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Arcturus Therapeutics (ARCT) investor relations material
Arcturus Therapeutics 44th Annual J.P. Morgan Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.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.
KOSTAIVE mRNA vaccine approved in over 30 countries, including Japan, UK, and EU; pipeline includes LUNAR-H5N1 for pandemic flu, ARCT-810 for OTC deficiency, and ARCT-032 for cystic fibrosis.
Key partnerships include CSL, Meiji, and the CF Foundation.
Over 500 patents and applications support a broad intellectual property portfolio.
Manufacturing expertise in mRNA production, purification, formulation, and lyophilization is a 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.
Phase II studies show safety, tolerability, and significant mucus plug and volume reduction at higher doses.
Fourth cohort, 12-week study in up to 20 participants, planned for H1 2026, focusing on Class I CF patients.
High-resolution CT scans and AI analytics demonstrate significant lung structure improvements.
~$25 million commitment from the CF Foundation; received multiple orphan and rare disease designations from FDA and EC.
Ornithine Transcarbamylase Deficiency (OTC) Program Highlights
mRNA therapy (ARCT-810) addresses about 10,000 patients in US/EU with severe unmet need, aiming to restore urea cycle function and reduce need for liver transplant.
Phase I and II studies 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.
Regulatory alignment with FDA on pivotal trial strategy for pediatric and adult populations targeted for H1 2026.
Orphan, Fast Track, and Rare Pediatric Disease designations granted by FDA and EC.
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