New positive donidalorsen data to be presented at AAAAI/WAO Joint Congress highlight sustained HAE attack rate reduction and disease control
Ionis Pharmaceuticals (NASDAQ: IONS) announced new data presentations for donidalorsen, their investigational RNA-targeted medicine for hereditary angioedema (HAE), at the 2025 AAAAI/WAO Joint Congress. The drug's New Drug Application is under FDA review with a PDUFA date of August 21, 2025.
The presentations will showcase additional data from the Phase 3 OASIS and OASISplus studies, along with three-year data from the Phase 2 open-label extension study. The company will present 11 posters covering various aspects including efficacy in adolescent patients, disease control after switching from prior treatments, quality-of-life improvements, and patient-reported outcomes.
The data demonstrates sustained HAE attack rate reduction and improved quality of life in patients previously on other prophylactic treatments, with the convenience of monthly or bi-monthly self-administration via autoinjector. If approved, donidalorsen would be Ionis' second independent commercial launch.
Ionis Pharmaceuticals (NASDAQ: IONS) ha annunciato nuove presentazioni di dati per donidalorsen, il loro medicinale sperimentale mirato all'RNA per l'angioedema ereditario (HAE), al Congresso Congiunto AAAAI/WAO del 2025. La Nuova Domanda di Farmaco è attualmente sotto revisione da parte della FDA con una data PDUFA fissata per il 21 agosto 2025.
Le presentazioni mostreranno dati aggiuntivi dagli studi di Fase 3 OASIS e OASISplus, insieme ai dati triennali dello studio di estensione open-label di Fase 2. L'azienda presenterà 11 poster che coprono vari aspetti, tra cui l'efficacia nei pazienti adolescenti, il controllo della malattia dopo il passaggio da trattamenti precedenti, i miglioramenti della qualità della vita e i risultati riportati dai pazienti.
I dati dimostrano una riduzione sostenuta della frequenza degli attacchi di HAE e un miglioramento della qualità della vita nei pazienti precedentemente trattati con altri farmaci profilattici, con la comodità di un'autoiniezione mensile o bimestrale tramite autoiniettore. Se approvato, donidalorsen rappresenterebbe il secondo lancio commerciale indipendente di Ionis.
Ionis Pharmaceuticals (NASDAQ: IONS) anunció nuevas presentaciones de datos para donidalorsen, su medicamento experimental dirigido al ARN para el angioedema hereditario (HAE), en el Congreso Conjunto AAAAI/WAO de 2025. La Solicitud de Nuevo Medicamento está bajo revisión de la FDA con una fecha PDUFA del 21 de agosto de 2025.
Las presentaciones mostrarán datos adicionales de los estudios de Fase 3 OASIS y OASISplus, junto con datos de tres años del estudio de extensión abierto de Fase 2. La compañía presentará 11 carteles que cubren varios aspectos, incluidos la eficacia en pacientes adolescentes, el control de la enfermedad después de cambiar de tratamientos previos, las mejoras en la calidad de vida y los resultados informados por los pacientes.
Los datos demuestran una reducción sostenida de la tasa de ataques de HAE y una mejora en la calidad de vida en pacientes que anteriormente recibían otros tratamientos profilácticos, con la conveniencia de la autoadministración mensual o bimestral a través de un autoinyector. Si se aprueba, donidalorsen sería el segundo lanzamiento comercial independiente de Ionis.
아이오니스 제약(Ionis Pharmaceuticals, NASDAQ: IONS)은 유전성 혈관부종(HAE)을 위한 RNA 표적 의약품인 도니다로르센에 대한 새로운 데이터 발표를 2025 AAAAI/WAO 공동 학술대회에서 발표할 예정이라고 발표했습니다. 이 약물의 신약 신청서는 FDA 검토 중이며 PDUFA 날짜는 2025년 8월 21일입니다.
이번 발표에서는 3상 OASIS 및 OASISplus 연구의 추가 데이터와 2상 오픈 라벨 확장 연구의 3년 데이터를 소개할 것입니다. 회사는 청소년 환자의 효능, 이전 치료에서의 전환 후 질병 관리, 삶의 질 개선 및 환자가 보고한 결과를 포함한 다양한 측면을 다룬 11개의 포스터를 발표할 것입니다.
데이터는 이전에 다른 예방 치료를 받은 환자에서 HAE 발작 비율 감소와 삶의 질 향상을 보여주며, 자가 주사기를 통한 월간 또는 격월 자가 투여의 편리함을 제공합니다. 승인된다면, 도니다로르센은 아이오니스의 두 번째 독립 상업 출시가 될 것입니다.
