European Commission Grants Orphan Medicinal Product Designation for Navenibart, Astria Therapeutics’ Investigational Therapy for the Treatment of Hereditary Angioedema

Rhea-AI Summary

Astria Therapeutics (Nasdaq:ATXS) announced that the European Commission has granted Orphan Medicinal Product Designation (OMPD) to navenibart for the treatment of hereditary angioedema (HAE). Navenibart is a monoclonal antibody inhibitor of plasma kallikrein designed for long-acting HAE attack prevention. Initial results from the Phase 1b/2 ALPHA-STAR clinical trial showed a favorable safety profile and 90-96% reduction in monthly attack rates over six months.

The OMPD status, along with the FDA's orphan drug designation in the US, highlights the global unmet need for HAE treatment and navenibart's potential impact. Astria Therapeutics plans to progress navenibart into a Phase 3 trial in Q1 2025. OMPD in the EU provides incentives for drug development, including regulatory and financial benefits, for rare conditions affecting no more than five in 10,000 persons in the EU.

Positive

• European Commission granted Orphan Medicinal Product Designation (OMPD) to navenibart for HAE treatment
• Initial clinical trial results showed 90-96% reduction in monthly HAE attack rates
• Favorable safety and tolerability profile demonstrated in Phase 1b/2 ALPHA-STAR trial
• Planned progression to Phase 3 trial in Q1 2025
• OMPD status provides regulatory and financial incentives for drug development in the EU

Negative

• None.

Insights

Medical Research Analyst

The European Commission's grant of Orphan Medicinal Product Designation (OMPD) for navenibart is a significant milestone for Astria Therapeutics. This designation acknowledges the potential of navenibart to address a critical unmet need in treating hereditary angioedema (HAE), a rare genetic disorder.

The Phase 1b/2 ALPHA-STAR trial results are particularly promising, showing a 90-96% reduction in monthly attack rates over six months. This level of efficacy, combined with a favorable safety profile, positions navenibart as a potentially game-changing therapy for HAE patients.

The OMPD status in Europe, coupled with the FDA's orphan drug designation in the U.S., provides Astria with significant regulatory and financial incentives. These include potential market exclusivity, reduced fees and development assistance, which could accelerate navenibart's path to market and enhance its commercial viability.

Investors should note the planned progression to Phase 3 trials in Q1 2025, which, if successful, could lead to a lucrative product launch in a market with high unmet needs. However, it's important to remember that drug development carries inherent risks and success in early-stage trials doesn't guarantee final approval or commercial success.

Financial Analyst

This regulatory milestone potentially enhances Astria Therapeutics' market position and financial outlook. The OMPD status in Europe, complementing the U.S. orphan drug designation, could significantly reduce development costs and time-to-market for navenibart.

Key financial implications include:

• Potential for 10 years of market exclusivity in the EU upon approval, protecting future revenue streams
• Eligibility for fee reductions and scientific advice from the EMA, potentially lowering R&D expenses
• Enhanced attractiveness to investors and potential partners, possibly improving access to capital

With a market cap of $648,326,490, Astria's valuation could see upward pressure as navenibart progresses. However, investors should consider that commercialization is still years away and the company will likely require additional funding to reach market launch.

The planned Phase 3 trial in Q1 2025 represents a critical value inflection point. Positive results could significantly boost the company's valuation, while negative outcomes could have a substantial adverse impact on the stock price.

BOSTON--(BUSINESS WIRE)-- Astria Therapeutics, Inc. (Nasdaq:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunologic diseases, today announced that the European Commission (EC) has granted Orphan Medicinal Product Designation (OMPD) to navenibart for the treatment of hereditary angioedema (HAE). Navenibart is a monoclonal antibody inhibitor of plasma kallikrein designed to provide long-acting attack prevention for HAE. Initial results from the Phase 1b/2 ALPHA-STAR clinical trial of navenibart have demonstrated a favorable safety and tolerability profile and a reduction of monthly attack rates by 90-96% when dosed once or twice over six months.

“We are very pleased to receive Orphan Medicinal Product Designation for navenibart in Europe,” said Jill C. Milne, Ph.D., Chief Executive Officer. “We believe that OMPD status, in combination with the FDA’s designation of navenibart as an orphan drug in the United States, reinforces both the global unmet need for people with HAE and the potential for navenibart to change the way that people live with HAE. We look forward to the expected progression of navenibart into a Phase 3 trial in the first quarter of 2025 and continuing our efforts to bring a new treatment option to people living with HAE around the world.”

In the European Union (EU), OMPD is granted by the EC following a recommendation from the European Medicines Agency (EMA) via its Committee for Orphan Medicinal Products (COMP). OMPD status provides incentives for drug development sponsors to develop products for diagnosing, preventing or treating life-threatening or very serious conditions that are rare and affect not more than five in 10,000 persons in the EU. Sponsors may receive regulatory and financial incentives as well as eligibility for EMA assistance in optimizing the candidate’s clinical development.

About Navenibart (STAR-0215):

Navenibart is a monoclonal antibody inhibitor of plasma kallikrein in development for the treatment of HAE. Our goal with navenibart is to provide rapid and sustained HAE attack prevention with a validated mechanism and trusted modality administered every 3 and 6 months. The U.S. Food and Drug Administration (FDA) has granted navenibart Orphan Drug and Fast Track Designations, and the EC has granted navenibart OMPD. For more information on navenibart, please visit www.astriatx.com.

