Arcturus Therapeutics Holdings Inc. · NASDAQ:ARCT
Arcturus Therapeutics — Q4 2025 Earnings Presentation
33 slides from Arcturus Therapeutics's earnings presentation, Mar 3, 2026
Slide 1: Next Generation RNA Medicines
NEXT GENERATION RNA MEDICINES March 2026
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements. These statements relate to future events and In some cases, you can identify forward-looking statements by terms such involve known and unknown risks, uncertainties and other factors which may cause our actual results,
Slide 3: Arcturus Therapeutics
Arcturus Therapeutics STARR® mRNA Vaccines Nasdaq: ARCT mRNA Therapeutic Candidates KOSTAIVE® Headquarters: San Diego, CA ARCT-810 (LUNAR-OTC) Approved in Japan, UK, EU Employees: 100 Ornithine Transcarbamylase Deficiency Founded: 2013 LUNAR-H5N1 ARCT-032 (LUNAR-CF) U.S. Pandemic Flu Cystic Fibrosis
Slide 4: Proprietary mRNA Technology
Proprietary mRNA Technology mRNA Technologies • mRNA for protein replacement Driving Therapeutic • Self-amplifying mRNA (STARR®) low-dose vaccine technology Programs LUNAR® Delivery Broad Intellectual Property Portfolio • Hepatocytes – intravenous • Airway Cells – inhaled • Myocytes – intramuscular
Slide 5: Pipeline of Arcturus-Owned mRNA Therapeutic Candidates
Pipeline of Arcturus-Owned mRNA Therapeutic Candidates Franchise Candidate Funded By Indication Global Prevalence Upcoming Milestone Ornithine Pivotal Trial Strategy LUNAR®-OTC Hepatic Transcarbamylase >10,000 Alignment with (ARCT-810) Deficiency (OTC) Regulatory Agencies H1 2026 Phase 2 LUNAR®-CF R
Slide 6: LUNAR-CF (ARCT-032)
LUNAR-CF (ARCT-032) Inhaled mRNA Therapeutic Candidate for Cystic Fibrosis
Slide 7: ARCT-032 Background
ARCT-032 Background An inhaled mRNA therapeutic that can build new CFTR protein in the lung cells of individuals with CF is desired Generally safe and well tolerated in Phase 2 studies after 28 daily treatments (up to 15 mg) Arcturus to initiate in H1 2026 a Phase 2 new cohort study in ~20 CF partic
Slide 8: Cystic Fibrosis: ARCT-032 Market Opportunity
Cystic Fibrosis ARCT-032 Market Opportunity LUNAR-CF Aims to Cystic Fibrosis Unmet Medical Need Restore CFTR Function >100,000 worldwide prevalence Effective CFTR modulators mRNA replacement therapy has the are not approved for treatment of all potential to produce wild-type CFTR in people with CF t
Slide 9: Class I CF: ARCT-032 Initial Target Population
Class I CF ARCT-032 Initial Target Population Class I CF Patients Unmet Medical Need Potential Uptake ~10,000 Class I CF patients globally Ineligibility for CFTR modulators is the Rapid adoption is expected among greatest area of unmet need in CF people with CF who carry ineligible mutations or who
Slide 10: ARCT-032 Clinical Summary
Phase 1 Study in Healthy Volunteers • N = 32 across 4 ascending single-dose cohorts • Safety, tolerability and PK data supported transition to Phase 1b study Phase 1b Study in Adults with Cystic Fibrosis ARCT-032 • N=7; Each CF participant received two administrations of ARCT-032 • Safety, tolerabil
Slide 11: ARCT-032 Phase 2 Interim Data
ARCT-032 Phase 2 Interim Data © 2026 Arcturus Therapeutics, Inc. 11
Slide 12: Mucus Plug Reduction is Important
Mucus Plug Reduction is Important Arcturus evaluated mucus plug reduction after 28 days of treatment with ARCT-032 High-Resolution CT Scans were analyzed utilizing Thirona’s FDA 510(k)-cleared AI Technology Lung structure changes are an important predictor and precursor of lung function improvements
Slide 13: Cohort 2: Mucus Reduction Summary
Cohort 2: Mucus Reduction Summary Subject Dose Number of Mucus Plugs Mucus Volume (mL) Number (mg) % Change @ Day 28 % Change @ Day 28 2 10 -38.5% -67.4% 4 10 -34.9% -27.5% 6 10 -28.5% -29.5% 5 10 -9.1% -6.1% 3 10 23.8% 9.1% 1 10 25.6% 60.9% 4 out of 6 participants in Cohort 2 exhibited Mucus Reduct
Slide 14: HRCT Imaging Background
HRCT Imaging Background Example of Adult Healthy Lung Example of Adult Class I CF Lung Mucus plugs are colored red © 2026 Arcturus Therapeutics, Inc. 14
Slide 15: HRCT Scans of Selected Class I CF Participants
HRCT Scans of Selected Class I CF Participants Cohort 2: Patient 2 Cohort 2: Patient 5 Reduction of mucus plugs after 28 days of treatment with ARCT-032 © 2026 Arcturus Therapeutics, Inc. 15
Slide 16: ARCT-032: Summary of Phase 2 Interim Data
ARCT-032: Summary of Phase 2 Interim Data • ARCT-032 generally safe and well tolerated at all tested dose levels (up to 15 mg) • Meaningful trends of clinical activity observed via high HRCT scans – After 28 days of treatment with ARCT-032 (10 mg), 4 out of 6 Class I CF participants exhibited encour
Slide 17: LUNAR-OTC (ARCT-810)
LUNAR-OTC (ARCT-810) Systemically Delivered mRNA for Ornithine Transcarbamylase (OTC) Deficiency
