Arcturus Therapeutics (NasdaqGM:ARCT) FY Conference Transcript

Summary of Arcturus Therapeutics FY Conference Call Company Overview - Company: Arcturus Therapeutics (NasdaqGM:ARCT) - Industry: Biotechnology, specializing in messenger RNA (mRNA) medicines and therapeutics - Founded: 2013 - Location: San Diego - Employee Count: Approximately 100 - Key Products: COVID vaccine Kostaive, H5N1 pandemic flu funding from BARDA [2][3] Core Products and Pipeline Kostaive COVID Vaccine - Approved in over 30 countries, including Japan, the UK, and the EU [2] - Partnered with CSL for distribution in Japan and BARDA for pandemic efforts in the U.S. [3] mRNA Therapeutic Candidates 1. Ornithine Transcarbamylase (OTC) Deficiency - Flagship liver program with a focus on replacing the missing OTC enzyme [19][20] - Phase 1 and 1B studies completed, transitioning to Phase 2 trials [20][21] - Received orphan drug designation and fast track designation from the FDA [21] 2. Cystic Fibrosis (CF) - Inhaled mRNA therapeutic candidate targeting Class I CF patients [9][10] - Phase 2 study ongoing with a focus on safety, tolerability, and preliminary efficacy [11][12] - CF Foundation has committed approximately $25 million to this asset [7] Key Milestones and Data Cystic Fibrosis Program - Phase 2 Study: - Initiating a fourth cohort study with 20 CF participants planned for the first half of 2026 [11][12] - Significant mucus plug reduction observed in the second cohort, with reductions of 28%-33% in volume [14] - High-resolution CT scans used to evaluate lung structure changes, which are predictors of lung function improvements [13] OTC Deficiency Program - Phase 2 Trials: - Focus on safety, tolerability, and biomarker responses (glutamine, ammonia, ureogenesis) [21][22] - Significant reduction in glutamine levels observed, indicating drug efficacy [22][23] - Ammonia levels maintained within normal range, supporting the drug's safety profile [23] Regulatory and Market Considerations - Regulatory Clarity: - Need for alignment with FDA on pivotal trial strategies for both pediatric and adult populations for OTC deficiency [24] - Importance of demonstrating clinical activity and safety to facilitate potential product approval [25] Financial Position - Cash Runway: Extended into 2028, providing flexibility to pursue clinical milestones and continue innovation on the LUNAR platform [34] - Operational Decisions: Restructuring and expense reduction have strengthened the balance sheet, allowing focus on key value-creating milestones [34][35] Conclusion - Arcturus Therapeutics is positioned to leverage its innovative mRNA delivery technology and strong financial position to advance its clinical programs in cystic fibrosis and OTC deficiency, with significant unmet medical needs in both areas. The upcoming regulatory discussions and clinical data will be critical for the company's future growth and market opportunities [24][35]