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March 2025 NEXT GENERATION RNA MEDICINES
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2© 2025 Arcturus Therapeutics, Inc. 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 performances or achievements expressed or implied by the forward-looking statements. Each of these statements is based only on current information, assumptions and expectations that are inherently subject to change and involve a number of risks and uncertainties. Forward-looking statements include, but are not limited to, statements about: our strategy, future operations, collaborations, the likelihood of success (including safety and efficacy) and promise of our pipeline, the development, manufacture or commercialization of our pipeline and partnered pipeline assets, the likelihood of success of, and achievement of revenues from, our partnered programs, the planned initiation, design or completion of clinical trials the likelihood that we will obtain clearance from regulatory authorities to proceed with planned clinical trials, the ability to enroll subjects in clinical trials, the timing for receipt of data, the likelihood that preclinical or clinical data will be predictive of future clinical results, the likelihood that clinical data will be sufficient for regulatory approval or completed in time to submit an application for regulatory approval within a particular timeframe, the anticipated timing for regulatory submissions, the timing of, and expectations for, any results of any preclinical or clinical studies or regulatory approvals, the potential administration regimen or dosage, or ability to administer multiple doses of, any of our drug candidates, our manufacturing methods and technologies (including purification, lyophilization and stability of our products), the likelihood that a patent will issue from any patent application, our current cash position and adequacy of our capital to support future operations, and any statements other than statements of historical fact. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential” and similar expressions (including the negative thereof) intended to identify forward looking statements. Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward- looking statements such as the foregoing, and you should not place undue reliance on such forward-looking statements. The forward-looking statements contained or implied in this presentation are subject to other risks and uncertainties, including those discussed under the heading "Risk Factors" in Arcturus’ most recent Annual Report on Form 10-K with the SEC and in other filings that Arcturus makes with the SEC. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise. Trademark Attribution: The Arcturus logo and other trademarks of Arcturus appearing in this presentation are the property of Arcturus. All other trademarks, services marks, and trade names in this presentation are the property of their respective owners.
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3© 2025 Arcturus Therapeutics, Inc. Arcturus Therapeutics Nasdaq: ARCT mRNA Medicine Candidates Headquarters: San Diego, CA Founded: 2013 LUNAR-OTC Ornithine Transcarbamylase Deficiency LUNAR-CF Cystic Fibrosis Additional Earlier Stage Programs Strategic Partners Global mRNA Medicines Company KOSTAIVE® Approved in Japan KOSTAIVE® Approved by the European Commission Active Clinical Trials on Five Continents
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4© 2025 Arcturus Therapeutics, Inc. Broad Intellectual Property Portfolio Proprietary mRNA Technologies Driving Therapeutic Programs 500+ Patents & Patent Applications mRNA Technology • mRNA for protein replacement • Self-amplifying mRNA (STARR®) low-dose vaccine technology Manufacturing Know-How • Drug Substance Production (mRNA & STARR® mRNA) • mRNA Purification • Drug Product Production (LUNAR ® Lipids + mRNA) • Fill Finish / Lyophilization LUNAR® Delivery • Hepatocytes – intravenous • Myocytes – intramuscular • Bronchial Cells – inhaled
