Latest news with #HAE
Business Wire
23-05-2025
- Business
- Business Wire
Astria Therapeutics to Present at Upcoming C1 Inhibitor Deficiency and Angioedema Workshop
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 it will present at the 14 th C1 Inhibitor Deficiency and Angioedema Workshop in Budapest, Hungary on May 31, 2025. Dr. Marc A. Riedl, Professor of Medicine and Clinical Director of the U.S. HAEA Angioedema Center at the University of California, San Diego, will present safety and efficacy data from the Phase 1a and Phase 1b/2 trials of navenibart in a presentation titled, 'Navenibart for Hereditary Angioedema (HAE): Analysis of Safety, Pharmacokinetic, and Pharmacodynamic Data From Phase 1a and Phase 1b/2 ALPHA-STAR Trial.' The presentation will take place at 2:45pm CET on Saturday, May 31 at the Ensana Thermal Hotel Margitsziget in Budapest. 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 an investigational monoclonal antibody OX40 antagonist in clinical development for the treatment of atopic dermatitis. Learn more about our company on our website, or follow us on Instagram @AstriaTx and on Facebook and LinkedIn.
Yahoo
16-05-2025
- Health
- Yahoo
BioCryst Presents New Real-world Evidence Showing Significant and Sustained Reductions in HAE Attack Rates in Adolescents and People with Severe HAE Following Initiation of ORLADEYO® (berotralstat)
RESEARCH TRIANGLE PARK, N.C., May 16, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new real-world evidence on the use of oral, once-daily ORLADEYO® (berotralstat) in adolescents and people with severe HAE showing significant and sustained reductions in HAE attack rates through 18 months of follow-up after beginning treatment with ORLADEYO in both patient populations. The real-world evidence was presented in two posters at the 2025 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR), which is being held in Montreal from May 13-16, 2025. 'The outcomes detailed in these posters show how ORLADEYO is making a difference for people living with HAE, in particular those with very severe disease and those who are adolescents. These two groups experienced far fewer attacks per month compared to baseline after starting ORLADEYO. These kinds of real-world results should give physicians as well as their HAE patients the additional confidence to improve control of their attacks,' said Dr. Raffi Tachdjian, associate clinical professor of medicine & pediatrics, division of allergy & clinical immunology, David Geffen School of Medicine, University of California Los Angeles. Significant and sustained reductions in attack rates after ORLADEYO initiation The results presented in two posters at ISPOR 2025 were from a retrospective pre-post study using outcomes collected from BioCryst's sole-source pharmacy from December 15, 2020, to January 8, 2024. The poster 'Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Patients with C1 Inhibitor Deficiency (Type I/II) and ≥8 Attacks/month' (#PCR182) detailed findings from 56 U.S. patients with HAE with C1-inhibitor deficiency (HAE-C1-INH) who received ORLADEYO. Patients experienced significantly lower HAE attack rates while on ORLADEYO in each 90-day follow-up period (1.24-1.90 attacks/month) compared to baseline (7.78-8.23 attacks/month). Patients experienced significantly fewer HAE attacks per month following ORLADEYO initiation during every 90-day follow-up period relative to baseline, including: 6.25 fewer attacks/month (95 percent confidence period (CI): [5.63, 6.87]; p<0.001) at 12 months (days 271-360) 6.43 fewer attacks/month (95 percent CI: [5.78, 7.09]; p<0.001) at 18 months (days 451-540) The poster 'Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Adolescents' (#PCR132) highlighted outcomes reported from 99 U.S. patients with HAE aged 12-17 years who received ORLADEYO. Patients had significantly lower HAE attack rates while on ORLADEYO during each 90-day follow-up period (0.36-0.76 attacks/month) compared to the mean baseline rate (2.07-2.30 attacks/month). Compared to baseline, adolescents experienced statistically significant and sustained reductions in HAE attack rates after ORLADEYO initiation during each 90-day follow-up period, including: 1.56 fewer attacks/month (95 percent CI: [0.89, 2.23]; p<0.001) at 12 months (days 271-360) 1.85 fewer attacks/month (95 percent CI: [1.12, 2.58]; p<0.001) at 18 months (days 451-540) 'We continue to generate evidence from real-world use of our oral, once-daily prophylactic therapy for HAE that supports its effectiveness in a wide range of people with HAE. Here, we show that ORLADEYO is having a positive impact on attack reduction for younger people and those with severe disease. These additional findings further underscore that ORLADEYO works well for many patients with HAE, regardless of their attack severity, age or other aspects,' said Dr. Donald S. Fong, chief medical officer of BioCryst. About ORLADEYO® (berotralstat)ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein. U.S. Indication and Important Safety Information INDICATIONORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. Limitations of useThe safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation. IMPORTANT SAFETY INFORMATIONAn increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent. The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C). Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John's wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO. ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO. The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established. There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production. To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or Please see full Prescribing Information. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance or achievements and statements relating to ORLADEYO performance and effectiveness. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst's ability to successfully implement or maintain its commercialization plans for ORLADEYO; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; and BioCryst's ability to successfully manage its growth and compete effectively. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst's forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
15-05-2025
- Health
- Yahoo
FDA awards BioCryst's Orladeyo NDA for paediatric HAE
The US Food and Drug Administration (FDA) has accepted BioCryst Pharmaceuticals' new drug application (NDA) for Orladeyo (berotralstat), granting it priority review for use in children aged two to 11 with hereditary angioedema (HAE). A Prescription Drug User Fee Act target action date was set by the agency for 12 September 2025. The application was supported by the APeX-P trial's positive interim data. This trial assessed a prophylactic HAE therapy in these subjects. The results indicated that the therapy was well tolerated and maintained a consistent safety profile in this young age group. It also led to sustained and early minimisations of monthly attack rates. If approved, this plasma kallikrein inhibitor will be the inaugural oral targeted prophylactic therapy for HAE patients under 12. Beyond the US, the company has also submitted a line extension application to the European Medicines Agency for the use of the therapy oral granules in this patient population. The company plans additional regulatory filings in other worldwide territories, including Canada and Japan. BioCryst Pharmaceuticals chief research and development officer Dr Helen Thackray stated: 'As detailed in the results from APeX-P, we observed that participants experienced serious HAE attacks at a very early age, with a median age of symptom onset of two years, which suggests there is a larger burden of disease at an earlier age than has been appreciated thus far. 'If approved, we believe this oral granule formulation of Orladeyo could help children with HAE and their families better manage their condition and avoid the traumatic experience of acute attacks with emergency care or hospital stays.' In December 2020, the therapy was approved by the US regulator initially for HAE attack prevention in the adult population and in children aged 12 and above. It is currently available commercially in 30 countries. in 2024, the company secured a contract worth up to $69m from the US Department of Health and Human Services for the procurement of Rapivab to treat influenza. "FDA awards BioCryst's Orladeyo NDA for paediatric HAE" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
13-05-2025
- Business
- Yahoo
Astria Therapeutics Reports First Quarter 2025 Financial Results and Provides a Corporate Update
-- ALPHA-ORBIT Pivotal Phase 3 Trial Evaluating Every 3- and Every 6-Month Administration of Navenibart (STAR-0215) in Hereditary Angioedema is Enrolling Patients -- -- Upcoming Navenibart and STAR-0310 Data: ALPHA-SOLAR Long-Term Extension Trial Results Expected Mid-2025 and Phase 1a STAR-0310 Healthy Subject Results Expected in Q3 2025 -- BOSTON, May 13, 2025--(BUSINESS WIRE)--Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunologic diseases, today reported financial results for the first quarter ended March 31, 2025, and provided a corporate update. "As a late-stage clinical company, we are focused on delivering on navenibart, a therapy with a trusted mechanism and modality, best-in-class profile, and the potential to change the way that people live with HAE," said Jill C. Milne, Ph.D., Chief Executive Officer at Astria Therapeutics. "Our goal is to ensure that navenibart reaches its full potential as the market-leading HAE therapy. We are thrilled with patient and physician enthusiasm for the program as we enroll patients in our trial. With STAR-0310, we anticipate early proof-of-concept results from the Phase 1a trial in Q3, which we expect to be informative on differentiation regarding efficacy, safety, and low treatment burden and next steps." Navenibart (STAR-0215) The ALPHA-ORBIT pivotal Phase 3 trial of navenibart in people with hereditary angioedema (HAE) is enrolling patients, with top-line results expected in early 2027. The ALPHA-ORBIT trial is a global, randomized, double-blind, placebo-controlled