Arcturus Therapeutics (ARCT) Jefferies London Healthcare Conference 2024 summary

Company overview and technology differentiation

• Focuses on next-generation mRNA medicines with commercialization initiated in Japan.

• Holds strong IP in self-amplifying mRNA and proprietary LUNAR lipid nanoparticle delivery platform.

• Demonstrates unique large-scale manufacturing capabilities for self-amplifying mRNA.

• Delivery technology optimized for myocytes, hepatocytes, and bronchial epithelial cells.

• LUNAR platform offers biodegradability and non-accumulation, proven in clinical trials.

Pipeline highlights and clinical milestones

• Flagship assets target ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF).

• Phase 2 interim data for both OTC and CF programs expected in the first half of next year.

• KOSTAIVE, a COVID-19 vaccine, approved and sold in Japan; MAA filed in Europe with CHMP opinion expected soon.

• Pandemic flu program, funded by BARDA, recently approved to proceed into phase 1.

• Multiple partnered self-amplifying mRNA vaccine programs in progress.

Strategic partnerships and commercialization

• CSL Seqirus partnership includes $3 billion in commercial milestones and 40% profit sharing in the U.S. and Europe.

• Meiji supports Japanese distribution and invested in joint venture Arcalis for mRNA manufacturing.

• Arcalis facility completed, nearing PMDA approval, with expansion planned.

• KOSTAIVE technology offers extended antibody durability and lower dosing.