Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication JOENJA is indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. JOENJA is a kinase inhibitor indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. ( 1 ) FDA label
Also known as Joenja
Leniolisib · 5 trials · 4 indications
To assess number of Participants with TEAEs, SAEs, and AEs leading to discontinuation of study drug
Number of Participants with change in clinical laboratory test results (hematology, blood
Number of Participants with change in vital signs
Number of Participants with change in physical examination findings
Number of Participants with change in electrocardiograms (ECGs)
Number of Participants with change in growth and physical development
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.
To assess change in the PDx effect of leniolisib will be assessed using ex vivo stimulated and unstimulated
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.
Adverse events (AEs)
To assess the number of AEs/SAEs and number of participants with AEs/SAEs
• To assess the number of AEs/SAEs and number of participants with AEs/SAEs
| Arm | Type | Description |
|---|---|---|
| Leniolisib | EXPERIMENTAL | Leniolisib 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. |
| Treatment Arm | EXPERIMENTAL | All subjects will receive leniolisib film-coated tablets (FCTs) |
| Name | Type | Description |
|---|---|---|
| Leniolisib | DRUG | The 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). |
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 ...
Leniolisib is an investigational oral small molecule being studied for activated phosphoinositide 3-kinase delta syndrome (APDS) and other primary immunodeficiency disorders, including common variable immunodeficiency (CVID) and PIDs linked to PI3K. It is also being evaluated in pediatric patients aged 1 to 6 years with APDS.
Leniolisib targets PIK3CD, the gene encoding the p110delta catalytic subunit of phosphoinositide 3-kinase delta. It acts as an inhibitor of this target. By inhibiting PI3K delta, leniolisib is designed to address the immune dysregulation that characterizes APDS and related primary immunodeficiency disorders.
Leniolisib is being developed by Pharming Group N.V., which trades under the ticker PHAR. The company is advancing the compound across multiple primary immunodeficiency indications, including APDS, CVID, and PIDs linked to PI3K.
Leniolisib is in Phase 3 development. A Phase 3 trial is evaluating the drug in pediatric patients aged 1 to 6 years with APDS. Additional Phase 2 studies are ongoing or completed in CVID and other primary immunodeficiency disorders. The drug has received a Priority Review designation from the FDA.
Leniolisib is being studied in several clinical trials. NCT05693129 is a Phase 3 trial in pediatric patients aged 1 to 6 years with APDS. NCT06990529 and NCT06549114 are Phase 2 trials in PIDs linked to PI3K and primary immunodeficiency disorders, respectively. NCT06897358 is a completed Phase 2 trial in CVID.
Leniolisib is a distinct small molecule developed specifically by Pharming Group N.V. for primary immunodeficiency disorders. It selectively targets PIK3CD and is not the same as other PI3K inhibitors, which may have different selectivity profiles and indications.