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5© 2025 Arcturus Therapeutics, Inc. STARR® self-replicating RNA-based prophylactic vaccine triggers rapid and prolonged antigen expression within host cells resulting in protective immunity against infectious pathogens STARR® Self-amplifying mRNA Vaccine Superior immune response: Induces higher neutralizing antibody response and increased immunogenicity Durable immune response: Requires less frequent boosters due to longer and more durable immune response Broad immune response: Demonstrated higher immune response to evolving variants of COVID-19 Lower dose level: Increase the potential for combined vaccines Manufacturing speed: Lower dose levels enabling vaccines do be produced more quickly and simply Potential advantages over conventional mRNA Vaccine
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6© 2025 Arcturus Therapeutics, Inc. Proprietary, Biodegradable, Optimized for Each Cell Type LUNAR® - Lipid Nanoparticle (LNP) Delivery Technology LUNAR® interacts with cell membrane LUNAR® internalized inside endosome mRNA release via engineered endosomolysis mRNA translated into protein of interest
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7© 2025 Arcturus Therapeutics, Inc. Pipeline of Arcturus-Owned mRNA Therapeutic Candidates Upcoming MilestoneFunded ByCandidate Indication Global Prevalence Phase 2 (EU & U.S.) Interim Data H1 2025 LUNAR®-OTC (ARCT-810) Ornithine Transcarbamylase Deficiency (OTC) > 10,000Hepatic Phase 2 Interim Data H1 2025 LUNAR®-CF (ARCT-032) Cystic Fibrosis 85,000-100,000Respiratory Franchise Each Arcturus-Owned Program Represents a Significant Commercial Opportunity
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8© 2025 Arcturus Therapeutics, Inc. Pipeline of Partnered STARR® mRNA Vaccines IndicationPartner StageCandidate COVID-19(i)KOSTAIVE® Approved JP Approved EU (ARCT-2138) LUNAR®-FLU Phase 1 Undisclosed Infectious Disease Preclinical 8 GREATER THAN $4 Billion in Potential Milestones & Profit Sharing/Royalties Seasonal Influenza (ARCT-2304) Pandemic LUNAR®-H5N1 Undisclosed Phase 1Pandemic Influenza Infectious Disease Preclinical (i) Commercialized in Japan
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9© 2025 Arcturus Therapeutics, Inc. CSL: Arcturus Therapeutics Global Vaccine Partner CSL Seqirus is one of the Three Core Businesses of CSL *CSL Full Year Results & ASX Information, August 2024 14.8 Billion USD Annual Revenue OPERATING IN 40+ Countries Worldwide 32,000+ Employees Worldwide 110 Million Influenza Doses Distributed in FY24 Focused on four strategic technology platforms – plasma protein; recombinant technology; cell and gene therapy; and vaccines Therapeutic areas of focus of immunology, hematology, respiratory, cardiovascular, transplant, nephrology and vaccines $2.13 Billion USD Annual Revenue* A World Leader in Flu Vaccines
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10© 2025 Arcturus Therapeutics, Inc. CSL Vaccine Partnership Collaboration combines CSL’s global vaccine commercial and manufacturing infrastructure with Arcturus’ expertise in mRNA design and modification, LUNAR® lipid nanoparticle (LNP) technology, Drug Substance and Drug Product manufacturing know-how. Deal terms encompass the development, manufacture, and commercialization of mRNA-based vaccines targeting COVID-19, Influenza and three additional respiratory infectious disease vaccines. 10 Partnership Terms 40% profit sharing for COVID-19 vaccines (defined as 40% of gross profits, less 40% of development costs) Up to double digit royalties for influenza and three additional infectious disease vaccines $200 million $1.3 billion $3.0 billion Upfront Payment Development Milestones Commercial Milestones Up to $4.3 Billion in Milestone Payments
