Arcturus Therapeutics (NASDAQ:ARCT) highlighted its mRNA technology platform, rare disease pipeline and vaccine programs during a presentation at the LD Micro conference in Los Angeles, emphasizing delivery and manufacturing capabilities as key points of differentiation in the mRNA field.
The company said it has an approved COVID-19 vaccine, KOSTAIVE, in 32 countries and has a relationship with BARDA for pandemic flu. Arcturus described itself as a San Diego-based company with about 100 employees and roughly 12 years of operating history.
Arcturus Emphasizes Delivery and Manufacturing Capabilities
Arcturus said its core differentiation is in delivery technology, including injectable, intravenous and inhaled mRNA. The presenter said inhaled mRNA has been a particularly difficult delivery challenge and that the company has invested “hundreds of millions of dollars” over the past decade in the area.
The company also pointed to mRNA manufacturing as a key capability, saying few companies can manufacture mRNA lipid nanoparticles at scale. The presenter said pandemic-era funding helped Arcturus expand its platform and manufacturing capabilities.
Cystic Fibrosis Program in Phase 2
Arcturus provided an update on its inhaled mRNA therapeutic for cystic fibrosis, which is designed to express a new CFTR protein in the lungs. The company is focusing initially on Class I cystic fibrosis patients, who the presenter said are missing the CFTR protein entirely and are not eligible for CFTR modulator drugs.
The presenter said the company has established safety and tolerability at 5 mg, 10 mg and 15 mg doses in prior studies and is now using a 10 mg daily dose in an ongoing Phase 2 open-label trial. The study is expected to enroll up to 20 patients and extend treatment to 12 weeks, compared with 28 days in earlier dosing.
Arcturus said it has been able to administer inhaled mRNA daily without steroid treatment before, during or after dosing. The presenter said earlier efforts by competitors historically required steroids to manage inflammatory responses.
The company showed high-resolution CT scan data that it said indicated reductions in mucus plugs after 28 days of treatment. However, the presenter noted that imaging alone is not sufficient for approval and said the current study is evaluating lung function measures, including FEV and lung clearance index, or LCI.
Arcturus said the Cystic Fibrosis Foundation is supporting the program and that the therapy has Rare Pediatric Disease and Orphan Drug designations in the U.S. and Europe. The presenter estimated there are about 10,000 Class I cystic fibrosis patients globally.
OTC Deficiency Program Advances Toward FDA Meeting
The company also discussed its ornithine transcarbamylase, or OTC, deficiency program, an mRNA therapeutic targeting a rare liver disease that affects the urea cycle and can lead to elevated ammonia levels.
Arcturus said the current standard of care involves ammonia scavengers, which the presenter described as burdensome and difficult for managing ammonia spikes. The company’s approach is intended to replace missing or dysfunctional OTC protein and restore urea cycle activity.
The presenter said the company completed a Phase 1 study in healthy subjects and continued dose escalation in patients in Phase 1b. Arcturus has also conducted Phase 2 studies in Europe and the U.S. and is completing an ongoing open-label Phase 2 study.
According to the presentation, the current U.S. Phase 2 study is evaluating 0.3 mg/kg and 0.5 mg/kg dosing through five biweekly intravenous infusions. Arcturus is tracking biomarkers including ammonia and glutamine. The presenter said interim data showed glutamine levels falling into the normal range during treatment and rising again after treatment discontinuation.
Arcturus said it recently discussed data with the FDA in a Type C meeting and received direction on how to advance the program, including in pediatric patients. The company expects an end-of-Phase 2 meeting in the second half of 2026. The program has Orphan Drug designation in the U.S. and Europe.
Vaccine Platform and Pandemic Flu
Arcturus also reviewed its vaccine platform, saying KOSTAIVE was developed using self-amplifying mRNA and advanced through approvals in Japan, Europe and the United Kingdom. The presenter said the company’s platform has shown stronger and longer-lasting immune responses than conventional mRNA in multiple Phase 3 studies, including data published in journals such as The Lancet.
The company also highlighted LUNAR-H5N1, its bird flu vaccine program, and said the U.S. government is funding pandemic flu vaccine efforts. The presenter said Arcturus believes it has a strong vaccine candidate for pandemic flu and a strong government relationship, while also expressing hope that pandemic flu does not become a public health issue.
In response to an audience question about Phase 3 studies, the presenter said rare disease programs with safety, tolerability and efficacy data in Phase 2 typically proceed to discussions with regulators at an end-of-Phase 2 meeting to determine Phase 3 requirements. The presenter added that Arcturus would still need to conduct Phase 3 trials.
About Arcturus Therapeutics (NASDAQ:ARCT)
Arcturus Therapeutics Holdings Inc is a clinical-stage biotechnology company dedicated to developing messenger RNA (mRNA) medicines that address a range of diseases. The company leverages its proprietary STARR® mRNA platform to enable precise control over mRNA expression, supported by its lipid nanoparticle delivery technology, LUNAR®. Arcturus’s approach is designed to address both therapeutic and prophylactic applications, with an emphasis on vaccines and treatments for rare genetic and infectious diseases.
The company’s pipeline includes ARCT-810, an mRNA therapeutic candidate for phenylketonuria (PKU), and ARCT-021 (also known as LUNAR-COV19), a COVID-19 vaccine candidate developed in collaboration with Duke-NUS Medical School in Singapore.
