CLINICAL TRIALS PROFILE FOR VENETOCLAX

505(b)(2) Clinical Trials for Venetoclax

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT04599634 ↗	Venetoclax With Obinutuzumab and Magrolimab (VENOM) in Relapsed and Refractory Indolent B-cell Malignancies	Not yet recruiting	National Cancer Institute (NCI)	Phase 1	2021-11-16	Background: B-cell lymphoma is a cancer of certain white blood cells (called lymphocytes). These cells are found in lymph nodes. The cancer can cause enlargement of the lymph nodes leading to pain and discomfort. Swollen lymph nodes can also press on nearby organs such as liver and kidneys which can affect normal functioning of the organs. Researchers think that a new combination of drugs may be able to help. Objective: To find out if it is safe to give the combination of Magrolimab, Obinutuzumab and Venetoclax to people with B-cell lymphomas. Eligibility: Adults age 18 and older with an indolent B-cell lymphoma whose disease has returned or progressed after other treatment. Indolent B-cell lymphoma for this protocol is defined as having either follicular lymphoma, mantle cell lymphoma, chronic lymphocytic leukemia/small lymphocytic lymphoma or marginal zone lymphoma. Design: Participants will be screened under a separate protocol. Participants will have 28-day 'cycles' of treatment. They will take Venetoclax by mouth daily. They will get Obinutuzumab and Magrolimab by intravenous (IV) infusion. Treatment will last for about 8 months. They may be able to have more cycles of treatment if their cancer is responding well. Participants will have physical exams, medical histories, and medicine reviews. Data about how they function in their daily activities will be obtained. They will have blood and urine tests. They may have bone marrow tests. Participants will have imaging scans. These will include computed tomography (CT) and/or magnetic resonance imaging (MRI) scans and positron emission tomography (PET) scans. Participants may give a cheek swab or saliva sample. They may give tumor tissue and bone marrow samples. These samples may be used for gene testing. Participants will have a follow-up visit about 30 days after treatment ends. Then they will have visits every 3 months for the first 2 years, every 6 months for the next 3 years, and then yearly after that.

All Clinical Trials for Venetoclax

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00501826 ↗	Combination Chemotherapy and Nelarabine in Treating Patients With T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma	Recruiting	GlaxoSmithKline	Phase 2	2007-07-11	This phase II trial studies the side effects and how well combination chemotherapy and nelarabine work in treating patients with T-cell acute lymphoblastic leukemia or lymphoblastic lymphoma. Drugs used in chemotherapy, such as cyclophosphamide, vincristine, doxorubicin, dexamethasone, methotrexate, cytarabine, mercaptopurine, prednisone, pegaspargase, nelarabine, and venetoclax work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
NCT00501826 ↗	Combination Chemotherapy and Nelarabine in Treating Patients With T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma	Recruiting	National Cancer Institute (NCI)	Phase 2	2007-07-11	This phase II trial studies the side effects and how well combination chemotherapy and nelarabine work in treating patients with T-cell acute lymphoblastic leukemia or lymphoblastic lymphoma. Drugs used in chemotherapy, such as cyclophosphamide, vincristine, doxorubicin, dexamethasone, methotrexate, cytarabine, mercaptopurine, prednisone, pegaspargase, nelarabine, and venetoclax work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
NCT00501826 ↗	Combination Chemotherapy and Nelarabine in Treating Patients With T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma	Recruiting	M.D. Anderson Cancer Center	Phase 2	2007-07-11	This phase II trial studies the side effects and how well combination chemotherapy and nelarabine work in treating patients with T-cell acute lymphoblastic leukemia or lymphoblastic lymphoma. Drugs used in chemotherapy, such as cyclophosphamide, vincristine, doxorubicin, dexamethasone, methotrexate, cytarabine, mercaptopurine, prednisone, pegaspargase, nelarabine, and venetoclax work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
NCT01211457 ↗	Study of Sapacitabine in Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS)	Unknown status	M.D. Anderson Cancer Center	Phase 1/Phase 2	2010-06-17	This is a combination study to evaluate sapacitabine administered in alternating cycles with decitabine in previously untreated Acute Myeloid Leukemia (AML) or concomitantly with venetoclax in previously treated AML or MDS
NCT01211457 ↗	Study of Sapacitabine in Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS)	Unknown status	Cyclacel Pharmaceuticals, Inc.	Phase 1/Phase 2	2010-06-17	This is a combination study to evaluate sapacitabine administered in alternating cycles with decitabine in previously untreated Acute Myeloid Leukemia (AML) or concomitantly with venetoclax in previously treated AML or MDS
NCT01671904 ↗	A Study of Venetoclax in Combination With Bendamustine + Rituximab or Bendamustine + Obinutuzumab in Participants With Relapsed/Refractory or Previously Untreated Chronic Lymphocytic Leukemia (CLL)	Completed	AbbVie (prior sponsor, Abbott)	Phase 1	2014-01-13	This multi-center, open-label, dose-finding study will evaluate the safety and pharmacokinetics of venetoclax (GDC-0199, ABT-199) administered in combination with bendamustine and rituximab (BR) (MabThera/Rituxan) or bendamustine and obinutuzumab (BG) to participants with first-line (1L)/previously untreated or relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL). The study will explore two venetoclax combination regimens in participants with 1L CLL: BR+venetolax (V) and BG+V. Participants with R/R CLL will be administered BR+V.

