Arcturus Therapeutics Holdings Inc. · NASDAQ:ARCT
Arcturus Therapeutics — Investor Presentation (June 2020)
38 slides from Arcturus Therapeutics's investor presentation, Jun 18, 2020
Slide 1: Arcturus Therapeutics
ARCTURUS’ THERAPEUTICS.
Slide 2: Forward Looking Statements
FORWARD LOOKING STATEMENTS x. 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 esults, performance or achievements tobe materially different from any futuee
Slide 3: Company Highlights
Company Highlights ong K Arcturus is a Clinical-Stage mRNA Vaccines and Medicines Company Publicly Traded (Nasdaq: ARCT) + Headquarters: San Diego, CA + Number of Employees: 97 * Founded: 2013 Promising Therapeutic Candidates + LUNAR-COV19 (COVID-19 Vaccine) + LUNAR-OTC (Ornithine Transcarbamylase D
Slide 4: Proprietary mRNA Technologies Driving Promising Therapeutic Programs
ARCTURUS THERAPEUTICS Proprietary mRNA Technologies Driving Promising Therapeutic Programs Broad and Strong Intellectual Property Portfolio Program Indication mRNA & STARR™ mRNA mea (anct-021) COVID-19 Vaccine MRNA Chemistry TRNA Design & Modifications TARNA Manufacturing Process LUNAR-OTC —_Omithin
Slide 5: Arcturus Pipeline of mRNA Medicines
ARCTURUS THERAPEUTICS Arcturus Pipeline of mRNA Medicines Toutest Tugetonan ——Proniece apne Nass eteatien: ‘Administration {Cell Type) Worldwide milestones Tae Tha WNAREOVIS —coypsovaccne nramutcd(in) (yore, al ite Dong Tracts nravenous ty 919600 tarcreiay Tanwar restores au o20 vg WHAREF yen ned av
Slide 6: Partnerships Maximize Platform
ARCTURUS THERAPEUTICS k Partnerships Maximize Platform ie he Program partner Indcation LUNAR-HBV Gofouonfofinuen Hepatitis B Virus (HBV) LUNAR-Nasi Nonalcoholic steatohepatit (NASH) vwnar-6s03 ultrageny’ —_—_ayeogen storage oisease Type LUNAR-RARE ultragenyx Undisclosed Rare Disease Lnar-e. aden Vac
Slide 7: LUNAR Delivery Technology
ARCTURUS THERAPEUTICS LUNAR® Delivery Technology LUNAR As with Celt Memb plfediated RNA Disruption in Cytosot = ©
Slide 8: STARR mRNA Superior to Conventional mRNA
ARCTURUS THERAPEUTICS STARR™ mRNA Superior to Conventional mRNA Self-Transcribing and Replicating mRNA (STARR) delivered with LUNAR® provides higher protein panes expression and potentially longer-lasting duration of protein expression in mouse Protein Expression Over 7 days STARR™ Technology : 30-F
Slide 9: LUNAR-COV19 (ARCT-021)
a." LUNAR-COV19 (ARCT-021) COVID-19 Vaccine Candidate
Slide 10: Arcturus COVID-19 Vaccine Candidate has Significant Advantages
ARGTURUS THERAPEUTICS Arcturus COVID-19 Vaccine Candidate has Significant Advantages /\ Duke-NUS Partnership DukeNUS Medical Schoo! mRNA Vaccine: Simple, No Adjuvants, No Viruses STARR™ mRNA: Produces 30X more Protein than Conventional mRNA LUNAR® Technology: non-viral Delivery System &. + Promising
Slide 11: Preclinical Data: Broad and Robust Immune Response
ARCTURUS THERAPEUTICS Preclinical Data: Broad and Robust Immune Response Humoral Immunity Cellular Immunity 'STARR™ induces more robust titers compared to conventional mRNA son ‘Adaptive Cellular (co8+ cells) "Neutralizing antibody titers and high seroconversion at low doses mate i a a u = a = immun
Slide 12: Clinical Development Plan to Rapidly Advance LUNAR-COV19
ARCTURUS THERAPEUTICS kX Clinical Development Plan to Rapidly Advance LUNAR-COV19 |.” * Initiate Phase 1/2 dosing this summer 2020 * Enroll up to 108 healthy volunteer adults, including the elderly, to evaluate safety and immune response * Trial design allows us to potentially rapidly select dose to
