BioCryst to Present New ORLADEYO® (berotralstat) Results from APeX-P Pediatric Trial at 2025 American Academy of Allergy, Asthma & Immunology / World Allergy Organization Joint Congress
BioCryst Pharmaceuticals (BCRX) has announced plans to present five abstracts featuring new clinical and real-world outcomes of ORLADEYO® (berotralstat) at the 2025 AAAAI/WAO joint congress in San Diego. The presentations will include a significant late-breaking abstract showcasing the first results from the APeX-P trial, which evaluates ORLADEYO in pediatric HAE patients aged 2 to <12 years.
The presentations, scheduled for March 2, 2025, will cover various aspects including: HAE attack rates in pediatric patients, real-world attack rates before and after berotralstat initiation in different patient groups, patient willingness to change long-term prophylaxis, and patient-reported impact of berotralstat as long-term prophylaxis. All abstracts are accessible through The Journal of Allergy and Clinical Immunology's online supplement.
BioCryst Pharmaceuticals (BCRX) ha annunciato piani per presentare cinque abstract contenenti nuovi risultati clinici e dati dal mondo reale su ORLADEYO® (berotralstat) al congresso congiunto AAAAI/WAO del 2025 a San Diego. Le presentazioni includeranno un abstract di grande rilevanza che mostrerà i primi risultati del trial APeX-P, che valuta ORLADEYO nei pazienti pediatrici con HAE di età compresa tra 2 e <12 anni.
Le presentazioni, programmate per il 2 marzo 2025, tratteranno vari aspetti tra cui: tassi di attacco HAE nei pazienti pediatrici, tassi di attacco nel mondo reale prima e dopo l'inizio di berotralstat in diversi gruppi di pazienti, la disponibilità dei pazienti a cambiare la profilassi a lungo termine e l'impatto segnalato dai pazienti di berotralstat come profilassi a lungo termine. Tutti gli abstract sono accessibili attraverso il supplemento online del Journal of Allergy and Clinical Immunology.
BioCryst Pharmaceuticals (BCRX) ha anunciado planes para presentar cinco resúmenes que presentan nuevos resultados clínicos y del mundo real de ORLADEYO® (berotralstat) en el congreso conjunto AAAAI/WAO 2025 en San Diego. Las presentaciones incluirán un resumen destacado que mostrará los primeros resultados del ensayo APeX-P, que evalúa ORLADEYO en pacientes pediátricos con HAE de 2 a <12 años.
Las presentaciones, programadas para el 2 de marzo de 2025, cubrirán varios aspectos, incluyendo: tasas de ataques de HAE en pacientes pediátricos, tasas de ataques en el mundo real antes y después del inicio de berotralstat en diferentes grupos de pacientes, disposición de los pacientes a cambiar la profilaxis a largo plazo y el impacto reportado por los pacientes de berotralstat como profilaxis a largo plazo. Todos los resúmenes son accesibles a través del suplemento en línea del Journal of Allergy and Clinical Immunology.
BioCryst Pharmaceuticals (BCRX)는 2025년 샌디에고에서 열리는 AAAAI/WAO 공동 회의에서 ORLADEYO® (berotralstat)의 새로운 임상 및 실제 결과를 포함한 다섯 개의 초록 발표 계획을 발표했습니다. 발표에는 APeX-P 시험의 첫 번째 결과를 전시하는 중요한 긴급 초록이 포함될 예정입니다. 이 시험은 2세에서 <12세> 사이의 소아 HAE 환자에서 ORLADEYO를 평가합니다.
2025년 3월 2일로 예정된 발표는 다음을 포함한 여러 측면을 다룰 것입니다: 소아 환자의 HAE 발작율, berotralstat 시작 전후의 실제 발작율, 장기 예방 조치를 변경하려는 환자의 의지, 장기 예방 조치로서의 berotralstat에 대한 환자 보고 영향. 모든 초록은 알레르기 및 임상 면역학 저널의 온라인 보조 자료를 통해 접근할 수 있습니다.
