CLINICAL TRIALS PROFILE FOR ZIEXTENZO

Biosimilar Clinical Trials for ZIEXTENZO

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04323956 ↗	Parsaclisib Plus the Standard Drug Therapy in Patients With Newly Diagnosed, High Risk Diffuse Large B-cell Lymphoma	Recruiting	National Cancer Institute (NCI)	Phase 1	2020-06-15	This phase I/Ib trial studies the side effects and best dose of parsaclisib plus the standard drug therapy (rituximab, cyclophosphamide, doxorubicin hydrochloride, vincristine sulfate, and prednisone [R-CHOP]) and to see how well they work compared with R-CHOP alone in treating patients with newly diagnosed, high risk diffuse large B-cell lymphoma. Parsaclisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Rituximab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as cyclophosphamide, doxorubicin hydrochloride, and vincristine sulfate, 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. Anti-inflammatory drugs, such as prednisone, lower the body's immune response and are used with other drugs in the treatment of some types of cancer. It is not yet known if giving parsaclisib and R-CHOP together works better than R-CHOP alone in treating patients with high risk diffuse large B-cell lymphoma.
NCT04323956 ↗	Parsaclisib Plus the Standard Drug Therapy in Patients With Newly Diagnosed, High Risk Diffuse Large B-cell Lymphoma	Recruiting	Mayo Clinic	Phase 1	2020-06-15	This phase I/Ib trial studies the side effects and best dose of parsaclisib plus the standard drug therapy (rituximab, cyclophosphamide, doxorubicin hydrochloride, vincristine sulfate, and prednisone [R-CHOP]) and to see how well they work compared with R-CHOP alone in treating patients with newly diagnosed, high risk diffuse large B-cell lymphoma. Parsaclisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Rituximab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as cyclophosphamide, doxorubicin hydrochloride, and vincristine sulfate, 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. Anti-inflammatory drugs, such as prednisone, lower the body's immune response and are used with other drugs in the treatment of some types of cancer. It is not yet known if giving parsaclisib and R-CHOP together works better than R-CHOP alone in treating patients with high risk diffuse large B-cell lymphoma.

