Arcturus says ARCT-810 Phase 2 interim data in OTC deficiency show safety, biomarker improvement

Arcturus reported interim Phase 2 data showing ARCT-810 was generally safe, with fasting ammonia maintained in-range and glutamine reduced.

• LUNAR 2.0 delivery platform showed about 40-fold higher protein expression versus LUNAR 1.0 in non-human primates.
• A separate primate study showed LUNAR 2.0 delivered 38-fold higher hOTC expression potency than ARCT-810’s key lipid ATX-95.
• FDA feedback supports adding ARCT-2601, a LUNAR 2.0-based OTCD candidate, into the ongoing Phase 2 study near year-end for ages 12+.
• ARCT-2601 is expected to enable lower or less frequent dosing versus ARCT-810, based on non-human primate data.