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).
APDS is a rare primary immunodeficiency disease caused by genetic variants affecting approximately 1-2 people per million. Clinical hallmarks of the disease are severe, recurrent sinopulmonary infections, significant lymphoproliferation and immune dysfunction, as well as increased risk of malignant lymphoma.
Read the full press release here.