New positive donidalorsen data to be presented at 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting
Ionis Pharmaceuticals (Nasdaq: IONS) will present new data on donidalorsen, its investigational RNA-targeted prophylactic medicine for hereditary angioedema (HAE), at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting. The presentations include:
1. Three-year data from the Phase 2 open-label extension study
2. Additional results from the pivotal Phase 3 OASIS and OASISplus studies
3. Impact on patient-reported outcomes
4. Safety, efficacy, and patient preference after switching to donidalorsen
5. Exposure-response analysis
6. Drug patterns, clinical outcomes, and economic costs among HAE patients
The new data demonstrate that donidalorsen significantly reduced HAE attacks, providing high levels of disease control and improved quality of life for up to three years with monthly or every two-month dosing.
Ionis Pharmaceuticals (Nasdaq: IONS) presenterà nuovi dati su donidalorsen, il suo farmaco profilattico sperimentale mirato all'RNA per l'angioedema ereditario (HAE), durante il Congresso Scientifico Annuale dell'American College of Allergy, Asthma & Immunology (ACAAI) del 2024. Le presentazioni includono:
1. Dati triennali dello studio di estensione open-label di Fase 2
2. Risultati aggiuntivi dagli studi pivotal di Fase 3 OASIS e OASISplus
3. Impatto sugli esiti riportati dai pazienti
4. Sicurezza, efficacia e preferenza dei pazienti dopo il passaggio a donidalorsen
5. Analisi esposizione-risposta
6. Modelli di trattamento, risultati clinici e costi economici tra i pazienti HAE
I nuovi dati dimostrano che donidalorsen ha significativamente ridotto gli attacchi di HAE, fornendo alti livelli di controllo della malattia e migliorando la qualità della vita per un periodo di fino a tre anni con dosaggi mensili o ogni due mesi.
Ionis Pharmaceuticals (Nasdaq: IONS) presentará nuevos datos sobre donidalorsen, su medicamento profiláctico en investigación dirigido al RNA para el angioedema hereditario (HAE), en la Reunión Científica Anual de 2024 del American College of Allergy, Asthma & Immunology (ACAAI). Las presentaciones incluyen:
1. Datos de tres años del estudio de extensión abierto de Fase 2
2. Resultados adicionales de los estudios pivotal de Fase 3 OASIS y OASISplus
3. Impacto en los resultados reportados por los pacientes
4. Seguridad, eficacia y preferencia del paciente tras cambiar a donidalorsen
5. Análisis de exposición-respuesta
6. Patrones de uso de medicamentos, resultados clínicos y costos económicos entre pacientes con HAE
Los nuevos datos demuestran que donidalorsen ha reducido significativamente los ataques de HAE, proporcionando altos niveles de control de la enfermedad y mejorando la calidad de vida durante hasta tres años con dosificación mensual o cada dos meses.
아이오니스 제약(Ionis Pharmaceuticals, Nasdaq: IONS)은 2024년 미국 알레르기, 천식 및 면역학회(ACAAI) 연례 과학 회의에서 유전성 혈관 부종(HAE)에 대한 RNA 표적 예방 의약품인 도니달로르센(donidalorsen)의 새로운 데이터를 발표할 예정입니다. 발표 내용은 다음과 같습니다:
1. 2상 오픈 라벨 확장 연구의 3년 데이터
2. 주요 3상 OASIS 및 OASISplus 연구의 추가 결과
3. 환자가 보고한 결과에 미치는 영향
4. 도니달로르센으로 전환 후 안전성, 효과 및 환자 선호도
5. 노출-반응 분석
6. HAE 환자 간의 약물 패턴, 임상 결과 및 경제적 비용
새로운 데이터는 도니달로르센이 HAE 발작을 상당히 감소시켜 질병 조절의 높은 수준과 3년 동안 매달 또는 두 달에 한 번 투약하여 삶의 질을 개선했음을 보여줍니다.
Ionis Pharmaceuticals (Nasdaq: IONS) présentera de nouvelles données sur le donidalorsen, son médicament prophylactique expérimental ciblant l'ARN pour l'angioedème héréditaire (HAE), lors de la Réunion Scientifique Annuelle 2024 du American College of Allergy, Asthma & Immunology (ACAAI). Les présentations incluent :
1. Données de trois ans de l'étude d'extension en ouvert de Phase 2
2. Résultats supplémentaires des études pivot de Phase 3 OASIS et OASISplus
3. Impact sur les résultats rapportés par les patients
4. Sécurité, efficacité et préférence des patients après passage au donidalorsen
5. Analyse exposition-réponse
6. Modèles de traitement, résultats cliniques et coûts économiques chez les patients HAE
Les nouvelles données démontrent que le donidalorsen a considérablement réduit les attaques de HAE, offrant des niveaux élevés de contrôle de la maladie et améliorant la qualité de vie pendant jusqu'à trois ans avec une posologie mensuelle ou tous les deux mois.
