Core Insights - Pharming Group N.V. is presenting Phase III clinical data for leniolisib, targeting pediatric patients aged 4-11 years with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) at the 2025 Annual Meeting of the Clinical Immunology Society [1][2] Presentations - The presentation titled "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)" will be led by Dr. Shanmuganathan Chandrakasan on May 2, 2025 [2] - Additional presentations include a systematic literature review on immune dysregulation in common variable immunodeficiency and insights into APDS from patient perspectives [2] About Leniolisib - Leniolisib is an oral small molecule PI3Kδ inhibitor approved in several countries as the first targeted treatment for APDS in patients aged 12 and older [3] - The drug has shown statistically significant improvement in co-primary endpoints in a Phase III clinical trial, indicating a favorable impact on immune dysregulation [3] - Leniolisib is under regulatory review in the European Economic Area, Canada, and other regions, with plans for approval in Japan [3] About Pharming Group N.V. - Pharming Group N.V. is a global biopharmaceutical company focused on developing innovative medicines for rare and life-threatening diseases [4] - The company is headquartered in Leiden, the Netherlands, and operates in over 30 markets worldwide [4]