Phase 3 clinical trial to evaluate the efficacy and safety of navenibart over 6 months and includes both every 3-month (Q3M) and every 6-month (Q6M) treatment arms. The primary endpoint is time-normalized monthly HAE attacks at 6 months, and a key secondary endpoint includes the proportion of participants who are attack-free at 6 months. After 6 months, patients may be eligible to enter a long-term trial called ORBIT-EXPANSE, in which all patients will be treated with navenibart and which will include a patient-centered flexible dosing period and assess the long-term safety and tolerability as well as efficacy of navenibart. The navenibart Phase 3 program consists of the ALPHA-ORBIT Phase 3 trial and the ORBIT-EXPANSE long-term trial, which are designed to support registration globally. Positive final top-line results from target enrollment in the Phase 1b/2 ALPHA-STAR trial of navenibart, announced in December 2024, showed rapid onset of robust and durable efficacy, favorable safety and tolerability, and pharmacokinetics and pharmacodynamics consistent with sustained plasma kallikrein inhibition for both Q3M and Q6M administration. Final results included reduction in mean monthly attack rate of 90-95% and up to a 67% attack-free rate over 6 months. All of the 16 target enrollment patients from ALPHA-STAR have entered the ALPHA-SOLAR long-term open-label trial. Initial safety and efficacy data from ALPHA-SOLAR, with long-term Q3M and Q6M administration, are expected mid-2025. Physician market research (n=50) reinforced the potential for navenibart to be the market-leading therapy for HAE. Physicians anticipated that offering both Q3M and Q6M options would gain 53% of their patient share for those initiating preventative therapy for the first time based on the existing treatment landscape, and 46% for those switching from currently available injectable and oral therapies. STAR-0310 Astria is developing STAR-0310, an investigational high-potency and long-acting monoclonal antibody OX40 antagonist that incorporates YTE technology, for the treatment of atopic dermatitis (AD) and potentially other indications. STAR-0310 was intentionally designed to capitalize on the learnings of OX40 receptor and OX40 ligand programs with the goal of having the best overall OX40 therapy. The Company initiated a Phase 1a clinical trial of STAR-0310 in healthy subjects in January 2025. The Phase 1a trial is intended to assess the safety, tolerability, pharmacokinetics, and immunogenicity of STAR-0310 in healthy adult participants, with early proof-of-concept (POC) results expected in the third quarter of 2025. Preclinical data supporting the differentiated profile of STAR-0310 was presented at AAAAI in March 2025. STAR-0310 demonstrated an extended half-life in cynomolgus monkeys and STAR-0310 preserved T-cells. STAR-0310 significantly reduced antibody-dependent cellular cytotoxicity (ADCC) on activated T-cells and regulatory T-cells when compared to other anti-OX40 monoclonal antibodies. Reduction in ADCC activity has the potential to support a more favorable safety profile and wider therapeutic window for STAR-0310, which the Company believes could drive greater efficacy. Corporate Updates Astria recently published its Corporate Responsibility Report. To learn more about Astria's Corporate Responsibility initiatives, please view the full report at this link: The Company will be attending the following upcoming investor conferences in June: Jefferies Global Healthcare Conference: June 3-5, 2025, New York, New York Oppenheimer Innovators in I&I Summit: June 25, 2025, New York, New York First Quarter 2025 Financial Results Cash Position: As of March 31, 2025, Astria had cash, cash equivalents and short-term investments of $295.1 million, compared to $328.1 million as of December 31, 2024. The Company expects that its cash, cash equivalents and short-term investments as of March 31, 2025 will be sufficient to fund its operations into mid-2027, including (i) for navenibart, support for all program activities through completion of our ALPHA-ORBIT Phase 3 trial, including activities related to the planned ORBIT-EXPANSE long-term trial and Phase 3 development and testing of drug device combinations for potential dosing of navenibart, and (ii) for STAR-0310, the completion of the ongoing Phase 1a clinical trial of healthy subjects. Net cash used in operating activities for the three months ended March 31, 2025 was $34.0 million, compared to $19.1 million for the three months ended March 31, 2024. R&D Expenses: Research and development expenses were $27.8 million for the three months ended March 31, 2025, compared to $15.7 million for the three months ended March 31, 2024. The increase in research and development expenses was attributed to an increase in external expenses related to the ALPHA-ORBIT and STAR-0310 Phase 1a clinical trials during the three months ended March 31, 2025. G&A Expenses: General and administrative expenses were $9.2 million for the three months ended March 31, 2025, compared to $8.4 million for the three months ended March 31, 2024. The increase in general and administrative expenses was attributable to stock-based compensation and company growth to support the advancement of our programs. Operating Loss: Loss from operations was $37.0 million for the three months ended March 31, 2025, compared to $24.2 million for the three months ended March 31, 2024. Net Loss: Net loss was $33.7 million for the three months ended March 31, 2025, compared to a net loss of $19.9 million for the three months ended March 31, 2024. Net Loss Per Share Basic and Diluted: Net loss per share basic and diluted was $0.58 for the three months ended March 31, 2025, compared to a net loss basic and diluted of $0.38 per share for the three months ended March 31, 2024. 