Arcturus Therapeutics
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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.
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44th Annual J.P. Morgan Healthcare Conference summary13 Jan, 2026

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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Frequently asked questions

Arcturus Therapeutics Holdings Inc. is a biotechnology company focused on developing RNA-based therapeutics for the treatment of infectious diseases, rare diseases, and other medical conditions. The company utilizes its proprietary self-amplifying mRNA technology and lipid nanoparticle delivery system to develop vaccines and therapies. Arcturus' pipeline includes treatments for diseases such as COVID-19 and other RNA-based therapies targeting unmet medical needs. The company is headquartered in San Diego, California, and its shares are listed on the NASDAQ.

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