An Open-Label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 4 to 11 Years) With APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-Label Long-Term Extension

Activated Phosphoinositide 3-Kinase Delta Syndrome

Part I: • Incidence of treatment-emergent AEs (TEAEs), SAEs, and AEs leading to discontinuation of study drug, Part I: • Change from baseline in clinical laboratory test results (hematology, blood chemistry, urinalysis), Part I: • Change from baseline in vital signs, Part I: • Change from baseline in physical examination findings, Part I: • Change from baseline in electrocardiograms (ECGs), Part I: • Change from baseline in growth and physical development, Part I: • Reduction in lymphoproliferation as measured by MRI or low-dose CT at end of 12 weeks of treatment, Part I: • Immunophenotype normalization assessed by changes from baseline in the proportion of naïve B cells among all B cells to end of 12 weeks of treatment, Part II: • All safety parameters (including TEAEs, SAEs, Aes leading to discontinuation of study drug, physical examination, vital signs, ECGs, growth and physical development, and clinical laboratory results)

Part I: • popPK model that describes the appropriate covariates (eg, body weight and age) that influence leniolisib PK in pediatric patients (from baseline to end of 12 weeks of treatment), Part I: • Frequency of infections, use of antibiotics, and Ig replacement therapy, Part I: • Phosphorylated protein kinase B (pAKT) inhibition in whole blood, Part II: • Reduction in lymphoproliferation as measured by MRI or low-dose CT at 1 year, as measured by SPD of index and measurable non-index lesions selected as per the Cheson methodology, 3D volume and 3D sizes of spleen and liver, where appropriate, Part II: • Incidence of infections, use of antibiotics, and use of Ig replacement therapy

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