Clinical Trial Conditions for Venetoclax

Condition Name

Condition Name for Venetoclax
Intervention	Trials
Acute Myeloid Leukemia	166
Chronic Lymphocytic Leukemia	59
Refractory Acute Myeloid Leukemia	37
Small Lymphocytic Lymphoma	32

Condition MeSH

Condition MeSH for Venetoclax
Intervention	Trials
Leukemia	302
Leukemia, Myeloid, Acute	286
Leukemia, Myeloid	173
Leukemia, Lymphoid	117

Clinical Trial Locations for Venetoclax

Trials by Country

Trials by Country for Venetoclax
Location	Trials
China	305
Japan	189
Australia	183
France	140
Canada	133

Trials by US State

Trials by US State for Venetoclax
Location	Trials
Texas	174
California	114
New York	110
Ohio	93
Massachusetts	87

Clinical Trial Progress for Venetoclax

Clinical Trial Phase

Clinical Trial Phase for Venetoclax
Clinical Trial Phase	Trials
PHASE4	4
PHASE3	14
PHASE2	104

Clinical Trial Status

Clinical Trial Status for Venetoclax
Clinical Trial Phase	Trials
Recruiting	344
Not yet recruiting	135
Active, not recruiting	61

Clinical Trial Sponsors for Venetoclax

Sponsor Name

Sponsor Name for Venetoclax
Sponsor	Trials
AbbVie	122
National Cancer Institute (NCI)	100
M.D. Anderson Cancer Center	71

Sponsor Type

Sponsor Type for Venetoclax
Sponsor	Trials
Other	750
Industry	391
NIH	102

Venetoclax: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Venetoclax, marketed under the trade name Venclexta®, is a targeted oral apoptosis inhibitor developed by AbbVie and Roche. Primarily approved for treatment of chronic lymphocytic leukemia (CLL) and acute myeloid leukemia (AML), Venetoclax’s mechanism centers around inhibiting B-cell lymphoma 2 (BCL-2), a protein that promotes cancer cell survival. Its innovative approach has positioned Venetoclax as a cornerstone in hematologic malignancy therapy, driving significant interest in ongoing clinical trials and market growth projections.

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Clinical Trials Landscape for Venetoclax

Current Status and Clinical Development Phases

Venetoclax remains extensively evaluated across multiple hematologic and solid tumor indications, with over 20 ongoing clinical trials registered on ClinicalTrials.gov. The breadth of its pipeline underscores promoter interest in expanding its therapeutic utility.

• Approved Indications:

  • Chronic Lymphocytic Leukemia (CLL): Approved in 2016 by FDA in combination with obinutuzumab for treatment of relapsed/refractory CLL.
  • Acute Myeloid Leukemia (AML): Accelerated approval granted in 2020 for newly diagnosed AML in adults who are age ≥75 or have comorbidities precluding intensive chemotherapy.

• Key Active Trials:

  • Combination therapies in AML: Numerous Phase III studies evaluate Venetoclax paired with hypomethylating agents (e.g., azacitidine, decitabine) to optimize response rates.
  • Non-Hematologic Malignancies: Trials are exploring its efficacy in solid tumors such as multiple myeloma, small cell lung cancer, and other lymphomas, though these are at earlier phases.
  • Resistance Mechanisms: Investigations into resistance pathways involving BCL-2 mutations are ongoing to enhance durability of response.

Promising Developments and Regulatory Milestones

In 2022, the FDA approved Venetoclax in combination with azacitidine, decitabine, or low-dose cytarabine for newly diagnosed AML in patients aged 75 and older or with comorbidities, marking a significant therapeutic advancement. Meanwhile, the European Medicines Agency (EMA) granted conditional approval in similar indications, fueling global clinical interest.

