Arcturus Therapeutics Reports Progress in Cystic Fibrosis Program

SAN DIEGO—Arcturus Therapeutics (Nasdaq: ARCT), a key player in the clinical development of mRNA-based medicines, has announced its upcoming presentation at the 47th European Cystic Fibrosis Conference on June 7, 2024. The company will showcase Phase 1 results from studies conducted on healthy volunteers and new interim data from Phase 1b trials involving cystic fibrosis (CF) patients treated with ARCT-032, an innovative inhaled mRNA therapeutic.

Promising Early Results

Joseph Payne, President & CEO of Arcturus Therapeutics, shared the initial findings, indicating that the first four CF patients who received ARCT-032 exhibited an average improvement of 4% in their Forced Expiratory Volume in 1 second (FEV1) after just two doses. Payne remarked, "The ARCT-032 treatments were generally safe and well tolerated, with no severe adverse events reported. We also noted a positive trend towards enhanced lung function."

Dr. Juergen Froehlich, Chief Medical Officer of Arcturus, elaborated on the quantitative improvements, highlighting the 4% mean increase in FEV1 observed in these early-stage participants by day eight. The forthcoming presentation at the European CF conference in Scotland will delve into these interim results, adding to the favorable outcomes seen in Phase 1 studies with healthy individuals.

Understanding Cystic Fibrosis

Cystic fibrosis is a genetic disorder that significantly shortens life expectancy. The condition arises from mutations in the CFTR gene, leading to deficient or dysfunctional CFTR protein in the airways. This defect hampers chloride transport, disrupting airway surface homeostasis and resulting in thick, sticky mucus that is difficult to expel. This compromised mucus clearance can lead to infections, inflammation, respiratory failure, and other severe complications. Current CFTR modulator therapies aim to enhance CFTR function but are ineffective for some patients due to underlying genetic diversity.

About ARCT-032

ARCT-032 has garnered Orphan Medicinal Product Designation from the European Medicines Agency and Orphan Drug Designation from the U.S. Food and Drug Administration for treating cystic fibrosis. This investigational therapy leverages Arcturus' LUNAR® lipid-mediated aerosolized delivery system to transport CFTR mRNA directly to the lungs. By expressing a functional CFTR mRNA, ARCT-032 aims to restore CFTR activity, potentially reversing the pathological effects that drive lung disease progression. Preclinical studies in various animal models, including rodents, ferrets, and primates, have shown promising results in restoring CFTR expression and function.

About Arcturus Therapeutics

Founded in 2013 and headquartered in San Diego, California, Arcturus Therapeutics Holdings Inc. is a global leader in mRNA medicines and vaccines. The company's technologies include LUNAR® lipid-mediated delivery, STARR® mRNA Technology, and expertise in mRNA drug substance and product manufacturing. Arcturus developed the world's first self-amplifying mRNA COVID vaccine, Kostaive®, which received regulatory approval. The company also collaborates globally with CSL Seqirus on innovative mRNA vaccines and operates a joint venture in Japan, ARCALIS, focusing on mRNA vaccine production. Arcturus' pipeline features RNA therapeutic candidates for conditions like ornithine transcarbamylase deficiency and cystic fibrosis, along with partnered mRNA vaccine programs for COVID-19 and influenza. The company's versatile RNA platforms accommodate various nucleic acid medicines, including mRNA, siRNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics.

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