About Astria Therapeutics:

Astria Therapeutics is a biopharmaceutical company, and our mission is to bring life-changing therapies to patients and families affected by allergic and immunologic diseases. Our lead program, navenibart (STAR-0215), is a monoclonal antibody inhibitor of plasma kallikrein in clinical development for the treatment of hereditary angioedema. Our second program, STAR-0310, is a monoclonal antibody OX40 antagonist in preclinical development for the treatment of atopic dermatitis. Learn more about our company on our website, www.astriatx.com, or follow us on X and Instagram @AstriaTx and on Facebook and LinkedIn.

Forward-Looking Statements:

This press release contains forward-looking statements within the meaning of applicable securities laws and regulations including, but not limited to, statements regarding: the expected timing of initiation of the planned navenibart Phase 3 trial; the expectation that navenibart will become a treatment option for HAE patients; the potential for navenibart in the HAE market and our overall vision and goals for the navenibart program; the potential unmet medical need for HAE; and the goal of bringing life changing therapies to patients and families affected by allergic and immunologic diseases and to become a leading allergy and immunology company. The use of words such as, but not limited to, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “goals,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” or "vision," and similar words expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Astria’s current beliefs, expectations and assumptions regarding the future of its business, future plans and strategies, future financial performance, results of pre-clinical and clinical results of the Astria’s product candidates and other future conditions. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the following risks and uncertainties: changes in applicable laws or regulations; the possibility that we may be adversely affected by other economic, business, and/or competitive factors; risks inherent in pharmaceutical research and development, such as: adverse results in our drug discovery, preclinical and clinical development activities, the risk that the results of preclinical studies, including of navenibart and STAR-0310, may not be replicated in clinical trials, that the preliminary or interim results from clinical trials may not be indicative of the final results, that the results of early stage clinical trials, such as the results from the navenibart Phase 1a clinical trial and the initial results from the ALPHA-STAR trial, may not be replicated in later stage clinical trials, including additional and final results from the ALPHA-STAR trial or the planned navenibart Phase 3 development program, the risk that we may not be able to enroll sufficient patients in our clinical trials on a timely basis, and the risk that any of our clinical trials may not commence, continue or be completed on time, or at all; decisions made by, and feedback received from, the U.S. Food and Drug Administration and other regulatory authorities on our regulatory and clinical trial submissions and other feedback from potential clinical trial sites, including investigational review boards at such sites, and other review bodies with respect to navenibart, STAR-0310, and any other future development candidates, and devices for such product candidates; our ability to manufacture sufficient quantities of drug substance and drug product for navenibart, STAR-0310, and any other future product candidates, and devices for such product candidates, on a cost-effective and timely basis, and to develop dosages and formulation for navenibart, STAR-0310, and any other future product candidates that are patient-friendly and competitive; our ability to develop biomarker and other assays, along with the testing protocols therefore; our ability to obtain, maintain and enforce intellectual property rights for navenibart, STAR-0310, and any other future product candidates; our potential dependence on collaboration partners; competition with respect to navenibart, STAR-0310, or any of our other future product candidates; the risk that survey results and market research may not be accurate predictors of the commercial landscape for HAE, the ability of navenibart to compete in HAE and the anticipated position and attributes of navenibart in HAE based on clinical data to date, its preclinical profile, pharmacokinetic modeling, market research and other data; risks with respect to the ability of STAR-0310 to compete in AD and the anticipated position and attributes of STAR-0310 in AD based on its preclinical profile; our ability to manage our cash usage and the possibility of unexpected cash expenditures; our ability to obtain necessary financing to conduct our planned activities and to manage unplanned cash requirements; the risks and uncertainties related to our ability to recognize the benefits of any additional acquisitions, licenses or similar transactions; and general economic and market conditions; as well as the risks and uncertainties discussed in the “Risk Factors” section of our Annual Report on Form 10-K for the period ended December 31, 2023 and in other filings that we may make with the Securities and Exchange Commission.

New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Astria may not actually achieve the forecasts or expectations disclosed in our forward-looking statements, and investors and potential investors should not place undue reliance on Astria’s forward-looking statements. Neither Astria, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing Astria’s views as of any date subsequent to the date hereof.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241016913098/en/

Astria Contact:

Investor Relations and Media:

Elizabeth Higgins

investors@astriatx.com

Source: Astria Therapeutics, Inc.

FAQ

What is the Orphan Medicinal Product Designation granted to Astria Therapeutics (ATXS) for navenibart?

The European Commission granted Orphan Medicinal Product Designation (OMPD) to Astria Therapeutics' navenibart for the treatment of hereditary angioedema (HAE).

What were the results of the Phase 1b/2 ALPHA-STAR clinical trial for navenibart (ATXS)?

The trial demonstrated a favorable safety and tolerability profile for navenibart, with a 90-96% reduction in monthly HAE attack rates when dosed once or twice over six months.

When is Astria Therapeutics (ATXS) planning to start the Phase 3 trial for navenibart?

Astria Therapeutics expects to progress navenibart into a Phase 3 trial in the first quarter of 2025.

What benefits does the OMPD status provide for Astria Therapeutics (ATXS) in the European Union?

OMPD status provides regulatory and financial incentives for drug development, as well as eligibility for EMA assistance in optimizing the clinical development of navenibart for HAE treatment.