Pharming Group N.V. announces that the first patient has been dosed in a Phase II, proof of concept, clinical trial evaluating leniolisib in common variable immunodeficiency (CVID) patients with immune dysregulation.

Pharming Group N.V. announces positive topline results of data from its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS).

This multinational Phase III study is evaluating leniolisib tablets in children aged 4 to 11 years with APDS, a rare primary immunodeficiency.

Initial development in PIDs with immune dysregulation linked to PI3Kẟ signaling Phase 2 clinical trial initiation planned for 2Q 2024

The multinational Phase III study is evaluating a new pediatric granulated formulation of leniolisib in children aged 1 to 6 years with APDS, a rare primary immunodeficiency

Single-arm Phase III study in Japan evaluating leniolisib in patients aged 12 years and older with APDS, a rare primary immunodeficiency

The multinational Phase III study is evaluating leniolisib tablets in children aged 4 to 11 years with APDS, a rare primary immunodeficiency.

V. Koneti Rao, MD, shared new evidence of long-term safety and hematologic response in patients who received leniolisib to treat APDS, a rare primary immunodeficiency. Interim analysis demonstrated leniolisib to be well tolerated and indicated the durability of the efficacy results seen in the Phase II/III randomized, controlled trial

Leniolisib was well tolerated and significant improvement over placebo was notable in the co-primary endpoints, reflecting a favorable impact on patients' immune dysregulation and deficiency. The peer-reviewed publication heightens international understanding of APDS, a rare and recently characterized immunodeficiency