAPEUTICS Delivery of LUNAR®-mRNA to Rodent Airways / Nebulization: Upper/Lower Airways | LUNAR?® Targets Mice Epithelial Airways ( ) and Ciliated Cells ( LUNAR® + Luciferase mRNA
Slide 27: LUNAR, an aerosolized delivery platform for lung
ARCTURUS THERAPEUTICS LUNAR®, an aerosolized delivery platform for lung Aerosolized LUNAR® Aerosolized LUNAR® -mRNA (EGFP) LUNAR®-mRNA (hCFTR) is biologically Particles are Breathable maintains activity active in vivo (NPD, Mouse) Pre-Nebulization al ZA 1 2 3 4 5 Breathable Range (microns) Post-Nebu
Slide 28: Delivery of LUNAR-mRNA into Epithelial Airways in Ferret
Delivery of LUNAR®-mRNA into Epithelial Airways in Ferret KX EGFP conversion in tracheal epithelial airways observed in the ROSA26TG Ferret model ¢ Ferrets are an excellent species for modeling certain human lung diseases* * Novel LUNAR® formulations of CRE mRNA were tested in a transgenic ROSA26T7G
Slide 29: Moving Forward
Moving Forward
Slide 30: Anticipated Milestones and Cash Position
ARCTURUS THERAPEUTICS Anticipated Milestones and Cash Position ARCT-021 (single shot COVID vaccine) Phase 3 Initiation H2 2021 ARCT-154 (COVID vaccine targeting variants of concern) Interim Phase 1/2/3 Data Q4 2021 ARCT-810 (LUNAR-OTC) Interim Phase 2 Data H2 2022 ARCT-032 (LUNAR-CF) Clinical Trial
Slide 31: Management Team and Board of Directors
ARCTURUS THERAPEUTICS Management Team e £2@ x Joseph E.Payne,MSc Pad Chivukula, Ph.D. Andrew Sassine, MBA _Steve Hughes, M.D. Lance Kurata, J.D. President & CEO CSO & COO cFO Chief Medical Officer Chief Legal Officer merck Nitto Fidelity &IONIS Mi . BUILDING INNOVATIVE RNA MEDICINES Board of Directo
Slide 32: Scientific Advisory Board
ARCTURUS THERAPEUTICS Scientific Advisory Board HPISHADDSE BUILDING INNOVATIVE RNA MEDICINES Jeff Colyer, Ooi Eng Eong, FrederickG. Hayden, Peter A. Patriarca, RobertT. Schooley, Jonathan Smith, Michael Hodges, Drew Weissman, Mo. BMBS, FRCPath, Ph.D. M.D.,FACP MOD. MO. Pho. M.D.,BSc. MD. Pho. CEO of
Slide 33: Slide 33
Slide 34: Appendix
Appendix
Slide 35: LUNAR-COV19 Preclinical Seroconversion Data
ARCTURUS THERAPEUTICS LUNAR-COV19 Preclinical Seroconversion Data Seroconversion Rate (% of Animals) — STARR™ mRNA vs. Conventional mRNA single LUNAR® Delivery : pase (i) STARR™ mRNA (%) Conventional mRNA (%) Day 10 Day 19 Day 10 Day 19 0.2 40 60 20 20 2 80 100 20 ie} 10 100 100 40 80 100% of mouse
Slide 36: Preclinical Data: Anti-Spike Protein Levels Continue to Increase Up to 50 Days
ARCTURUS THERAPEUTICS kX Preclinical Data: Anti-Spike Protein Levels Continue to Increase Up to 50 Days 7 7 RNA MEDICINES Single Administration of LUNAR-COV19 0.2 ng Dose 2.0 ng Dose 10.0 ng Dose 000 00. £ -- STARR, © STARR £ “© STARR Ba a mRNA mRNA Bs oe mRNA 4 = 6000 8 = 6000. N BE] eas ue va Ny 3
Slide 37: Preclinical Data: Neutralizing Antibodies Continue to Increase for 60 Days
ARCTURUS THERAPEUTICS Preclinical Data: Neutralizing Antibodies Continue to Increase for 60 Days Single Administration (small dose, 2ug) of LUNAR-COV19 PRNT50 1001 Virus neutralization assay: Serum dilutions are incubated with SARS-CoV-2 virus, then added to cells. The cells die forming plaques, whi
