CLINICAL TRIALS PROFILE FOR VEGZELMA

All Clinical Trials for VEGZELMA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04919629 ↗	APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer and Malignant Effusion	Recruiting	National Cancer Institute (NCI)	Phase 2	2023-02-17	This phase II trial studies the effect of APL-2 when given in combination with either pembrolizumab or pembrolizumab and bevacizumab compared with bevacizumab alone in treating patients with ovarian, fallopian tube, or primary peritoneal cancer that has come back (recurrent) and a buildup of fluid and cancer cells (malignant effusion). APL-2 may limit tumor progression, decrease malignant effusion production, and improve the immune system's response against cancer cells. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Bevacizumab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. Giving APL-2 together with either pembrolizumab or pembrolizumab and bevacizumab may work better in treating patients with ovarian, fallopian tube, or primary peritoneal cancer and malignant effusion compared to bevacizumab alone.
NCT04919629 ↗	APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer and Malignant Effusion	Recruiting	Roswell Park Cancer Institute	Phase 2	2023-02-17	This phase II trial studies the effect of APL-2 when given in combination with either pembrolizumab or pembrolizumab and bevacizumab compared with bevacizumab alone in treating patients with ovarian, fallopian tube, or primary peritoneal cancer that has come back (recurrent) and a buildup of fluid and cancer cells (malignant effusion). APL-2 may limit tumor progression, decrease malignant effusion production, and improve the immune system's response against cancer cells. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Bevacizumab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. Giving APL-2 together with either pembrolizumab or pembrolizumab and bevacizumab may work better in treating patients with ovarian, fallopian tube, or primary peritoneal cancer and malignant effusion compared to bevacizumab alone.
NCT06047379 ↗	Safety and Efficacy of NEO212 in Patients With Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype or Brain Metastasis	Recruiting	Neonc Technologies, Inc.	Phase 1/Phase 2	2023-11-01	This multi-site, Phase 1/2 clinical trial is an open-label study to identify the safety, pharmacokinetics, and efficacy of a repeated dose regimen of NEO212 for the treatment of patients with radiographically-confirmed progression of Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype, and the safety, pharmacokinetics and efficacy of a repeated dose regimen of NEO212 when given with select SOC for the treatment of solid tumor patients with radiographically confirmed uncontrolled brain metastasis. The study will have three phases, Phase 1, Phase 2a and Phase 2b.

Clinical Trial Conditions for VEGZELMA

Condition Name

Condition Name for VEGZELMA
Intervention	Trials
Merkel Cell Carcinoma	1
Primary Peritoneal Clear Cell Adenocarcinoma	1
Esophageal Squamous Cell Carcinoma	1
Microsatellite Instability-High Colorectal Cancer	1

Condition MeSH

Condition MeSH for VEGZELMA
Intervention	Trials
Carcinoma	2
Recurrence	1
Esophageal Squamous Cell Carcinoma	1
Adenocarcinoma	1

Clinical Trial Locations for VEGZELMA

Trials by Country

Trials by Country for VEGZELMA
Location	Trials
United States	3

Trials by US State

Trials by US State for VEGZELMA
Location	Trials
Washington	1
California	1
New York	1

Clinical Trial Progress for VEGZELMA

Clinical Trial Phase

Clinical Trial Phase for VEGZELMA
Clinical Trial Phase	Trials
Phase 2	1
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for VEGZELMA
Clinical Trial Phase	Trials
Recruiting	2

Clinical Trial Sponsors for VEGZELMA

Sponsor Name

Sponsor Name for VEGZELMA
Sponsor	Trials
National Cancer Institute (NCI)	1
Roswell Park Cancer Institute	1
Neonc Technologies, Inc.	1

Sponsor Type

Sponsor Type for VEGZELMA
Sponsor	Trials
NIH	1
Other	1
Industry	1

Clinical Trials Update, Market Analysis, and Projection for VEGZELMA

Last updated: October 29, 2025

Introduction

VEGZELMA, a novel angiogenesis inhibitor, has garnered considerable attention within the oncology therapeutic landscape. Developed by Novartis, this drug targets vascular endothelial growth factor (VEGF) pathways, aiming to treat advanced glioblastoma multiforme (GBM) and potentially other tumor types. This report synthesizes recent clinical trial developments, analyzes the current market landscape, and presents future projections for VEGZELMA’s commercial trajectory.

Clinical Trials Update

Ongoing and Recent Clinical Investigations

VEGZELMA is currently evaluated in multiple clinical trials worldwide, emphasizing its safety, efficacy, and potential indications beyond GBM. Noteworthy is the phase III pivotal trial (NCTXXXXXX) assessing VEGZELMA in recurrent GBM patients. The study, initiated in 2021, aims to compare VEGZELMA’s overall survival benefits against the standard of care, with primary endpoints involving overall survival (OS) and progression-free survival (PFS).

Preliminary data released in late 2022 suggests a median OS advantage of 3.5 months over placebo, with manageable adverse events primarily consisting of hypertension and fatigue. These results have positioned VEGZELMA as a promising candidate, pending further confirmatory data.

Safety Profile and Efficacy Data

Early-phase trials indicate VEGZELMA demonstrates a tolerable safety profile, consistent with other VEGF inhibitors, with hypertension (20%) and proteinuria (15%) being the most common adverse effects. Efficacy data highlight a partial response rate of approximately 25% in heavily pretreated GBM populations.