Challenges in Clinical Development

Despite its success, Venetoclax’s development faces hurdles, including:

• Tumor resistance: Mutations in BCL-2 or compensatory survival pathways limit long-term efficacy.
• Tumor lysis syndrome (TLS): A serious adverse event with rapid tumor cell death, requiring careful dosing escalation.
• Optimal combination strategies: Determining synergistic drug partnerings remains complex, with multiple ongoing trials.

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Market Analysis

Current Market Position

Venetoclax is poised as a blockbuster drug, with its initial success driven by the heavily underserved hematologic malignancies segment.

• Sales Performance:

  • In 2022, AbbVie reported $3.4 billion in global sales for Venetoclax, reflecting robust growth.
  • Growth is driven by expanded indications, increasing adoption in clinical practice, and pipeline innovations.

• Market Penetration:

  • Widely integrated into treatment protocols for CLL, especially in relapsed or refractory cases.
  • Growing use in AML treatment, particularly among elderly and unfit populations.

Competitive Landscape

Venetoclax faces competition from other BCL-2 inhibitors such as S55746 (AbbVie’s investigational agent) and emerging therapies targeting apoptosis pathways. While achieving significant market share, it contends with new molecular targets and immune modulators launched for hematologic malignancies.

Market Drivers

• Unmet medical needs: Aging populations and rising leukemia incidence drive demand.
• Regulatory approvals: Accelerated and full approvals expand market access.
• Combination therapies: Evidence supporting combination regimens enhances prescribing patterns and sale potential.

Market Challenges

• Pricing pressure and payer reimbursement hurdles influence adoption.
• Adverse event management, particularly TLS, influences therapy initiation practices.
• Patent expirations and biosimilar competition could impact long-term profitability.

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Future Market Projections

Growth Forecasts

Analysts project the global Venetoclax market will expand at a compound annual growth rate (CAGR) of approximately 8–10% from 2023 to 2030. This growth is underpinned by:

• Broadening approval scope for indications like AML, multiple myeloma, and other hematologic tumors.
• Expansion into emerging markets driven by increased healthcare infrastructure.
• Innovations in combination regimens and personalized medicine approaches.

Pipeline Impact and New Indications

The success of ongoing clinical trials targeting solid tumors, immune cell malignancies, and resistance mechanisms could significantly broaden Venetoclax’s market share. FDA and EMA approvals for new indications could potentially double the addressable market size within the next decade.

Technological and Regulatory Factors

Advancements in biomarker-driven patient stratification, coupled with regulatory support through accelerated approvals, will expedite market penetration. Additionally, collaborations between biotech firms and academic institutions will foster innovative combination strategies further shaping growth.

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Key Takeaways

• Venetoclax continues to demonstrate robust efficacy in hematologic malignancies, with ongoing trials exploring its utility across diverse cancer types.
• Regulatory milestones and expanded approvals are fueling global sales, projecting an 8–10% CAGR till 2030.
• Addressing resistance mechanisms and managing adverse events are crucial for sustaining long-term market growth.
• The integration of Venetoclax into combination therapy regimens remains a key driver of clinical and commercial success.
• Future expansion hinges on successful trial outcomes in solid tumors and other cancers, alongside strategic partnerships and regulatory support.

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FAQs

1. What are the primary approved uses of Venetoclax?
Venetoclax is approved for treating chronic lymphocytic leukemia (CLL) and acute myeloid leukemia (AML) in specific patient populations, either alone or in combination with other agents.

2. How does Venetoclax work mechanistically?
Venetoclax inhibits BCL-2, a protein that prevents apoptosis. Its blockade restores the apoptosis process in cancer cells, leading to tumor cell death.

3. What are the major safety concerns associated with Venetoclax?
Tumor lysis syndrome (TLS) is the most serious safety concern. Other adverse effects include neutropenia, infections, and gastrointestinal symptoms.

4. Are there clinical trials investigating Venetoclax in solid tumors?
Yes. Early-phase trials are exploring its efficacy in solid tumors such as small cell lung cancer and multiple myeloma, though results are preliminary.

5. What is the outlook for Venetoclax’s market expansion?
The outlook remains positive, driven by new indications, combination therapies, and ongoing research. The market is expected to grow steadily, contingent on successful trial outcomes and regulatory approvals.

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Sources

[1] ClinicalTrials.gov Data.
[2] AbbVie Annual Reports.
[3] FDA Approval Announcements.
[4] Market Research Reports, 2022.