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Venetoclax (VEN)
Venetoclax is a selective BCL-2 inhibitor that promotes apoptosis by blocking the anti-apoptotic protein BCL-2 in cancer cells.
Venetoclax is a selective BCL-2 inhibitor that promotes apoptosis by blocking the anti-apoptotic protein BCL-2 in cancer cells. Used for Chronic lymphocytic leukemia (CLL), Acute myeloid leukemia (AML), Small lymphocytic lymphoma (SLL).
At a glance
| Generic name | Venetoclax (VEN) |
|---|---|
| Sponsor | Nanfang Hospital, Southern Medical University |
| Drug class | BCL-2 inhibitor |
| Target | BCL-2 |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Venetoclax binds directly to BCL-2, preventing it from sequestering pro-apoptotic proteins like BAX and BAK. This allows the intrinsic mitochondrial apoptotic pathway to proceed, leading to programmed cell death in malignant cells that depend on BCL-2 for survival. It is particularly effective in hematologic malignancies with high BCL-2 expression.
Approved indications
- Chronic lymphocytic leukemia (CLL)
- Acute myeloid leukemia (AML)
- Small lymphocytic lymphoma (SLL)
Common side effects
- Neutropenia
- Thrombocytopenia
- Anemia
- Tumor lysis syndrome
- Nausea
- Diarrhea
- Fatigue
Key clinical trials
- BLAST MRD AML-2: BLockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia 2- A Randomized Phase 2 Study of Anti-PD-1 Pembrolizumab in Combination With Azacitidine and Venetoclax as Frontline Therapy in Unfit Patients With Acute Myeloid Leukemia (PHASE2)
- A Multicenter RCT of "3+7" vs Venetoclax + CACAG in Newly Diagnosed Mid/High-Risk AML Patients (PHASE2)
- A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) (PHASE3)
- Front-line VenObi Combination Followed by Ven or VenZan Combination in Patients With Residual Disease: a MRD Tailored Treatment for Young Patients With High-risk CLL (PHASE2)
- Safety and Efficacy of Venetoclax With Escalating Doses of Omacetaxine in Patients With Acute Myeloid Leukemia Safety and Efficacy of Venetoclax With Escalating Doses of Omacetaxine in Patients With Acute Myeloid Leukemia (AML) (PHASE1)
- De-Escalation Study Evaluating Venetoclax and Azacitidine Discontinuation in AML Responding Patients (PHASE2)
- Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML (PHASE3)
- A Study of Bleximenib in Combination With Acute Myeloid Leukemia (AML) Directed Therapies (PHASE1)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |