Pharming Group Reports Positive Phase II Topline Data for Leniolisib in PIDs
核心洞察
Pharming reported positive topline data from a Phase II single-arm study of oral PI3Kδ (搜索) inhibitor leniolisib in 13 patients with genetically defined primary immunodeficiencies.
Treatment showed a favorable safety and tolerability profile with no new safety signals and clinical improvements in immune dysregulation.
The data include a mean 26.4% reduction in spleen volume and smaller lymphoproliferative lesions, with nine enrolled patients also diagnosed with common variable immunodeficiency (搜索).
Pharming Group has reported positive topline data from a Phase II single-arm study of its oral PI3Kδ (搜索) inhibitor leniolisib in 13 patients with genetically defined primary immunodeficiencies linked to dysregulated PI3Kδ signaling. The treatment showed a favorable safety and tolerability profile, with no new safety signals and clinical improvements in immune dysregulation, including a mean 26.4% reduction in spleen volume and smaller lymphoproliferative lesions.
The abstract has been accepted as a late-breaking presentation at October's European Society for Immunodeficiencies meeting. The findings point to growing clinical interest in leniolisib beyond its current approval in activated PI3Kδ syndrome (搜索).
Nine enrolled patients were also diagnosed with common variable immunodeficiency (搜索), and separate Phase II CVID data are expected in the fourth quarter of 2026. Pharming said the results may help define a path toward a potential registrational program in a broader primary immunodeficiency population.