Arcturus Therapeutics (ARCT) Corporate presentation summary

Pipeline and platform

• Two first-in-class mRNA programs: inhaled ARCT-032 for cystic fibrosis and hepatic ARCT-810/ARCT-2601 for OTC deficiency; Phase 2 data expected Q2 2027 and H2 2027, respectively.

• LUNAR 2.0 produced 40x higher hEPO expression than LUNAR 1.0 in NHPs at 0.3 mg/kg; only the ionizable lipid differed between study arms.

• LUNAR 2.0 produced 38x higher hOTC expression than ATX-95 in NHPs at 0.3 mg/kg; ARCT-2601 uses the next-generation platform.

• Platform includes 100 mRNA technologies, 500+ patents and patent applications, and proprietary end-to-end manufacturing know-how.

Cystic fibrosis program

• ARCT-032 is an inhaled mRNA candidate intended to produce CFTR protein in lung cells; Phase 2 daily dosing for 28 days was generally safe and well tolerated up to 15 mg.

• In Phase 2 cohort 2, 4 of 6 participants on 10 mg daily showed mucus reduction; among these four, mucus plugs fell 27.8% and mucus volume fell 32.6% at Day 28.

• A 12-week open-label study is enrolling up to 20 people with Class I CF at 10 mg; endpoints include safety, tolerability, lung function, quality of life, and HRCT imaging.

• The Cystic Fibrosis Foundation committed $25M to ARCT-032; the candidate has U.S. Rare Pediatric Disease and Orphan Drug designations and EC Orphan Medicinal Product designation.

OTC deficiency programs

• ARCT-810 U.S. Phase 2 enrolled 8 participants across 0.3 and 0.5 mg/kg cohorts; multiple dosing was generally safe and well tolerated, with no serious AEs.

• At Day 60, mean ammonia was 19% below baseline at 0.3 mg/kg and 42% below baseline at 0.5 mg/kg; all participants maintained normal ammonia levels after treatment.

• Mean glutamine fell 17% at 0.3 mg/kg and 25% at 0.5 mg/kg; protein intake generally increased above baseline, supporting potential improvement in urea-cycle function and protein tolerance.

• FDA agreed to evaluate ARCT-2601 in the ongoing ARCT-810 Phase 2 study; planned IND filing is 4Q 2026, with a pediatric Phase 2/3 study targeted for H2 2027.