Arcturus Therapeutics(US:ARCT)2025-05-12 21:30Financial Data and Key Metrics Changes - For Q1 2025, revenues were $29.4 million, down from $38 million in Q1 2024, primarily due to lower development milestone revenues from the CSL collaboration as the COVID vaccine transitions to commercialization [17][18] - Research and development expenses decreased to $34.9 million from $53.6 million year-over-year, driven by lower manufacturing costs, partially offset by increased costs for CF and OTC programs [18][19] - General and administrative expenses were $11.3 million, down from $14.9 million in the same period last year, mainly due to reduced share-based compensation costs [19][20] - The net loss for Q1 2025 was approximately $14.1 million, or $0.52 per diluted share, compared to a net loss of $3 million, or $1 per diluted share, in Q1 2024 [20] Business Line Data and Key Metrics Changes - The mRNA therapeutics pipeline is being prioritized, with significant focus on CF and OTC programs, while the COVID vaccine program is transitioning to commercialization [10][16] - The company expects to complete Phase II enrollment for ARCT032 by the end of 2025 and provide interim data for the first two cohorts in mid-2025 [6][9] - The ARCT-810 program for OTC deficiency is also progressing, with interim data expected in Q2 2025 [7][9] Market Data and Key Metrics Changes - The company received an initial milestone payment from CSL related to the EU approval of the COVID vaccine, Costave, and anticipates further milestone payments as the program progresses [10][17] - The company is preparing for a Marketing Authorization Application (MAA) filing in the UK in Q2 2025 and a U.S. BLA filing in Q3 2025 [11] Company Strategy and Development Direction - The company has made a strategic decision to focus resources on mRNA therapeutics, particularly CF and OTC programs, due to current market conditions and regulatory uncertainties [10][16] - The STAR self-amplifying mRNA platform continues to receive positive feedback from publications, enhancing the company's position in the market [11][13] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming clinical data from CF and OTC programs, indicating a strong cash runway extending into 2028 [21] - The company remains committed to advancing its therapeutic programs and believes that the science will support its regulatory interactions [100] Other Important Information - The company has received FDA Fast Track designation for ARCT2304, its mRNA vaccine candidate for pandemic influenza A [14] - The 15N Ureogenesis Assay is expected to provide important data for monitoring the effect of ARCT-810 in clinical development [9][108] Q&A Session Summary Question: Can you provide more details on extending the cash runway and potential cash flows? - Management indicated that focusing on critical programs and making cost reductions contributed to extending the cash runway, with a conservative approach to cash burn forecasts [26][27] Question: What milestones should we expect related to UK and U.S. approvals for the COVID vaccine? - There are no milestones associated with UK or U.S. approvals; however, a milestone is anticipated with the first U.S. revenues from Costave, expected in 2028 [31][60] Question: What is the expected size of the interim cohort for ARCT032? - The interim data set is expected to include 6 to 9 subjects, with a bar for success set at a 3% improvement in lung function [36][37] Question: How does ARCT032 differ from competitor programs? - The key differentiators include the exclusive LUNAR lipid nanoparticle delivery technology and the high purity of the mRNA construct, which may enhance safety and tolerability [44][45] Question: What are the thresholds for FEV1 improvement in the CF program? - A 3% improvement in FEV1 is considered a significant threshold, with higher improvements potentially allowing for smaller Phase III trial sizes [86][87] Question: Will other biomarkers be measured in the OTC program? - In addition to glutamine, orotic acid and other amino acids will also be measured as part of the biomarker strategy [88][89]