Arcturus Therapeutics Holdings Inc. · NASDAQ:ARCT
Arcturus Therapeutics — Investor Presentation (August 2024)
26 slides from Arcturus Therapeutics's investor presentation, Aug 13, 2024
Slide 1: Next Generation RNA Medicines
Next Generation RNA Medicines ~ August 2024
Slide 2: Forward Looking Statements
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 materially different from any future p
Slide 3: Arcturus Therapeutics
Arcturus Therapeutics Global mRNA Medicines Nasdaq: ARCT Company Headquarters: San Diego, CA Founded: 2013 Strategic Partners MRNA Medicine Candidates LUNAR-OTC Ornithine Transcarbamylase Deficiency LUNAR-CF Cystic Fibrosis Additional Earlier Stage Programs -aa®8 CYSTIC FIBROSIS FOUNDATION’
Slide 4: Proprietary mRNA Technologies Driving Therapeutic Programs
A mrcrurus c Proprietary mRNA Technologies Driving Therapeutic Programs Broad Intellectual Property Portfolio mRNA Technology LUNAR® Delivery Manufacturing Know-How mRNA for protein replacement Hepatocytes — intravenous mRNA Drug Substance Production Self-amplifying mRNA (STARR®) Myocytes — intramus
Slide 5: LUNAR - Lipid Nanoparticle (LNP) Delivery Technology
LUNAR® - Lipid Nanoparticle (LNP) Delivery Technology LUNAR? interacts with LUNAR® internalized cell membrane inside endosome 5 » mRNA translated into mRNA release protein of interest a a q va f 6 ( oobi ptel
Slide 6: Pipeline of Arcturus-Owned mRNA Therapeutic Candidates
A mrcrurus c Pipeline of Arcturus-Owned mRNA Therapeutic Candidates Global Pr Omithine Phase 2 Hepatic Ree Ac anctupus Transcarbamylase > 10,000 Interim Data Deficiency (OTC) Q4 2024 Phase 2 a LUNAR-CF ~ a Cystic Respiratory (ARCT-032) evsricFisnosis Fibrosis 85,000-100,000 Inbation Each Arcturus-Ow
Slide 7: Pipeline of Partnered Self-amplifying mRNA Vaccines
A mrcrurus c Pipeline of Partnered Self-amplifying mRNA Vaccines Candidate Partner Stage Kostaive® Approved (JP) O@RCT 154) CSL Covipr2 MAA Filed (EU) Monovalent: Ancestral Kostaive® Bivalent (ARCT-2301) CSL COVID-19 Phase 3 Ancestral / Omicron BA.4/5 Kostaive® XBB.1.5 (ARCT-2303) CSL COVID-19 Phase
Slide 8: CSL: Arcturus Therapeutics Global Vaccine Partner
ARCTURUS CSL: Arcturus Therapeutics Global Vaccine Partner = = = 2 CSL $13.3 Billion USD Annual Revenue Operating in 40+ Countries Worldwide * 32,000+ Employees Worldwide * 13 Phase III programs — including Kostaive® + Focused on four strategic technology platforms — plasma protein; recombinant tech
Slide 9: CSL Vaccine Partnership
CSL Vaccine Partnership Up to $4.3 billion in Milestone Payments Partnership Terms * Collaboration combines CSL’s global ° vaccine commercial and manufacturing CSL ARCTURUS . . ; on therapeutics infrastructure with Arcturus’ expertise in mRNA design and modification, LUNAR® $200 million Upfront Paym
Slide 10: Meiji Background Information
A mrcrurus c i: Background Information Meij meiji The Meiji Group provides food and pharmaceuticals indispensable to their customers MeijiHoldings Co.Ltd. . $7 9 Billion USD Net Sales (As of March 31, 2023) * 113 Locations Worldwide with 17,290 Employees see mejji Meiji Seika Pharma provides antibac
Slide 11: ARCALIS: Arcturus' Joint Venture mRNA Manufacturing Partner
ARCTURUS’ ARCALIS: Arcturus’ Joint Venture mRNA Manufacturing Partner Acrseus * ARCALIS is a CDMO Specializing in Manufacturing of mRNA Vaccines and Therapeutics = oy + Joint Venture Founded in 2021 + Major Equity Owners: Axcelead & Arcturus, subject to dilution Meiji Seika Pharma is collaborating w
Slide 12: Kostaive Phase 3 Clinical Studies
Kostaive® Phase 3 Clinical Studies Acarcturus Kostaive® (Monovalent, Kostaive®) Phase 3 Non-inferiority safety and immunogenicity trial + Kostaive® administered at an 83.3% lower dose than Comirnaty® (N = 828) + 50% of participants received Kostaive® (5 mcg); 50% of participants received Comirnaty®
Slide 13: Historic Approval of World's First sa-mRNA Product
Historic Approval of World’s First sa-mRNA Product Ac aserunus E CSL-Arcturus Collaboration Results in Groundbreaking Approval of Kostaive® First Arcturus Approval \ Enduring Vaccine with Strong Clinical Data Kostaive® self-amplifying mRNA COVID Approval based on positive clinical data from several
