Japan’s Ministry of Health, Labor and Welfare Approves CSL and Arcturus Therapeutics’ Updated Self-amplifying mRNA COVID-19 Vaccine for Protection against JN.1 Strain, to be Distributed in Japan by Meiji Seika Pharma

Rhea-AI Summary

CSL and Arcturus Therapeutics announced that Japan's Ministry of Health, Labor and Welfare has approved their updated self-amplifying mRNA (sa-mRNA) COVID-19 vaccine, KOSTAIVE®, for adults 18 and older. The vaccine is tailored to protect against the JN.1 lineage of Omicron subvariants. Meiji Seika Pharma, CSL's exclusive partner in Japan, will distribute the vaccine for the October 2024 vaccination campaign.

KOSTAIVE® is the world's first commercially available sa-mRNA COVID-19 vaccine for adults. Clinical data showed superior immunogenicity to Omicron BA 4/5 compared to conventional mRNA vaccines and immunity lasting up to one year. This approval aligns with recent World Health Organization recommendations and demonstrates CSL and Arcturus' commitment to innovative vaccine technology for viral respiratory diseases.

Positive

• Approval of KOSTAIVE® for adults 18+ in Japan, targeting JN.1 Omicron subvariants
• Distribution planned for October 2024 vaccination campaign in Japan
• Clinical data shows superior immunogenicity compared to conventional mRNA vaccines
• Immunity lasting up to one year demonstrated in follow-up data
• World's first commercially available sa-mRNA COVID-19 vaccine for adults

Negative

• None.

Medical Research Analyst

The approval of KOSTAIVE®, the world's first self-amplifying mRNA (sa-mRNA) COVID-19 vaccine, for the JN.1 strain in Japan is a significant milestone in vaccine technology. This innovative approach offers potential advantages over conventional mRNA vaccines:

• Enhanced durability: Data suggests immunity lasting up to one year, potentially reducing the need for frequent boosters.
• Superior immunogenicity: Demonstrated better immune response against Omicron variants compared to conventional mRNA vaccines.
• Targeted protection: Specifically designed for the JN.1 lineage, aligning with current WHO recommendations.

The sa-mRNA technology's ability to produce more antigen with a lower dose could lead to improved efficacy and potentially reduced side effects. This approval could pave the way for sa-mRNA platforms in developing vaccines for other respiratory diseases, representing a paradigm shift in vaccine development.

Financial Analyst

This approval marks a significant commercial opportunity for CSL and Arcturus Therapeutics in the lucrative Japanese vaccine market. Key financial implications include:

• First-mover advantage: Being the first sa-mRNA COVID-19 vaccine positions them ahead of competitors in this technology space.
• Revenue potential: Distribution for the October vaccination campaign could lead to substantial near-term revenue.
• Market expansion: Success in Japan could open doors for approvals and sales in other countries, expanding the global market reach.
• Long-term growth: The sa-mRNA platform's versatility could lead to development of vaccines for other diseases, diversifying the product pipeline.

Investors should monitor vaccination uptake rates and any potential manufacturing or distribution challenges. The durability advantage could impact future sales patterns compared to traditional vaccines requiring more frequent administration.

Public Health Expert

The approval of KOSTAIVE® in Japan represents a significant advancement in COVID-19 vaccination strategies. Key public health implications include:

• Targeted protection: Specifically designed for the JN.1 lineage, addressing current variant concerns.
• Potential for improved compliance: Longer-lasting immunity could increase public willingness to vaccinate.
• Reduced logistical burden: Less frequent vaccination needs could ease pressure on healthcare systems.
• Adaptability: The sa-mRNA platform allows for rapid updates to address new variants.

However, public health officials should closely monitor real-world effectiveness and any rare adverse events. Clear communication about the new technology will be important for public acceptance. The success of this vaccine could influence global vaccination strategies and potentially accelerate the end of the pandemic phase of COVID-19.

Approval underscores CSL and Arcturus Therapeutics’ commitment to delivering disruptive technologies to combat COVID-19 and other respiratory viral diseases to protect public health

• KOSTAIVE^®, the world’s first self-amplifying mRNA (sa-mRNA) COVID-19 vaccine, was approved for adults 18 years of age and older in November 2023
• The updated sa-mRNA COVID-19 vaccine is tailored to protect against the JN.1 lineage of Omicron subvariants
• Meiji Seika Pharma, CSL’s exclusive partner in Japan, will begin vaccine distribution in time for the October COVID-19 vaccination campaign
• CSL and Arcturus Therapeutics’ partnership delivers on the promise to advance innovative vaccine technology for viral respiratory diseases

KING OF PRUSSIA, PA & SAN DIEGO--(BUSINESS WIRE)--

Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) and sa-mRNA pioneer Arcturus Therapeutics today announced that Japan's Ministry of Health, Labor and Welfare (MHLWs) granted approval and authorization for their updated self-amplifying mRNA (sa-mRNA) COVID-19 vaccine, KOSTAIVE^®. The updated vaccine is targeted to protect against the JN.1 lineage of Omicron subvariants for adults 18 years of age and older.