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11© 2025 Arcturus Therapeutics, Inc. Meiji: Background Information Meiji Holdings Co., Ltd., IR Team, Corporate Communications Dept. Data Book, Fiscal Year 2024, October 9, 2024 Meiji Holdings Co., Ltd., IR Team, Corporate Communications Dept. Data Book, H1 of FYE March 2024, November 11, 2024 The Meiji Group provides food and pharmaceuticals indispensable to their customers 113 Locations Worldwide with 17,270 Employees $7.3 Billion USD Net Sales (As of March 31, 2024) • Announced investment in ARCALIS • Submitted application to amend Approval for KOSTAIVE® to include domestic manufacturing sites in Japan in July 2024 • Received approval in Japan for KOSTAIVE® by the MHLW in November 2023 • Entered into agreement with CSL Seqirus April 2023, responsible for obtaining regulatory approval, distribution, sales and marketing of KOSTAIVE® in Japan $1.36 Billion USD Net Sales (As of March 31, 2024) Meiji Seika Pharma provides antibacterial drugs, vaccines, central nervous system drugs, and generic drugs Meiji Seika Pharma, a Subsidiary of Meiji Holdings Co. Ltd., Funded and Conducted the KOSTAIVE ® Phase 3 Comparator Booster Study and Obtained Regulatory Approval in Japan
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12© 2025 Arcturus Therapeutics, Inc. ARCALIS: Arcturus’ Joint Venture mRNA Manufacturing Partner ARCALIS is a CDMO Specializing in Manufacturing of mRNA Vaccines and Therapeutics • Joint Venture Founded in 2021 • Major Equity Owners: Axcelead & Arcturus & Meiji • Meiji Seika Pharma is collaborating with ARCALIS for domestic mRNA vaccine production • Meiji established an equity position in ARCALIS through an investment in November 2024 • In January, Meiji Seika Pharma and ARCALIS received Ministry of Health, Labour and Welfare (MHLW) approval for commercial manufacturing sites in Japan for KOSTAIVE® ARCALIS’ cGMP mRNA Drug Substance Manufacturing Plant • Completed July 2023; Located in Minamisoma City, Japan • Capacity: Up to 5 kg in bulk mRNA drug substance per year • 78,059 sq ft (7,252 sq m) floor space ARCALIS’ cGMP mRNA Drug Product Manufacturing Expansion • Capacity: 30 L (3 Lines); building to 100 L (2 Lines) ARCALIS Awarded with $165 Million in Grants from the Japanese Government ARCALIS Major Equity Owners
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13© 2025 Arcturus Therapeutics, Inc. KOSTAIVE® Phase 3 Clinical Studies 1 Yoshiaki Oda, Yuji Kumagai, Manabu Kanai, Yasuhiro Iwama, Iori Okura, Takeshi Minamida, Yukihiro Yagi, Toru Kurosawa, Benjamin Greener, Ye Zhang, Judd L Walson. Immunogenicity and safety of a booster dose of a self-amplifying RNA COVID-19 vaccine KOSTAIVE ® versus BNT162b2 mRNA COVID-19 vaccine: a double-blind, multicentre, randomised, controlled, phase 3, non-inferiority trial, The Lancet Infectious Diseases, 2023, https://doi.org/10.1016/S1473-3099(23)00650-3. 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® (30 mcg) • Conducted in Japan KOSTAIVE® is Approved in Japan and the European Union—total of 31 countries KOSTAIVE® (Monovalent) KOSTAIVE® (Bivalent, ARCT-2301) Achieved Secondary Endpoint of superiority of KOSTAIVE® in neutralizing antibody response against SARS-CoV-2 Omicron BA.4/5 variant; increased immunogenicity associated with KOSTAIVE® versus Comirnaty® at Day 29 Achieved Primary Endpoint of non-inferiority of neutralizing antibody response against SARS-CoV-2 Ancestral strain compared to Comirnaty ® Generally safe and well tolerated Phase 3 Study published in The Lancet Infectious Diseases 1 © 2025 Arcturus Therapeutics, Inc. Bivalent KOSTAIVE® (ARCT-2301: ancestral D614G and Omicron BA.4-5) • Results consistent with monovalent KOSTAIVE ® • Phase 3 clinical booster vaccination study was also conducted in Japan Bivalent KOSTAIVE ® was assessed in comparison with bivalent conventional mRNA vaccine (Comirnaty®): • Day 29 superiority of neutralizing antibody response against SARS-CoV-2 Ancestral strain was established • Day 29 superiority of neutralizing antibody response against SARS-CoV-2 Omicron BA.4/5 subvariant was established • Day 29 neutralizing immune response against SARS-CoV-2 Omicron XBB.1.5 subvariant was higher compared to Comirnaty KOSTAIVE Represents a Significant Advancement in Vaccine Technology, Demonstrating Superior Immunogenicity and Antibody Persistence for up to 12 Months Post-Vaccination Compared to Conventional mRNA COVID-19 Vaccines in Clinical Trials