Ionis Pharmaceuticals (NASDAQ: IONS) a annoncé de nouvelles présentations de données pour le donidalorsen, leur médicament expérimental ciblant l'ARN pour l'angioœdème héréditaire (HAE), lors du Congrès conjoint AAAAI/WAO de 2025. La demande de nouveau médicament est actuellement en cours de révision par la FDA avec une date PDUFA fixée au 21 août 2025.
Les présentations mettront en avant des données supplémentaires des études de phase 3 OASIS et OASISplus, ainsi que des données sur trois ans de l'étude d'extension en ouvert de phase 2. L'entreprise présentera 11 affiches couvrant divers aspects, y compris l'efficacité chez les patients adolescents, le contrôle de la maladie après un changement de traitement antérieur, les améliorations de la qualité de vie et les résultats rapportés par les patients.
Les données démontrent une réduction soutenue du taux d'attaques de HAE et une amélioration de la qualité de vie chez les patients ayant précédemment reçu d'autres traitements prophylactiques, avec la commodité d'une auto-administration mensuelle ou bimestrielle via un autoinjecteur. Si approuvé, le donidalorsen serait le deuxième lancement commercial indépendant d'Ionis.
Ionis Pharmaceuticals (NASDAQ: IONS) hat neue Datenpräsentationen für Donidalorsen, ihr experimentelles RNA-zielgerichtetes Medikament für hereditäres Angioödem (HAE), auf dem 2025 AAAAI/WAO Joint Congress angekündigt. Der Antrag auf Zulassung eines neuen Medikaments wird derzeit von der FDA geprüft, mit einem PDUFA-Datum vom 21. August 2025.
Die Präsentationen werden zusätzliche Daten aus den Phase-3-Studien OASIS und OASISplus sowie dreijährige Daten aus der offenen Verlängerungsstudie der Phase 2 zeigen. Das Unternehmen wird 11 Poster präsentieren, die verschiedene Aspekte abdecken, darunter die Wirksamkeit bei jugendlichen Patienten, die Krankheitskontrolle nach dem Wechsel von vorherigen Behandlungen, Verbesserungen der Lebensqualität und patientenberichtete Ergebnisse.
Die Daten zeigen eine nachhaltige Reduzierung der HAE-Attackenrate und eine verbesserte Lebensqualität bei Patienten, die zuvor andere prophylaktische Behandlungen erhalten hatten, mit der Bequemlichkeit der monatlichen oder zwei monatlichen Selbstverabreichung über einen Autoinjektor. Wenn genehmigt, wäre Donidalorsen Ionis' zweiter unabhängiger kommerzieller Launch.
- FDA review in progress with clear PDUFA date of August 21, 2025
- Positive clinical data showing sustained HAE attack rate reduction
- Demonstrated improvements in quality-of-life measures
- Convenient monthly or bi-monthly self-administration option
- None.
Insights
The extensive data presentation for donidalorsen at the AAAAI/WAO Joint Congress represents a important milestone for Ionis Pharmaceuticals' commercial expansion strategy. The breadth of clinical evidence, spanning 11 presentations, strategically addresses key market differentiators that could position donidalorsen as a leading prophylactic treatment for HAE.
Several aspects of the data are particularly compelling from a market perspective:
- The demonstration of sustained attack rate reduction in both Phase 3 OASIS and Phase 2 studies strengthens the drug's efficacy profile
- The convenience of monthly or bi-monthly self-administration via autoinjector addresses a critical patient need
- Positive quality-of-life data and successful switching studies from other prophylactic treatments suggest strong potential for market adoption
The timing of this data presentation, ahead of the August 21, 2025, PDUFA date, is strategically important. As Ionis' second independent commercial launch, donidalorsen represents a significant opportunity in the HAE market, where current prophylactic treatments often require more frequent dosing schedules. The first-in-class RNA-targeted approach could provide Ionis with a competitive advantage in a market that continues to seek more effective and convenient treatment options.
The comprehensive nature of the clinical program, including long-term safety data and specific analyses in adolescent populations, positions donidalorsen favorably for regulatory review and subsequent market acceptance. The focus on patient-reported outcomes and quality-of-life measures aligns with increasing emphasis on patient-centric healthcare delivery, potentially supporting favorable reimbursement decisions.
