Arcturus Therapeutics (ARCT) investor relations material

Company overview and technology

• Focuses on next-generation mRNA medicines with differentiated delivery technologies for inhaled and injectable applications.

• Vaccine enterprise, including an approved COVID vaccine in 32 countries, funds therapeutic programs.

• Two flagship assets: inhaled mRNA for cystic fibrosis (CF) and injectable mRNA for ornithine transcarbamylase (OTC) deficiency.

Cystic fibrosis (CF) program (ARCT-032)

• ARCT-032 delivers mRNA to express new CFTR protein in lungs, targeting Class I CF patients with high unmet need.

• Uses LUNAR lipid nanoparticle technology for inhaled delivery via nebulizer.

• Phase II study enrolling up to 20 subjects in the US, Turkey, and Israel, focusing on lung function and quality-of-life endpoints.

• Decision to proceed to phase III expected in Q4, triggering up to $40 million in support from Thermo Fisher.

• Data will include lung function, quality-of-life, and high-resolution CT scans, with comparison to CF Foundation normative study.

Thermo Fisher partnership

• Thermo Fisher to provide up to $40 million for phase III if decision to proceed is made.

• Deal grants Thermo Fisher commercial manufacturing rights in exchange for phase III support.

• Thermo Fisher had access to data before deal; ongoing periodic updates are provided.