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 an investigational monoclonal antibody inhibitor of plasma kallikrein in clinical development for the treatment of hereditary angioedema. Our second program, STAR-0310, is an investigational monoclonal antibody OX40 antagonist in clinical development for the treatment of atopic dermatitis. Learn more about our company on our website, or follow us on 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 receipt of topline results from the navenibart ALPHA-ORBIT Phase 3 trial; the goals and objectives of the ALPHA-ORBIT Phase 3 trial and the ORBIT-EXPANSE long-term trial, including that they are designed to support registration of Q3M and Q6M navenibart administration; the expected timing of release of initial safety and efficacy data from the ALPHA-SOLAR trial; our goal of developing two dosing options for navenibart; the potential for navenibart in the HAE market, including the potential to be the market leading treatment in HAE, the potential therapeutic and other benefits of navenibart as a treatment for HAE, and our vision and goals for the program; the potential therapeutic benefits and potential attributes of STAR-0310 as a treatment for AD; expectations regarding the nature and timing of receipt of early proof-of-concept results from such trial, including our expectation that such results will inform on STAR-0310's differentiated profile; the potential to develop STAR-0310 in additional indications; our goals and vision for STAR-0310; anticipated cash runway; and the goal of bringing life changing therapies to patients and families affected by allergic and immunological 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 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 FDA 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, 2024 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. Astria Therapeutics, Inc. Consolidated Statements of Operations (In thousands, except share and per share data) (Unaudited) Three Months Ended March 31, 2025 2024 Operating expenses: Research and development $ 27,786 $ 15,726 General and administrative 9,209 8,424 Total operating expenses 36,995 24,150 Loss from operations (36,995 ) (24,150 ) Other income (expense): Interest and investment income 3,346 4,241 Other expense, net (60 ) (19 ) Total other income, net 3,286 4,222 Net loss (33,709 ) (19,928 ) Net loss per share attributable to common shareholders - basic and diluted $ (0.58 ) $ (0.38 ) Weighted-average common shares outstanding used in net loss per share - basic and diluted 58,005,312 52,294,765 Astria Therapeutics, Inc. Selected Consolidated Balance Sheets Data (In thousands) (Unaudited) March 31, December 31, 2025 2024 Assets Cash and cash equivalents $ 54,385 $ 59,820 Short-term investments 240,679 268,312 Right-of-use asset 4,833 5,114 Other current and long-term assets 10,490 9,117 Total assets 310,387 342,363 Liabilities and stockholders' equity Current portion of operating lease liabilities 1,392 1,384 Long term portion of operating lease liabilities 3,671 3,969 Other current and long-term liabilities 16,034 17,747 Total liabilities 21,097 23,100 Total stockholders' equity $ 289,290 $ 319,263 Astria Therapeutics, Inc. Selected Consolidated Statements of Cash Flows Data (In thousands) (Unaudited) Three Months Ended March 31, 2025 2024 Net cash used in operating activities $ (34,018 ) $ (19,094 ) Net cash provided by (used in) investing activities 28,583 (126,231 ) Net cash provided by financing activities - 141,807 Net decrease in cash, cash equivalents and restricted cash $ (5,435 ) $ (3,518 ) View source version on Contacts Astria Contact: Investor Relations and Media: Elizabeth Higginsinvestors@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
13-05-2025
- Business
- Business Wire
Astria Therapeutics Reports First Quarter 2025 Financial Results and Provides a Corporate Update
