Arcturus Therapeutics (ARCT) 44th Annual J.P. Morgan Healthcare Conference summary

Company and Strategic Overview

• Transitioned to a leaner organization with about 100 employees, focusing on value-creating assets and next-generation RNA medicines leveraging proprietary mRNA and LUNARⓇ delivery technologies.

• COVID vaccine product approved in over 30 countries, including KOSTAIVEⓇ in Japan, UK, and EU, with key partnerships for distribution and pandemic flu funding.

• Over 500 patents and applications support a broad intellectual property portfolio.

• Manufacturing expertise in mRNA production, purification, formulation, and lyophilization supports scalability and safety.

• Differentiated LUNAR lipid nanoparticle delivery technology is a core competitive advantage.

Cystic Fibrosis (CF) Program Highlights

• Inhaled mRNA therapeutic (ARCT-032) targets over 100,000 CF patients globally, especially those ineligible for CFTR modulators, addressing a high unmet need.

• Phase II studies show ARCT-032 is safe and well tolerated, with significant mucus plug and volume reduction at 10 mg dose, using AI-analyzed HRCT scans.

• Achieved safety and tolerability at high doses (up to 15 mg daily), a first in the field.

• Fourth cohort (N=20, 12-week daily dosing) to begin in H1 2026, targeting Class I CF patients with highest unmet need.

• Cystic Fibrosis Foundation committed ~$25M; received multiple orphan and rare disease designations from FDA and EC.

Ornithine Transcarbamylase (OTC) Deficiency Program Highlights

• mRNA therapy (ARCT-810) replaces missing OTC enzyme, aiming to improve urea cycle function and reduce toxic ammonia and glutamine levels for ~10,000 patients in US/EU.

• Phase I and II studies in healthy volunteers and patients show safety, normalization of glutamine, increased ureagenesis, and stable ammonia levels.

• Ongoing US Phase II open-label study in severe patients; European Phase II completed in stable patients.

• Orphan, Fast Track, and Rare Pediatric Disease designations granted by FDA and EC.

• FDA meetings planned to align on pivotal study design for both pediatric and adult populations, with glutamine as a primary biomarker for adults and ammonia for severe pediatric cases.