Arcturus Therapeutics (ARCT) H.C. Wainwright 28th Annual Global Investment Conference summary

Corporate overview and differentiation

• Focuses on next-generation mRNA medicines with unique delivery and purification technologies, including a proprietary nebulizer for inhaled treatments.

• Holds 100% strategic control of an approved COVID vaccine (KOSTAIVE), now commercialized in 32 countries, and has a strong partnership with BARDA for bird flu vaccine development.

• Differentiates itself with self-amplifying mRNA technology and scalable GMP manufacturing, being the only company to commercialize self-amplifying mRNA products.

• Maintains a stable revenue stream from vaccines, supporting investment in therapeutic programs.

Therapeutics pipeline and clinical progress

• Leads in rare disease therapeutics for ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF), with both inhaled and intravenous mRNA delivery platforms.

• CF program targets Class I CF patients, a group not responsive to modulators, with a phase III go/no-go decision expected in Q4, potentially triggering up to $40 million in additional funding from Thermo Fisher.

• Demonstrated safety and tolerability for inhaled mRNA therapy in CF, with self-administration at home and no need for steroids.

• Ongoing 12-week open-label CF study enrolling up to 20 Class I patients, collecting lung function and quality of life data, with strong support from the Cystic Fibrosis Foundation.

• Phase II data readout for OTC deficiency program expected later this month, with Fast Track designations and promising interim biomarker results.

Commercial and regulatory outlook

• Regained global control of KOSTAIVE, a COVID vaccine with demonstrated superiority in antibody response and duration over market leaders.

• COVID vaccine market remains significant, with ongoing commercial build-out and upcoming publications on bird flu vaccine data.

• CF and OTC programs have received rare pediatric disease and Fast Track designations, indicating regulatory momentum.

• Board and management team positioned to guide key decisions, including the pivotal CF phase III advancement and vaccine commercialization.