Arcturus Therapeutics (ARCT) 16th Annual LD Micro Invitational Conference summary

Company overview and differentiation

• Leading next-generation mRNA medicines with an approved COVID vaccine in 32 countries, focusing on unique delivery and manufacturing technologies.

• Specializes in injectable, intravenous, and inhaled mRNA, with a strong emphasis on cystic fibrosis (CF) and rare liver diseases.

• Proprietary LUNAR lipid nanoparticle technology enables safe, effective delivery and high-dose administration.

• Self-amplifying mRNA platform is 30 times more efficient than conventional mRNA, with advanced manufacturing capabilities scaled during the pandemic.

Flagship programs and clinical progress

• Inhaled mRNA therapeutic for CF is in phase II, targeting Class I CF patients with no current standard of care.

• Achieved high-dose, daily administration without steroids, supported by a unique purification process and delivery technology.

• Phase II study extends treatment to 12 weeks, with support from the CF Foundation and orphan drug designations in the US and Europe.

• Demonstrated mucus plug reduction and promising safety/tolerability, with rapid adoption expected if successful.

Clinical data and future milestones

• Phase II CF study evaluates lung function improvements (FEV, LCI) over 12 weeks in 20 subjects.

• Ornithine transcarbamylase deficiency (OTC) program in phase II, focusing on restoring urea cycle function in rare liver disease.

• Interim data show normalization of glutamine and ammonia biomarkers, with end of phase II meeting planned for the second half of the year.

• Both programs have orphan drug status and rare pediatric disease designations, streamlining regulatory and commercial pathways.