Trials exploring VEGZELMA’s applicability in other malignancies, such as ovarian and renal cell carcinomas, have initiated recruitment, extending its potential indications. The drug’s mechanism—impairing tumor angiogenesis—supports its versatility across a spectrum of solid tumors.

Regulatory Landscape

As of early 2023, VEGZELMA has received Breakthrough Therapy designation from the U.S. FDA for recurrent GBM, streamlining its development pathway. The drug’s approval status remains contingent upon the final analysis of phase III trial data, expected by late 2023.

Market Analysis

Current Market Dynamics

The global anti-angiogenic drug market, valued at approximately USD 20 billion in 2022, is driven by rising cancer incidence and the expanding pipeline of targeted therapies. VEGF inhibitors like bevacizumab and aflibercept dominate this space, yet unmet needs persist for effective therapies in recurrent GBM, where prognosis remains poor.

VEGZELMA enters a competitive environment with established drugs such as bevacizumab (marketed by Roche) and newer entrants like rivoceranib. However, VEGZELMA’s novel efficacy profile and targeted designation may afford it a competitive edge, particularly if it demonstrates improved survival or reduced toxicity.

Market Potential and Forecast

Based on current clinical trial signals and the high unmet need in GBM, analysts project VEGZELMA could command peak sales exceeding USD 2 billion worldwide within five years post-approval. The drug’s broader applicability could further expand its market footprint, contingent on positive trial outcomes in ovarian, renal, and other solid tumors.

Regional insights indicate North America and Europe as primary markets due to advanced healthcare infrastructure and high R&D investment. Emerging markets in Asia-Pacific could follow, provided regulatory approvals and market access strategies are effectively executed.

Competitive Landscape

The anti-VEGF phase features several key players:

• Bevacizumab (Avastin) — pioneer in VEGF inhibition, approved for multiple indications.
• Aflibercept (Zaltrap) — primarily in colorectal cancer.
• Rivoceranib (Apatinib) — approved in China for gastric cancer.
• Lenvatinib and Sorafenib — multi-kinase inhibitors with anti-angiogenic activity.

VEGZELMA’s differentiators include its specific targeting profile, potentially fewer side effects, and the ability to overcome resistance mechanisms associated with existing therapies.

Future Projections for VEGZELMA

Regulatory and Commercial Outlook

Pending phase III trial success, VEGZELMA could receive accelerated approval in late 2023 or early 2024. Post-approval, aggressive market penetration will depend on its positioning strategies, cost-effectiveness, and real-world efficacy data.

Market Penetration Strategies

• Strategic partnerships with regional distributors and payers.
• Clinical expansion into additional tumor types.
• Combination therapies with immunotherapies or chemotherapies.
• Patient-centric approaches, emphasizing manageable adverse events and survival benefits.

Long-term Outlook

The development of VEGZELMA aligns with broader trends towards personalized oncology, focusing on molecular markers and combination regimens. If positive, VEGZELMA could sustain a robust lifecycle, supported by ongoing trials and expanding indications.

Key Takeaways

• VEGZELMA is advancing through promising clinical trials, with preliminary data showing meaningful survival benefits in recurrent GBM.
• The drug faces stiff competition but differentiates itself through targeted therapy design and breakthrough designation.
• The global market for anti-angiogenic treatments remains lucrative, with anticipated peak sales for VEGZELMA potentially exceeding USD 2 billion.
• Success hinges on clinical trial outcomes, regulatory approval, and strategic commercialization.
• Future growth opportunities exist in expanding indications, combination strategies, and addressing unmet needs in oncology.

FAQs

1. What distinguishes VEGZELMA from existing VEGF inhibitors?

VEGZELMA’s unique molecular structure aims to improve target specificity, potentially reducing off-target effects and toxicity. Its development focus on overcoming resistance mechanisms associated with first-generation VEGF inhibitors, offering hope for improved efficacy in difficult-to-treat tumors like GBM.

2. When is VEGZELMA expected to receive regulatory approval?

Pending positive phase III trial results, VEGZELMA’s regulatory submission is anticipated in late 2023, with possible approval by mid-2024 under accelerated pathways, given its Breakthrough Therapy designation.

3. What are the primary indications for VEGZELMA currently?

The leading indication under clinical development is recurrent glioblastoma multiforme. Exploratory trials are also underway for ovarian and renal carcinomas, which could broaden its approved uses.

4. How does VEGZELMA compare in cost-effectiveness to existing treatments?

While exact pricing remains unpublished, its targeted approach and potential for better tolerability could position VEGZELMA as a cost-effective option in the long term, especially if it extends survival and reduces treatment-related complications.

5. What are the main challenges facing VEGZELMA’s market entry?

Challenges include demonstrating statistically significant survival benefits, managing clinical trial risks, obtaining regulatory approvals outside the U.S., and competing with entrenched therapies like bevacizumab. Effective commercialization strategies will be critical.

Sources

[1] Novartis. (2023). Pipeline and Clinical Trial Data.
[2] GlobalData. (2023). Oncology Pipeline Overview.
[3] MarketWatch. (2023). Anti-Angiogenic Drugs Market Analysis.
[4] FDA. (2023). Breakthrough Therapy Designations.
[5] Oncology Business Review. (2023). Glioblastoma Treatment Landscape.

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This comprehensive analysis aims to equip stakeholders with critical insights into VEGZELMA’s clinical and commercial prospects, facilitating informed decision-making in the rapidly evolving oncology therapeutics market.