Arcturus Therapeutics Holdings Inc. · NASDAQ:ARCT
Arcturus Therapeutics — Investor Presentation (November 2019)
25 slides from Arcturus Therapeutics's investor presentation, Nov 7, 2019
Slide 1: Building the Next Generation of RNA Medicines
Building the Next Generation of RNA Medicines ARCTURUS THERAPEUTICS November 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: 15.1M; Employees: 85; Insider Ownership: 33% BUILDING INNOVATIVE RNA MEDICINES Broad and Strong Intellectual Property Portfolio 177 Patents & Patent Applicat
Slide 4: Pipeline: Arcturus mRNA Medicines Key Value Drivers: Platform & Pipeline
Pipeline: Arcturus mRNA Medicines BUILDING INNOVATIVE RNA MEDICINES Key Value Drivers: Platform & Pipeline ARCTURUS THERAPEUTICS Platform: LUNAR® Delivery, mRNA Drug Substance, and STARR (Self-Transcribing And Replicating RNA) Technology ™ Strategic Partners: More than $1 Billion in Potential Milest
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 Expected IND Date LUNAR-HBV Hepatitis B RNA LUNAR® Hepatocytes ARCT TBD LUNAR-NASH NASH RNA LUNAR® Stellate Cells ARCT TBD LUNAR-GSD3 Glycogen Storage Disease Type III mRNA LUNAR® Hepatocytes ARCT 202
Slide 7: Arcturus Pipeline of mRNA Medicines
Arcturus Pipeline of mRNA Medicines Name Indication Expected 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 2021 Nebulized
Slide 8: 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 9: Target Opportunities for LUNAR Delivery Platform Exceed $100 Billion in Potential Value
Target Opportunities for LUNAR® Delivery Platform Exceed $100 Billion in Potential Value Immuno-oncology Vaccines Immune System Infectious Disease mRNA geRNA DNA Lung Eye Oral Kidney Spleen Brain Liver mRNA geRNA DNA mRNA geRNA DNA mRNA geRNA DNA mRNA geRNA DNA mRNA geRNA DNA mRNA geRNA DNA geRNA DN
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: Disease Normalization Following Single and Repeat Dosing in OTC Mouse Model
BUILDING INNOVATIVE RNA MEDICINES Disease Normalization Following Single and Repeat Dosing in OTC Mouse Model LUNAR®-OTC ARCTURUS THERAPEUTICS Survival of OTC-deficient Mice on High Protein Diet - Weekly LUNAR-OTC Treatment
Slide 12: LUNAR-OTC Exceeds Therapeutic Target of 10% Enzyme Replacement at all Doses
LUNAR-OTC 11 BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS Exceeds Therapeutic Target of 10% Enzyme Replacement at all Doses in OTC-Deficient Mouse Model Therapeutic Target = 10% OTCD impacts ureagenesis (ammonia detoxification) The main site of ureagenesis is the periportal region of the
Slide 13: ATX Lipids are Effective and Degrade Rapidly
ATX Lipids are Effective and Degrade Rapidly ARCTURUS THERAPEUTICS BUILDING INNOVATIVE RNA MEDICINES 12 Protein Expression (in vivo) Esterase Catalyzed Degradation (in vitro) Next Generation ATX Lipids Retain Degradability & Improve Delivery Efficiency
Slide 14: ATX 2.0 Lipid Rapidly Clears in vivo
ATX 2.0 Lipid Rapidly Clears in vivo 13 BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS Plasma Liver ATX Lipid (the major component in LUNAR® technology) is rapidly degraded in vivo ATX Lipid Half-Life in the Liver is Approximately 20 hours
Slide 15: External Validation: 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 Top Safety Concern for RNA Medicines is Delivery Arcturus
Slide 16: mRNA Drug Substance
Arcturus Therapeutics Lots produced at Arcturus Lots produced at CMO as part of recent GMP campaign mRNA Drug Substance 15 Three 12.5 g lots produced in recent campaign are of equivalent quality and yield
Slide 17: LUNAR-mRNA Drug Product
BUILDING INNOVATIVE RNA MEDICINES LUNAR®-mRNA Drug Product Arcturus Therapeutics 16 Scalability of Drug Product demonstrated from milligram to multigram scale with yields > 85% Multiple batches (10g) of LUNAR®-OTC mRNA manufactured
Slide 18: 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 19: LUNAR Targeting Lung
LUNAR® Targeting Lung LUNAR + Luciferase mRNA LUNAR® Delivery into Bronchial Epithelial Cells (BECs) Nebulization TdTomato / FoxJ1 / Dapi BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS Functional Nebulized Delivery of LUNAR + mRNA into Lung Epithelial Cells
Slide 20: Drug Substance: mRNA Design
Drug Substance: mRNA Design Weekly Dosing in Non-Human Primates Arcturus’ proprietary mRNA optimization platform Optimize mRNA sequence Chemistry Process Sustained hEPO activity in NHPs upon repeat dosing BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS Proprietary mRNA Optimization Platform
Slide 21: Arcturus mRNA Manufacturing
Arcturus mRNA Manufacturing 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 Efficient Entire Process Less Than One Week Scalable to > 1Kg Access L
Slide 22: Drug Product: LUNAR Formulation & Production
BUILDING INNOVATIVE RNA MEDICINES Drug Product: LUNAR® Formulation & Production LUNAR® Reproducibility LUNAR® Scalability ARCTURUS THERAPEUTICS Proprietary, Reproducible & Scalable Drug Product Production Process LUNAR-Formulated mRNA Successfully Scaled From Milligram to Multigram Batch Sizes Parti
Slide 23: Board of Directors
Board of Directors ARCTURUS THERAPEUTICS Dr. Peter Farrell Chairman of the Board James Barlow, MA Director of the Board Dr. Magda Marquet Director of the Board Joseph E. Payne, MSc Director of the Board, President & CEO Founder & Chairman of ResMed Former CAO of Allergan Chairman & Co-Founder of Alt
Slide 24: 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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