BOSTON--(BUSINESS WIRE)--Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunologic diseases, today reported financial results for the first quarter ended March 31, 2025, and provided a corporate update. 'As a late-stage clinical company, we are focused on delivering on navenibart, a therapy with a trusted mechanism and modality, best-in-class profile, and the potential to change the way that people live with HAE,' said Jill C. Milne, Ph.D., Chief Executive Officer at Astria Therapeutics. 'Our goal is to ensure that navenibart reaches its full potential as the market-leading HAE therapy. We are thrilled with patient and physician enthusiasm for the program as we enroll patients in our trial. With STAR-0310, we anticipate early proof-of-concept results from the Phase 1a trial in Q3, which we expect to be informative on differentiation regarding efficacy, safety, and low treatment burden and next steps.' Navenibart (STAR-0215) The ALPHA-ORBIT pivotal Phase 3 trial of navenibart in people with hereditary angioedema (HAE) is enrolling patients, with top-line results expected in early 2027. The ALPHA-ORBIT trial is a global, randomized, double-blind, placebo-controlled Phase 3 clinical trial to evaluate the efficacy and safety of navenibart over 6 months and includes both every 3-month (Q3M) and every 6-month (Q6M) treatment arms. The primary endpoint is time-normalized monthly HAE attacks at 6 months, and a key secondary endpoint includes the proportion of participants who are attack-free at 6 months. After 6 months, patients may be eligible to enter a long-term trial called ORBIT-EXPANSE, in which all patients will be treated with navenibart and which will include a patient-centered flexible dosing period and assess the long-term safety and tolerability as well as efficacy of navenibart. The navenibart Phase 3 program consists of the ALPHA-ORBIT Phase 3 trial and the ORBIT-EXPANSE long-term trial, which are designed to support registration globally. Positive final top-line results from target enrollment in the Phase 1b/2 ALPHA-STAR trial of navenibart, announced in December 2024, showed rapid onset of robust and durable efficacy, favorable safety and tolerability, and pharmacokinetics and pharmacodynamics consistent with sustained plasma kallikrein inhibition for both Q3M and Q6M administration. Final results included reduction in mean monthly attack rate of 90-95% and up to a 67% attack-free rate over 6 months. All of the 16 target enrollment patients from ALPHA-STAR have entered the ALPHA-SOLAR long-term open-label trial. Initial safety and efficacy data from ALPHA-SOLAR, with long-term Q3M and Q6M administration, are expected mid-2025. Physician market research (n=50) reinforced the potential for navenibart to be the market-leading therapy for HAE. Physicians anticipated that offering both Q3M and Q6M options would gain 53% of their patient share for those initiating preventative therapy for the first time based on the existing treatment landscape, and 46% for those switching from currently available injectable and oral therapies. STAR-0310 Astria is developing STAR-0310, an investigational high-potency and long-acting monoclonal antibody OX40 antagonist that incorporates YTE technology, for the treatment of atopic dermatitis (AD) and potentially other indications. STAR-0310 was intentionally designed to capitalize on the learnings of OX40 receptor and OX40 ligand programs with the goal of having the best overall OX40 therapy. The Company initiated a Phase 1a clinical trial of STAR-0310 in healthy subjects in January 2025. The Phase 1a trial is intended to assess the safety, tolerability, pharmacokinetics, and immunogenicity of STAR-0310 in healthy adult participants, with early proof-of-concept (POC) results expected in the third quarter of 2025. Preclinical data supporting the differentiated profile of STAR-0310 was presented at AAAAI in March 2025. STAR-0310 demonstrated an extended half-life in cynomolgus monkeys and STAR-0310 preserved T-cells. STAR-0310 significantly reduced antibody-dependent cellular cytotoxicity (ADCC) on activated T-cells and regulatory T-cells when compared to other anti-OX40 monoclonal antibodies. Reduction in ADCC activity has the potential to support a more favorable safety profile and wider therapeutic window for STAR-0310, which the Company believes could drive greater efficacy. Corporate Updates Astria recently published its Corporate Responsibility Report. To learn more about Astria's Corporate Responsibility initiatives, please view the full report at this link: The Company will be attending the following upcoming investor conferences in June: Jefferies Global Healthcare Conference: June 3-5, 2025, New York, New York Oppenheimer Innovators in I&I Summit: June 25, 2025, New York, New York First Quarter 2025 Financial Results Cash Position: As of March 31, 2025, Astria had cash, cash equivalents and short-term investments of $295.1 million, compared to $328.1 million as of December 31, 2024. The Company expects that its cash, cash equivalents and short-term investments as of March 31, 2025 will be sufficient to fund its operations into mid-2027, including (i) for navenibart, support for all program activities through completion of our ALPHA-ORBIT Phase 3 trial, including activities related to the planned ORBIT-EXPANSE long-term trial and Phase 3 development and testing of drug device combinations for potential dosing of navenibart, and (ii) for STAR-0310, the completion of the ongoing Phase 1a clinical trial of healthy