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Pharming presents positive Phase II data for leniolisib in genetic PIDs with immune dysregulation
PHARPharming Group N.V.
Pharming Group N.V. presented positive topline data from a single-arm, open-label Phase II trial of leniolisib in patients with genetically identifiable primary immunodeficiencies (PIDs) linked to altered PI3Kδ signaling. The study enrolled 13 participants across four genotypes (CTLA4, SOCS1, NFKB1, FAS), nine of whom also had common variable immunodeficiency (CVID). Leniolisib was well-tolerated with no new safety signals. Key efficacy results included a mean spleen volume reduction of 26.4% in evaluable participants and a mean lymph node size reduction of 33.7%. Pulmonary function improved, with DLCO increasing by 8.0%, and liver stiffness improved in two of three participants. All 12 completers enrolled in a long-term extension study. The data support the potential of targeted PI3Kδ inhibition to address immune dysregulation beyond APDS. Pharming Group N.V. is a global biotechnology company that develops and commercializes innovative medicines for rare immune and genetic diseases.