Arcturus Therapeutics (ARCT) investor relations material

Corporate and technology overview

• Focus on next-generation messenger RNA medicines, with a proven COVID vaccine approved in 32 countries and ongoing partnerships for pandemic preparedness.

• Differentiation through LUNAR lipid nanoparticle delivery, which is biodegradable and optimized for specific cell types, enhancing safety for repeated therapeutic use.

• Manufacturing expertise enables large-scale, reproducible production of mRNA therapeutics, a key advantage for clinical and commercial supply.

Pipeline and clinical progress

• Lead respiratory program for cystic fibrosis (CF) targets Class I CF patients with inhaled mRNA, partnered with the CF Foundation and currently in a 12-week phase II open-label study.

• Flagship liver program for ornithine transcarbamylase (OTC) deficiency is progressing toward an end of phase II meeting with the FDA in the second half of the year.

Cystic fibrosis program details

• Inhaled mRNA therapy aims to build new CFTR protein in the lungs, addressing unmet needs for Class I CF patients who lack modulator options.

• Phase I and Ib studies demonstrated safety and tolerability; phase II study is ongoing, measuring lung function improvements and mucus reduction.

• Early phase II data show a 28% reduction in mucus plugs and 33% reduction in mucus volume after 28 days, with high-resolution CT scans supporting clinical activity.