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14© 2025 Arcturus Therapeutics, Inc. CSL-Arcturus Collaboration Results in Groundbreaking Approval of KOSTAIVE® Historic Approval of World’s First sa-mRNA Product © 2025 Arcturus Therapeutics, Inc. Unprecedented approval paves the way for additional sa-mRNA vaccines First Arcturus Approval Enduring Vaccine with Strong Clinical Data KOSTAIVE® self-amplifying mRNA COVID vaccine was approved in Japan by the MHLW in November 2023 and the centralized marketing authorization of KOSTAIVE is valid in all EU member states and in the EEA countries Feb 2025 1 The STARR® vaccine was created, optimized, clinically developed and approved in under 4 years Approval based on positive clinical data from several KOSTAIVE ® studies 18,000+ subjects have received sa-mRNA COVID vaccines Partner Meiji Seika Pharma advanced the MHLW approval and is the exclusive distributor of KOSTAIVE ® in Japan 1 The centralized marketing authorization of KOSTAIVE provided by the EC is valid in all 27 European Union (EU) member states and 3 additional European Economic Area (EEA) countries summarized here: Austria, Belgium, Bulgaria, Croatia, Republic of Cyprus, Czech Republic, Denmark, Estonia, Finland, France, Germany, Greece, Hungary, Iceland, Ireland, Italy, Latvia, Liechtenstein, Lithuania, Luxembourg, Malta, The Netherlands, Norway, Poland, Portugal, Romania, Slovakia, Slovenia, Spain and Sweden KOSTAIVE® is Approved in 31 Countries
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15© 2025 Arcturus Therapeutics, Inc. Phase 3 Persistence Data Comparing KOSTAIVE® (5 mcg) to Comirnaty® (30 mcg) COMIRNATY® is the brand name of BNT162b2 KOSTAIVE®: More Durable Immune Response KOSTAIVE® sa-mRNA Booster Shows Higher Durability of Immune Response Compared to Approved mRNA Vaccine KOSTAIVE® (5 mcg) COMIRNATY® (30 mcg) Wuhan-Hu-1 Omicron BA.4/5 Surrogate Neutralizing Antibody Titer (log scale) Day 1 Day 29 Day 91 Day 181 Day 1 Day 29 Day 91 Day 181 10000 1000 100 813 866 3738 2899 1861 1624 275 292 888 495 2125 1892 1119 5390 5928 4119 Oda Y et al. Lancet Infect Diseases; February 1, 2024 DOI: (10.1016/S1473-3099 mcg(mcg 24)00060-4)
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LUNAR-H5N1 (ARCT-2304) Avian Influenza Program U.S. Pandemic Preparedness Initiative
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17© 2025 Arcturus Therapeutics, Inc. LUNAR-H5N1 (ARCT-2304) Phase 1 Randomized placebo-controlled trial (NCT06602531) • Designed to enroll approximately 200 healthy adults • 120 participants 18-59 years old; 80 participants 60-80 years old • Clinical study is fully funded by BARDA • Immune responses are measured by hemagglutination inhibition (HAI), virus microneutralization (MN) and neuraminidase enzyme-linked lectin assays (ELLA) Received Clearance from FDA to Begin H5N1 Pandemic Flu Vaccine Clinical Trial in Nov 2024 Initiated Phase 1 Dosing in December 2024 Third STARR® mRNA Vaccine Candidate to Enter Clinical Development LUNAR-H5N1 H5N1 Influenza H5N1 influenza is a significant concern in animal health. To date, H5N1 flu has affected over 10,000 wild birds, nearly a thousand dairy cows, and over 130 million poultry. Elevated H5N1 infections in animals have led to increasing numbers of human infections including two confirmed severe cases in the United States and one death. Most of the confirmed 67 human infections were due to exposure of U.S. dairy and poultry workers to infected dairy cows and poultry Primary objective Evaluate safety and immune responses of three different dose levels and two different vaccination schedules of ARCT-2304 vaccine ARCT-2304 Utilizes clinically validated LUNAR® delivery and STARR® mRNA platform technologies. STARR mRNA has demonstrated in multiple clinical trials its ability to elicit a robust immune response at very low dose levels, with extended persistence of neutralizing antibodies compared to approved conventional mRNA vaccines LUNAR-H5N1 First Clinical Data Anticipated H2 2025
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LUNAR-CF (ARCT-032) Inhaled mRNA Therapeutic Candidate for Cystic Fibrosis