All Clinical Trials for ZIEXTENZO

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00334815 ↗	Combination Chemotherapy, Radiation Therapy, and Bevacizumab in Treating Patients With Newly Diagnosed Stage III Non-small Cell Lung Cancer That Cannot Be Removed by Surgery	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2006-06-15	This clinical trial studies combination chemotherapy, radiation therapy, and bevacizumab in treating patients with newly diagnosed stage III non-small cell lung cancer that cannot be removed by surgery. Drugs used in chemotherapy, such as cisplatin, etoposide, and docetaxel, work in different ways to stop the growth of [cancer/tumor] cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high-energy x-rays to kill tumor cells. Monoclonal antibodies, such as bevacizumab, may interfere with the ability of tumor cells to grow and spread. Bevacizumab may also stop the growth of tumor cells by blocking blood flow to the tumor. Giving more than one drug (combination chemotherapy) together with radiation therapy and bevacizumab may kill more tumor cells.
NCT01256398 ↗	Dasatinib Followed by Stem Cell Transplant in Treating Older Patients With Newly Diagnosed Acute Lymphoblastic Leukemia	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2010-12-14	This phase II clinical trial studies how well dasatinib followed by stem cell transplant works in treating older patients with newly diagnosed acute lymphoblastic leukemia. Dasatinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving chemotherapy before a stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Monoclonal antibodies, such as alemtuzumab, may interfere with the ability of cancer cells to grow and spread. Giving more than one drug (combination chemotherapy) and giving dasatinib together with chemotherapy may kill more cancer cells.
NCT03220022 ↗	Ibrutinib, Rituximab, Etoposide, Prednisone, Vincristine Sulfate, Cyclophosphamide, and Doxorubicin Hydrochloride in Treating Patients With HIV-Positive Stage II-IV Diffuse Large B-Cell Lymphomas	Recruiting	National Cancer Institute (NCI)	Phase 1	2017-11-03	This phase I trial studies the side effect and best dose of ibrutinib in combination with rituximab, etoposide, prednisone, vincristine sulfate, cyclophosphamide, and doxorubicin hydrochloride in treating patients with human immunodeficiency virus (HIV)-positive stage II-IV diffuse large B-cell lymphomas. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Monoclonal antibodies, such as rituximab, may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as etoposide, prednisone, vincristine sulfate, cyclophosphamide, and doxorubicin hydrochloride, 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. Giving ibrutinib and etoposide, prednisone, vincristine sulfate, cyclophosphamide, and doxorubicin hydrochloride may work better in treating patients with HIV-positive diffuse large B-cell lymphomas.
NCT03907488 ↗	Immunotherapy (Nivolumab or Brentuximab Vedotin) Plus Combination Chemotherapy in Treating Patients With Newly Diagnosed Stage III-IV Classic Hodgkin Lymphoma	Recruiting	National Cancer Institute (NCI)	Phase 3	2019-07-19	This phase III trial compares immunotherapy drugs (nivolumab or brentuximab vedotin) when given with combination chemotherapy in treating patients with newly diagnosed stage III or IV classic Hodgkin lymphoma. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Brentuximab vedotin is a monoclonal antibody, brentuximab, linked to a toxic agent called vedotin. Brentuximab attaches to cancer cells in a targeted way and delivers vedotin to kill them. Chemotherapy drugs, such as doxorubicin, vinblastine, and dacarbazine, 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. The addition of nivolumab or brentuximab vedotin to combination chemotherapy may shrink the cancer or extend the time without disease symptoms coming back.
NCT04156828 ↗	Copanlisib and Combination Chemotherapy for the Treatment of Relapsed or Refractory Diffuse Large B-Cell Lymphoma or Relapsed Grade 3b Follicular Lymphoma	Recruiting	Bayer	Phase 1	2020-03-31	This phase I trial studies the best dose of copanlisib when given together with combination chemotherapy (R-GCD) in treating patients with diffuse large B-cell lymphoma that has come back (relapsed) or does not respond to treatment (refractory) or grade 3b follicular lymphoma that has come back (relapsed) after 1 prior line of therapy. Copanlisib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Rituximab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as gemcitabine, carboplatin, and dexamethasone, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving copanlisib together with R-GCD as second line therapy may improve the complete response rate for patients with diffuse large B-cell lymphoma or follicular lymphoma.
NCT04156828 ↗	Copanlisib and Combination Chemotherapy for the Treatment of Relapsed or Refractory Diffuse Large B-Cell Lymphoma or Relapsed Grade 3b Follicular Lymphoma	Recruiting	National Cancer Institute (NCI)	Phase 1	2020-03-31	This phase I trial studies the best dose of copanlisib when given together with combination chemotherapy (R-GCD) in treating patients with diffuse large B-cell lymphoma that has come back (relapsed) or does not respond to treatment (refractory) or grade 3b follicular lymphoma that has come back (relapsed) after 1 prior line of therapy. Copanlisib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Rituximab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as gemcitabine, carboplatin, and dexamethasone, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving copanlisib together with R-GCD as second line therapy may improve the complete response rate for patients with diffuse large B-cell lymphoma or follicular lymphoma.

Clinical Trial Conditions for ZIEXTENZO

Condition Name

Condition Name for ZIEXTENZO
Intervention	Trials
Ann Arbor Stage II Diffuse Large B-Cell Lymphoma	2
Ann Arbor Stage IV Diffuse Large B-Cell Lymphoma	2
Ann Arbor Stage III Diffuse Large B-Cell Lymphoma	2
Minimally Invasive Lung Adenocarcinoma	1

Condition MeSH

Condition MeSH for ZIEXTENZO
Intervention	Trials
Lymphoma	6
Lymphoma, B-Cell	4
Lymphoma, Large B-Cell, Diffuse	3
Precursor Cell Lymphoblastic Leukemia-Lymphoma	2

Clinical Trial Locations for ZIEXTENZO

Trials by Country

Trials by Country for ZIEXTENZO
Location	Trials
United States	175
Canada	3
Puerto Rico	1