Slide 14: Kostaive: More Durable Post-boost Immune Response
Kostaive®: More Durable Post-boost Immune Response ARCTURUS: Phase 3 Persistence Data Comparing Kostaive® (5 mcg) to Comirnaty® (30 mcg) @ Kostaive® (5 mcg) © Comirnaty® (30 mcg) THE LANC ET Infectious Diseases Oda Y etal. Lancet infec Diseases; February 1, 2024 Wuhan-Hu-1 Omicron BA.4/5 200) DOK: (
Slide 15: ARCT-032 Inhaled mRNA Therapeutic Candidate for Cystic Fibrosis
ARCT-032 Inhaled mRNA Thera apeutic Candidate for Cystic Fibrosis
Slide 16: Cystic Fibrosis: ARCT-032 Market Opportunity
Cystic Fibrosis ARCT-032 Market Opportunity Cystic Fibrosis 85,000-100,000 worldwide prevalence Caused by mutations in the CFTR gene, resulting in poor chloride transport and dehydrated, sticky mucus in the airways Chronic airway obstruction leads to infection and inflammation, which causes progress
Slide 17: ARCT-032 Clinical Update
ARCT-032 Clinical Update Acarcturus Phase 1 Study in Healthy Volunteers (New Zealand) * Objectives: Assess safety, tolerability and PK of ARCT-032 in healthy adults + Status: Completed dosing across 4 ascending single-dose cohorts (8 subject per cohort) + Total number of subjects N = 32 + Safety and
Slide 18: LUNAR-mRNA in Healthy Animals (four different species)
: . F ‘ ARCTURUS’ LUNAR®-mRNA in Healthy Animals (four different species) “44ers F Successful delivery to airway epithelium; transduction demonstrated by Brown and Green staining LUNAR® Delivery to Airway Epithelium is Demonstrated in Rodent and Non-Rodent Species
Slide 19: LUNAR-mRNA in Cystic Fibrosis Ferret Model
r LUNAR®-mRNA in Cystic Fibrosis Ferret Model ES Bronchus Luminal Mucus In collaboration with Univ. of lowa; presented at North American CF Conference Nov 2023 n Model (G55
Slide 20: ARCT-032 in a Kalydeco-responsive CF Ferret Model (G551D)
ARCT-032 in a Kalydeco®-responsive CF Ferret Model (G551D) Ke Proof of Activity: Mucociliary clearance improves after single administration of ARCT-032 100 90 80 70 60 50 40 30 % Clearance ARCT-032 (LUNAR®-CF) Kalydeco® (Positive Control) LUNAR-TdTomato (Negative Control) Off Kalydeco® (Baseline) Mi
Slide 21: ARCT-032 Restores CFTR Expression & Function
Acarcturus ARCT-032 Restores CFTR Expression & Function — High Expression Levels of CFTR protein Restored chloride activity (chloride gradient) gs + £2 ¢ [i ncrtR mRNA g $ E $ =— Ey Control mRNA > SS GO S € o o £ O oO Non-CF KPa » -C < Range 150 B CFTR = ig ii 9 o 100 — <J i —— +e e--- Tubulin F508d
Slide 22: ARCT-810 Systemically Delivered mRNA for OTC Deficiency
ARCT-810 Systemically Delivered mRNA for Ornithine Transcarbamylase (OTC c
Slide 23: Ornithine Transcarbamylase (OTC) Deficiency: ARCT-810 Market Opportunity
Ornithine Transcarbamylase (OTC) Deficiency ARCT-810 Market Opportunity (6) 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 neurological damage, coma,
Slide 24: LUNAR-OTC Exceeds Target of 5% Enzyme Replacement in Mouse Model
A mrcrurus c LUNAR-OTC Exceeds Target of 5% Enzyme Replacement in OTC-Deficient Mouse Model Periportal Expression in the Liver of OTC Protein * OTC deficiency impacts ureagenesis Hd . (ammonia detoxification) 90. + The main site of ureagenesis is the periportal 0 70 region of the liver* 60 + The cri
Slide 25: ARCT-810 Clinical Update
ARCT-810 Clinical Update Aucums I Phase 1 (NZ) Study in Healthy Volunteers + Completed dosing up to 0.4 mg/kg, total number of subjects N = 24, generally safe and well tolerated Phase 1b (U.S.) Single Ascending Dose (SAD) Study in OTCD Adults * Completed enrollment and dosing of all cohorts (N=16) +
Slide 26: Arcturus Board of Directors
° ARCTURUS” Arcturus Board of Directors AX ancturus & ® @ & Peter Farrell, Ph.D. Joseph E. Payne, MSc John Markels, Ph.D. Moncef Slaoui, Ph.D. Chairman Board Member Board Member Board Member President & CEO ResMep €& MERCK & MERCK GSK James Barlow, MA Magda Marquet, Ph.D. Edward W. Holmes, M.D. Jing
Arcturus Therapeutics Holdings — Investor Presentation (August 2024) on 6ix