FDA Clears Arcturus Therapeutics' ARCT-032 Inhaled mRNA Drug for Cystic Fibrosis

SAN DIEGO, CA, USA I September 03, 2024 I Arcturus Therapeutics Holdings Inc. (Arcturus, Nasdaq: ARCT), a company specializing in messenger RNA (mRNA) medicines, has announced that the U.S. Food and Drug Administration (FDA) has given the green light for their Investigational New Drug (IND) application for ARCT-032, aimed at treating cystic fibrosis (CF). This approval allows the company to commence a Phase 2 study to examine the safety, tolerability, and effectiveness of ARCT-032 in CF patients.

Dr. Juergen Froehlich, Chief Medical Officer of Arcturus Therapeutics, emphasized the significance of the Phase 2 approval, stating that it enables the company to explore ARCT-032 as a potential CF treatment. The study will evaluate ARCT-032's safety and efficacy over multiple weeks and at various dose levels in CF patients who cannot benefit from or are not suitable for CFTR modulator therapy.

Cystic fibrosis is a serious condition affecting many people globally. It is caused by mutations in the CFTR gene, which lead to a lack or dysfunction of the CFTR protein in the airways. This disrupts chloride transport, resulting in thick, sticky mucus that clogs the airways and can cause severe complications like infections, inflammation, and respiratory failure. Standard treatments often include CFTR modulators, but about 15% of CF patients do not benefit from these due to either dysfunctional or absent CFTR protein.

ARCT-032 is an investigational mRNA therapeutic designed to deliver functional CFTR protein directly to the lungs via inhalation. The treatment has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA. The drug utilizes Arcturus’ LUNAR® lipid-mediated platform to transport CFTR mRNA to the lungs. Lung disease is the primary cause of mortality among CF patients, and ARCT-032 aims to restore CFTR activity in the lungs, potentially mitigating the disease's progression. Preclinical studies have shown promising results in various animal models and human bronchial epithelial cells, demonstrating the restoration of CFTR expression and function.

Founded in 2013 and headquartered in San Diego, California, Arcturus Therapeutics Holdings Inc. is a global leader in mRNA medicines and vaccine development. The company boasts several proprietary technologies, including the LUNAR® lipid-mediated delivery system, STARR® mRNA Technology (sa-mRNA), and advanced mRNA manufacturing capabilities. Arcturus made history with KOSTAIVE®, the first self-amplifying mRNA COVID-19 vaccine to receive approval. The company is also involved in a global collaboration with CSL Seqirus for innovative mRNA vaccines and has a joint venture in Japan named ARCALIS focused on mRNA vaccine and therapeutic production.

Arcturus' pipeline features RNA therapeutic candidates targeting various diseases, including ornithine transcarbamylase (OTC) deficiency and cystic fibrosis, as well as partnered mRNA vaccine programs for COVID-19 and influenza. The company’s versatile RNA therapeutics platforms are adaptable to multiple types of nucleic acid medicines such as messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’ innovation is protected by an extensive patent portfolio, consisting of over 400 patents and applications worldwide.

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