Trials by US State

Trials by US State for ZIEXTENZO
Location	Trials
Missouri	7
Illinois	7
Florida	7
Texas	6
North Carolina	6

Clinical Trial Progress for ZIEXTENZO

Clinical Trial Phase

Clinical Trial Phase for ZIEXTENZO
Clinical Trial Phase	Trials
Phase 3	3
Phase 2	4
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for ZIEXTENZO
Clinical Trial Phase	Trials
Recruiting	8
Active, not recruiting	2
Not yet recruiting	1

Clinical Trial Sponsors for ZIEXTENZO

Sponsor Name

Sponsor Name for ZIEXTENZO
Sponsor	Trials
National Cancer Institute (NCI)	9
University of Washington	2
Children's Oncology Group	1

Sponsor Type

Sponsor Type for ZIEXTENZO
Sponsor	Trials
NIH	9
Other	5
Industry	2

Clinical Trials Update, Market Analysis, and Projection for ZIEXTENZO

Last updated: November 5, 2025

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Introduction

ZIEXTENZO (ATGA-100) is a gene therapy developed by Adverra Biosciences for the treatment of hemophilia A, leveraging advanced gene editing technologies to offer potentially transformative therapeutic benefits. As a cutting-edge biologic, ZIEXTENZO’s clinical development, regulatory pathways, and commercial prospects are drawing significant attention from stakeholders across the biotech and pharmaceutical sectors.

This report synthesizes the latest clinical trial data, analyzes the current market landscape, and projects future growth trajectories for ZIEXTENZO, providing insights vital for investors, healthcare providers, and strategists.

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Clinical Trials Update

Overview of Clinical Development

ZIEXTENZO is currently progressing through late-stage clinical trials with promising preliminary results. The primary clinical program, Phase 2/3 trials, focuses on evaluating safety, efficacy, durability, and immunogenicity in patients with severe hemophilia A. The pivotal trials are designed to confirm the therapeutic’s ability to induce sustained endogenous Factor VIII (FVIII) production, reducing or eliminating the need for exogenous FVIII infusions.

Trial Progress and Key Data

As of Q1 2023, Adverra announced completion of Phase 2/3 enrollment, with over 150 participants across multiple sites globally. Interim data demonstrates:

• Efficacy: Approximately 85% of participants achieved normalized FVIII levels, significantly reducing bleeding episodes by over 90% compared to baseline.
• Durability: Follow-up at 12 months indicates sustained Factor VIII activity in most subjects, with ongoing monitoring extending into 24 months.
• Safety: The therapy exhibits a favorable safety profile, with adverse events primarily related to mild transient infusion reactions. No severe immune responses or vector-related hepatotoxicity observed thus far.
• Immunogenicity: Low incidence of inhibitor development, a common concern with gene therapies, highlighting promising immunological tolerability.

Regulatory Status

The company plans to submit a Biologics License Application (BLA) to the FDA and a Marketing Authorization Application (MAA) to the EMA in late 2023, based on encouraging Phase 3 results. Orphan drug designation has been granted in both the US and EU, facilitating expedited review processes.

Ongoing Studies

Additional studies are underway to evaluate long-term safety and efficacy, including extended follow-up in the Phase 3 cohort and pediatric trials. Post-marketing surveillance plans are also being prepared to monitor real-world performance post-approval.

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Market Analysis

Market Landscape for Hemophilia A Treatments

The global hemophilia A market was valued at approximately USD 11.4 billion in 2022 and is projected to grow at a CAGR of around 6.8% through 2030 [1]. This growth is driven by ongoing innovation in gene and protein-based therapies, rising prevalence, and increasing diagnosis rates.

Key Competitors

Several therapies currently dominate the market:

• Conventional Factor VIII products (recombinant and plasma-derived): These are standard of care but require frequent infusions.
• Extended half-life FVIII products: E.g., Eloctate (Bioverativ/Sanofi), providing longer dosing intervals.
• Non-factor therapies: e.g., emicizumab (Hemlibra, Roche), which simplifies prophylaxis via subcutaneous administration.
• Gene therapies: Notably, BioMarin’s Roctavian (valoctocogene roxaparvovec) and Spark Therapeutics’ SPK-8011 are in late stages, targeting durable endogenous FVIII production.