Slide 18: Ornithine Transcarbamylase (OTC) Deficiency
Ornithine Transcarbamylase (OTC) Deficiency ARCT-810 Market Opportunity The most common LUNAR-OTC Aims to Unmet Medical Need urea cycle disorder Restore Enzyme Function ~10,000 prevalence in U.S./ EU Present standard of care Establishing expression of OTC involves a strict diet (low protein, enzyme
Slide 19: ARCT-810 Clinical Summary
Phase 1 Study in Healthy Volunteers • Completed dosing up to 0.4 mg/kg, total participants N = 24, generally safe and well tolerated Phase 1b Single Ascending Doseaaaaa Study in OTCD Adults ARCT-810 • Completed enrollment and dosing of all cohorts (N=16) • Dose cohorts were 0.2, 0.3, 0.4 and 0.5 mg/
Slide 20: ARCT-810: European and U.S. Phase 2 Trials
ARCT-810: European and U.S. Phase 2 Trials Objectives • Establish safety and tolerability in OTC deficient adolescents and adults • Evaluate biomarker responses – glutamine, ammonia, ureagenesis European Phase 2 Study – Completed (N = 8; 6 ARCT-810 / 2 placebo) • Randomized, placebo-controlled • 0.3
Slide 21: ARCT-810 Phase 2 Interim Data
ARCT-810 Phase 2 Interim Data © 2026 Arcturus Therapeutics, Inc. 21
Slide 22: Glutamine Normalization Following ARCT-810 Administration
Glutamine Normalization Following ARCT-810 Administration Glutamine normal values range from ~400 to 800 umol/L Phase 2 (EU - 6 dose) Phase 2 (US - 5 dose) (Interim) 1000 1300 1200 900 Glutamine Normal Glutamine Normal Range Range Mean Glutamine (umol/L) 1100 Glutamine (umol/L) 800 1000 Final Dose D
Slide 23: ARCT-810 Significantly Increases 15N-Ureagenesis
ARCT-810 Significantly Increases 15N-Ureagenesis Interim Phase 2 Data U.S. Open Label Study (N = 3): Mean Relative Ureagenesis Function (RUF) increased +14.7% from baseline to 28 days post-fifth dose-- from 29.0% (SD 9.1%) to 43.7% (SD 21.7%) Mean RUF increase is statistically significant -- p-value
Slide 24: ARCT-810: Ammonia Stable and Within Normal Range
ARCT-810: Ammonia Stable and Within Normal Range Phase 2 (Europe) Interim Phase 2 (U.S.) 120 120 Mean Ammonia (umol/L) Mean Ammonia (umol/L) Hyperammonemia ≥100 μmol/L Hyperammonemia ≥100 μmol/L 100 100 80 REF ≤72 μmol/L 80 REF ≤72 μmol/L 60 60 40 40 20 20 0 0 1 15 29 43 57 71 85 1 15 29 43 57 71 85
Slide 25: ARCT-810: Summary of Phase 2 Interim Data
ARCT-810: Summary of Phase 2 Interim Data1 • Significant and consistent reduction in glutamine levels in both Phase 2 studies – from abnormal to normal levels • Significant increase in 15N-ureagenesis in the U.S. Phase 2 study – additional evidence of urea cycle improvement • Ammonia – stable and wi
Slide 26: Key Near Term Objectives H1 2026
Key Near Term Objectives H1 2026 Arcturus mRNA Therapeutic Clinical Programs ARCT-810 (OTC Deficiency) Establish clarity and alignment with FDA on the regulatory development paths • For severe pediatric OTC Deficient patients (0-6 years of age) • For OTC deficient adolescents and adults with stable
Slide 27: Vaccines
VACCINES therapeutics
Slide 28: STARR Self-amplifying mRNA Vaccine
STARR® Self-amplifying mRNA Vaccine STARR® self-replicating RNA-based prophylactic vaccine triggers rapid and prolonged antigen expression within host cells resulting in protective immunity against infectious pathogens Potential advantages over conventional mRNA Vaccine Superior immune response: Ind
Slide 29: KOSTAIVE Phase 3 Clinical Studies
KOSTAIVE® is Approved in KOSTAIVE 31 Countries ® (Monovalent) Phase 3 Non-inferiority safety and immunogenicity trial KOSTAIVE® • KOSTAIVE® administered at an 83.3% lower dose than Comirnaty® • Adults ≥18 years who previously received 3 doses of mRNA vaccines (≥3 mo before enrollment) • Conducted in
Slide 30: KOSTAIVE: More Durable Immune Response
KOSTAIVE®: More Durable Immune Response Phase 3 Persistence Data Comparing KOSTAIVE® (5 mcg) to Comirnaty® (30 mcg) ✓ A booster dose of ARCT-154 induced a similar or higher immune response against 4 epidemiologically relevant SARS-CoV-2 variants – Delta, Omicron BA.2, Omicron XBB.1.5.6 and Omicron B
Slide 31: LUNAR-H5N1 (ARCT-2304)
LUNAR-H5N1 (ARCT-2304) Avian Influenza Program U.S. Pandemic Preparedness Initiative
Slide 32: LUNAR-H5N1 (ARCT-2304)
LUNAR-H5N1 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 LUNAR-H5N1 low dose levels, with extended persistence of neutralizing antibodies co
Slide 33: Arcturus Board of Directors
Arcturus Board of Directors Moncef Slaoui, Ph.D. Joseph E. Payne, MSc John Markels, Ph.D. Peter Farrell, Ph.D. Chairman Board Member; President & CEO Board Member Board Member James Barlow, MA Magda Marquet, Ph.D. Edward W. Holmes, M.D. Jing L. Marantz, M.D., Ph.D., MBA Board Member Board Member Boa
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