Pharming Group announces upcoming presentations at the 2025 Annual Meeting of the Clinical Immunology Society (CIS)
Pharming Group (Euronext: PHARM/Nasdaq: PHAR) has announced multiple presentations at the 2025 Annual Meeting of the Clinical Immunology Society (CIS) in Philadelphia, scheduled for May 1-4, 2025. The presentations focus on leniolisib, including Phase III clinical data for pediatric patients aged 4-11 years with activated phosphoinositide 3-kinase delta syndrome (APDS).
The presentations include six posters covering various aspects:
- Phase 3 study outcomes of leniolisib in pediatric APDS patients
- Impact of immune dysregulation in Common Variable Immunodeficiency
- APDS-like endotype within common variable immunodeficiency
- Leniolisib's effects in autoimmune lymphoproliferative syndrome
- Demographic characteristics from the APDS-CHOIR registry
- Patient insights on APDS
Note: Leniolisib's effectiveness and safety for pediatric patients aged 4-11 years with APDS has not yet received regulatory approval.
Pharming Group (Euronext: PHARM/Nasdaq: PHAR) ha annunciato diverse presentazioni al 2025 Annual Meeting of the Clinical Immunology Society (CIS) che si terrà a Philadelphia dal 1 al 4 maggio 2025. Le presentazioni si concentrano su leniolisib, inclusi i dati clinici di Fase III per pazienti pediatrici di età compresa tra 4 e 11 anni affetti da sindrome da attivazione della fosfoinositide 3-chinasi delta (APDS).
Le presentazioni comprendono sei poster che trattano vari aspetti:
- Risultati dello studio di Fase 3 su leniolisib in pazienti pediatrici con APDS
- Impatto della disregolazione immunitaria nell'immunodeficienza comune variabile
- Endotipo simile ad APDS nell'immunodeficienza comune variabile
- Effetti di leniolisib nella sindrome linfoproliferativa autoimmune
- Caratteristiche demografiche dal registro APDS-CHOIR
- Percezioni dei pazienti sull’APDS
Nota: L'efficacia e la sicurezza di leniolisib nei pazienti pediatrici di 4-11 anni con APDS non hanno ancora ottenuto l'approvazione regolatoria.
Pharming Group (Euronext: PHARM/Nasdaq: PHAR) ha anunciado múltiples presentaciones en la Reunión Anual 2025 de la Clinical Immunology Society (CIS) en Filadelfia, programada del 1 al 4 de mayo de 2025. Las presentaciones se centran en leniolisib, incluyendo datos clínicos de fase III para pacientes pediátricos de 4 a 11 años con síndrome de activación de la fosfoinosítido 3-quinasa delta (APDS).
Las presentaciones incluyen seis pósters que abordan diversos aspectos:
- Resultados del estudio de fase 3 de leniolisib en pacientes pediátricos con APDS
- Impacto de la desregulación inmunitaria en la inmunodeficiencia común variable
- Endotipo similar a APDS dentro de la inmunodeficiencia común variable
- Efectos de leniolisib en el síndrome linfoproliferativo autoinmune
- Características demográficas del registro APDS-CHOIR
- Perspectivas de los pacientes sobre APDS
Nota: La eficacia y seguridad de leniolisib en pacientes pediátricos de 4 a 11 años con APDS aún no ha recibido aprobación regulatoria.
Pharming Group (유로넥스트: PHARM/나스닥: PHAR)는 2025년 5월 1일부터 4일까지 필라델피아에서 열리는 임상면역학회 연례회의(CIS) 2025에서 여러 발표를 진행할 예정입니다. 발표 내용은 레니올리십(leniolisib)에 초점을 맞추고 있으며, 4세에서 11세 사이의 소아 환자를 대상으로 한 활성화된 포스포이노시티드 3-키나제 델타 증후군(APDS)의 3상 임상 데이터가 포함됩니다.
발표는 다음과 같은 여섯 개의 포스터로 구성됩니다:
- 소아 APDS 환자를 대상으로 한 레니올리십 3상 연구 결과
- 일반 가변 면역결핍증에서 면역 조절 장애의 영향
- 일반 가변 면역결핍증 내 APDS 유사 내분류형
- 자가면역 림프증식 증후군에서 레니올리십의 효과
- APDS-CHOIR 등록부의 인구통계학적 특성
- APDS 환자의 인식
참고: 4세에서 11세 소아 APDS 환자에 대한 레니올리십의 효능 및 안전성은 아직 규제 당국의 승인을 받지 못했습니다.
