Product Description
Leniolisib is an orally active, potent phosphatidylinositol 3-kinase inhibitor (PI3K) inhibitor, with selectivity for the PI3Kdelta isoform [2]. Novartis originally developed CDZ173 as a treatment for autoimmune diseases, but has rapidly repurposed it for its potential in patients with Activated PI3K Delta Syndrome (APDS, a.k.a. p110 delta activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency, or PASLI). APDS is a rare genetic immunodeficiency disease characterised by lymphoproliferation, recurrent infections from childhood, and an increased risk of EBV-associated lymphoma. APDS is known to be caused by autosomal dominant, gain-of-function mutations in the PIK3CD gene which encodes the PI3Kdelta protein [3,5]. (Sourced from: https://www.guidetopharmacology.org/GRAC/LigandDisplayForward?ligandId=9424)
Mechanisms of Action: PI3K Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Approved
Approved Countries: Czech | United States
Approved Indications: None
Known Adverse Events: None
Company: Pharming Technologies B.V.
Company Location:
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Belarus, Czech Republic, France, Germany, Italy, Japan, Netherlands, Portugal, Russia, Spain, United Kingdom, United States, Unknown Location
Active Clinical Trial Count: 11
Highest Development Phases
Phase 3: Colitis|Lymphadenopathy|Other
Phase 2: Deficiency Diseases
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| CCDZ173X2201E1 | P3 |
Active, not recruiting |
Lymphadenopathy |
2027-01-21 |
79% |
| jRCT2031230417 | P3 |
Recruiting |
Other |
2025-12-31 |
|
| LE 3302 | P3 |
Unknown Status |
Other |
2025-12-23 |
|
| LE 7201 | P2 |
Recruiting |
Deficiency Diseases |
2025-10-01 |