Press release
Arcturus Therapeutics Provides Updates for Ornithine Transcarbamylase (OTC) Deficiency and Cystic Fibrosis (CF) Programs
ARCT-810 Phase 2 study completed enrollment at 0.3 mg/kg dose level in Europe with data expected later this year ARCT-810 to expand Phase 2 clinical program
About this update from Arcturus Therapeutics Holdings Inc.
[{"type":"text","content":"\nARCT-810 Phase 2 study completed enrollment at 0.3 mg/kg dose level in Europe with data expected later this year\n\n\nARCT-810 to expand Phase 2 clinical program in the U.S. to enroll patients with more severe disease\n\n\nARCT-032 IND for Phase 2 multiple ascending dose study to be submitted in the next 60 days\n\n\n SAN DIEGO--(BUSINESS WIRE)--\nArcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global messenger RNA medicines company focused on the development of infectious disease vaccines and medicines to treat unmet medical needs within liver and respiratory rare diseases, today is providing mid-year updates for ARCT-810, an investigational mRNA therapeutic to treat ornithine transcarbamylase (OTC) deficiency, and ARCT-032, an investigational inhaled mRNA therapeutic to treat cystic fibrosis (CF).\n\n\n“CFTR replacement using mRNA therapeutics is an area of significant medical interest, as it may benefit null patients and potentially many other CF patients,” said Dr. Juergen Froehlich, Chief Medical Officer of Arcturus Therapeutics. “The safety profile of ARCT-032 observed in Phase 1 and after two administrations in Phase 1b for patients dosed to date, including the trend of lung function improvement in the Class I participant, is encouraging. These data are supportive of expanding our clinical program to the U.S. and other regions with a Phase 2 multiple ascending dose protocol, and I look forward to a successful conduct and outcome of this planned study. We are also excited to have started patient screening for the expansion of our ARCT-810 clinical program in the U.S. to more severely affected OTC deficiency patients.”\n\n\nARCT-810 (OTC Deficiency) Update\n\n\nThe double blind ARCT-810 Phase 2 study in the EU and UK has completed enrollment of eight (8) subjects, including adolescents and adults, at the 0.3 mg/kg dose level. The participants in this group are randomized 3:1 and receive 6 doses of ARCT-810 or placebo administered every 14 days. Treatment and follow-up are ongoing with the safety and complete set of biomarker data expected later this year.\n\n\nARCT-810 is expanding the Company’s clinical program in the U.S. by enrolling patients with more severe disease. Patient screening has been initiated and the Company expects the Phase 2 clinical program to be completed in the United ...