Pharming Group (Euronext : PHARM/Nasdaq : PHAR) a annoncé plusieurs présentations lors de la réunion annuelle 2025 de la Clinical Immunology Society (CIS) à Philadelphie, prévue du 1er au 4 mai 2025. Les présentations portent sur leniolisib, incluant des données cliniques de phase III chez des patients pédiatriques âgés de 4 à 11 ans atteints du syndrome d’activation de la phosphoinositide 3-kinase delta (APDS).
Les présentations comprennent six posters couvrant différents aspects :
- Résultats de l’étude de phase 3 sur leniolisib chez les patients pédiatriques atteints d’APDS
- Impact de la dysrégulation immunitaire dans l’immunodéficience commune variable
- Endotype de type APDS au sein de l’immunodéficience commune variable
- Effets de leniolisib dans le syndrome lymphoprolifératif auto-immun
- Caractéristiques démographiques issues du registre APDS-CHOIR
- Perceptions des patients concernant l’APDS
Note : L’efficacité et la sécurité de leniolisib chez les patients pédiatriques de 4 à 11 ans atteints d’APDS n’ont pas encore reçu d’approbation réglementaire.
Pharming Group (Euronext: PHARM/Nasdaq: PHAR) hat mehrere Präsentationen auf dem Jahrestreffen 2025 der Clinical Immunology Society (CIS) in Philadelphia angekündigt, das vom 1. bis 4. Mai 2025 stattfindet. Die Präsentationen konzentrieren sich auf leniolisib, einschließlich Phase-III-Klinikdaten für pädiatrische Patienten im Alter von 4 bis 11 Jahren mit aktiviertem Phosphoinositid-3-Kinase-Delta-Syndrom (APDS).
Die Präsentationen umfassen sechs Poster, die verschiedene Aspekte behandeln:
- Ergebnisse der Phase-3-Studie zu leniolisib bei pädiatrischen APDS-Patienten
- Auswirkungen der Immunregulationsstörung bei der Gemeinsamen Variablen Immundefizienz
- APDS-ähnlicher Endotyp innerhalb der Gemeinsamen Variablen Immundefizienz
- Wirkungen von leniolisib beim Autoimmun-Lymphoproliferationssyndrom
- Demografische Merkmale aus dem APDS-CHOIR-Register
- Patientenperspektiven zu APDS
Hinweis: Die Wirksamkeit und Sicherheit von leniolisib bei pädiatrischen Patienten im Alter von 4 bis 11 Jahren mit APDS wurde bisher noch nicht von den Zulassungsbehörden genehmigt.
- None.
- None.
Leiden, the Netherlands, April 25, 2025: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM/Nasdaq: PHAR) announces the following presentations by the Company or its collaborators at the 2025 Annual Meeting of the Clinical Immunology Society (CIS), taking place May 1-4, 2025 in Philadelphia, PA. These presentations include Phase III clinical data for leniolisib for pediatric patients aged 4-11 years with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) and insights into APDS and additional primary immunodeficiencies (PIDs) with immune dysregulation.