CSL's exclusive partner in Japan, Meiji Seika Pharma, will begin distributing the updated vaccine in time for the October COVID-19 vaccination campaign, marking the world's first commercially available sa-mRNA COVID-19 vaccine for adults 18 and older.

In May 2024, a Japanese health ministry panel recommended that COVID-19 vaccines be updated to target the JN.1 lineage of Omicron subvariants for the 2024/2025 national immunization program. This aligns with recent recommendations from the World Health Organization.

“We are pleased to receive the updated approval from Japan’s Ministry of Health, Labor and Welfare, and remain on track to distribute KOSTAIVE^® in time for the October COVID-19 vaccination campaign,” said Emmanuelle Lecomte-Brisset, Senior Vice President and Head of Global Regulatory Affairs, CSL. “We look forward to introducing KOSTAIVE^® in Japan as a safe, tolerable and more durable option in protecting against COVID-19.”

“We believe KOSTAIVE^® has the potential to change the paradigm for COVID-19 vaccines in Japan,” said Jonathan Edelman, M.D., Senior Vice President, Vaccines Innovation Unit, CSL. “Today’s approval further demonstrates CSL's promise to pursue, develop and deliver new innovative treatment options to protect public health.”

The approval is based on clinical evidence supporting the safety and effectiveness of CSL and Arcturus Therapeutics’ sa-mRNA COVID-19 vaccine, including published data demonstrating superior immunogenicity to Omicron BA 4/5 compared to a conventional mRNA COVID-19 vaccine booster and follow-up data demonstrating duration of immunity lasting up to one year.

“We are pleased that our sa-mRNA technology will be available to vaccinate people in Japan against COVID-19, which remains an ongoing risk to public health around the world,” said Joseph Payne, Chief Executive Officer of Arcturus Therapeutics. “We look forward to continuing to work with CSL and regulators to explore the development of KOSTAIVE^® in additional countries.”

About sa-mRNA

mRNA vaccines help protect against infectious diseases by providing a blueprint for cells in the body to make a protein to help our immune systems recognize and fight the disease. Unlike conventional mRNA vaccines, self-amplifying mRNA vaccines instruct the body to make more mRNA and protein to boost the immune response.

About CSL

CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSL. For more information about CSL, visit www.CSL.com.

About Arcturus

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global 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.

About Meiji Seika Pharma Co., Ltd.

Meiji Seika Pharma, since it launched penicillin in 1946, has been providing efficacious and high-quality pharmaceutical products such as therapeutics and vaccines for infectious diseases, therapeutics for central nervous system diseases as well as generic drugs in response to various medical needs. As a leading company in the field of infectious diseases, we are strengthening our platform for infection control and prevention with vaccines and antimicrobial agents.

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 strategy, future operations, the planned launch of KOSTAIVE^® in Japan, the distribution of the updated vaccine by Meiji Seika Pharma and timing thereof, the timing and likelihood of availability of doses of the updated vaccine in Japan, the continued demonstration of KOSTAIVE^® as a safe, tolerable and more durable option against COVID-19, that preclinical or clinical data will be predictive of future clinical results, 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240913667150/en/

CSL Media Contacts

Sue Thorn

Mobile: 617 799 3151

Email: sue.thorn@cslbehring.com

Australia:

Jimmy Baker

Jimmy.Baker@csl.com.au

+61 450 909 211

Asia Pacific:

Hamish Walsh

+61 422 424 338

hamish.walsh@seqirus.com

Arcturus Media Contact:

Neda Safarzadeh

VP, Head of IR/PR/Marketing

IR@arcturusrx.com

Source: Arcturus Therapeutics Holdings Inc.

FAQ

What is KOSTAIVE® and when was it approved in Japan?

KOSTAIVE® is a self-amplifying mRNA (sa-mRNA) COVID-19 vaccine developed by CSL and Arcturus Therapeutics. It was initially approved in Japan for adults 18 and older in November 2023, with an updated version targeting the JN.1 Omicron subvariants approved in 2024 for the October vaccination campaign.

How does KOSTAIVE® (ARCT) compare to conventional mRNA COVID-19 vaccines?

Clinical data shows that KOSTAIVE® demonstrates superior immunogenicity to Omicron BA 4/5 compared to conventional mRNA COVID-19 vaccine boosters. Additionally, follow-up data indicates that KOSTAIVE® provides immunity lasting up to one year.

When will KOSTAIVE® (ARCT) be available in Japan?

Meiji Seika Pharma, CSL's exclusive partner in Japan, will begin distributing KOSTAIVE® in time for the October 2024 COVID-19 vaccination campaign in Japan.

What age group is KOSTAIVE® (ARCT) approved for in Japan?

KOSTAIVE® is approved in Japan for adults 18 years of age and older.