– Data also show significant and clinically meaningful improvements in quality-of-life measures in new analyses from Phase 3 and Phase 2 clinical program –
–With a PDUFA date of August 21, 2025, donidalorsen positioned to be Ionis’ second independent commercial launch –
The New Drug Application (NDA) for donidalorsen to prevent attacks of HAE in adult and pediatric patients 12 years of age and older is currently under review with the
“While there’s been notable advancement in the HAE treatment landscape, there is still an urgent need for a medicine that effectively reduces attacks, is well tolerated and simple to administer. Across the breadth of presentations at the congress, we believe the totality of the clinical evidence underscores the potential of donidalorsen to be the prophylactic treatment of choice for people living with HAE,” said Kenneth Newman, M.D., senior vice president, head of clinical development, Ionis. “In new analyses from our OASISplus prospective switch cohort, donidalorsen continued to demonstrate the ability to reduce the HAE attack rate burden and improve quality of life in patients previously on other prophylactic treatments, with the simplicity of monthly or every two-month self-administration via an autoinjector. As a first-in-class RNA-targeted medicine, we believe donidalorsen has the potential to advance the prophylactic treatment paradigm for HAE.”
Ionis will have 11 presentations, as follows:
-
Efficacy and Safety of Donidalorsen In Adolescent Patients with Hereditary Angioedema: A Subanalysis of the Phase 3 OASIS-HAE Study
- Featured Poster Presentation: March 2, 3:30-5:00pm PT (Poster 890)
- Presenting Author: Joshua Jacobs
-
Hereditary Angioedema Disease Control after Switching to Donidalorsen from Prior Long-Term Prophylaxis: Results from the OASISplus Open-Label Extension Study
- Poster Presentation: March 2, 2025, 9:45-10:45am PT (Poster 601)
- Presenting Author: Marc Reidl
-
Improvements in Quality-of-Life in Patients with HAE Receiving Donidalorsen: Post Hoc Analysis from the OASIS-HAE Study
- Poster Presentation: March 2, 2025, 9:45-10:45am PT (Poster 648)
- Presenting Author: Danny Cohn
-
Patient-Reported Injection-Site Pain and Treatment Satisfaction after Switching from Long-Term Prophylaxis to Donidalorsen for the Treatment of Hereditary Angioedema: Results from the OASISplus Study
- Poster Presentation: March 2, 2025, 9:45-10:45am PT (Poster 599)
- Presenting Author: Marc Reidl
-
Psychometric Validation of Angioedema Quality-of-Life Questionnaire in Hereditary Angioedema: Results from the OASIS-HAE Study
- Poster Presentation: February 28, 2025, 2:45-3:45pm PT (Poster 216)
- Presenting Author: Aaron Yarlas
-
Long-Term Analysis of the Phase 2 Open-Label Extension of Donidalorsen in Patients With Hereditary Angioedema
- Poster Presentation: March 2, 2025, 9:45-10:45am PT (Poster 654)
- Presenting Author: Michael Manning
-
Correlation between Subjective and Objective Disease Control in Hereditary Angioedema: Association between the Angioedema Control Test and Attack Rate
- Poster Presentation: March 2, 2025, 9:45-10:45am PT (Poster 647)
- Presenting Author: Danny Cohn
-
Long-Term Prophylaxis for Hereditary Angioedema: Real-World Experience in Selected US Allergy Clinics
- Poster Presentation: March 2, 2025, 9:45-10:45am PT (Poster 739)
- Presenting Author: Huamin Henry Li
-
The Patient Experience of Hereditary Angioedema: Findings from a Racially Diverse Sample of Adult Patients
- Poster Presentation: February 28, 2025, 2:45-3:45pm PT (Poster 215)
- Presenting Author: Aaron Yarlas
-
Patient Preferences for Attributes of Prophylactic Treatment in Hereditary Angioedema: A Discrete-Choice Experiment
- Poster Presentation: March 2, 2025, 9:45-10:45am PT (Poster 696)
- Presenting Author: Kathleen Villa
-
Participant Reported Ease-of-Use with a Prefilled, Single-dose, Disposable Autoinjector for the Treatment of Hereditary Angioedema
- Poster Presentation: March 2, 2025, 9:45-10:45am PT (Poster 699)
- Presenting Author: Hetal Khatri
Ionis previously reported positive results from the Phase 3 OASIS-HAE and OASISplus studies. Data from both studies were first presented at the 2024 European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress in
About Hereditary Angioedema (HAE)
HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect more than 20,000 people in the
About Donidalorsen
Donidalorsen is an investigational RNA-targeted medicine designed to target prekallikrein (PKK), which plays an important role in activating inflammatory mediators associated with acute attacks of hereditary angioedema (HAE). By reducing the production of PKK, donidalorsen could be an effective prophylactic approach to preventing HAE attacks, if approved.
Donidalorsen is an investigational medicine that has not been approved for the treatment of any disease by regulatory authorities.
About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram.
Ionis Forward-looking Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of our commercial medicines, donidalorsen, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc.
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Ionis Investor Contact:
D. Wade Walke, Ph.D.
IR@ionis.com
760-603-2331
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media@ionis.com
760-603-4679
Source: Ionis Pharmaceuticals, Inc.
FAQ
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