Slide 13: LUNAR-OTC (ARCT-810)
a." LUNAR-OTC (ARCT-810) Ornithine Transcarbamylase (OTC) Deficiency
Slide 14: OTC Deficiency Market Opportunity
ARCTURUS THERAPEUTICS OTC Deficiency Market Opportunity Ornithine Transcarbamylase (OTC) Deficiency: The most common urea cycle disorder ‘The urea cycle converts neurotoxic ammonia to water-soluble urea that can be excreted in urine Deficiency in OTC causes elevated blood ammonia, which can lead to
Slide 15: LUNAR-OTC
ARCTURUS THERAPEUTICS LUNAR-OTC Disease Normalization Following Single and Repeat Dosing in OTC Mouse Model "i Survival of OTC-deficient Mice on High Protein Diet - Weekly LUNAR-OTC Treatment fe un anore tm ester nnae = seeks pe wich Prosein Diet teontea ths anae
Slide 16: LUNAR-OTC
ARCTURUS THERAPEUTICS LUNAR-OTC Exceeds Therapeutic Target of 10% Enzyme Replacement at all Doses in OTC-Deficient Mouse Model + OTCD impacts ureagenesis {ammonia detoxification) + The main site of ureagenesis is the periportal region of the liver* + Establishing 10% of natural enzyme levels is expe
Slide 17: ARCT-810 Phase 1/1b Study Ongoing
ARCTURUS THERAPEUTICS ARCT-810 Phase 1/1b Study Ongoing ‘Two Single Dose Studies + New Zealand Phase 1 clinical trial underway, up to 30 healthy volunteers ~ Clinical Trial Application (CTA) approved + U.S. Phase 1b clinical trial in up to 12 stable OTC-deficient patients ~ IND allowed to proceed Pr
Slide 18: LUNAR-CF
a." LUNAR-CF Cystic Fibrosis
Slide 19: Cystic Fibrosis Market Opportunity
Cystic Fibrosis Market Opportunity KX Cystic Fibrosis: The most common rare disease in the United States + Caused by genetic mutations in the CFTR gene, resulting in aberrant flux of ions in and out of cells, causing thick mucus buildup in lung airways + Chronic airway obstruction leads to infection
Slide 20: Delivery of LUNAR-mRNA to Rodent Airways
ARCTURUS THERAPEUTICS Delivery of LUNAR®-mRNA to Rodent Airways Nebulization: Upper/Lower Airways ee ets Mice Epithelial Airways ( Cals ( i
Slide 21: LUNAR, an aerosolized delivery platform for lung
ARCTURUS THERAPEUTICS LUNAR®, an aerosolized delivery platform for lung Aerosolized LUNAR? ‘Aerosolized LUNAR®-mRNA (EGFP) [J LUNAR®-mRNA (hCFTR) is biologically Particles are Breathable maintains activity active in vivo (NPD, Mouse) Pre-Nebulization Breathable Range (microns) Post Nebulization Po i
Slide 22: Delivery of LUNAR-mRNA into Epithelial Airways in Ferret
ARCTURUS THERAPEUTICS Delivery of LUNAR®-mRNA into Epithelial Airways in Ferret 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 t
Slide 23: Moving Forward
a." Moving Forward
Slide 24: Anticipated Milestones and Cash Position
ARCTURUS THERAPEUTICS k Anticipated Milestones and Cash Position LUNAR-COV19 (ARCT-021) Phase 1/2 Initiation ‘Summer 2020 Initial Clinical Date 03/04 2020 CSE Oba $59.5 million as of March 31, 2020. $9.6 million added in Q2 2020 for Ultragenyx Option ibeoe eC Cane SSe CO ie) Sufficient to support op
Slide 25: Management Team and Board of Directors
AncTURUS THERAPEUTICS A Management Team Joseph Pay Sc Pad Chiehua, PLD. Andie Sassi NBA Steve Hughes, MO, ‘resdintecso GSO8C00 0 ‘it Cavey Oar > merck Nitto Fidelity k!ONIS Board of Directors £10080 £O6¢ i \wultragenyr Sikicoons Allergan. OALTHEA © enex Fidelity ultragenyA ResMed ee 2s
Slide 26: Slide 26
Slide 27: Appendix
Appendix
Slide 28: Higher and More Robust Antibody Titers
