SAN DIEGO–(BUSINESS WIRE)–May 12, 2025 Arcturus Therapeutics declared its strategic pivot towards experimental mRNA drugs, emphasizing programs for cystic fibrosis and the rare ornithine transcarbamylase (OTC) deficiency. This focus on mRNA technology was projected to extend the company’s financial runway into 2028.
Investors reacted positively to this mRNA-centric strategy. Arcturus’ stock ($ARCT) saw an initial surge, reflecting confidence in their mRNA approach for rare diseases. Analysts noted that this shift could position Arcturus as a significant player in the rare disease space, diversifying beyond their partnered vaccine programs.
Arcturus’ existing vaccine portfolio, including an approved COVID-19 shot and experimental flu vaccines, contrasts with their growing mRNA therapeutic ambitions. The cystic fibrosis mRNA program, ARCT-032, generated considerable interest, particularly after a competitor’s mRNA-based cystic fibrosis drug encountered tolerability issues. Both Arcturus and the competitor are developing mRNA therapies for patients unresponsive to standard treatments, utilizing lipid nanoparticles for mRNA delivery. Arcturus’ CEO expressed confidence in their distinct LNP and mRNA purification processes to mitigate such issues.
The potential for accelerated approval for their mRNA cystic fibrosis therapy hinges on strong Phase 2 data, expected around “mid-2025.” Separately, the mRNA program for OTC deficiency, ARCT-810, was in Phase 2, with results anticipated by the end of June of the previous year. Arcturus’ commitment to mRNA therapeutics signals a significant strategic evolution.
Source:
https://endpts.com/arcturus-to-prioritize-mrna-therapeutics-in-bid-to-boost-rare-disease-programs/
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