Arcturus Therapeutics Holdings Inc. · NASDAQ:ARCT
Arcturus Therapeutics — Investor Presentation (February 2019)
25 slides from Arcturus Therapeutics's investor presentation, Feb 11, 2019
Slide 1: Enabling the Next Generation of RNA Medicines
“ARCTURUS ~ THERAPEUTICS. : Buin Next Cen neration of the RNA Medi icines
Slide 2: Forward Looking Statements
FORWARD LOOKING STATEMENTS = This presentation contains forward-looking statements, These statements relate to fulure events and involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially diferent from any future
Slide 3: Board of Directors
ARCTURUS THERAPEUTICS XK Board of Directors aaa £O00Q8 Dr. Peter Farrell Andrew Sassine, MBA James Barlow, MA Dr. Magda Marquet Joseph E. Payne, MSc rman ofthe Board Director a the Basra irate of he Boars actor of the Bases Directoy of ne Bost @ALTHEA — €& MERCK ResMed Founder and Chairman Former Po
Slide 4: Management Team
ARCTURUS THERAPEUTICS Management Team Dr. Pad Chivukula ‘Andrew Sassine, MBA Founder, C50 COO cro merck (Nitto Fidelity
Slide 5: Corporate Overview
Corporate Overview nau + HQ: San Diego, CA + Founded: 2013 + Nasdaq: ARCT + Outstanding shares: 10.8 M + Employees: 80 + Insider Ownership: 24% Arcturus is an RNA Medicines Company Focused on Significant Opportunities in Rare, Liver, and Respiratory Diseases
Slide 6: Investment Highlights
Investment Highlights _Xx = RNA Medi Manufacturing Expertise: in both messenger RNA Drug Substance & LUNAR® Nanoparticle Drug Product ines Drug Development Company Diverse Pipeline 7 Programs: 2 Wholly-owned, 5 Pharma Collaborations Intellectual Property LUNAR’ Delivery Technology, UNA Oligomer Chem
Slide 7: Pipeline of RNA Medicines
ARCTURUS THERAPEUTICS kK Pipeline of RNA Medicines bas Name Indication Partner Partnership Structure IND Date LUNAR-OTC Ornithine Transcarbamylase = : (ARCT-810) maNA (OTC) Deficiency MuBH ets tes os 201s —s holly-Owne : LUNancr mnRNA eystier boss Whol Owned by ARCT va.2020 unded by =: LUNAR-HBV RNA
Slide 8: Pipeline of RNA Medicines
ARCTURUS THERAPEUTICS Pipeline of RNA Medicines sida ae ame REWURISRNA atm = Partnership e__Routeof Tareet Target caus Prevalence mi Modality met fare structure "NP paministration organ T™8E*C* Worldwide Tae Oring reset mRNA Transearbamyiaee PUM wholly-owned 62019, w Liver Hepatocytes > 10.000, Defi
Slide 9: LUNAR Lipid-Mediated Delivery
ARCTURUS THERAPEUTICS XK LUNAR® Lipid-Mediated Delivery — Feature Benefit Diverse Library of over 150 Proprietary Lipids Compatibty Formulated with multiple RNA modalities Rational Design to Maximize Efficacy and Increase Tolerabiliy Route of © Ot tion ‘Ws IM, Nebulzation Formulation Compositions Cu
Slide 10: LUNAR Mechanism of Delivery
ARCTURUS THERAPEUTICS LUNAR® Mechanism of Delivery LUNAR Associ with Gell Lipid Particle in
Slide 11: OTC Deficiency Market Opportunity
ARCTURUS THERAPEUTICS LUNAR AND mRNA PLATFORMS ARE DRIVING DEVELOPMENT PIPELINE K 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
Slide 12: LUNAR-OTC
ARCTURUS THERAPEUTICS LUNAR-OTC Disease Normalization Following Single and Repeat Dosing in OTC Mouse Model Urinary Orotic Acid Single Dose hase - eterase conte igh dose roti AcidlCreatinine Normalized to Baseline velop tne Days Post Dose sin NCEOR SI sleet Lever man CTC mRNA mn ina Or Aid ln ‘hath
Slide 13: LUNAR-OTC
ARCTURUS THERAPEUTICS LUNAR-OTC Exceeds Therapeutic Target of 10% Enzyme Replacement at all Doses in OTC-Deficient Mouse Model + OTCD impacts ureagenesis Periportal Expression in the Liver of OTC Protein (ammonia detoxification) + The main site of ureagenesis is the periportal region of the liver* +