Slide 38: Preclinical Data: Balanced Cell Mediated Immune Response
ARCTURUS THERAPEUTICS Preclinical Data: Arcturus Vaccine elicits a Balanced Cell Mediated Immune Response %of CDS T cells h BUILDING INI RNA MEDIC CD8* IFN-y Th1/Th2 ratio RNA Dose | % IFN-y+ CD8* T Cells 4+ Th1/Th2 (ue) . 0 4.0 4.6 Ly + 0.2 4.5 53 f. = . 2.0 6.0 5.0 10.0 8.0 6.0 Ong O2n9 2p9 1049 O
Slide 39: Arcturus Safety Profile
ARCTURUS THERAPEUTICS Arcturus Safety Profile k External Validation * Multiple strategic partnerships over many years confirms the positive potential safety profile of Arcturus LUNAR® and mRNA Arcturus is committed to developing safe mRNA products * 15 studies over several years with strategic partn
Slide 40: ATX Lipids are Effective and Biodegradable
ARCTURUS THERAPEUTICS ATX Lipids are Effective and Biodegradable K BUILDING INNOVATIVE RNA MEDICINES Protein Expression (in vivo) Esterase Catalyzed Degradation (in vitro) Z 300: 120% z mm PBS 2 mE ATX 10 100% & 200 mm ATX 2.0 B 80% o & 3 E 60% —O—%ATXLO E 100 & “i Oe %ATX2.0 © —e% mcs 2 20% 3 = o{_
Slide 41: ATX 2.0 Lipid is Biodegradable and Clears in vivo
ARCTURUS THERAPEUTICS K ATX 2.0 Lipid is Biodegradable and Clears in vivo ounuono naovarwe foi “= 0.5 mg/kg 250000 “© 0.5 mgikg = 1mgk _ -& 1 mgfkg 00000 wa/kg = 30000. 3 £ > 2 E 150000. © 20000: Ps a S 100000. x z & 10000. = S000. ot = + oF = 3 ay 7 3 * sf we we FUP OM Oe Time (hours) Time (hours)
Slide 42: Key Existing Country Relationships
ARCTURUS THERAPEUTICS Key Existing Country Relationships Singapore DukeNUS Research Partnership with Duke-NUS Medical School Medical School Financial Support from the Economic Development Board of Singapore EDB + $10 M Grant for Research and Preclinical Work singap@re « $6.7 M Grant for Phase 1/2 Cl
Slide 43: Drug Substance: mRNA Design
ARCTURUS THERAPEUTICS Drug Substance: mRNA Design Arcturus’ proprietary mRNA optimization platform Improve mRNA sequence . Protein Expression Chemistry Duration Process Functional Activity S'cap | 5’ UTR Coding Region 3° UTR Poly(A) tail Proprietary mRNA Opti x BUILDING INNOVATIVE RNA MEDICINES Sust
Slide 44: Drug Substance (mRNA) Manufacturing
Drug Substance (mRNA) Manufacturing K BUILDING INNOVATIVE RNA MEDICINES |A Template IVT and Capping re Buffer Exchange & Purification Process Caneanietiion Features Benefits Optimized IVT Method Reduced Cost; Higher Purity Improved Capping Reaction Reduced Cost of Goods Proprietary Purification Proc
Slide 45: Drug Substance (mRNA) Manufacturing
ARCTURUS THERAPEUTICS Drug Substance (mRNA) Manufacturing INNOVATIVE RNA Yield RNA Purity 6 4100 5 90 3: E w cP > a a Bs 5 7 2 a 1 60 0 50 125 125 125 125 125 125 scale [g] scale [gl Hi Non-GMP Lots Produced at Arcturus GMP Lots Produced at CMO as part of recent GMP campaign ee 12.5 g lots produced
Slide 46: LUNAR Versatility
ARCTURUS THERAPEUTICS LUNAR® Versatility K Compatible with RNA of Various size BUILDING INNOVATIVE RNA MEDICINES % Encapsulated RNA Particle Size 15 2 100 im Yr E % E 2” 2 Rg 2 ° ca S00 5 ui a R25 28 0 0 20 1800 © 1@00:«=«-2000»««5000-»««900 © 10000 © 12000 20 1800 © 1800» 2000» 5000» 9800» 10000» 1
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