Arcturus Therapeutics Holdings Inc. · NASDAQ:ARCT
Arcturus Therapeutics — Investor Presentation (February 2019)
21 slides from Arcturus Therapeutics's investor presentation, Feb 27, 2019
Slide 1: Building the Next Generation of RNA Medicines
ARCTURUS THERAPEUTICS 4 Builai ig Next Generation of the RNA Medi icines
Slide 2: Forward Looking Statements
FORWARD LOOKING STATEMENTS saa 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 futur
Slide 3: Investment Highlights
ARCTURUS THERAPEUTICS Investment Highlights Arcturus is an mRNA Medicines Drug Development Company Focused on Rare Diseases LUNAR® Delivery Platform Validated by Multiple Strategic Partners + More than $1 Billion in potential milestones and royalties Broad and Strong Intellectual Property Portfolio
Slide 4: Key Value Drivers: mRNA Medicines & Platform
ARCTURUS THERAPEUTICS Key Value Drivers: mRNA Medicines & Platform Arcturus LUNAR® Delivery Platform: Enabling Genetic Medicines fohsensfonon Utrageny~ ue Qo Strategic Partners: More than $1 Billion in Potential Milestones & Royalties Arcturus mRNA Me ines LUNAR-OTC (ARCT-810) to treat Ornithine Tra
Slide 5: LUNAR Mechanism of Delivery
ARCTURUS THERAPEUTICS LUNAR® Mechanism of Delivery
Slide 6: Arcturus Platform: Enabling Genetic Medicines
ARCTURUS THERAPEUTICS K Arcturus Platform: Enabling Genetic Medicines Biseasaocn Year of _ ‘Arcturus ‘Arcturus mRNA xme Partner tndicetion Chemistry Delivery __ Process UNARHBY folate 2088 gate rn LUNAR-NASH 2017 NASH ANA snag RCT ) Glycogen Storage LUNAR® wnar-csos—_ultragenyX 2016 Disease Type I m
Slide 7: LUNAR Platform Substantial Preclinical Proof-of-Concept Demonstrated
LUNAR® Platform Substantial Preclinical Proof-of-Concept Demonstrated K en * — a TRINA aN TinaRoTe Takeda =208 a i iramescutor Hivinvitro {ARCT-810) LUNAR-NASH LUNA GSO RNA 4 4 LUNARCF mRNA mRNA inretreal sirsachesl Subvetinal Tnnalston ultragenyx LUNAR? Platform Preclinical Proof-of-Concept Demonst
Slide 8: Target Opportunities for LUNAR Delivery Platform Exceed $100 Billion
Target Opportunities for LUNAR® Delivery Platform Exceed $100 Billion in Potential Value Eom oc om ‘fa tp |) =) }
Slide 9: Arcturus Pipeline of mRNA Medicines
ARCTURUS THERAPEUTICS kK Arcturus Pipeline of mRNA Medicines preeyeorte 7” Toa Prevalence Nome inden INDDate Teast Tagetca_—_Prevae orate LNAR-OTE —ycerhpase 2019 Intravenous (in) Wer Hepototes > 4000 (OTC) Deficiency ebuled bronchi v8 Aerosol to Lung, 6 Epithelial Cells " LUWAR-2020 Rarer Disease 2
Slide 10: OTC Deficiency Market Opportunity
ARCTURUS THERAPEUTICS A OTC Deficiency Market Opportunity pen © ) Omithine 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
Slide 11: LUNAR-OTC
ARCTURUS THERAPEUTICS LUNAR-OTC Disease Normalization Following Single and Repeat Dosing in OTC Mouse Model Survival of OTC-deficient Mice on High Protein Diet Weekly LUNAR- Surviving Animals (9) OTC Treatment 1.0 mg/Kg Time (Days) fe tt t LUNAR-OTC LV. Dosing Schedule LUNAR-OTC 1.0m¢/Kg (N=10) LUNA
Slide 12: LUNAR-OTC
ARCTURUS THERAPEUTICS LUNAR-OTC Exceeds Therapeutic Target of 10% Enzyme Replacement at all Doses in OTC-Deficient Mouse Model + OTCD impacts ureagenesis Periportsl Expression in the Liver of OTC Protein (ammonia detoxification) + The main site of ureagenesis is the periportal region of the liver* +
Slide 13: Arcturus Safety Profile
Arcturus Safety Profile External Validation Multiple strategic partnerships over many years confirms the positive safety profile of Arcturus LUNAR® and mRNA Arcturus is committed to developing safe mRNA products + 15 studies over several years with strategic partners * Over $3 Million invested to da
Slide 14: Cystic Fibrosis Market Opportunity
Cystic Fibrosis Market Opportunity x ® @ 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 infect
Slide 15: Nebulization and Delivery to Lung
ARCTURUS THERAPEUTICS NEBULIZATION AND DELIVERY TO LUNG LUNAR® Targeting Lung LUNAR® Delivery into Bronchial Epithelial Cells (BECs) TdTomato/Fox!1/Dapi LUNAR-Luc mRNA Functional Delivery of LUNAR®-mRNA into Lung Epithelial Cells 4
Slide 16: Internal RNA Development Platforms
ARCTURUS THERAPEUTICS INTERNAL RNA DEVELOPMENT PLATFORMS Drug Substance: mRNA Design Optimized conditions Improved protein Weekly Dosing in Non-Human Primates expression and + mRNA sequence gg duration 180 Mm PRS control + Chemistry = + Improved functional = = UNAREPO mRNA + Process optimization act
Slide 17: Arcturus mRNA Manufacturing
Arcturus mRNA Manufacturing x DNA Template IVT and Capping aaa = 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 Scalable to
Slide 18: Proprietary Reproducible & Scalable Production Process
ARCTURUS THERAPEUTICS PROPRIETARY REPRODUCIBLE & SCALABLE PRODUCTION PROCESS Drug Product: LUNAR® Formulation & Production “ pee see (in rare} % Encapsulated RNA Particle Size ‘Yo Eneapeulated RNA atch Sal (RNA Grams) atch Seale (RWAIn Grams) Botch scale aNAin Grams) re) 4 EncapsulatedNulele Act Par
Slide 19: Board of Directors
ARCTURUS THERAPEUTICS Board of Directors EGO Et Dr. Peter Farrell Andrew Sassine, MBA James Barlow, MA Dr. Magda Marquet Joseph E. Payne, MSc rman of he Board Director of the Board Directo f he Boars Dvectr ofthe Boars Disctor of he Boar, Four, Pesto & CEO @ALTHEA 2 MERCK ResMed Founder and Chairman
Slide 20: Management Team
ARCTURUS THERAPEUTICS XK Management Team £ 8&9 Joseph E. Payne, MSc Dr. Pad Chivukula Andrew Sassine, MBA Founder, Prostent & CEO ‘under, C50 & COO Dr. Suezanne Parker \Por Translational Biscay merck Nitto Fidelity !ONIS Shire 6
Slide 21: Slide 21
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