Presentations:
Title: Primary and Safety Outcomes of a Phase 3 Open-Label, Single-Arm, 12-Week Study of Treatment With PI3Kδ Inhibitor Leniolisib in Pediatric Patients Aged 4-11 Years With Activated PI3Kδ Syndrome (APDS)
Presenting Author: Shanmuganathan Chandrakasan, MD, Division of Bone Marrow Transplant, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA, USA
Session Type: Poster
Session Date/Time: Friday, May 2, 2025, 1:30-2:30 pm (ET)
Abstract/Poster Number: 70
Title: The Impact of Immune Dysregulation on Clinical Outcomes in Common Variable Immunodeficiency: A Systematic Literature Review
Presenting Author: Jocelyn R. Farmer, MD, PhD, Department of Medicine, UMass Chan Medical School, Worcester, MA, USA, and Clinical Immunodeficiency Program of Beth Israel Lahey Health, Division of Allergy and Immunology, Lahey Hospital & Medical Center, Burlington MA, USA
Session Type: Poster
Session Date/Time: Saturday, May 3, 2025, 1:30-2:30 pm (ET)
Abstract/Poster Number: 30
Title: Defining an activated PI3K-delta syndrome-like endotype within broader common variable immunodeficiency
Presenting Author: Jocelyn R. Farmer, MD, PhD, Department of Medicine, UMass Chan Medical School, Worcester, MA, USA, and Clinical Immunodeficiency Program of Beth Israel Lahey Health, Division of Allergy and Immunology, Lahey Hospital & Medical Center, Burlington MA, USA
Session Type: Poster
Session Date/Time: Friday, May 2, 2025, 1:30-2:30 pm (ET)
Abstract/Poster Number: 32
Title: Leniolisib, a PI3Kδ inhibitor, improves lymphoproliferative disease in a murine model of autoimmune lymphoproliferative syndrome (ALPS-FAS)
Presenting Author: Kevin Thorneloe, PhD, Pharming Healthcare, Inc., Warren, NJ, USA
Session Type: Poster
Session Date/Time: Saturday, May 3, 2025, 1:30-2:30 pm (ET)
Abstract/Poster Number: 37
Title: Demographic and clinical characteristics of people with activated phosphoinositide 3-kinase delta syndrome (APDS) in the APDS-Characterization and Clinical Outcomes Immunologic Registry (APDS-CHOIR)
Presenting Author: Nicholas Hartog, MD, Cornwell Health Pediatric Allergy and Immunology, Grand Rapids, MI, USA
Session Type: Poster
Session Date/Time: Friday, May 2, 2025, 1:30-2:30 pm (ET)
Abstract/Poster Number: 49
Title: Patient insights expand understanding of APDS
Presenting Author: Kristie Cline, MBA, Pharming Healthcare, Inc., Warren, NJ, USA
Session Type: Poster
Session Date/Time: Saturday, May 3, 2025, 1:30-2:30 pm (ET)
Abstract/Poster Number: 40
The effectiveness and safety of leniolisib for pediatric patients aged 4-11 years with APDS has not been reviewed or approved by a regulatory authority. Preclinical data in ALPS-FAS may not be predictive of clinical results.
About leniolisib
Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved in the U.S., U.K., Australia and Israel as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which serves as an important cellular messenger and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Results from a randomized, placebo-controlled Phase III clinical trial demonstrated statistically significant improvement in the coprimary endpoints, reflecting a favorable impact on the immune dysregulation and deficiency seen in these patients, and interim open label extension data has supported the safety and tolerability of long-term leniolisib administration.1,2 Leniolisib is currently under regulatory review in the European Economic Area, Canada and several other countries for APDS, with plans to pursue regulatory approval in Japan. Leniolisib is also being evaluated in two Phase III clinical trials in children with APDS and in two Phase II clinical trials in primary immunodeficiencies (PIDs) with immune dysregulation. The safety and efficacy of leniolisib has not been established for PIDs with immune dysregulation beyond APDS.
About Pharming Group N.V.
Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. We are commercializing and developing a portfolio of innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.
For more information, visit www.pharming.com and find us on LinkedIn.
References
- Rao VK, et al Blood. 2023 Mar 2;141(9):971-983.
- Rao VK, et al. J Allergy Clin Immunol 2024;153:265-74.
For further public information, contact:
Pharming Group, Leiden, the Netherlands
Michael Levitan, VP Investor Relations & Corporate Communications
T: +1 (908) 705 1696
E: investor@pharming.com
FTI Consulting, London, UK
Simon Conway/Alex Shaw/Amy Byrne
T: +44 203 727 1000
LifeSpring Life Sciences Communication, Amsterdam, the Netherlands
Leon Melens
T: +31 6 53 81 64 27
E: pharming@lifespring.nl
US PR
Christina Skrivan
T: +1 (636) 352-7883
E: Christina.Skrivan@precisionaq.com
Attachment
FAQ
What are the key findings from Pharming's Phase 3 study of leniolisib in pediatric APDS patients?
When will Pharming (PHARM) present its leniolisib clinical data at the 2025 CIS Meeting?
How many research presentations will Pharming deliver at the 2025 CIS Annual Meeting?
What is the regulatory status of leniolisib for pediatric APDS patients?
What conditions is Pharming's leniolisib being studied for?
