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

Pharming Group announces positive results from the pivotal Phase II/III blinded randomized, placebo-controlled registration-enabling study of leniolisib for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) also known as PASLI (p110δ-activating mutation causing senescent T cells, lymphadenopathy, and immunodeficiency).