Astria Therapeutics Announces Design of ALPHA-ORBIT Pivotal Phase 3 Trial of Navenibart in HAE
Astria Therapeutics (NASDAQ:ATXS) has announced the design of ALPHA-ORBIT, a pivotal Phase 3 trial for navenibart in hereditary angioedema (HAE). The trial will evaluate both 3-month (Q3M) and 6-month (Q6M) dosing schedules over a 6-month treatment period.
The global, randomized, double-blind, placebo-controlled study will enroll up to 145 patients with Type 1 or 2 HAE. Patients will be randomized into three navenibart dose arms: 600mg initial dose followed by 300mg Q3M, 600mg Q6M, and 600mg Q3M, or placebo. The primary endpoint is time-normalized monthly HAE attacks at 6 months.
The Phase 3 trial initiation is expected in Q1 2025, with top-line results anticipated in early 2027. The program design incorporates feedback from regulators and is intended to support global registration. Previous Phase 1b/2 ALPHA-STAR trial results showed 90-95% reduction in mean monthly attack rate and up to 67% attack-free rate over 6 months.
Astria Therapeutics (NASDAQ:ATXS) ha annunciato il design di ALPHA-ORBIT, uno studio clinico di fase 3 fondamentale per navenibart nel trattamento dell'angioedema ereditario (HAE). Lo studio valuterà sia schemi di dosaggio di 3 mesi (Q3M) che di 6 mesi (Q6M) su un periodo di trattamento di 6 mesi.
Lo studio globale, randomizzato, in doppio cieco e controllato con placebo arruolerà fino a 145 pazienti con HAE di tipo 1 o 2. I pazienti saranno randomizzati in tre gruppi di dosaggio di navenibart: 600 mg di dose iniziale seguita da 300 mg Q3M, 600 mg Q6M, e 600 mg Q3M, oppure placebo. L'obiettivo primario è il numero di attacchi di HAE mensili normalizzati nel tempo a 6 mesi.
Si prevede che l'inizio della fase 3 dello studio avverrà nel primo trimestre del 2025, con risultati preliminari attesi all'inizio del 2027. Il design del programma incorpora il feedback degli enti regolatori ed è destinato a supportare la registrazione globale. I risultati precedenti della fase 1b/2 dello studio ALPHA-STAR hanno mostrato una riduzione del 90-95% nella media degli attacchi mensili e un tasso di attacchi assenti fino al 67% dopo 6 mesi.
Astria Therapeutics (NASDAQ:ATXS) ha anunciado el diseño de ALPHA-ORBIT, un ensayo clínico pivotal de fase 3 para navenibart en el angioedema hereditario (HAE). El ensayo evaluará tanto esquemas de dosificación de 3 meses (Q3M) como de 6 meses (Q6M) durante un periodo de tratamiento de 6 meses.
El estudio global, aleatorizado, doble ciego y controlado con placebo inscribirá hasta 145 pacientes con HAE tipo 1 o 2. Los pacientes se asignarán al azar a tres grupos de dosis de navenibart: una dosis inicial de 600 mg seguida de 300 mg Q3M, 600 mg Q6M, y 600 mg Q3M, o placebo. El objetivo principal es el número de ataques de HAE mensuales normalizados por tiempo a los 6 meses.
Se espera que el inicio del ensayo de fase 3 ocurra en el primer trimestre de 2025, con resultados preliminares anticipados a principios de 2027. El diseño del programa incorpora comentarios de los reguladores y está destinado a apoyar el registro global. Los resultados previos del ensayo ALPHA-STAR de fase 1b/2 mostraron una reducción del 90-95% en la tasa media de ataques mensuales y hasta un 67% de tasa libre de ataques durante 6 meses.
Astria Therapeutics (NASDAQ:ATXS)는 유전성 혈관부종(HAE) 치료를 위한 navenibart의 3상 임상시험 ALPHA-ORBIT의 설계를 발표했습니다. 이 시험에서는 3개월(Q3M) 및 6개월(Q6M) 투여 계획을 6개월 치료 기간 동안 평가할 것입니다.
이번 글로벌, 무작위, 이중 맹검, 위약 대조 연구에는 1형 또는 2형 HAE 환자 최대 145명이 등록될 예정입니다. 환자들은 600mg의 초기 용량, 이후 300mg Q3M, 600mg Q6M, 그리고 600mg Q3M 또는 위약의 세 가지 navenibart 용량 그룹으로 무작위 배정됩니다. 주요 평가 지표는 6개월 후 월별 HAE 공격 수의 시간 정규화입니다.
3상 임상시험은 2025년 1분기에 시작될 것으로 예상되며, 주요 결과는 2027년 초에 발표될 것으로 보입니다. 프로그램 설계는 규제 기관의 피드백을 반영하며, 글로벌 등록을 지원하기 위한 것입니다. 이전 1b/2 ALPHA-STAR 임상시험 결과는 평균 월별 공격률이 90-95% 감소했으며 6개월 동안 최대 67%의 공격 없음 비율을 보였습니다.
