Arcturus Therapeutics Receives Clearance from FDA to Begin H5N1 Pandemic Flu Vaccine Clinical Trial

Rhea-AI Summary

Arcturus Therapeutics (Nasdaq: ARCT) has received FDA clearance to proceed with a clinical trial for ARCT-2304, a self-amplifying mRNA vaccine candidate targeting H5N1 pandemic influenza. The Phase 1 clinical trial, funded by BARDA, will evaluate safety, reactogenicity, and immunogenicity in approximately 200 healthy adults in the United States. The vaccine utilizes Arcturus' STARR® self-amplifying mRNA technology and is part of the company's preparation efforts for potential future pandemics.

Positive

• Received FDA clearance to proceed with Phase 1 clinical trial
• Secured BARDA funding for the clinical study
• Trial will enroll approximately 200 participants

Negative

• None.

Insights

Medical Research Analyst

The FDA clearance for ARCT-2304's clinical trial marks a significant milestone in pandemic preparedness. This self-amplifying mRNA vaccine candidate represents cutting-edge technology, potentially offering advantages over traditional flu vaccines. The BARDA funding support adds credibility and financial backing to the program.

The planned Phase 1 trial with 200 participants will provide important early safety and immunogenicity data. H5N1 (bird flu) poses a serious pandemic threat with mortality rates up to 60% in human cases, making vaccine development strategically important. The STARR® technology's self-amplifying nature could potentially enable lower doses and better immune responses compared to conventional mRNA platforms.

SAN DIEGO--(BUSINESS WIRE)-- Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a commercial messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has issued a “Study Can Proceed” notification for the Company’s Investigational New Drug (IND) application, ARCT-2304, a self-amplifying mRNA (sa-mRNA) vaccine candidate for active immunization to prevent pandemic influenza disease caused by H5N1 virus. The clinical study is funded by Biomedical Advanced Research and Development Authority (BARDA) and designed to enroll approximately 200 healthy adults in the United States.

“Arcturus is actively engaged with the U.S. government to prepare for the next pandemic, and clearance to proceed into the clinic with our STARR® self-amplifying mRNA technology is a key step in this important process,” said Joseph Payne, President & CEO of Arcturus Therapeutics. “The Phase 1 clinical trial is designed to evaluate the safety, reactogenicity, and immunogenicity of ARCT-2304 as a potential vaccine to protect against the highly pathogenic H5N1 avian influenza.”

About ARCT-2304

ARCT-2304 is a sa-mRNA vaccine candidate formulated within a lipid nanoparticle (LNP). The sa-mRNA vaccine candidate is designed to make many copies of mRNA within the host cell after intramuscular injection to achieve enhanced expression of haemagglutinin (HA) and neuraminidase (NA) antigens, thereby enabling lower doses than conventional mRNA vaccines. Utilizing a mRNA-based platform for pandemic influenza vaccine development offers further options for meeting domestic vaccine manufacturing surge capacity goals. The technology may make vaccines available much sooner than egg- and cell-based technologies. The lyophilized vaccine formulation is stable in refrigerators, thereby simplifying cold-chain storage and reducing distribution risks.

About Arcturus

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a commercial mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR® mRNA Technology (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus developed KOSTAIVE®, the first self-amplifying messenger RNA (sa-mRNA) COVID vaccine in the world to be approved. Arcturus has an ongoing global collaboration for innovative mRNA vaccines with CSL Seqirus, and a joint venture in Japan, ARCALIS, focused on the manufacture of mRNA vaccines and therapeutics. Arcturus' pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF), along with its partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and influenza. Arcturus' versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus' technologies are covered by its extensive patent portfolio (over 400 patents and patent applications in the U.S., Europe, Japan, China, and other countries). For more information, visit www.ArcturusRx.com. In addition, please connect with us on Twitter and LinkedIn.

Forward Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact included in this press release, are forward-looking statements, including those regarding the initiation and enrollment of a Phase 1 clinical trial of ARCT-2304, the likelihood of success of the Company’s development and related efforts for an influenza vaccine candidate, the future activities under and fulfillment of the Company’s contract with BARDA, the ability of the Company’s influenza vaccine technologies to support U.S. government pandemic preparedness goals, and the impact of general business and economic conditions. Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements such as the foregoing and you should not place undue reliance on such forward-looking statements. These statements are only current predictions or expectations, and are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels of activity, performance or achievements to be materially different from those anticipated by the forward-looking statements, including those discussed under the heading "Risk Factors" in Arcturus’ most recent Annual Report on Form 10-K, and in subsequent filings with, or submissions to, the SEC, which are available on the SEC’s website at www.sec.gov. Except as otherwise required by law, Arcturus disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.

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IR and Media Contacts

Arcturus Therapeutics

Neda Safarzadeh

VP, Head of IR/PR/Marketing

(858) 900-2682

IR@ArcturusRx.com

Source: Arcturus Therapeutics Holdings Inc.

FAQ

What is the purpose of Arcturus Therapeutics' ARCT-2304 vaccine candidate?

ARCT-2304 is designed as a self-amplifying mRNA vaccine candidate for active immunization to prevent pandemic influenza disease caused by the H5N1 virus.

How many participants will be enrolled in Arcturus' H5N1 vaccine Phase 1 trial?

The clinical study is designed to enroll approximately 200 healthy adults in the United States.

What will Arcturus' Phase 1 trial for ARCT-2304 evaluate?

The Phase 1 clinical trial will evaluate the safety, reactogenicity, and immunogenicity of ARCT-2304 as a potential vaccine against H5N1 avian influenza.

Who is funding Arcturus Therapeutics' H5N1 vaccine clinical trial?

The clinical study is funded by the Biomedical Advanced Research and Development Authority (BARDA).