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Investing.com -- Arcturus Therapeutics Holdings Inc (NASDAQ:ARCT) stock rose 2% following positive interim results from two Phase 2 clinical trials of its ARCT-810 mRNA therapeutic for ornithine transcarbamylase (OTC) deficiency.
The company reported that ARCT-810 demonstrated significant biological effects across both studies, including consistent reduction and normalization of elevated glutamine levels, an important biomarker for urea cycle function. In the ongoing U.S. Phase 2 study, patients showed the first significant improvements in relative ureagenesis function using a new optimized assay.
In the combined analysis from both Phase 2 studies, patients receiving ARCT-810 while maintaining their standard care therapy showed significantly decreased glutamine levels (p-value = 0.0055) following multiple administrations. All three participants in the U.S. open-label study experienced sustained decreases in glutamine reaching normal levels after the first three doses.
The U.S. study also demonstrated statistically significant increases in relative ureagenesis function, rising from a baseline of 29.0% to 43.7% at 28 days after the fifth dose. Two of the three participants achieved levels above 50%, which the company considers clinically meaningful improvement.
Patients in both studies maintained ammonia levels within normal range after receiving at least two doses of ARCT-810, with stability continuing for approximately 28 days after completing treatment.
The treatment has been generally safe and well-tolerated across all studies to date, which have included 40 participants, 20 of whom have OTC deficiency. The company has improved the infusion regimen without requiring corticosteroid pre-treatment, and no serious infusion-related reactions have been observed using the improved 3-hour IV protocol.
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