Arcturus Therapeutics Receives Orphan Medicinal Product Designation from the European Commission (EC), for ARCT-032, for the Treatment of Cystic Fibrosis

Rhea-AI Summary

Arcturus Therapeutics Holdings Inc. receives orphan medicinal product designation from the European Commission and FDA for ARCT-032 to treat Cystic Fibrosis. The designation provides various incentives, including market exclusivity and fee reductions.

Insights

Pharmaceutical Industry Analyst

The European Commission's granting of orphan medicinal product designation to ARCT-032 for treating Cystic Fibrosis (CF) is a significant development for Arcturus Therapeutics. This status is pivotal for biotech companies as it provides a range of incentives that can reduce the cost and risk associated with drug development. These incentives include protocol assistance, which can streamline the clinical trial process, fee reductions that lower the financial burden of regulatory submissions and 10 years of market exclusivity post-approval, which can protect the product from direct competition, thus potentially leading to increased revenue streams.

From an industry perspective, orphan drug status is often associated with a positive market reaction, particularly for smaller biotech firms, due to the potential for higher pricing power and reduced competition. For stakeholders, the long-term benefits may include a strengthened product pipeline and enhanced company valuation. However, the path to market for orphan drugs can still be fraught with clinical and regulatory challenges and the actual impact on the company's financials will depend on the successful progression of ARCT-032 through clinical trials and eventual market adoption.

Medical Research Analyst

ARCT-032's orphan drug designation by both the European Commission and the U.S. FDA underscores its potential as a novel therapeutic for Cystic Fibrosis, a disease characterized by the buildup of thick mucus that can lead to severe respiratory and digestive problems. The designation is particularly noteworthy given that CF affects a relatively small patient population and treatments that can work across different genetic mutations are highly sought after.

The successful completion of the single-ascending-dose study in healthy adults and the enrollment of CF patients in the Phase 1b study are crucial steps forward. The medical community will be closely watching the interim Phase 1b data expected in H1 2024, as it will provide insights into the drug's safety profile and efficacy. The ability of ARCT-032 to address the needs of CF patients regardless of genetic mutation could represent a significant advancement in the treatment landscape and could lead to improved patient outcomes.

Healthcare Economist

The economic implications of orphan drug designations are profound, particularly within the European Union where healthcare systems are often publicly funded. The incentives provided by the designation can encourage investment in treatments for rare diseases, which might otherwise be neglected due to the smaller market size. The 10 years of market exclusivity granted by the EC is a critical factor in the potential return on investment for Arcturus Therapeutics, as it allows the company to set prices that reflect the high costs of research and development without immediate generic competition.

However, it is important to consider the balance between incentivizing drug development and the resulting cost to healthcare systems. High prices of orphan drugs can strain healthcare budgets, necessitating negotiations between pharmaceutical companies and payers. The overall economic impact on the business will depend on the drug's pricing, reimbursement decisions and the prevalence of CF in the EU. These factors will ultimately influence the stock market performance of Arcturus Therapeutics, as investors assess the potential revenue against the costs of ongoing development.

Access to protocol assistance, centralized authorization process, fee reductions and 10 years of market exclusivity

SAN DIEGO--(BUSINESS WIRE)-- Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and medicines to treat unmet medical needs within liver and respiratory rare diseases, today announced that the European Commission (EC), based on a positive opinion issued by the European Medicines Agency (EMA), has granted orphan medicinal product designation for the Company’s product candidate ARCT-032 to treat Cystic Fibrosis (CF). In November 2023, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to ARCT-032 for CF.

Orphan medicinal product designation by the EC is available to novel therapeutics that prevent or treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the EU. The designation provides significant incentives to promote the development of the drug including protocol assistance, access to the centralized authorization procedure, fee reductions, and research grants, as well as 10 years of market exclusivity.

“We are pleased to receive orphan medicinal product designation from the EC for ARCT-032, as it represents a significant milestone for our CF program,” said Joseph Payne, President & CEO of Arcturus Therapeutics. “We will continue to advance ARCT-032 as a potential new treatment option for people with CF regardless of their genetic mutations.”

After successful completion of the single-ascending-dose portion of the study in healthy adults, the first participant with CF in the Phase 1b study part enrolled and completed two administrations of ARCT-032. Arcturus remains on track to share interim Phase 1b data in H1 2024.

About Cystic Fibrosis

Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein and/or function in the airways, causing insufficient chloride transport to maintain airway surface homeostasis. CF mucus is more difficult to clear, thus clogging the airways and leading to infection, inflammation, respiratory failure, or other life-threatening complications. Currently approved CFTR modulator therapies are designed to increase function of the CFTR channel to help reduce symptoms yet are ineffective in some people with CF because of their underlying mutations.

About ARCT-032

ARCT-032 utilizes Arcturus' LUNAR^® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. Expression of a functional copy of the CFTR mRNA in the lungs of people with CF has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease. The ARCT-032 program is supported by preclinical data in rodents, ferrets and primates, as well as demonstrating restoration of CFTR expression and function in human bronchial epithelial cells.

About Arcturus Therapeutics

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global late-stage clinical 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 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 deficiency and cystic fibrosis, 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 issued 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 continued clinical development of ARCT-032, the likelihood of sharing and timing for sharing interim Phase 1b data, likelihood of success (including safety and efficacy) of ARCT-032, the Company’s strategy and future operations, 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 orphan medicinal product designation granted to Arcturus Therapeutics for ARCT-032?

The European Commission and FDA granted orphan medicinal product designation to ARCT-032 to treat Cystic Fibrosis.

What are the benefits of orphan medicinal product designation?

The designation provides incentives such as protocol assistance, centralized authorization process, fee reductions, research grants, and 10 years of market exclusivity.

What is the significance of receiving orphan medicinal product designation for Arcturus Therapeutics?

It represents a significant milestone for Arcturus Therapeutics' CF program, advancing ARCT-032 as a potential new treatment option for people with CF.

What is the timeline for sharing interim Phase 1b data for ARCT-032?

Arcturus remains on track to share interim Phase 1b data in H1 2024 after successful completion of the single-ascending-dose portion of the study in healthy adults and enrolling the first participant with CF.