Astria Therapeutics (NASDAQ:ATXS) a annoncé la conception d'ALPHA-ORBIT, un essai clinique de phase 3 crucial pour navenibart dans l'angioœdème héréditaire (HAE). L'essai évaluera à la fois des protocoles de dosage de 3 mois (Q3M) et de 6 mois (Q6M) sur une période de traitement de 6 mois.
L'étude mondiale, randomisée, en double aveugle et contrôlée par placebo recrutera jusqu'à 145 patients atteints de HAE de type 1 ou 2. Les patients seront répartis au hasard en trois groupes de doses de navenibart : une dose initiale de 600 mg suivie de 300 mg Q3M, 600 mg Q6M et 600 mg Q3M, ou placebo. L'objectif principal est le nombre d'attaques mensuelles d'HAE normalisé dans le temps à 6 mois.
Le début de l'essai de phase 3 est prévu au premier trimestre 2025, avec des résultats préliminaires attendus début 2027. La conception du programme intègre les retours des régulateurs et vise à soutenir l'enregistrement mondial. Les résultats précédents de l'essai ALPHA-STAR de phase 1b/2 ont montré une réduction de 90-95% du taux moyen d'attaques mensuelles et un taux sans attaque allant jusqu'à 67% sur 6 mois.
Astria Therapeutics (NASDAQ:ATXS) hat das Design von ALPHA-ORBIT angekündigt, einer entscheidenden Phase-3-Studie für navenibart bei hereditärem Angioödem (HAE). Die Studie wird sowohl 3-Monats- (Q3M) als auch 6-Monats- (Q6M) Dosierungsschemata über einen Zeitraum von 6 Monaten evaluieren.
Die globale, randomisierte, doppelt-blinde, placebokontrollierte Studie wird bis zu 145 Patienten mit Typ-1- oder Typ-2-HAE einschreiben. Die Patienten werden in drei Gruppen für navenibart-Dosen randomisiert: 600 mg Initialdosis, gefolgt von 300 mg Q3M, 600 mg Q6M und 600 mg Q3M oder Placebo. Der primäre Endpunkt ist die zeitnormalisierte monatliche HAE-Attacke nach 6 Monaten.
Der Beginn der Phase-3-Studie wird im ersten Quartal 2025 erwartet, mit den Hauptergebnissen, die Anfang 2027 erwartet werden. Das Programmdesign berücksichtigt das Feedback der Regulierungsbehörden und ist darauf ausgelegt, die globale Zulassung zu unterstützen. Frühere Ergebnisse der Phase 1b/2 ALPHA-STAR-Studie zeigten eine Reduktion der durchschnittlichen monatlichen Angriffshäufigkeit um 90-95% und eine bis zu 67% angreifungsfreie Rate über 6 Monate.
- Phase 1b/2 trial showed strong efficacy with 90-95% reduction in monthly attack rate
- Up to 67% attack-free rate achieved over 6 months in previous trial
- Company is funded through expected Phase 3 top-line results
- Trial design supports potential global registration for both Q3M and Q6M administration
- Phase 3 trial results not expected until early 2027
- Large trial size of 145 patients may extend recruitment period
Insights
The Phase 3 ALPHA-ORBIT trial design for navenibart represents a significant advancement in HAE treatment development. The trial's innovative approach includes three dosing arms (300mg Q3M following 600mg loading dose, 600mg Q6M and 600mg Q3M) against placebo, with 145 patients. The Phase 1b/2 ALPHA-STAR results showing 90-95% reduction in monthly attack rates and 67% attack-free rate over 6 months provide strong foundational data.
The trial's differentiated feature lies in its patient-centric dosing flexibility, potentially allowing for both quarterly and semi-annual administration options - a first in the HAE space. The primary endpoint focusing on time-normalized monthly attacks at 6 months, coupled with the attack-free rate secondary endpoint, aligns well with clinically meaningful outcomes. The planned long-term extension study with flexible dosing will provide important safety and durability data.
With trial initiation in Q1 2025 and top-line results expected in early 2027, this timeline suggests efficient trial execution. The regulatory engagement with both FDA and EMA strengthens the registration strategy.
This Phase 3 trial announcement carries substantial market implications. The HAE therapeutics market is competitive and navenibart's potential flexible dosing schedule could be a significant differentiator. Current treatments typically require more frequent dosing, creating a clear market opportunity for a less burdensome option.
The company's strong financial position through expected top-line Phase 3 results reduces near-term funding risks. With a market cap of
The emphasis on patient-centric design and potential "market-leading, first-choice therapy" positioning suggests a well-thought-out commercial strategy. The Q3M and Q6M dosing options could capture both treatment-experienced patients seeking longer intervals between doses and newly diagnosed patients looking for optimal first-line therapy.