ARCTURUS THERAPEUTICS Higher and More Robust Antibody Titers Single Dose Single Dose Single Dose A > STARR 0.2 pg g + sme = yg =] + sme 10g $n] > nn Seno] = mm Swe] = m0 oe u ig Shaw She Sham $3 x FE ms $2 wom. = z z aaa aie ae “4 me 8) Time ny) ie ny) + Higher titers (anti-SARS-CoV-2 Spike Glycopro
Slide 29: LUNAR-COV19 Positive Preclinical Data
LUNAR-COV19 Positive Preclinical Data kX Arcturus COVID-19 vaccine to begin human dosing this Summer nat ameyan Seroconversion Rate (% of Animals) - STARR™ mRNA vs. Conventional mRNA Single LUNAR® Delivery Deke ual STARR™ mRNA (%) Conventional mRNA (%) Day 10 Day 19 Day 10 Day 19 02 40 60 20 20 2 80
Slide 30: Arcturus Vaccine elicits a Balanced Cell Mediated Immune Response
Arcturus Vaccine elicits a Balanced Cell Mediated kK Immune Response cD" IFNy Ta1rTh2 ratio RNA Dose | %IFN-g+CD8*TCells | CD4+ Thi/Th2 3 (ug) (IFN-g/1L4) 2 e 0.0 40 46 5 zi 8 <q Ea q 0.2 45 53 2 yd 20 60 50 10.0 80 60 ° Ono Oya 249 1949 y9 0299 240 10¥a Dose (3015) Results Summary * RNA dose depend
Slide 31: LUNAR-COV19 Data Summary
LUNAR-COV19 Data Summary ‘ Very low dose: Strong neutralizing antibody response with just a single dose of 0.2 - 10 ug STARR™ RNA Strong humoral response continuous increase in neutralizing antibodies beyond Day 30 Strong T-cell response: dose responsive increase in IFN-g positive CD8* T-cells Poten
Slide 32: Arcturus Safety Profile
Arcturus Safety Profile kK 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 partners, ‘Top Safety Con
Slide 33: ATX Lipids are Effective and Biodegradable
ARCTURUS THERAPEUTICS ATX Lipids are Effective and Biodegradable x Protein Expression (in vivo) Esterase Catalyzed Degradation (in vitro) J s00- i Z i 1106 § mo me arxza | |p so 3 4 cox names E i 7 Foo Fo Eo. Pes ATK1O ATK2.0 bei s ws ms Next Generation ATX Lipids Retain Degradability & Improve Deli
Slide 34: ATX 2.0 Lipid is Biodegradable and Clears in vivo
ARCTURUS THERAPEUTICS ATX 2.0 Lipid is Biodegradable and Clears in vivo a = 051% | = teats oF 8 «© & § SF (the major component in LUNAR® technology) is degra he Approximately 20 hours
Slide 35: Drug Substance: mRNA Design
ARCTURUS THERAPEUTICS Drug Substance: mRNA Design Weekly Dosing in Non-Human Primates (NHPs) Optimize improve 1 mS cont MRNA sequence», Protein Expression = pm LUNAR.EPO mRNA Chemistry Duration $ 2 al Si neas. Process Functional Activity, ee ip cap (UTR UESANGRESSRINN SUR) Pobiatat 2
Slide 36: Drug Substance (mRNA) Manufacturing
Drug Substance (mRNA) Manufacturing kK INA Template Production ntratic Features Benefits Optimized IVT Method Reduced Cost; Higher Purity Improved Capping Reaction Reduced Cost of Goods Proprietary Purification Process Higher Purity in a Shorter Time Efficient Entire Process Less Than One Week Scala
Slide 37: Drug Substance (mRNA) Manufacturing
Drug Substance (mRNA) Manufacturing RNA Yield RNA Purity Scale {g} scale (e} Yield [¢/1] Purity Hi Non-Gii Lots Produced at Arcturus GMP Lots Produced at CMO as part of recent GMP campaign Three 12.5 g lots produced in recent GMP campaign are of equivalent quality and yield
Slide 38: Drug Product (LUNAR + mRNA) Manufacturing
Parl sie (om) ARCTURUS THERAPEUTICS Drug Product (LUNAR® + mRNA) Manufacturing Particle Size ‘%hEncapsulated mRNA Bath Size (mR in rama). Bate Sie RA grams) + Manufacturing of Drug Product Demonstrated up to Multigram Scale with Yields + GMP Batch of LUNAR®-OTC (ARCT-810) Drug Product Manufactured a
Arcturus Therapeutics Holdings — Investor Presentation (June 2020) on 6ix