Arcturus Therapeutics (ARCT) The 38th Annual Roth Conference summary

Strategic focus and technology differentiation

• Refocusing on rare disease applications post-pandemic, leveraging mRNA and proprietary LUNAR lipid nanoparticle delivery technology.

• Approved COVID vaccine in Europe and Japan, but current value creation is centered on rare diseases.

• Manufacturing capabilities enable production of highly pure mRNA, supporting repeated dosing for rare diseases.

ARCT-810 for OTC deficiency: clinical and regulatory outlook

• Multiple phase I and II trials completed in the US and Europe, showing safety, tolerability, and biomarker changes.

• Priority is pediatric patients with severe disease; regulatory discussions ongoing with FDA, aiming for clarity in Q2 2024.

• Exploring single-arm trial design for children under six, with regulatory feedback expected soon.

• Adult development may proceed in parallel or sequentially, with initial focus on non-responders to gene therapy.

• Market opportunity is significant, especially in pediatrics, with potential for label expansion to adults.

Competitive positioning and market opportunity

• mRNA therapy offers a transient, genotype-agnostic approach, potentially applicable to most OTC deficiency patients.

• Differentiation from gene therapy competitors, targeting unmet needs in both pediatric and adult populations.

• Commercial strategy initially targets children, with potential to capture a large share of the market if successful.