Arcturus Therapeutics(US:ARCT)2021-03-02 02:43Financial Data and Key Metrics Changes - For the year ended December 2020, the company reported revenues of $9.5 million, down from $20.8 million in the prior year, primarily due to a decrease in collaboration revenues [30] - Total revenue for Q4 was $2.2 million, relatively flat compared to $2.3 million in Q3 [30] - Total operating expenses for the year were $81.1 million, compared to $46.3 million in the prior year, driven by increased clinical and manufacturing expenditures related to COVID-19 and OTC programs [31] - Cash balance totaled $463 million as of December 31, 2020, up from $301.1 million on September 30, 2020, mainly due to a public offering [32] Business Line Data and Key Metrics Changes - The company is advancing its lead clinical pipeline candidates, including ARCT-021 for COVID-19 and ARCT-810 for OTC deficiency, with significant progress reported in 2020 [11][24] - ARCT-021 is a self-amplifying mRNA vaccine candidate, with ongoing Phase 2 studies involving over 500 subjects [18][56] - ARCT-810 has completed a Phase 1 dose escalation study in healthy volunteers and has initiated a Phase 1 study in patients with OTC deficiency [26] Market Data and Key Metrics Changes - The COVID-19 pandemic is expected to require vaccination of billions of individuals for years, with a focus on vaccines that can be easily updated to address variants [9][12] - The company is preparing to produce hundreds of millions of doses of ARCT-021 annually, with ongoing manufacturing campaigns [33] Company Strategy and Development Direction - The company aims to develop differentiated mRNA-based medicines, with a focus on vaccines and therapeutics for serious diseases [10] - ARCT-021 is positioned as a single administration vaccine, which could provide a competitive advantage in the market [35] - The company is also advancing ARCT-032 for cystic fibrosis, utilizing aerosolized administration of mRNA technology [27][36] Management's Comments on Operating Environment and Future Outlook - Management acknowledges the ongoing challenges posed by COVID-19 variants and emphasizes the importance of cellular immunity in providing broader protection [9][42] - The company is optimistic about obtaining emergency use authorization for ARCT-021 in the second half of 2021 [33][35] - Management is focused on rapidly progressing clinical trials and expects to report important milestones from various programs throughout the year [35] Other Important Information - The company received a $46 million loan from Singapore EDB to support the manufacturing of its COVID-19 vaccine program [33] - The company has laid the foundation for high-purity mRNA vaccine manufacturing, which is crucial for its various therapeutic applications [63] Q&A Session Summary Question: Can you comment on T cell responses post day 28? - Management has looked at longer time points for ARCT-021 and observed some contraction of T cells, which is normal as they switch to memory [39][41] Question: Is Arcturus ready to incorporate new constructs for variants in Phase III? - The company is closely monitoring COVID variants and is prepared to update the vaccine if necessary, leveraging its mRNA technology [42][46] Question: Will Phase II data be available before starting Phase III? - Phase II data will be available before Phase III, with interim analyses planned for safety and immunogenicity [44] Question: Can you discuss your solution to variants and Phase 3 trial design? - The company plans a randomized placebo-controlled Phase 3 study with several thousand participants, focusing on high COVID incidence areas [52][60] Question: What is the difference in your technology that allows for lyophilization? - The company has developed specific know-how for lyophilization, which allows for easier distribution compared to liquid formulations [71][72]