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19© 2025 Arcturus Therapeutics, Inc. ARCT-032 Market Opportunity Cystic Fibrosis 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 progressive airway damage and ultimately, respiratory failure Unmet Medical Need LUNAR-CF Aims to Restore CFTR Function An mRNA replacement therapy has the potential to produce wild-type CFTR into the lungs of CF patients, independent of genotype 1 Cystic Fibrosis Foundation. (2024). 2023 CYSTIC FIBROSIS FOUNDATION PATIENT REGISTRY HIGHLIGHTS. In https://www.cff.org/medical-professionals/patient-registry. Highly effective CFTR modulators are not approved for treatment of all people with CF and may not be tolerated in others Standard of care therapies do not prevent the chronic, progressive loss of lung function that ultimately requires lung transplantation or leads to early death 8% of CF patients have genotypes making them ineligible for modulators 1 Additional 10% of CF patients are eligible but not prescribed modulators 1 Functional CFTR protein can restore chloride efflux in the airways, reducing mucus accumulation and airway damage and minimizing the progressive respiratory impairment observed in people with CF
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20© 2025 Arcturus Therapeutics, Inc. Successful delivery to airway epithelium; transduction demonstrated by Brown and Green staining LUNAR®-mRNA in Healthy Animals (four different species) LUNAR® Delivery to Airway Epithelium is Demonstrated in Rodent and Non-Rodent Species
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21© 2025 Arcturus Therapeutics, Inc. Successfully Transduces Epithelium in the Presence of CF Mucus LUNAR®-mRNA in Cystic Fibrosis Ferret Model LUNAR® Effectively Delivers mRNA Expressing Cre in a Ferret CF Model (G551D) High-res Low-res Luminal Mucus In collaboration with Univ. of Iowa; presented at North American CF Conference Nov 2023 Green denotes functional expression of protein (Cre) Trachea Bronchus
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22© 2025 Arcturus Therapeutics, Inc. ARCT-032 in a Kalydeco®-responsive CF Ferret Model (G551D) In collaboration with Univ. of Iowa; presented at North American CF Conference Nov 2023 Proof of Activity: Mucociliary clearance improves after single administration of ARCT-032 -10 0 10 20 30 40 50 60 70 80 90 100 1.5 2.5 3.5 4.5 5.5 6.5 7.5 8.5 9.5 10.5 11.5 12.5 13.5 14.5 % Clearance Minutes ARCT-032 Functionally Restores Mucociliary Clearance to Normal Levels in CF Ferrets ARCT-032 (LUNAR®-CF) Kalydeco® (Positive Control) LUNAR-TdTomato (Negative Control) Off Kalydeco® (Baseline)
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23© 2025 Arcturus Therapeutics, Inc. Restoration of CFTR Expression and Function in CF Human Bronchial Epithelial Cells In collaboration with Univ. of Alabama-Birmingham; presented at North American CF Conference Nov 2022 ARCT-032 Restores CFTR Expression & Function F508del Non-CF Control mRNA hCFTR mRNA Control mRNA Buffer F508del/ARCT-032F508del/TdTomato WT/TdTomato Isc (µA/cm2) WT Range ✱✱ Non-CF Range F508del Non-CF ∆ hCFTR mRNA Control mRNA In collaboration with UAB CFRC and Javier Campos-Gomez **P<0.01; Data from two F508del donors; Using chamber studies performed with chloride secretory gradient High Expression Levels of CFTR Protein Restored Chloride Activity (Chloride Gradient)
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ARCT-032 Clinical Update ARCT-032 Phase 2 Interim Data Expected H1 2025 Phase 1 Study in Healthy Volunteers • Completed dosing across 4 ascending single-dose cohorts (8 subject per cohort) • Total number of subjects N = 32 • Safety, tolerability and PK data supported transition to Phase 1b study Phase 1b Study in Adults with Cystic Fibrosis • CF Participants received two administrations of ARCT-032 • Total number of CF participants N = 7 • Safety, tolerability and PK data supported transition to Phase 2 study Phase 2 Study in CF Participants – Initiated December 2024 • Multiple ascending dose open-label study • Evaluating safety, tolerability and efficacy – including FEV improvements in lung function • Each participant is expected to receive daily treatments of ARCT-032 over a period of 28 days • Recruiting individuals that do not qualify or benefit from CFTR modulator therapy • The trial involves a relatively low number of study visits and 12 weeks of follow up ARCT-032 received Rare Pediatric Disease Designation and Orphan Drug Designation from the U.S. FDA and Orphan Medicinal Product Designation from the European Commission (EC) The Cystic Fibrosis Foundation has committed ~$25 Million to advance ARCT-032 24© 2025 Arcturus Therapeutics, Inc.
