Arcturus Therapeutics Highlights mRNA Pipeline, CF Data and Vaccine Expansion at Jefferies

Key Points

• Arcturus Therapeutics is expanding beyond vaccines and is now emphasizing therapeutic mRNA programs, especially its differentiated LUNAR delivery platform and scalable manufacturing capabilities.
• The company said its lead cystic fibrosis candidate, ARCT-032, has shown safety/tolerability across early studies and encouraging mucus-plug reductions, with a Phase 2 study underway and more data expected later this year.
• Arcturus also highlighted progress in OTC deficiency and vaccines: ARCT-810 is heading toward an FDA end-of-Phase 2 meeting, while its COVID vaccine Kostaive is approved in 32 countries and the firm continues work on an H5N1 bird flu program with BARDA.

Arcturus Therapeutics NASDAQ: ARCT President and CEO Joseph Payne outlined the company’s messenger RNA programs, vaccine approvals and upcoming clinical milestones during a presentation at the Jefferies Global Healthcare Conference in New York.

Payne described Arcturus as a “next generation messenger RNA medicines company” based in San Diego with about 100 employees. He said the company used its technology during the pandemic to support vaccination efforts in Singapore, Vietnam and Japan, and that its COVID-19 vaccine, Kostaive, is now approved in 32 countries, including Japan, the United Kingdom and the European Union.

Payne said Arcturus also has an ongoing relationship with the U.S. Biomedical Advanced Research and Development Authority, or BARDA, for an H5N1 bird flu vaccine program. He also noted that Meiji supports distribution in Japan.

Company Emphasizes Differentiated mRNA Delivery

Payne said Arcturus’ primary focus going forward is on therapeutic applications of mRNA, including a liver disease program and a lung disease program. He highlighted the company’s LUNAR delivery platform, a lipid nanoparticle technology that he said is chemically distinct from older lipid systems.

According to Payne, Arcturus uses thiocarbamate cores in its lipid nanoparticles, which he said allow the body to break the lipids down. He said the company has shown in multiple human trials that its lipids biodegrade and do not accumulate, which he called important for therapeutic applications that require regular dosing and larger administered volumes than vaccines.

Payne also cited manufacturing know-how as a differentiating factor, saying the company is among a small number of firms capable of making mRNA at scale. He said this capability is particularly important for therapeutic programs, which require more material than vaccine applications.

Cystic Fibrosis Program Advances in Phase 2

Payne said Arcturus’ lead respiratory program is ARCT-032, an inhaled mRNA therapeutic for cystic fibrosis that is partnered with the Cystic Fibrosis Foundation. He said the foundation has committed $25 million to the program and understands the cystic fibrosis patient community, including Class I cystic fibrosis patients in the U.S. and Europe.

The program is initially focused on Class I cystic fibrosis patients, who Payne said are ineligible for CFTR modulators because they do not have CFTR protein in their lungs to modulate. He said Arcturus’ approach differs from existing cystic fibrosis therapies because it aims to build a new CFTR protein in the lung through inhaled mRNA delivered to bronchial epithelial cells.

Payne said ARCT-032 completed a Phase 1 study in 32 subjects and a Phase 1b study in adults with cystic fibrosis, including Class I patients. He said the therapy was found to be safe and tolerable in those studies. He added that a Phase 2 dose-response study tested daily dosing over 28 days at 5 mg, 10 mg and 15 mg, with all three cohorts considered successful on safety and tolerability.

The company is currently enrolling up to 20 participants in a 12-week open-label Phase 2 study in the U.S. and abroad using a 10 mg dose. Payne said the study is evaluating safety, tolerability and efficacy, including lung function measures such as FEV and lung clearance index, or LCI. He described both as potential approvable endpoints.

Payne also discussed high-resolution CT scan data from a previous Phase 2 cohort, saying Arcturus observed reductions in mucus plugs after 28 days of treatment. He said four of six participants in the second cohort showed a noticeable reduction, including an approximately 28% reduction in mucus plugs and a 33% reduction in mucus volume among responders. Payne said this imaging measure is not an approvable endpoint but was encouraging to the company and clinical investigators.

Payne said the company has “a high level of confidence” that it will have data later this year sufficient to make decisions about the next steps for the cystic fibrosis program.

OTC Deficiency Program Heads Toward FDA Meeting

Payne also highlighted ARCT-810, the company’s program for ornithine transcarbamylase deficiency, or OTC deficiency, a rare liver disease and the most common urea cycle disorder. He said patients with OTC deficiency lack or have insufficient ornithine transcarbamylase enzyme, leading to elevated ammonia levels that can cause serious neurological damage.

Payne said the current standard of care includes ammonia scavengers, but he noted that these therapies do not address glutamine, which can cross the blood-brain barrier and convert to ammonia. Arcturus’ mRNA approach is intended to produce a new ornithine transcarbamylase enzyme in the liver.

According to Payne, ARCT-810 completed a Phase 1 study in healthy volunteers and a Phase 1b study up to 0.5 mg/kg with no severe adverse events. He said the company has also established safety, tolerability and biomarker correction and normalization in multiple Phase 2 studies in stable adults in the United Kingdom, European Union and United States.

Payne said Arcturus is preparing for an end-of-Phase 2 meeting with the U.S. Food and Drug Administration in the second half of the year. He said the company is also holding a Type C meeting this month, after which it expects to provide more specific timing for the end-of-Phase 2 meeting.

Vaccine Programs Include COVID and Bird Flu

Payne said Kostaive uses self-amplifying mRNA technology, which he said allows for a substantially lower dose than conventional mRNA vaccines. He said the vaccine is administered at five micrograms and that multiple Phase 3 studies comparing the technology to conventional mRNA have been published, including in The Lancet.

Payne said the company has also generated data with BARDA in bird flu and believes its self-amplifying mRNA technology could be relevant if H5N1 were to become airborne. He emphasized that he hopes such a scenario does not occur.

Payne concluded by noting recent additions to the company’s board, including Chairman Moncef Slaoui, who led Operation Warp Speed and previously held a senior role at GSK, and John Markels, former president of Merck Vaccines.

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.

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