Leniolisib is an orally active, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor. It is a targeted small-molecule therapy used for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency associated with dysregulated PI3Kδ signaling.
By selectively inhibiting PI3Kδ, leniolisib helps modulate abnormal immune-cell signaling and can improve clinical manifestations associated with APDS.
Leniolisib is a specialized immunology pharmaceutical API manufactured through controlled chemical synthesis, purification and crystallization.
Important quality attributes include identity, assay, related substances, water content, residual solvents, elemental impurities, polymorphic form and stability.
CAS Number: 1354690-24-8
Molecular Formula: C18H17F3N6O2
Molecular Weight: 406.37 g/mol
Drug Class: Immunomodulatory Agent
Pharmacological Class: Selective PI3Kδ Inhibitor
Therapeutic Class: Targeted Immunomodulator
Therapeutic Area: Immunology / Rare Diseases
Leniolisib selectively inhibits the delta isoform of phosphoinositide 3-kinase (PI3Kδ).
PI3Kδ is highly expressed in leukocytes and plays an important role in immune-cell signaling. In APDS, excessive PI3Kδ activity contributes to abnormal immune-cell development and function.
Leniolisib reduces this signaling and can:
Leniolisib is used for the treatment of activated PI3Kδ syndrome (APDS) in approved patient populations.
APDS is a rare genetic primary immunodeficiency characterized by abnormalities in the PI3Kδ signaling pathway and recurrent infections, lymphoproliferation and other immune-system complications.
Leniolisib is distinguished by its selectivity for PI3Kδ, rather than broad inhibition of multiple PI3K isoforms.
This targeted mechanism makes it relevant to therapeutic research involving immune-cell signaling and primary immunodeficiency disorders.
Typical API manufacturing may involve:
Leniolisib API quality evaluation may include:
Leniolisib should be stored in a tightly closed container under controlled pharmaceutical storage conditions, protected from excessive heat, moisture and direct light. Final storage conditions should be established through validated stability studies.
Leniolisib is relevant to research involving:
Leniolisib is a selective PI3Kδ inhibitor with CAS Number 1354690-24-8, molecular formula C18H17F3N6O2, and molecular weight 406.37 g/mol. It selectively modulates PI3Kδ signaling and is used for the treatment of activated PI3Kδ syndrome (APDS) in approved patient populations.
| Parameter | Specification |
|---|---|
| Appearance | White to off-white crystalline powder |
| Identification | IR & HPLC compliant |
| Assay (HPLC) | 98.0% – 102.0% |
| Loss on Drying | NMT 1.0% |
| Residue on Ignition | NMT 0.5% |
| Individual Impurity | NMT 0.5% |
| Total Impurities | NMT 1.0% |
| Water Content | NMT 1.0% |