-- Single Pivotal Trial Designed to Demonstrate Efficacy and Safety of Every 3- and Every 6-Month Administration in a 6-Month Treatment Period --
-- Pioneering Patient-Centric Dosing Flexibility in HAE with Potential Market-Leading First-Choice Profile --
-- Phase 3 Initiation On-Track, Expected in Q1 2025 --
-- Strong Financial Position, Funded Through Expected Top-Line Phase 3 Results --
“We are thrilled to announce our planned Phase 3 design, which reflects feedback from regulators and is intended to support global registration for both Q3M and Q6M administration,” said Jill C. Milne, Ph.D., Chief Executive Officer at Astria. “With navenibart, we are pioneering patient-centric dosing flexibility in HAE with the goal of maximizing attack rate reduction with a compellingly low burden of treatment. Assuming approval, we believe navenibart will become the market-leading, first-choice therapy for HAE.”
“Our Phase 3 program was designed in collaboration with the patient community and physicians, is based on input from global regulatory authorities, and addresses the importance of providing options to patients for a disease that’s highly variable,” said Christopher Morabito, M.D., Chief Medical Officer at Astria. “Phase 3 preparations are underway, with trial initiation on-track and expected for this quarter. We are driven by the goal of bringing a potentially life-changing therapy to patients with HAE.”
ALPHA-ORBIT is designed as a global, randomized, double-blind, placebo-controlled Phase 3 pivotal clinical trial to evaluate the efficacy and safety of navenibart over a 6-month treatment period in up to 145 patients with Type 1 or Type 2 HAE. Patients will be randomized to receive one of three navenibart dose arms: 1) an initial 600 mg dose and followed by 300 mg Q3M, 2) 600 mg Q6M, and 3) 600 mg Q3M, or placebo. The dose arms support the potential to provide patient-centric dosing flexibility to people with HAE. 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 extension trial, in which all patients will be treated with navenibart (open-label) and which will include an open-label, patient-centric flexible dosing period. The navenibart Phase 3 program will consist of the ALPHA-ORBIT Phase 3 trial and the long-term extension trial, which are designed to support registration globally. The Phase 3 program was designed with input from the European Medicines Agency and the Company’s end of Phase 2 meeting with the
Planned doses for the Phase 3 ALPHA-ORBIT program were selected based on positive final top-line results from target enrollment in the Phase 1b/2 ALPHA-STAR trial of navenibart, announced in December 2024, which 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
Additional details regarding the Company’s planned Phase 3 program and other business updates are contained in the Company’s Corporate Presentation, which is available on the “Events and Presentations” page of the “For Investors” section of the Company’s website.
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 Instagram @AstriaTx and on Facebook and LinkedIn.
About Navenibart:
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. We aim to empower people with HAE to live life without limitations from their disease.
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 design, timing of initiation and receipt of topline results from the ALPHA-ORBIT trial; the goals and objectives of the ALPHA-ORBIT trial and the long-term extension trial, including that they would support registration of Q3M and Q6M administration, and potentially accelerate the availability of Q6M administration; our expectations for the dosing regimens of navenibart and the efficacy data of navenibart in the ALPHA-ORBIT trial; the potential therapeutic benefits of navenibart as a treatment for HAE; the potential attributes and profile of navenibart as a treatment for HAE, including our expectation that it will be the market-leading, first choice and a potentially life-changing treatment for patients with HAE; our overall vision and goals for the navenibart program; expectations about being funded through top-line Phase 3 results; and our corporate strategy and vision, including our mission to bring life-changing therapies to patients and families affected by allergic and immunologic diseases. 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 and 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 studies and clinical trials 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 risks and uncertainties related to: 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 may not be replicated in clinical trials, that the preliminary, initial 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 ALPHA-STAR Phase 1b/2 clinical trial, may not be replicated in later stage clinical trials, such as the ALPHA-ORBIT trial and the open-label extension trial, 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, including ALPHA-ORBIT, may not commence, continue or be completed on our anticipated timelines, or at all; decisions made by, and feedback received from, the FDA and other regulatory authorities on our clinical trial design, including for ALPHA-ORBIT, and on our regulatory and clinical trial submissions, including receipt of FDA minutes from our December 2024 end of Phase 2 meeting, 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, decisions that we make about the design of clinical trials in response to regulatory feedback, including the design of the ALPHA-ORBIT trial and the long-term extension trial; our ability to manufacture sufficient quantities of drug substance and drug product for navenibart, STAR-0310, and any other future product candidates on a cost-effective and timely basis, and to develop dosages and formulations for navenibart, STAR-0310, and any other future product candidates that are patient-friendly and competitive; our ability to develop sufficient data to enable the use of planned devices with navenibart, STAR-0310 and any other future product candidates at commercial launch or otherwise as planned; our ability to develop biomarker and other assays, along with the testing protocols therefor; 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, modeling data 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 that any of our clinical trials of STAR-0310 may not commence, continue or be completed on time, or at all; risks that results of preclinical studies of STAR-0310 will not be replicated in clinical trials; 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.
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Source: Astria Therapeutics, Inc.
FAQ
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