Ionis Pharmaceuticals (Nasdaq: IONS) wird auf dem jährlichen wissenschaftlichen Treffen des American College of Allergy, Asthma & Immunology (ACAAI) 2024 neue Daten zu Donidalorsen präsentieren, einem experimentellen RNA-zielgerichteten prophylaktischen Medikament für hereditäres Angioödem (HAE). Die Präsentationen umfassen:
1. Dreijährige Daten aus der offenen Verlängerungsstudie der Phase 2
2. Zusätzliche Ergebnisse aus den pivotalen Phase-3-Studien OASIS und OASISplus
3. Auswirkungen auf patientenberichtete Ergebnisse
4. Sicherheit, Wirksamkeit und Patientenpräferenz nach dem Wechsel zu Donidalorsen
5. Expositions-Wirkungs-Analyse
6. Arzneimittelgebrauch, klinische Ergebnisse und wirtschaftliche Kosten bei HAE-Patienten
Die neuen Daten zeigen, dass Donidalorsen HAE-Anfälle erheblich reduziert hat, was hohe Kontrollniveaus der Krankheit und eine verbesserte Lebensqualität über einen Zeitraum von bis zu drei Jahren mit monatlicher oder alle zwei Monate verabreichten Dosen ermöglicht.
- Donidalorsen demonstrated significant and sustained reductions in HAE attacks for up to three years
- High levels of disease control achieved with monthly or every two-month dosing
- Significant improvements in quality-of-life measures for a majority of patients
- Positive results from Phase 3 OASIS-HAE and OASISplus studies previously reported
- None.
– Data continue to demonstrate donidalorsen potential to deliver significant and sustained reductions in HAE attacks up to three years with monthly or every two-month dosing
New data at ACAAI demonstrate donidalorsen significantly reduced HAE attacks, translating to high levels of disease control and significant and clinically meaningful improvements in quality-of-life across multiple measures in a vast majority of patients, sustained up to three years. Presentations include:
- Phase 2 Open-Label Extension of Donidalorsen In Patients with Hereditary Angioedema: A Week 197 Analysis
- e-Poster Presentation: October 25, 2024 (Monitor 21, Exhibit Hall A)
- Presenting Author: Michael Manning
- Impact of Donidalorsen on Patient-Reported Outcomes: Results from the Phase 3 OASIS-HAE Study
- e-Poster Presentation: October 25, 2024 (Monitor 21, Exhibit Hall A)
- Presenting Author: Aaron Yarlas
- Treatment Of Hereditary Angioedema: Safety, Efficacy, and Patient Preference after Switching to Donidalorsen (OASISplus Study)
- e-Poster Presentation: October 25, 2024 (Monitor 21, Exhibit Hall A)
- Presenting Author: Danny Cohn
- Donidalorsen for Hereditary Angioedema: Results from the OASISplus Open-Label Extension Study
- e-Poster Presentation: October 25, 2024 (Monitor 21, Exhibit Hall A)
- Presenting Author: Raffi Tachdjian
- Donidalorsen Exposure-Response Analysis: Hereditary Angioedema Attack Rate versus Plasma Prekallikrein Concentration Relationship
- e-Poster Presentation: October 25, 2024 (Monitor 19, Exhibit Hall A)
- Presenting Author: Pratap Singh
- Drug Patterns, Clinical Outcomes, and Economic Costs among Hereditary Angioedema Patients Initiating Long-Term Prophylaxis
- e-Poster Presentation: October 25, 2024 (Monitor 19, Exhibit Hall A)
- Presenting Author: William R. Lumry
Ionis previously reported positive results from the Phase 3 OASIS-HAE and OASISplus studies. Data from both studies were presented at the 2024 European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress in Valencia, Spain and results from OASIS-HAE were published in The New England Journal of Medicine. The company also previously reported positive two-year results from the Phase 2 OLE study last year.
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 patients 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 five marketed medicines and a leading pipeline in neurology, cardiology, and other 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) and LinkedIn.
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, 2023, 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.
Ionis Pharmaceuticals Investor Contact:
D. Wade Walke, Ph.D. – IR@ionis.com – 760-603-2331
Ionis Pharmaceuticals Media Contact:
Hayley Soffer – media@ionis.com – 760-603-4679
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SOURCE Ionis Pharmaceuticals, Inc.
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