Arcturus Therapeutics (NASDAQ:ARCT) expects clinical data from two mRNA drug programs in 2027, including a full presentation of Phase 2 cystic fibrosis results in the second quarter and top-line data from a reformulated treatment for a rare liver disorder in the second half of the year.
President and CEO Joe Payne outlined the milestones during a fireside chat with Evan Wang, alongside Chief Medical Officer Alan Cohen. Payne said the company plans to share top-line cystic fibrosis data when it makes a decision about advancing the program to Phase 3, but did not provide a specific date.
Cystic Fibrosis Study Nears Data Readout
Arcturus’ inhaled mRNA therapy is designed to produce a new CFTR protein in lung cells. Payne said the approach is particularly relevant to people with Class I cystic fibrosis, who do not produce the transporter targeted by existing modulator therapies.
Cohen said earlier four-week dose-ranging cohorts tested daily doses of 5, 10 and 15 milligrams. At the 10-milligram dose, high-resolution CT scans showed improvements in mucus plugging and reductions in airway obstruction, he said. The company selected that dose for the current 12-week study.
The study is enrolling patients in the United States, Turkey and Israel. Cohen said enrollment remained active and would continue through year-end. In addition to spirometry, the study is assessing lung clearance, quality of life and CT scans.
Cohen said the company narrowed the eligible range of predicted lung function to 45% to 85% to improve its ability to detect a clinical signal. It also tightened requirements for consistency between screening and baseline spirometry measurements after variability complicated analysis in an earlier cohort.
Payne said a Phase 3 decision would depend on the collective data, including lung function, imaging and quality-of-life measures. A decision to proceed could trigger up to a $40 million contribution from Thermo Fisher to support the trial, he said. Arcturus did not provide timing guidance for a potential Phase 3 start.
Reformulated Liver Treatment in Focus
Arcturus also plans to begin a clinical trial of ARCT-2601, its reformulated mRNA candidate for ornithine transcarbamylase, or OTC, deficiency, early in 2027. Payne said top-line data are expected in the second half of the year.
Payne said the company’s earlier candidate, ARCT-810, normalized ammonia and glutamine levels at a dose of 0.5 milligrams per kilogram in stable adults. ARCT-2601 uses Arcturus’ newer LUNAR 2.0 lipid nanoparticle formulation, which Payne said performed 38 times better than the first-generation formulation in nonhuman primates. He noted that the primate data involved small numbers of animals.
The company hopes the reformulation could allow lower, less frequent doses and potentially home administration, rather than clinic infusions every two weeks. Those changes remain goals for the new candidate, not established clinical results.
Payne said Arcturus plans to evaluate the reformulated treatment in roughly six or fewer patients before discussing potential pivotal-trial paths with the Food and Drug Administration for stable adults and pediatric patients. The company also plans an investigational new drug filing for a phenylketonuria program in the second half of 2027, he said.
About Arcturus Therapeutics (NASDAQ:ARCT)
Arcturus Therapeutics Holdings Inc is a biotechnology company headquartered in San Diego, California, focused on developing medicines based on messenger RNA (mRNA) and other RNA technologies. The company’s platform includes LUNAR, a lipid-mediated delivery system designed to transport nucleic acids into cells, as well as technologies for producing and administering mRNA and self-amplifying RNA.
The company’s development programs address infectious diseases and rare genetic disorders. Arcturus has developed KOSTAIVE, a self-amplifying mRNA COVID-19 vaccine known as ARCT-154, in collaboration with CSL.
