U.S. FDA Grants Fast Track to Arcturus' mRNA Vaccine ARCT-2304 for H5N1 Flu

Arcturus Therapeutics Holdings Inc., a company specializing in messenger RNA medicines, recently announced that the U.S. Food and Drug Administration (FDA) has awarded Fast Track Designation to its innovative vaccine candidate, ARCT-2304. This self-amplifying mRNA (sa-mRNA) vaccine is aimed at active immunization against the influenza A virus H5N1 subtype, included in the vaccine formulation. This designation is a recognition of ARCT-2304's potential in addressing the unmet medical needs in preventing diseases caused by the pandemic influenza A virus H5N1, which poses a significant risk to global health. The clinical Phase 1 trials for this vaccine began in November 2024.

The Fast Track Designation by the FDA is reserved for vaccines targeting serious conditions caused by infectious diseases. This designation facilitates an accelerated development and review process, offering additional benefits like improved communication with the FDA, eligibility for priority review, and the possibility of a rolling review.

Joseph Payne, the President and CEO of Arcturus Therapeutics, expressed satisfaction with this development. He emphasized the company's dedication to collaborating with the U.S. government to create safe and effective next-generation mRNA vaccines to safeguard U.S. citizens from potential future pandemic threats. The FDA's designation is a crucial milestone in Arcturus' mission to develop solutions for global health challenges.

The ARCT-2304 vaccine, also known as LUNAR-H5N1, is a product of Arcturus' proprietary LUNAR® sa-mRNA technology. This vaccine candidate is formulated to produce numerous mRNA copies within the host cell following intramuscular injection. This method enhances the expression of key antigens such as haemagglutinin (HA) and neuraminidase (NA), allowing for lower dosages compared to traditional mRNA vaccines. The sa-mRNA platform provides additional options for meeting domestic vaccine production surge capacity goals, potentially making vaccines available more quickly than those developed using egg- and cell-based technologies. The vaccine's lyophilized formulation remains stable in standard refrigeration, simplifying cold storage and reducing distribution risks.

Established in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. is a commercial company focused on mRNA medicines and vaccines, utilizing its proprietary technologies. These include LUNAR® lipid-mediated delivery, STARR® mRNA technology (sa-mRNA), and expertise in both the production of mRNA drug substances and drug products. Arcturus achieved a significant milestone with the development of KOSTAIVE®, the world's first self-amplifying mRNA COVID vaccine to receive approval.

Arcturus is engaged in a global collaboration with CSL Seqirus, aimed at developing innovative mRNA vaccines, and has a joint venture in Japan named ARCALIS, focusing on the production of mRNA vaccines and therapeutics. The company's pipeline includes RNA therapeutic candidates for treating conditions such as ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF), in addition to its partnered mRNA vaccine programs targeting SARS-CoV-2 (COVID-19) and influenza. Arcturus' versatile RNA therapeutics platforms can be applied to various nucleic acid medicines, including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus' technological innovations are protected by a robust patent portfolio, comprising over 500 patents and applications worldwide.