Arcturus Therapeutics has announced interim Phase 2 results for ARCT-810 in OTC deficiency. The company also introduced its LUNAR 2.0™ mRNA delivery platform. The update highlights progress across Arcturus Therapeutics’ liver-focused mRNA therapeutics pipeline. Additionally, the company outlined plans for ARCT-2601 and further liver pipeline programs. ARCT-810 generally remained safe and well tolerated during the ongoing Phase 2 study. The treatment also showed encouraging activity across important urea cycle biomarkers.
Moreover, Arcturus reported strong preclinical results for its next-generation delivery platform. LUNAR 2.0™ produced more than 30-fold higher protein expression across multiple non-human primate studies. The company said these results could expand the therapeutic opportunities available through mRNA medicines. Consequently, Arcturus plans to advance additional programs using the new platform.
“The ARCT-810 Phase 2 interim results validate Arcturus’ mRNA liver therapeutics platform and the introduction of LUNAR 2.0™ marks the beginning of a new and exciting chapter for Arcturus,” said Joseph Payne, President and CEO of Arcturus. “We are very pleased that ARCT-2601, utilizing LUNAR 2.0™, is positioned to be efficiently integrated into our ongoing OTC deficiency Phase 2 study later this year.”
ARCT-810 Shows Encouraging Phase 2 Activity
In the ongoing Phase 2 study, ARCT-810 demonstrated a generally safe and well-tolerated profile. The treatment reduced or maintained first morning fasting ammonia within the normal range This result also continued during periods of increased protein intake among participants. In addition, ARCT-810 reduced glutamine levels during treatment.
Participants receiving the 0.5 mg/kg dose achieved mean glutamine values within the normal range. However, weight gain occurred in all participants during the study. The findings support continued evaluation of ARCT-810 in OTC deficiency. They also provide clinical data for Arcturus’ broader mRNA liver therapeutics strategy.
“The greater than 30-fold improvement demonstrated by the next-generation platform is exciting and has the potential to translate into a more effective and more convenient therapy,” said Dr. Marshall Summar, former founding member and Executive Committee member of the NIH Urea Cycle Disorders Consortium, a recognized expert in rare diseases and OTC deficiency. “I believe ARCT-2601 has the potential to become a new standard of care for OTCD patients, particularly in severe onset, if approved, and I am excited to see the potential impact of Arcturus’ next-generation mRNA delivery platform across other programs.”
LUNAR 2.0™ Expands mRNA Delivery Potential
LUNAR 2.0™ represents Arcturus Therapeutics’ next-generation mRNA delivery platform. The platform uses improved lipids and an enhanced formulation process. Arcturus developed the formulation process specifically for these improved lipids. In non-human primate studies, LUNAR 2.0™ delivered significant improvements in protein expression. The platform achieved a 40-fold improvement over LUNAR 1.0. This result involved expression of human erythropoietin, also known as hEPO.
In another non-human primate study, LUNAR 2.0™ showed 38-fold greater potency than ATX-95. ATX-95 serves as the key lipid in ARCT-810. That study measured expression of human ornithine transcarbamylase, or hOTC. Therefore, the results strengthen the platform’s potential for liver-focused mRNA therapeutics. LUNAR 2.0™ could also support therapeutic areas that were previously inaccessible. These areas include Phenylketonuria, or PKU, and Gout.
“Validating clinical data, a compellingly differentiated LUNAR 2.0™ mRNA delivery platform, and a proprietary AI computational engine, support the continued advancement of Arcturus’ growing mRNA therapeutics pipeline, including ARCT-2601, and the initiation of new programs, such as Phenylketonuria (PKU) and Gout,” said Dr. Pad Chivukula, Chief Scientific Officer of Arcturus.
ARCT-2601 Moves Toward Phase 2 Integration
Arcturus is developing ARCT-2601 as a next-generation investigational mRNA therapeutic for OTCD. The candidate uses the LUNAR 2.0™ delivery platform. So far, ARCT-2601 has shown a safety profile similar to ARCT-810. These findings came from non-GMP preclinical studies. The U.S. Food and Drug Administration also provided favorable feedback on ARCT-2601. The feedback followed a Type C meeting held in June this year.
Furthermore, the FDA provided regulatory-path clarity for the program. Based on the available data, Arcturus plans to begin dosing near year-end. The planned study will include participants with OTCD who are 12 years and older. Arcturus intends to use an amended Phase 2 protocol. The amended protocol will integrate ARCT-2601 into the existing ARCT-810 Phase 2 study. Based on non-human primate data, ARCT-2601 could support lower or less frequent dosing.
Arcturus Therapeutics Expands AI Capabilities
Arcturus Therapeutics has also entered a definitive agreement to acquire myNEO. The transaction remains subject to customary closing conditions. The companies expect the acquisition to close in October. Arcturus has worked closely with myNEO since 2024. The acquisition will add a differentiated computational engine to Arcturus’ technology capabilities. The engine also includes proprietary intellectual property. As a result, Arcturus expects to strengthen its AI infrastructure. The company plans to improve mRNA design quality through the technology.
The acquisition will also enhance Arcturus’ target identification capabilities. These capabilities could support future mRNA drug development and pipeline expansion. Together, the clinical results, LUNAR 2.0™ platform, and AI capabilities support Arcturus’ ongoing development strategy. The company continues to advance mRNA therapeutics across liver and respiratory applications. Arcturus also plans to explore new opportunities in rare diseases and other therapeutic areas. The latest developments mark another step in the company’s mRNA medicine strategy.
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News Source: Businesswire.com
