Astria Therapeutics receives FDA orphan drug designation for Navenibart (STAR-0215) for the treatment of hereditary angioedema

BOSTON–(BUSINESS WIRE)–Astria Therapeutics, Inc. (Nasdaq:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunological diseases, today announced that Navenibart (STAR-0215) has received orphan drug designation for the treatment from the U.S. Food and Drug Administration of hereditary angioedema (HAE) and Drug Administration (FDA). Navenibart is a monoclonal antibody inhibitor of plasma kallikrein intended to provide long-acting prevention of HAE attacks. Initial results from the Phase 1b/2 ALPHA-STAR clinical trial with Navenibart have shown a favorable safety and tolerability profile and a 90-96% reduction in monthly seizure rates when administered once or twice over six months.

“Receiving orphan drug designation for Navenibart is an important validation of our belief that there is a significant unmet need for people with HAE,” said Jill C. Milne, Ph.D., Chief Executive Officer. “We believe that Navenibart has the potential to become the market-leading HAE treatment due to its proven mechanism and modality, the efficacy observed to date, and the low treatment burden at infrequent dosing and believe that Navenibart will change the way people living with your HAE could change.” . We expect to report additional results from the ALPHA-STAR trial in the fourth quarter and advance Navenibart to a Phase 3 trial beginning in the first quarter of 2025.”

The FDA's Orphan Drug Designation program grants orphan designation to drugs intended for the safe and effective prevention, diagnosis, or treatment of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan status provides sponsors with development and commercial incentives for certain compounds and drugs.

About Navenibart (STAR-0215):

Navenibart is a monoclonal antibody inhibitor of plasma kallikrein that is being developed for the treatment of HAE. Our goal with Navenibart is to provide rapid and sustained prevention of HAE attacks with a validated mechanism and a trusted modality administered every 3 and 6 months. The FDA has granted Orphan Drug and Fast Track designations to Navenibart. For more information about Navenibart, see www.astriatx.com.

About Astria Therapeutics:

Astria Therapeutics is a biopharmaceutical company and our mission is to provide life-changing therapies to patients and families affected by allergic and immunological 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. Find out more about our company on our website, www.astriatx.comor follow us on X and Instagram @AstriaTx as well as 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: our expectations regarding the potential significance of the initial results of the ALPHA-STAR Phase 1b/2 clinical trial of Navenibart ; the expected timing of publication of additional data from the ALPHA-STAR study; the expected timing of initiation of the proposed Navenibart Phase 3 trial and receipt of topline results from that trial, subject to feedback from regulatory authorities; the potential of Navenibart in the HAE market, including the potential to be the market-leading treatment, the potential therapeutic benefits of Navenibart for the treatment of HAE, the timing of administration of Navenibart and our overall vision and objectives for the Navenibart program; the potential unmet medical need for HAE; and the goal of providing life-changing therapies to patients and families affected by allergic and immunological diseases and becoming a leader in allergy and immunology. The use of words such as, but not limited to, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “goals,” “intend,” “could,” “could.” . “Plan,” “potential,” “predict,” “project,” “should,” “goal,” “will,” “would,” or “vision” and similar 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, preclinical and clinical results of its product candidates Astria and other future conditions. Actual results may differ materially from those reflected in such forward-looking statements due to 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 associated with 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 Navenibart and STAR-0310, will be affected in clinical trials Studies may not be reproduced The preliminary or preliminary results of clinical trials may not be indicative of the final results, and the results of early clinical trials, such as the results of the Navenibart Phase 1a clinical trial and the initial results of the ALPHA-STAR study, may not be indicative of the final results being replicated in subsequent 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 enroll enough patients in a timely manner our ability to commence clinical trials and the risk that any of our clinical trials will not commence, continue or be completed on a timely basis or at all; Decisions and feedback from the U.S. Food and Drug Administration and other regulatory authorities regarding our regulatory and clinical trial submissions and other feedback from potential clinical trial sites, including review boards at such sites, and other review bodies regarding Navenibart, STAR-0310 and any other future development candidates and devices for such product candidates; our ability to produce sufficient quantities of drug substances and drug products for Navenibart, STAR-0310 and any other future product candidates, as well as devices for such product candidates, in a cost-effective and timely manner, and to develop dosages and formulations for Navenibart, STAR-0310 and any other future product candidates that are patient-friendly and are competitive; our ability to develop biomarker and other tests and related testing protocols; our ability to obtain, maintain and enforce intellectual property rights for Navenibart, STAR-0310 and any other future product candidates; our potential dependence on cooperation 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 accurately predict the commercial landscape for HAE, Navenibart's ability to compete in HAE, and Navenibart's expected position and characteristics in HAE based on clinical data to date, its preclinical profile and its Pharmacokinetics are modeling, market research and other data; risks relating to STAR-0310's ability to compete in AD and the expected position and properties 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 financing necessary to carry out our planned activities and meet unplanned cash needs; the risks and uncertainties associated with our ability to realize the benefits of further acquisitions, licenses or similar transactions; and general economic and market conditions; and 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 we may make with the Securities and Exchange Commission.

New risks and uncertainties may arise from time to time and it is not possible to predict all risks and uncertainties. Astria may not actually achieve the projections or expectations set forth 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, consultants or agents undertake any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as reflecting the views of Astria as of any date subsequent to this date.

Contacts

Astria:
Investor relations and media:
Elizabeth Higgins
[email protected]