Slide 14: Cystic Fibrosis Market Opportunity
ARCTURUS THERAPEUTICS A LUNAR AND mRNA PLATFORMS ARE DRIVING DEVELOPMENT PIPELINE Cystic Fibrosis Market Opportunity — ® 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
Slide 15: Proof of Concept in CF Models of Disease
ARCTURUS THERAPEUTICS PROOF OF CONCEPT IN GF MODELS OF DISEASE LUNAR-CF Protein and Functional Profile of Arcturus’ CFTR mRNAs Validated Protein Expression: Western-Bilot detection Functional Assay: Transepithelial Conductance of mature CFTR protein in an epithelial cell model (FRT) Natural Human CF
Slide 16: Nebulization and Delivery to Lung
ARCTURUS THERAPEUTICS NEBULIZATION AND DELIVERY TO LUNG LUNAR® Targeting Lung LUNAR® Delivery into Bronchial Epithelial Cells (BECs) at Es Functional Delivery of LUNAR®-mRNA into Lung Epithelial Cells TdTomato/FoxJ1/Dapi LUNAR-Luc mRNA 6
Slide 17: GSD Type III Market Opportunity
ARCTURUS THERAPEUTICS LUNAR AND mRNA PLATFORMS ARE DRIVING DEVELOPMENT PIPELINE GSD Type Ill Market Opportunity ~— © Glycogen Storage Disease type Ill + Caused by genetic mutations in the glycogen debranching enzyme (AGL), which leads to a toxic glycogen-byproduct (limit dextrin) accumulation in liv
Slide 18: LUNAR Targeting Liver
LUNAR® Targeting Liver _X : mRNA delivery to Liver LUNAR-SiRNA delivery to Liver Cell Subtypes Target Knockdown in Liver Cell Types LUNVAR-GEP. Hepatocytes Stellate Cells Cell Type LUNAR-Luc LUNAR-SiIRNA. LUNAR¢I dalivers to Hepatocytes LUNAR-2 delivers to Hepatocytes and Stllate cells Functional De
Slide 19: Nebulization and Delivery to Lung
ARCTURUS THERAPEUTICS NEBULIZATION AND DELIVERY TO LUNG LUNAR® Targeting Lung LUNAR-GEP mRNA. Control PBS LUNAR-Luc mRNA Functional Delivery of LUNAR®-mRNA into Lung Epithelial Cells ®
Slide 20: IM Delivery and Vaccine Proof of Concept with HA mRNA
ARCTURUS THERAPEUTICS Im DELIVERY AND VAGGINE PROOF OF CONCEPT WITH HA mRNA LUNAR® Targeting Muscle (Vaccines) Intramuscular (IM) administration HA Inhibition Titer and T-Cell responses Drug Product LUNAR® Manufacturing Hemagglutination CD8+ T-Cell Responses Inhibition Titer (Day 35) (Day 35) Serum
Slide 21: Internal RNA Development Platforms
ARCTURUS THERAPEUTICS INTERNAL RNA DEVELOPMENT PLATFORMS UNA Oligomer Efficacy in LUNAR-HBV (siRNA) LUNAR-HBV (siRNA) effectively knocks-down Inhibition of HBsAg Secretion in HBV-infected HBV Gene Products in AAV-HBV Mouse Model Primary Human Hepatocytes ‘Serum HBsAg ‘Serum HBV DNA. = una oe unaz i
Slide 22: Drug Substance: mRNA Design
ARCTURUS THERAPEUTICS Drug Substance: mRNA Design “sane Sustained hEPO actly in NHPS upon repeat dosing Optimized conditions Improved protein Weekly Dosing in Non-Human Primates expression and MRNA sequence gue, duration 486 Mm PRS contol Chemistry = + Improved functional = = CONARERO TARA, Process
Slide 23: Arcturus mRNA Manufacturing
Arcturus mRNA Manufacturing x DNA Template IVT and Capping Runtcaion Peseess 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
Slide 24: Proprietary Reproducible & Scalable Production Process
ARCTURUS THERAPEUTICS PROPRIETARY REPRODUCIBLE & SCALABLE PRODUCTION PROCESS Drug Product: LUNAR® Formulation & Production” Pantcle size % Encapsulated RNA Particle Size ‘Encapsulated RNA tcl nk ah Batch ele (NAn Gram) atch Scala (RNAn Grams) ath Seale (Rn Grams) 8 Particle size nm) ‘6 Encapsulated
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