Arcturus Therapeutics (ARCT) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Platform technology and differentiation

• Self-amplifying mRNA technology enables longer protein expression (1–2 weeks) and higher antibody durability at much lower doses than conventional mRNA, reducing toxicity and enabling multi-antigenic vaccines.

• Proprietary purification methods and biodegradable, non-accumulating LUNAR lipid nanoparticles enhance safety and delivery efficiency.

• Delivery platforms are optimized for specific cell and organ types, including bronchial, hepatic, and muscle cells.

Cystic fibrosis (CF) program updates

• Phase I-B data showed a 4% FEV improvement after two doses in CF patients, including a Class I patient with no transporter, supporting phase II recruitment.

• No severe adverse events observed in 39 subjects across phase I and I-B; phase II trial design avoids placebo and lung brushing, improving recruitment.

• Phase II will test two dose levels, with daily or every-other-day dosing, using an optimized nebulizer for efficient delivery.

• Primary endpoint is improved lung function (FEV); even stabilization is meaningful for Class I patients with no current treatment options.

• Data from phase II expected next year, with ongoing recruitment and support from the CF Foundation.

COVID vaccine and partnerships

• Self-amplifying mRNA COVID vaccine (Kostaive) is approved in Japan, with four million doses shipped and commercial launch in October.

• Partnerships with CSL (global, except Japan) and Meiji (Japan) include profit-sharing; CSL and Meiji also collaborate.

• CSL holds 60% profit share in US/EU, Meiji has rights in Japan; milestone payments are incorporated into financial runway.

• Additional manufacturing capacity in Japan is being qualified for future production, with Meiji forecasting potential 10 million dose orders for 2025.

• Twelve-month durability and bivalent phase III data to be presented soon; global regulatory submissions ongoing, with EMA and BLA applications planned.