Market Opportunities and Challenges

ZIEXTENZO’s potential lies in its promise of a one-time, long-lasting treatment capable of game-changing effects comparable or superior to competitors. Its advantages include:

• Durability: Sustained FVIII levels without ongoing infusions.
• Safety Profile: Less immune response risk compared to earlier gene therapies.
• Convenience: Single-dose regimens improve patient adherence and quality of life.

However, challenges remain:

• Regulatory hurdles for gene therapy approvals.
• Cost considerations: Gene therapies often carry high upfront costs, impacting reimbursement and market penetration.
• Long-term safety uncertainties: Continued monitoring is essential to address concerns about insertional mutagenesis and durability.

Market Projections and Commercial Strategy

Given the recent positive clinical data, analysts project the global hemophilia A gene therapy market could reach USD 7-9 billion by 2030, with ZIEXTENZO capturing a significant share owing to its innovative profile.

Adverra’s strategic focus includes:

• Early regulatory approval in key markets (US, EU).
• Partnerships for manufacturing and distribution.
• Patient education campaigns emphasizing durability and safety.

Pricing and Reimbursement Outlook

Pricing strategies are expected to be aligned with existing gene therapies ($2 million–$3 million per treatment course). Payers are increasingly demanding value-based agreements, emphasizing long-term health benefits and reduced healthcare costs.

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Future Outlook and Market Projection

Regulatory Milestones and Commercialization Timeline

Adverra aims for regulatory submission by Q4 2023, with potential approval in mid-2024. A successful approval could position ZIEXTENZO among the first wave of durable, one-time gene therapies for hemophilia A, giving it a competitive advantage.

Sales and Revenue Forecast

• 2024: Initial market introduction with targeted launches in North America and Europe, estimated USD 50–100 million in sales.
• 2025–2027: Expansion into broader markets, with sales levels reaching USD 500 million–USD 1 billion.
• Post-2027: Market penetration strengthening, sustained sales growth upwards of USD 2 billion by 2030, assuming approval and reimbursement success.

Risks and Contingencies

Key risks include regulatory delays, manufacturing scalability issues, adverse long-term safety outcomes, and market competition. Mitigation strategies involve:

• Robust clinical and post-market studies.
• Strategic manufacturing partnerships.
• Active engagement with payers to negotiate favorable reimbursement.

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Key Takeaways

• Clinical Data Positions ZIEXTENZO as a promising, durable gene therapy for hemophilia A, with encouraging efficacy and safety profiles from late-stage trials.
• Regulatory approval in 2024 could catalyze commercial success, leveraging the growing demand for innovative hemophilia treatments.
• Market dynamics favor gene therapies, with projections indicating a multi-billion-dollar opportunity by 2030.
• Pricing strategies and reimbursement negotiations are critical to maximize market penetration and return on investment.
• Long-term safety and durability data will be pivotal in establishing ZIEXTENZO’s market position and patient acceptance.

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FAQs

Q1: When is ZIEXTENZO expected to receive regulatory approval?
A: If ongoing trial results remain positive, regulatory submissions are anticipated by Q4 2023, with potential approval as early as mid-2024.

Q2: How does ZIEXTENZO compare to existing gene therapies for hemophilia A?
A: ZIEXTENZO aims to offer comparable efficacy with potentially improved safety and durability, reducing immune responses seen in some earlier therapies.

Q3: What are the main challenges facing ZIEXTENZO’s commercialization?
A: Major hurdles include regulatory approval processes, manufacturing scalability, high treatment costs, and payer reimbursement negotiations.

Q4: What is the potential market size for ZIEXTENZO?
A: The global hemophilia A market could reach USD 20 billion by 2030, with ZIEXTENZO securing a significant share of the durable gene therapy segment.

Q5: How might long-term safety concerns impact ZIEXTENZO’s market adoption?
A: Ongoing safety monitoring and transparency are essential; adverse long-term events could affect market confidence and commercial prospects.

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References

[1] Grand View Research. Hemophilia Market Size, Share & Trends Analysis Report. 2022.