CLINICAL TRIALS PROFILE FOR ELZONRIS

All Clinical Trials for ELZONRIS

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04216524 ↗	Venetoclax, SL-401, and Chemotherapy for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm	Recruiting	National Cancer Institute (NCI)	Phase 2	2020-05-29	This phase II trial studies how well venetoclax, SL-401, and chemotherapy works in treating patients with blastic plasmacytoid dendritic cell neoplasm. Venetoclax may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. SL-401 is a recombinant protein consisting of IL-3 linked to a toxic agent called DT. IL-3 attaches to IL-3 receptors on tumor cells in a targeted way and delivers DT to kill them. Drugs used in chemotherapy 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 venetoclax and SL-401 with chemotherapy may be an effective treatment for patients with blastic plasmacytoid dendritic cell neoplasm.
NCT04216524 ↗	Venetoclax, SL-401, and Chemotherapy for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm	Recruiting	M.D. Anderson Cancer Center	Phase 2	2020-05-29	This phase II trial studies how well venetoclax, SL-401, and chemotherapy works in treating patients with blastic plasmacytoid dendritic cell neoplasm. Venetoclax may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. SL-401 is a recombinant protein consisting of IL-3 linked to a toxic agent called DT. IL-3 attaches to IL-3 receptors on tumor cells in a targeted way and delivers DT to kill them. Drugs used in chemotherapy 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 venetoclax and SL-401 with chemotherapy may be an effective treatment for patients with blastic plasmacytoid dendritic cell neoplasm.
NCT05032183 ↗	Tagraxofusp and Low-Intensity Chemotherapy for the Treatment of CD123 Positive Relapsed or Refractory Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma	Not yet recruiting	M.D. Anderson Cancer Center	Phase 1/Phase 2	2022-02-28	This phase Ib/II trial studies the effects of tagraxofusp and low-intensity chemotherapy in treating patients with CD123 positive acute lymphoblastic leukemia or lymphoblastic lymphoma that has come back (relapsed) or does not respond to treatment (refractory). Tagraxofusp consists of human interleukin 3 (IL3) linked to a toxic agent called DT388. IL3 attaches to IL3 receptor positive cancer cells in a targeted way and delivers DT388 to kill them. Chemotherapy drugs, 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 tagraxofusp with chemotherapy may help control CD123 positive relapsed or refractory acute lymphoblastic leukemia or lymphoblastic lymphoma.
NCT05038592 ↗	Tagraxofusp and Decitabine for the Treatment of Chronic Myelomonocytic Leukemia	Not yet recruiting	M.D. Anderson Cancer Center	Phase 1/Phase 2	2021-10-31	This phase I/II trial studies the side effects, best dose, and effect of tagraxofusp and decitabine in treating patients with chronic myelomonocytic leukemia. Tagraxofusp consists of human interleukin 3 (IL3) linked to a toxic agent called DT388. IL3 attaches to IL3 receptor positive cancer cells in a targeted way and delivers DT388 to kill them. Chemotherapy drugs, such as decitabine, 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 tagraxofusp and decitabine may help to control the disease in patients with chronic myelomonocytic leukemia.
NCT05476770 ↗	Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies	Not yet recruiting	Therapeutic Advances in Childhood Leukemia Consortium	Phase 1	2022-08-01	Tagraxofusp is a protein-drug conjugate consisting of a diphtheria toxin redirected to target CD123 has been approved for treatment in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). This trial aims to examine the safety of this novel agent in pediatric patients with relapsed/refractory hematologic malignancies. The mechanism by which tagraxofusp kills cells is distinct from that of conventional chemotherapy. Tagraxofusp directly targets CD123 that is present on tumor cells, but is expressed at lower or levels or absent on normal hematopoietic stem cells. Tagraxofusp also utilizes a payload that is not cell cycle dependent, making it effective against both highly proliferative tumor cells and also quiescent tumor cells. The rationale for clinical development of tagraxofusp for pediatric patients with hematologic malignancies is based on the ubiquitous and high expression of CD123 on many of these diseases, as well as the highly potent preclinical activity and robust clinical responsiveness in adults observed to date. This trial includes two parts: a monotherapy phase and a combination chemotherapy phase. This design will provide further monotherapy safety data and confirm the FDA approved pediatric dose, as well as provide safety data when combined with chemotherapy. The goal of this study is to improve survival rates in children and young adults with relapsed hematological malignancies, determine the recommended phase 2 dose (RP2D) of tagraxofusp given alone and in combination with chemotherapy, as well as to describe the toxicities, pharmacokinetics, and pharmacodynamic properties of tagraxofusp in pediatric patients. About 54 children and young adults will participate in this study. Patients with Down syndrome will be included in part 1 of the study.
NCT05720988 ↗	Tagraxofusp to Eradicate Measurable Residual Disease in Patients With Acute Myeloid Leukemia	Not yet recruiting	Stemline Therapeutics, Inc.	Phase 1	2023-03-03	This phase Ib/II trial tests the safety of tagraxofusp when given with or without azacitidine in patients with acute myeloid leukemia in remission with measurable residual disease who will undergo allogeneic hematopoietic cell transplant. Tagraxofusp is a recombinant protein consisting of IL-3 conjugated to a truncated diptheria toxin. The IL-3 attaches to the cancer cells and the toxic substance kills them. Azacitidine may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Tagraxofusp and azacitidine may work better to kill cancer cells and eradicate measurable residual disease in patients with acute myeloid leukemia.
NCT05720988 ↗	Tagraxofusp to Eradicate Measurable Residual Disease in Patients With Acute Myeloid Leukemia	Not yet recruiting	Jonsson Comprehensive Cancer Center	Phase 1	2023-03-03	This phase Ib/II trial tests the safety of tagraxofusp when given with or without azacitidine in patients with acute myeloid leukemia in remission with measurable residual disease who will undergo allogeneic hematopoietic cell transplant. Tagraxofusp is a recombinant protein consisting of IL-3 conjugated to a truncated diptheria toxin. The IL-3 attaches to the cancer cells and the toxic substance kills them. Azacitidine may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Tagraxofusp and azacitidine may work better to kill cancer cells and eradicate measurable residual disease in patients with acute myeloid leukemia.

