Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) has announced its third-quarter 2024 financial results and provided updates on its mRNA medicines and vaccine pipeline. The company's lead product, KOSTAIVE®, a self-amplifying mRNA COVID-19 vaccine, achieved its first commercial sale in Japan, triggering a $25 million milestone payment from CSL Seqirus.
Pipeline Progress
"I am thrilled about the approval of KOSTAIVE® for the COVID-19 JN.1 strain in Japan and for the continued success of the STARR® platform in multiple Phase 3 trials, underscoring CSL and Arcturus’ commitment to deliver disruptive technologies for protection against respiratory viral diseases," said Joseph Payne, President & CEO of Arcturus Therapeutics.
Cystic Fibrosis Program
ARCT-032, an inhaled mRNA therapeutic for cystic fibrosis (CF), is currently in a Phase 2 multiple ascending dose study. The study is evaluating the safety, tolerability, and efficacy of ARCT-032 in CF patients who do not qualify for or benefit from CFTR modulator medicines. Interim proof-of-concept data is expected in the first half of 2025. The Phase 2 study is screening individuals with CF who do not qualify for, or benefit from, CFTR modulator medicines due to dysfunctional or absent CFTR protein and/or drug intolerance.
Ornithine Transcarbamylase (OTC) Deficiency Program
ARCT-810, an mRNA therapeutic for ornithine transcarbamylase (OTC) deficiency, is also in Phase 2 clinical development. The program has been expanded into the United States with an open-label, multiple-dose study evaluating pharmacodynamics and safety in adults and adolescents requiring clinical management for OTC deficiency. Interim Phase 2 proof-of-concept data from both U.S. and European studies are anticipated in the first half of 2025. The placebo-controlled Phase 2 European study has completed the dosing phase (N = 8; 0.3 mg/kg) in OTC deficient individuals.
KOSTAIVE® Clinical Data
Arcturus and its partners CSL and Meiji Seika Pharma presented new 12-month post-vaccination data for KOSTAIVE® at the OPTIONS XII for the Control of Influenza conference. Results from a head-to-head study demonstrated that KOSTAIVE® maintained superior immunogenicity compared to the conventional mRNA vaccine COMIRNATY® for up to one year against Wuhan-Hu-1, Omicron BA.4/5, and certain other variants, at one-sixth the dose (5 μg vs 30 μg, respectively). These results were published in The Lancet Infectious Diseases. Additional Phase 3 data showed that bivalent KOSTAIVE®, ARCT-2301, induced superior immunogenicity over conventional bivalent mRNA vaccine COMIRNATY® that persisted against key variants up to six months post-vaccination.
Financial Results
For the third quarter ended September 30, 2024, Arcturus reported:
- Revenue: $41.7 million, a slight decrease from $45.2 million in the same period in 2023, primarily due to lower milestone achievement from the CSL agreement.
- Operating Expenses: $52.4 million, compared to $64.5 million for the three months ended September 30, 2023.
- Research and Development Expenses: $39.1 million, compared to $51.1 million for the three months ended September 30, 2023, driven by a decrease in manufacturing costs for the COVID program.
- Net Loss: Approximately $6.9 million, or ($0.26) per diluted share, compared to a net loss of $16.2 million, or ($0.61) per diluted share in the three months ended September 30, 2023.
- Cash Position: Cash, cash equivalents, and restricted cash were $294.1 million as of September 30, 2024, compared to $348.9 million on December 31, 2023. The company expects its cash runway to extend through the first quarter of fiscal year 2027.
"I am happy to report our first commercial milestone achieved from our CSL partnership for the first commercial sale of KOSTAIVE in Japan," said Andy Sassine, Chief Financial Officer of Arcturus. "We anticipate another milestone related to potential European approval in the first quarter of 2025. I am also happy to announce that Arcturus is planning to transfer our cystic fibrosis manufacturing process technology to ARCALIS."
Regulatory and Manufacturing Updates
Meiji Seika Pharma, CSL’s exclusive partner in Japan, began KOSTAIVE® commercial sales in September 2024, triggering the $25 million commercial milestone. Arcturus is also planning to file a Biologics License Application (BLA) for KOSTAIVE® in the U.S. in the first half of 2025, supported by positive results from multiple Phase 3 studies.
Earlier this year, CSL Seqirus’s partner Meiji Seika Pharma announced that it submitted a partial change application for an amendment to the manufacturing and marketing approval of KOSTAIVE® to include manufacturing sites in Japan, including ARCALIS, Inc., Arcturus’ manufacturing joint venture in Japan. When approved, Meiji Seika Pharma will begin selling domestically produced KOSTAIVE® this season.
Arcturus' versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics.
