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Leniolisib

Phase 3

APDS | Small molecule | Other |Pharming Group N.V.|Last Updated: May 6, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment31
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05693129Pediatric Patients Aged 1 to 6 Years With APDSPHASE3 ACTIVE NOT_RECRUITING 16 — — Aug 30, 2023Oct 28, 2026Oct 28, 20259 United States, Japan +3
NCT05438407Pediatric Patients Aged 4 to 11 Years With APDSPHASE3 ACTIVE NOT_RECRUITING 15 — — Feb 1, 2023Dec 30, 2026May 6, 20267 United States, France +1
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Study Endpoints
Primary Endpoints
Part I & II: Number of Participants with Treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs) , and Adverse Events (AEs)
From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year, plus 30 days

To assess number of Participants with TEAEs, SAEs, and AEs leading to discontinuation of study drug

Part I & II: Change from baseline in clinical laboratory test results
From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year

Number of Participants with change in clinical laboratory test results (hematology, blood

Part I & II: Change from baseline in vital signs
From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year

Number of Participants with change in vital signs

Part I & II: Change from baseline in physical examination findings
From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year

Number of Participants with change in physical examination findings

Part I & II: Change from baseline in electrocardiograms (ECGs)
From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year, plus 30 days

Number of Participants with change in electrocardiograms (ECGs)

Part I & II: Change from baseline in growth and physical development
From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year

Number of Participants with change in growth and physical development

Part I & II: Reduction in lymphadenopathy as measured by MRI or low-dose CT
Part I: Baseline and Day 85 Part II: at Day 252, through study completion, an average of 1 year

To assess the impact of leniolisib on lymphadenopathy, patients will be scanned in an MRI or a CT scanner as based on clinical practice and local regulation. Index lesions will be selected from measurable nodal and extra nodal lesions as per the Cheson methodology. The same imaging modality will be used throughout the study for the same patient. Patients will be assessed by MRI, or in sites where local practice and local authorities/IECs/IRBs approve CT scans for research purposes using a low-dose CT scan.

Part I: A Percentage of Inhibition of Unstimulated and Stimulated pAkt Levels in B Cells
Part I: Baseline, Days 29, 57 and 85

To assess change in the PDx effect of leniolisib will be assessed using ex vivo stimulated and unstimulated

Part I & II: Reduction in lymphoproliferation as measured by MRI or low-dose CT
Part I: Baseline and Day 85 Part II: at Day 252, through study completion, an average of 1 year

For the assessment of the impact of leniolisib on lymphoproliferation, patients will be scanned in an MRI or a CT scanner as based on clinical practice and local regulation. Index lesions will be selected from measurable nodal and extra nodal lesions as per the Cheson methodology. The same imaging modality will be used throughout the study for the same patient. Patients will be assessed by MRI, or in sites where local practice and local authorities/IECs/IRBs approve CT scans for research purposes using a low-dose CT scan.

Secondary Endpoints
Part I: To assess the total drug exposure (AUC) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
From baseline to end of 12 weeks of treatment
Part I: To assess the maximum concentration (Cmax) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
From baseline to end of 12 weeks of treatment
Part I: To assess the time to maximum concentration (Tmax) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
From baseline to end of 12 weeks of treatment
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LeniolisibEXPERIMENTALLeniolisib film-coated granules in 10, 15 and 20 mg strengths administered orally BID by body weight for 12 weeks for Part I and for 1 year for Part II.
Interventions
NameTypeDescription
LeniolisibDRUGThe doses selected will range from 10 to 50 mg twice daily (BID) (resulting in total daily doses ranging from 20 to 100 mg per day).
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Eligibility Criteria
Age Range1 Year — 6 Years
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: 1. Patient is male or female and between the age of 1 to 6 years old at time of the first study procedure. 2. Patient weighs ≥8 and ≤37 kg at baseline. 3. Patient has a confirmed PI3Kδ genetic mutation of either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene. 4. Patient has at least ...

Countries:United StatesJapanPortugalSpainUnited KingdomFrance
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