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LUNAR-OTC (ARCT-810) Systemically Delivered mRNA for Ornithine Transcarbamylase (OTC) Deficiency
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26© 2025 Arcturus Therapeutics, Inc. ARCT-810 Market Opportunity Ornithine Transcarbamylase (OTC) Deficiency The most common urea cycle disorder Unmet Medical Need LUNAR-OTC Aims to Restore Enzyme Function Establishing expression of OTC enzyme in liver has potential to restore urea cycle activity to detoxify ammonia, preventing neurological damage and potentially removing need for liver transplantation 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, and death Present standard of care involves a strict diet (low protein, high fluid intake) plus ammonia scavengers Present standard of care does not effectively prevent life- threatening spikes of ammonia Severe OTC Deficiency patients are referred for liver transplant, currently the only cure 10,000 prevalence in U.S./Europe
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27© 2025 Arcturus Therapeutics, Inc. LUNAR-OTC Treatment Increases OTC Expression in Mouse Periportal Hepatocytes (Main Site of Ureagenesis) LUNAR-OTC Periportal Expression of OTC Protein in Mouse Liver 0 10 20 30 40 50 60 70 80 90 100 110 Low Mid High % Natural Levels Dose Level 5% Threshold * Li, L. et al. PGC-1 Promotes Ureagenesis in Mouse Periportal Hepatocytes through SIRT3 and SIRT5 in Response to Glucagon. Scientific Reports. 6:24156 | DOI: 10.1038/srep24156, April 2016 * Lamers, W.H., Hakvoort, T.B.M., and Köhler, E.S. 'Molecular Pathology of Liver Diseases' in Monga S.P.S. (ed.), MOLECULAR PATHOLOGY LIBRARY SERIES, Springer Publishing, New York, pp. 125-132 | DOI: 10.1007/978-1-4419-7107-4 * Scharre, Svenja. “In vitro enzyme activity predicts phenotypic severity in male individuals with ornithine transcarbamylase deficiency.” SSIEM Annual Symposium 2022, Freiburg, Germany. 30 August – 2 September 2022. Poster Presentation. α Exceeds Target of 5% Enzyme Replacement in OTC-Deficient Mouse Model OTC deficiency impacts ureagenesis (ammonia detoxification) The critical threshold of 5% residual enzymatic OTC activity helps avoid severe manifestations of the disease (neonatal coma, mortality)* The main site of ureagenesis is the periportal region of the liver* 27
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ARCT-810 Clinical Update ARCT-810 Phase 2 (EU & U.S.) Interim Data Expected H1 2025 28© 2025 Arcturus Therapeutics, Inc. aaaaa aa Phase 1 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 Single Ascending Dose Study in OTCD Adults • Completed enrollment and dosing of all cohorts (N=16) • Dose cohorts were 0.2, 0.3, 0.4 and 0.5 mg/kg; no serious or severe adverse events Phase 2 (UK & EU) Single and Multiple Ascending Dose, Placebo- controlled Study in OTCD Adolescents & Adults • Completed enrollment of 8 subjects at the 0.3 mg/kg dose level • Up to 6 bi-weekly doses for each participant with the following endpoints ‒ Primary Endpoints: Safety and tolerability ‒ Secondary Endpoints: PK and PD (ureagenesis assay, plasma ammonia: 24-hr profile and peak level) ‒ Exploratory Endpoints: Plasma amino acids and OTC enzyme activity; urine orotic acid ARCT-810 received Orphan Drug Designation, Fast Track Designation & Rare Pediatric Disease Designation from the U.S. FDA and Orphan Medicinal Product Designation from the European Commission (EC) Phase 2 Open Label Expansion Study (U.S.) • Enrolling adults and adolescent participants with more severe disease • Multiple dose levels to be evaluated • Each participant is expected to receive five intravenous infusions administered over two months • First participant dosed at 0.5 mg/kg in December 2024
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29© 2025 Arcturus Therapeutics, Inc. Arcturus Board of Directors Jing L. Marantz, M.D., Ph.D., MBA Board Member Edward W. Holmes, M.D. Board Member Magda Marquet, Ph.D. Board Member James Barlow, MA Board Member Andrew Sassine, MBA Board Member; CFO Joseph E. Payne, MSc Board Member; President & CEO John Markels, Ph.D. Board Member Moncef Slaoui, Ph.D. Chair Designate Peter Farrell, Ph.D. Chairman