Arcturus Therapeutics (ARCT) Earnings Call Presentation

ARCT-810 Overview - Arcturus is a commercial mRNA medicines company with a pipeline of multiple therapeutic candidates in advanced clinical trial development[7] - ARCT-810 aims to restore OTC enzyme function, potentially preventing neurological damage and the need for liver transplantation in Ornithine Transcarbamylase (OTC) Deficiency patients[9] - ARCT-810 has received Orphan Drug Designation (FDA), Orphan Medicinal Product Designation (EMA), Fast Track Designation (FDA), and Rare Pediatric Disease Designation (FDA)[14, 15, 16, 17] Clinical Trial Results - European Phase 2 study (N = 8; 6 ARCT-810 / 2 placebo) is completed, and the U S Phase 2 study (N = 3 completed to date) is ongoing[20] - Combined analysis of both Phase 2 studies showed mean glutamine levels decreased significantly (N = 8, p-value = 0 0055)[25] - In the Phase 2 Open-label U S Study, mean glutamine levels decreased significantly (N = 3, p-value = 0 004), with all subjects achieving normal levels after three administrations[28] - Interim Phase 2 data from the U S Open Label Study (N = 3) showed a mean RUF (relative ureagenesis function) increase of +14 7% from baseline to 28 days post-fifth dose, from 29 0% (SD 9 1%) to 43 7% (SD 21 7%), which is statistically significant (p-value = 0 026)[31] Safety and Tolerability - ARCT-810 was generally safe and well-tolerated in Phase 2 studies at all tested dose levels, comprising 40 participants to date, including 20 OTC deficient participants[34, 35] KOL Insights - Glutamine rises earlier than ammonia in OTC deficiency and has lower variability, making it a reliable indicator of urea cycle stress[47] - 15N-Ureagenesis assay directly reflects cycle function, providing a clear readout of therapeutic efficacy[47]