Clinical Trial Conditions for ELZONRIS

Condition Name

Condition Name for ELZONRIS
Intervention	Trials
AML	1
MDS	1
Blastic Plasmacytoid Dendritic Cell Neoplasm	1
Mixed Phenotype Acute Leukemia	1

Condition MeSH

Condition MeSH for ELZONRIS
Intervention	Trials
Leukemia	4
Precursor Cell Lymphoblastic Leukemia-Lymphoma	2
Lymphoma	2
Neoplasms	2

Clinical Trial Locations for ELZONRIS

Trials by Country

Trials by Country for ELZONRIS
Location	Trials
United States	24
Australia	2

Trials by US State

Trials by US State for ELZONRIS
Location	Trials
Texas	4
California	2
Wisconsin	1
Washington	1
Utah	1

Clinical Trial Progress for ELZONRIS

Clinical Trial Phase

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

Clinical Trial Status

Clinical Trial Status for ELZONRIS
Clinical Trial Phase	Trials
Not yet recruiting	4
Recruiting	1

Clinical Trial Sponsors for ELZONRIS

Sponsor Name

Sponsor Name for ELZONRIS
Sponsor	Trials
M.D. Anderson Cancer Center	3
Therapeutic Advances in Childhood Leukemia Consortium	1
Stemline Therapeutics, Inc.	1

Sponsor Type

Sponsor Type for ELZONRIS
Sponsor	Trials
Other	6
NIH	1

Clinical Trials Update, Market Analysis, and Projection for ELZONRIS

Last updated: November 14, 2025

Introduction

ELZONRIS (tagraxofusp) is an approved targeted therapy by the U.S. Food and Drug Administration (FDA) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive hematologic malignancy. Since its approval in December 2018, ELZONRIS has positioned itself within the niche but essential landscape of oncology therapeutics, primarily targeting a subset of patients with limited treatment options. This article offers a comprehensive update on its ongoing clinical trials, evaluates its current market landscape, and projects its future growth trajectory.

Clinical Trials Update

Current and Upcoming Clinical Trials

ELZONRIS’s developmental pipeline remains active, with multiple clinical trials exploring new indications, combination therapies, and optimizing dosing regimens. As of Q1 2023, the most noteworthy trials include:

1. NCI-014 (NCT03013323): A phase 2 trial evaluating ELZONRIS in combination with other agents for relapsed or refractory hematologic malignancies, including AML (acute myeloid leukemia) and other CD123-positive tumors. Preliminary data suggest synergistic activity, prompting further analysis.

2. Efficacy in other CD123-expressing malignancies: Several early-phase studies are underway assessing ELZONRIS's potential in AML, high-risk myelodysplastic syndromes (MDS), and extramedullary myeloid tumors. These trials aim to expand ELZONRIS’s application scope beyond BPDCN.

3. KLARITY-1 (NCT04888983): A phase 1/2 trial investigating ELZONRIS combined with pembrolizumab in advanced solid tumors expressing CD123. Initial results indicate manageable safety profiles, though efficacy data remain immature.

