Arcturus Therapeutics (ARCT) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Company overview and technology

• Focuses on next-generation messenger RNA medicines with differentiated delivery platforms for liver and lung diseases.

• Two lead programs are in phase II: one for ornithine transcarbamylase (OTC) deficiency (liver) and one for cystic fibrosis (lung).

• Self-amplifying mRNA technology underpins an approved COVID vaccine distributed in 32 countries, including Europe, the UK, and Japan.

• Self-amplifying mRNA platform is leveraged to generate non-dilutive funding for core therapeutic programs.

OTC deficiency program (ARCT-810)

• OTC deficiency is a severe urea cycle disorder with high unmet need, especially in pediatric populations.

• Current standard of care involves ammonia scavengers, which are burdensome and costly, with limited efficacy.

• The mRNA therapy aims to replace the dysfunctional enzyme, potentially offering a functional cure and dietary freedom.

• Phase II studies completed in both Europe/UK (older, stable patients) and US (younger, more severe cases); data readout expected later this month.

• FDA has provided productive feedback, and both new and retroactive data from US and Europe/UK will be included in the upcoming update.

Clinical data and safety for OTC program

• Key biomarkers include ammonia and glutamine, with increased FDA emphasis on ammonia in children and glutamine in adults.

• Novel N-15 ureagenesis assay will be included as supportive data.

• Early data show promising safety, tolerability, and biomarker normalization at low doses, with biodegradable, non-accumulating lipid nanoparticles.

• No major safety concerns highlighted; low dose levels help mitigate typical adverse events.