Arcturus Therapeutics Holdings Inc

Arcturus Therapeutics said interim phase 2 results for ARCT-810 showed the candidate reduced and/or maintained first morning fasting ammonia within the normal range in patients with ornithine transcarbamylase deficiency, and also reduced glutamine, with some individuals reaching the normal range. Based on those results and a June meeting with the US FDA, the company aims to begin dosing of ARCT-2601 close to the end of the year in participants 12 years and older under an amended phase 2 protocol that integrates ARCT-2601 into the current ARCT-810 phase 2 study. Both ARCT-810 and ARCT-2601 are mRNA therapeutics for OTC deficiency, but ARCT-810 uses the LUNAR platform while ARCT-2601 uses the next-generation LUNAR 2.0 platform, which Arcturus says produces greater than 30-fold higher protein expression and could allow for lower or less frequent dosing. OTC deficiency is a rare genetic disorder in which the body stops breaking down and removing nitrogen, leading to dangerous ammonia levels in the blood. Arcturus is also acquiring AI discovery company myNeo, with which it has worked since 2024, and the deal is expected to close in October.

Arcturus Therapeutics Holdings Inc. announced it will host a virtual presentation on Wednesday, September 23, 2026, at 4:30 p.m. ET covering the ARCT-810 Phase 2 clinical program for Ornithine transcarbamylase deficiency and the company's mRNA liver therapeutics platform. The company will issue a press release summarizing the presentation prior to the call. Marshall Summar, M.D., a founding member and Executive Committee member of the NIH UCD Consortium and a recognized expert in rare diseases and OTC deficiency, will participate in the presentation. ARCT-810 is an intravenously administered investigational mRNA therapeutic designed to express normal functional OTC enzyme in the liver of individuals with OTC deficiency, and it holds Orphan Medicinal Product Designation and an approved pediatric investigation plan from the European Medicines Agency, plus Orphan Drug Designation, Fast Track Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. In Europe and the U.S., approximately 10,000 people have OTC deficiency.

Moderna and Merck announced positive Phase 3 results for an individualized mRNA cancer vaccine, marking the first successful late-stage trial of its kind. The treatment showed significant improvement in recurrence-free and metastasis-free survival in melanoma patients when combined with Merck's Keytruda. Elon Musk said mRNA technology could fundamentally change how diseases are treated, making medicine more of a software problem than a biological one. Moderna shares skyrocketed 176.97% to close at $174.38, while Merck closed 12.6% higher at $152.20. BioNTech gained 21.96% and Arcturus Therapeutics closed 25.21% higher as peers rallied on the news.

Arcturus Therapeutics has entered a strategic collaboration with Thermo Fisher Scientific to support Phase 3 development and potential commercialization of ARCT-032, its investigational mRNA therapy for cystic fibrosis. Under the agreement, Thermo Fisher will provide Phase 3 manufacturing, clinical research, and related services, leveraging its Accelerator Drug Development solutions. If Phase 2 yields positive results, Arcturus expects to conduct the Phase 3 program through Thermo Fisher's PPD clinical research business. Subject to regulatory approval, Thermo Fisher will receive exclusive commercial manufacturing rights under a separate commercial agreement. The collaboration aims to accelerate development of ARCT-032 while preparing for potential commercial supply.

The global mRNA cancer vaccine market is projected to exceed US$1.20 billion by 2026, according to a new report from ResearchAndMarkets.com. The market is expected to see continued robust growth through 2036, driven by advances in precision oncology, sequencing technologies, and an expanding pipeline of clinical candidates. Key players such as BioNTech, Moderna, Arcturus Therapeutics, and Sana Biotechnology are advancing platform and clinical innovations, while innovations like self-amplifying mRNA constructs and improved lipid nanoparticle delivery systems are enhancing scalability. The report also highlights that production relies on globally sourced inputs like lipid nanoparticles and nucleotides, making supply chains vulnerable to tariffs and disruptions, which could impact costs and development timelines. The analysis covers market segmentation by vaccine type, therapeutic approach, clinical phase, indication, and end-user across regions including North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.