BioCryst Pharmaceuticals (BCRX) a annoncé des projets de présentation de cinq résumés contenant de nouveaux résultats cliniques et du monde réel sur ORLADEYO® (berotralstat) lors du congrès conjoint AAAAI/WAO 2025 à San Diego. Les présentations incluront un résumé important à divulgation tardive montrant les premiers résultats de l'
Les présentations, programmées pour le 2 mars 2025, aborderont divers aspects, notamment : les taux d'attaques HAE chez les patients pédiatriques, les taux d'attaques dans le monde réel avant et après le début du berotralstat dans différents groupes de patients, la disposition des patients à changer de prophylaxie à long terme et l'impact signalé par les patients de berotralstat en tant que prophylaxie à long terme. Tous les résumés sont accessibles via le supplément en ligne du Journal of Allergy and Clinical Immunology.
BioCryst Pharmaceuticals (BCRX) hat Pläne angekündigt, fünf Abstracts mit neuen klinischen und realen Ergebnissen zu ORLADEYO® (berotralstat) auf dem gemeinsamen Kongress AAAAI/WAO 2025 in San Diego zu präsentieren. Die Präsentationen umfassen ein wichtiges „late-breaking“ Abstract, das die ersten Ergebnisse der APeX-P-Studie zeigt, die ORLADEYO bei pädiatrischen HAE-Patienten im Alter von 2 bis <12 Jahren> bewertet.
Die Präsentationen, die für den 2. März 2025 geplant sind, decken verschiedene Aspekte ab, darunter: HAE-Attacken bei pädiatrischen Patienten, reale Angriffsraten vor und nach Beginn von berotralstat in verschiedenen Patientengruppen, die Bereitschaft der Patienten, die langfristige Prophylaxe zu ändern, und die von den Patienten berichteten Auswirkungen von berotralstat als langfristige Prophylaxe. Alle Abstracts sind über das Online-Supplement des Journal of Allergy and Clinical Immunology zugänglich.
- None.
- None.
RESEARCH TRIANGLE PARK, N.C., Feb. 10, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company will present five abstracts featuring new clinical and real-world outcomes with oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) joint congress.
Included among these is a late-breaking abstract that highlights the first presentation of results from the APeX-P trial evaluating oral, once-daily ORLADEYO in pediatric patients with HAE who are 2 to <12 years of age. The congress will take place in San Diego from February 28-March 3, 2025.
BioCryst will present the following five abstracts on Sunday, March 2 from 9:45-10:45 a.m. PT in Hall A at the San Diego Convention Center:
- HAE Attack Rates in Pediatric Patients 2 to <12 Years of Age with Prophylactic Berotralstat: Results from Interim Analysis of APeX-P; Poster #L55
- Real-World Attack Rates Before and After Berotralstat Initiation Among Patients with Hereditary Angioedema with C1-Inhibitor Deficiency (Type I/II) Stratified by Monthly Baseline HAE Attack Frequency; Poster #603
- Real-World Attack Rates Before and After Berotralstat Initiation Among Patients with Hereditary Angioedema without C1-Inhibitor Deficiency (HAE-nl-C1-INH) Stratified by Monthly Baseline HAE Attack Frequency; Poster #607
- Exploring the Role of Disease Burden, Treatment Effectiveness, and Administration Preference on Willingness of Patients With HAE to Change Long-Term Prophylaxis; Poster #608
- Patient-Reported Impact of Berotralstat as Long-Term Prophylaxis on Hereditary Angioedema Attack Frequency and Attack Severity; Poster #655
The abstracts are available to view in an online supplement to The Journal of Allergy and Clinical Immunology (JACI) at jacionline.org.
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
INDICATION
ORLADEYO® (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 use
The 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 INFORMATION
An 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 (≥
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 www.fda.gov/medwatch.
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 www.biocryst.com 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. 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, which could take longer or be more expensive than planned; 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; BioCryst’s ability to successfully manage its growth and compete effectively; and risks related to the international expansion of BioCryst’s business. 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 7910
jbluth@biocryst.com
FAQ
What are the key findings from BioCryst's APeX-P pediatric trial for ORLADEYO presented at AAAAI 2025?
How many abstracts will BCRX present at the 2025 AAAAI/WAO congress?
What is the focus of BCRX's real-world attack rate studies for ORLADEYO?
When and where will BCRX present the ORLADEYO research findings?