subjects. Net cash used in operating activities for the three months ended March 31, 2025 was $34.0 million, compared to $19.1 million for the three months ended March 31, 2024. R&D Expenses: Research and development expenses were $27.8 million for the three months ended March 31, 2025, compared to $15.7 million for the three months ended March 31, 2024. The increase in research and development expenses was attributed to an increase in external expenses related to the ALPHA-ORBIT and STAR-0310 Phase 1a clinical trials during the three months ended March 31, 2025. G&A Expenses: General and administrative expenses were $9.2 million for the three months ended March 31, 2025, compared to $8.4 million for the three months ended March 31, 2024. The increase in general and administrative expenses was attributable to stock-based compensation and company growth to support the advancement of our programs. Operating Loss: Loss from operations was $37.0 million for the three months ended March 31, 2025, compared to $24.2 million for the three months ended March 31, 2024. Net Loss: Net loss was $33.7 million for the three months ended March 31, 2025, compared to a net loss of $19.9 million for the three months ended March 31, 2024. Net Loss Per Share Basic and Diluted: Net loss per share basic and diluted was $0.58 for the three months ended March 31, 2025, compared to a net loss basic and diluted of $0.38 per share for the three months ended March 31, 2024. 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 an investigational monoclonal antibody inhibitor of plasma kallikrein in clinical development for the treatment of hereditary angioedema. Our second program, STAR-0310, is an investigational monoclonal antibody OX40 antagonist in clinical development for the treatment of atopic dermatitis. Learn more about our company on our website, or follow us on 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 receipt of topline results from the navenibart ALPHA-ORBIT Phase 3 trial; the goals and objectives of the ALPHA-ORBIT Phase 3 trial and the ORBIT-EXPANSE long-term trial, including that they are designed to support registration of Q3M and Q6M navenibart administration; the expected timing of release of initial safety and efficacy data from the ALPHA-SOLAR trial; our goal of developing two dosing options for navenibart; the potential for navenibart in the HAE market, including the potential to be the market leading treatment in HAE, the potential therapeutic and other benefits of navenibart as a treatment for HAE, and our vision and goals for the program; the potential therapeutic benefits and potential attributes of STAR-0310 as a treatment for AD; expectations regarding the nature and timing of receipt of early proof-of-concept results from such trial, including our expectation that such results will inform on STAR-0310's differentiated profile; the potential to develop STAR-0310 in additional indications; our goals and vision for STAR-0310; anticipated cash runway; and the goal of bringing life changing therapies to patients and families affected by allergic and immunological 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 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 FDA 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, 2024 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. Astria Therapeutics, Inc. Consolidated Statements of Operations (In thousands, except share and per share data) (Unaudited) Three Months Ended March 31, 2025 2024 Operating expenses: Research and development $ 27,786 $ 15,726 General and administrative 9,209 8,424 Total operating expenses 36,995 24,150 Loss from operations (36,995 ) (24,150 ) Other income (expense): Interest and investment income 3,346 4,241 Other expense, net (60 ) (19 ) Total other income, net 3,286 4,222 Net loss (33,709 ) (19,928 ) Net loss per share attributable to common shareholders - basic and diluted $ (0.58 ) $ (0.38 ) Weighted-average common shares outstanding used in net loss per share - basic and diluted 58,005,312 52,294,765 Expand Astria Therapeutics, Inc. Selected Consolidated Balance Sheets Data (In thousands) (Unaudited) March 31, December 31, 2025 2024 Assets Cash and cash equivalents $ 54,385 $ 59,820 Short-term investments 240,679 268,312 Right-of-use asset 4,833 5,114 Other current and long-term assets 10,490 9,117 Total assets 310,387 342,363 Liabilities and stockholders' equity Current portion of operating lease liabilities 1,392 1,384 Long term portion of operating lease liabilities 3,671 3,969 Other current and long-term liabilities 16,034 17,747 Total liabilities 21,097 23,100 Total stockholders' equity $ 289,290 $ 319,263 Expand Astria Therapeutics, Inc. Selected Consolidated Statements of Cash Flows Data (In thousands) (Unaudited) Three Months Ended March 31, 2025 2024 Net cash used in operating activities $ (34,018 ) $ (19,094 ) Net cash provided by (used in) investing activities 28,583 (126,231 ) Net cash provided by financing activities - 141,807 Net decrease in cash, cash equivalents and restricted cash $ (5,435 ) $ (3,518 ) Expand
