Arcturus Therapeutics Holdings Inc. · NASDAQ:ARCT
Arcturus Therapeutics — Investor Presentation (June 2019)
20 slides from Arcturus Therapeutics's investor presentation, Jun 19, 2019
Slide 1: Building the Next Generation of RNA Medicines
Building the Next Generation of RNA Medicines ARCTURUS THERAPEUTICS June 2019 Exhibit 99.1
Slide 2: Forward Looking Statements
FORWARD LOOKING STATEMENTS ARCTURUS THERAPEUTICS 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 diffe
Slide 3: Arcturus is an mRNA Medicines Drug Development Company Focused on Rare Diseases
Arcturus is an mRNA Medicines Drug Development Company Focused on Rare Diseases HQ: San Diego; Founded: 2013; Nasdaq: ARCT Outstanding Shares: 10.8 M; Employees: 85; Insider Ownership: 24% BUILDING INNOVATIVE RNA MEDICINES Broad and Strong Intellectual Property Portfolio 152 Patents & Patent Applica
Slide 4: Arcturus mRNA Medicines: Key Value Drivers
Arcturus mRNA Medicines BUILDING INNOVATIVE RNA MEDICINES Key Value Drivers: mRNA Medicines & Platform ARCTURUS THERAPEUTICS Arcturus LUNAR® Delivery Platform: Enabling Genetic Medicines Strategic Partners: More than $1 Billion in Potential Milestones & Royalties OTC Deficiency market potential $500
Slide 5: LUNAR Mechanism of Delivery
LUNAR® Mechanism of Delivery LUNAR Associates with Cell Membrane Enters Cell Via Endocytosis Lipid Particle in Endosome pH-Mediated Disruption Rapid Biodegradation of Vehicle RNA in Cytosol RNA Processing and Translation Increased Acidity as Endosome Ages ARCTURUS THERAPEUTICS BUILDING INNOVATIVE RN
Slide 6: Arcturus Platform: Enabling Genetic Medicines
BUILDING INNOVATIVE RNA MEDICINES Name Partner Indication Arcturus Chemistry Arcturus Delivery mRNA Process LUNAR-HBV Hepatitis B RNA LUNAR® Hepatocytes ARCT LUNAR-NASH NASH RNA LUNAR® Stellate Cells ARCT LUNAR-GSD3 Glycogen Storage Disease Type III mRNA LUNAR® Hepatocytes ARCT LUNAR-RARE Undisclose
Slide 7: LUNAR Platform Substantial Preclinical Proof-of-Concept Demonstrated
LUNAR® Platform Substantial Preclinical Proof-of-Concept Demonstrated Vaccines Respiratory Ophthalmology Liver Cancer Inf. Disease Infectious Disease LUNAR-CF mRNA Intratracheal Inhalation Gene Editing RNA mRNA Animal Health mRNA Shared Antigens Ultragenyx LUNAR-GSD3 mRNA Takeda LUNAR-NASH LUNAR-OTC
Slide 8: Target Opportunities for LUNAR Delivery Platform Exceed $100 Billion
Target Opportunities for LUNAR® Delivery Platform Exceed $100 Billion in Potential Value Transcription Factors Immuno-oncology (non-vaccine) Vaccines Respiratory Ophthalmology CNS Liver Cancer Inf. Disease Oral Intracellular Targets Gene Editing Immune System Transcription Factors Transcription Fact
Slide 9: Arcturus Pipeline of mRNA Medicines
Arcturus Pipeline of mRNA Medicines Name Indication IND Date Route of Administration Target Organ Target Cells Prevalence Worldwide LUNAR-OTC (ARCT-810) Ornithine Transcarbamylase (OTC) Deficiency Q1 2020 Intravenous (i.v.) Liver Hepatocytes > 10,000 LUNAR-CF Cystic Fibrosis H2 2020 Nebulized Aeroso
Slide 10: OTC Deficiency Market Opportunity
OTC Deficiency Market Opportunity LUNAR-OTC Aims to Restore Enzyme Function Expression of OTC enzyme in liver has potential to restore normal urea cycle activity to detoxify ammonia, preventing neurological damage and removing need for liver transplantation Unmet Medical Need Present standard of car
Slide 11: LUNAR-OTC Disease Normalization in OTC Mouse Model
LUNAR-OTC 10 BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS Disease Normalization Following Single and Repeat Dosing in OTC Mouse Model Survival of OTC-deficient Mice on High Protein Diet Weekly LUNAR-OTC Treatment
Slide 12: LUNAR-OTC Exceeds Therapeutic Target of 10% Enzyme Replacement
LUNAR-OTC 11 BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS Exceeds Therapeutic Target of 10% Enzyme Replacement at all Doses in OTC-Deficient Mouse Model LUNAR-OTC treatment increases OTC expression in mouse periportal hepatocytes (main site of ureagenesis) Therapeutic Target = 10% OTCD im
Slide 13: Arcturus Safety Profile External Validation
Arcturus Safety Profile 12 BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS 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
Slide 14: LUNAR-CF Aims to Restore CFTR Function
LUNAR-CF Aims to Restore CFTR Function An mRNA replacement therapy has the potential to deliver a new copy of CFTR into the lungs of CF-patients, independent of any genotype A functional CFTR protein can restore chloride channel efflux in the airways, reducing mucus accumulation, tissue scarring and
Slide 15: LUNAR Targeting Lung
LUNAR® Targeting Lung NEBULIZATION AND DELIVERY TO LUNG Functional Delivery of LUNAR®-mRNA into Lung Epithelial Cells LUNAR-Luc mRNA LUNAR® Delivery into Bronchial Epithelial Cells (BECs) Nebulization TdTomato/FoxJ1/Dapi BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS
Slide 16: Drug Substance: mRNA Design
Drug Substance: mRNA Design INTERNAL RNA DEVELOPMENT PLATFORMS Proprietary mRNA Optimization Platform Demonstrates Sustained Activity Upon Repeat Dosing in NHPs Weekly Dosing in Non-Human Primates Arcturus’ proprietary mRNA optimization platform Optimized conditions mRNA sequence Chemistry Process o
Slide 17: Arcturus mRNA Manufacturing
Arcturus mRNA Manufacturing Arcturus’ Internal mRNA Production: Up to 30 g in Less Than One Week ARCTURUS THERAPEUTICS 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 E
Slide 18: Drug Product: LUNAR Formulation & Production
BUILDING INNOVATIVE RNA MEDICINES Drug Product: LUNAR® Formulation & Production PROPRIETARY REPRODUCIBLE & SCALABLE PRODUCTION PROCESS LUNAR® Has Been Successfully Scaled From Milligram to Multigram Batch Sizes LUNAR® Reproducibility LUNAR® Scalability Particle Size % Encapsulated RNA % Encapsulated
Slide 19: Management Team
Management Team ARCTURUS THERAPEUTICS Joseph E. Payne, MSc Founder, President & CEO Andrew Sassine, MBA CFO Dr. Pad Chivukula Founder, CSO & COO Kevin Skol, MBA Sr. VP of Business Development & Alliance Management Dr. Suezanne Parker VP of Translational Biology BUILDING INNOVATIVE RNA MEDICINES
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