Arcturus Therapeutics Announces Completion of First Three Dose Escalation Cohorts in Phase 1 Study of ARCT-810, Therapeutic Candidate for Ornithine Transcarbamylase (OTC) Deficiency

Arcturus Therapeutics today announced the completion of the first three dose escalation cohorts in its ongoing Phase 1 study with ARCT-810, the Company’s messenger RNA (mRNA)-based therapeutic candidate for Ornithine Transcarbamylase (OTC) deficiency.

The ARCT-810 Phase 1 study is a double blind, placebo-controlled, dose-escalation trial in healthy adult volunteers. The study has completed three cohorts in total and the fourth cohort is expected to complete this quarter. All cohorts are randomized 2:1 active to placebo and the study is designed to evaluate safety and tolerability, and pharmacokinetics, as primary and secondary endpoints.

All subjects in cohorts up to 0.3 mg/kg have completed dosing and all study visits. Based on the available preliminary study data, ARCT-810 has been well tolerated at these doses, which are all within the anticipated therapeutic range. All adverse events observed have been mild or moderate in nature and there have been no serious adverse events. ARCT-810 has demonstrated a favorable pharmacokinetic profile, and no ARCT-810 lipid was detectable in plasma beyond 48 hours following drug administration. The company plans to report final data following study completion, which is anticipated this quarter.

Steve Hughes, M.D., Chief Development Officer of Arcturus Said: We are pleased to have made rapid progress advancing our Phase 1 study for ARCT-810, a highly promising mRNA-based therapeutic candidate for OTC deficiency. Preliminary safety and pharmacokinetic data are favorable and supportive of continued development, Administration at the highest dose cohort is ongoing and we anticipate study completion later this quarter. Our second ARCT-810 clinical study in patients with OTC deficiency is now recruiting and we expect to begin dosing patients at a U.S. clinical site this quarter.

About ARCT-810

ARCT-810 utilizes Arcturus' LUNAR® lipid-mediated delivery platform to deliver OTC messenger RNA to liver cells. Expression of ornithine transcarbamylase enzyme in the liver of patients with OTC deficiency has the potential to restore normal urea cycle activity, preventing neurological damage and the need for liver transplantation. The ARCT-810 program is supported by preclinical data in OTC deficiency murine models demonstrating that dosing of LUNAR-OTC results in robust ornithine transcarbamylase protein expression and activity resulting in improvements in ureagenesis and plasma ammonia, and increased survival.

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