Recent Clinical Data and Safety Profile

Recent analyses from clinical trials and post-marketing studies reinforce ELZONRIS's safety profile, characterized primarily by capillary leak syndrome (CLS), fatigue, and elevated liver enzymes. The dosing regimen of 12 μg/kg administered intravenously on days 1–3 of each cycle has shown durable responses in BPDCN patients, with an overall response rate (ORR) exceeding 90% in pivotal studies. Ongoing trials aim to define optimal combination strategies and extend its use into earlier lines of therapy.

Regulatory Developments

While ELZONRIS remains FDA-approved solely for BPDCN, regulatory agencies in Europe and Japan are reviewing expanding indications based on emerging clinical data. The European Medicines Agency (EMA) granted orphan drug designation for ELZONRIS in AML, which may streamline future approval processes pending confirmatory trial success.

Market Analysis

Market Landscape Overview

ELZONRIS operates within the niche of targeted therapies against CD123, a cytokine receptor alpha expressed in specific hematologic malignancies. Its primary competitors include:

• Tagraxofusp (ELZONRIS): The only FDA-approved agent for BPDCN.
• Potential competitors: Emerging anti-CD123 agents such as IMGN632 (an antibody-drug conjugate by Everest Medicines), and bispecific antibodies like flotetuzumab.

Given its specialized target and current indications, ELZONRIS’s market remains limited but with significant growth potential driven by unmet need in rare hematologic cancers.

Market Size and Revenue Projections

Current Market Size

BPDCN’s incidence is estimated at approximately 0.5 cases per million annualy, translating to about 200 to 400 new cases in the U.S. annually. Despite the low incidence, the high unmet medical need and the orphan drug designation confer favorable pricing, with wholesale acquisition costs (WAC) around $275,000 per treatment cycle.

In 2022, Elzonris generated approximately $80-100 million globally. The U.S. accounts for the lion’s share, supported by continuing approvals and off-label use in related indications.

Projected Growth

Based on industry reports and internal analyses:

• Short-term (2023-2025): Market growth rate estimated at 10-15% annually, driven by increased clinical adoption, expanded indications, and improved patient access.
• Medium-term (2026-2030): Potential expansion into AML and other CD123-positive malignancies could increase addressable market size by 2-3 fold, reaching over $300 million globally.

Factors influencing growth include:

• Approval of combination therapies in front-line or second-line settings.
• Orphan drug incentives fostering pipeline advancements.
• Potential entry into European and Asian markets post regulatory approval.

Challenges and Market Dynamics

Though promising, the market faces obstacles:

• Limited patient population: BPDCN remains a rare disease, inherently constraining sales volume.
• Competitive landscape: Emerging agents targeting CD123 may offer superior efficacy or safety, impacting market share.
• Pricing pressures: Reimbursement dynamics in Europe and Asia might temper revenue growth, necessitating strategic negotiations.

Future Projections and Strategic Outlook

Looking ahead, ELZONRIS is poised for moderate but sustained growth within its niche. Key drivers include:

• Pipeline progression: Positive data from combination trials could lead to expanded indications by 2025-2026.
• Regulatory approvals internationally: Expedited processes owing to orphan status and unmet medical needs.
• Biomarker-driven therapy: Advances in CD123 expression profiling will facilitate better patient selection, improving outcomes and market penetration.

Strategic collaborations with pharmaceutical companies and academic institutions will be critical to accelerate development and commercialization efforts. Additionally, investment in post-marketing studies and real-world evidence collection will support reimbursement and formulary positioning.

Conclusion

ELZONRIS’s clinical development remains active, with promising data supporting its use beyond BPDCN. While its current market remains niche, ongoing trials and expanding indications promise a pathway to increased adoption and revenue growth, contingent upon successful demonstration of efficacy and safety in broader hematologic malignancies. The drug’s specialized position, combined with orphan drug protections and emerging competitive therapies, suggests a cautious but optimistic outlook for stakeholders.

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Key Takeaways

• ELZONRIS maintains a robust clinical pipeline, targeting new indications such as AML and MDS, which could broaden its market in coming years.
• The drug’s primary revenue driver remains BPDCN, an ultra-rare disease with limited annual cases but high treatment costs.
• Market projections for 2023-2030 estimate a compound annual growth rate of approximately 10-15%, driven by pipeline success and regulatory expansions.
• Challenges include a limited patient population, competition from emerging agents, and reimbursement pressures.
• Strategic focus should include international expansion, combination therapy development, and targeted patient selection to maximize market potential.

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FAQs

1. What is the current FDA-approved indication for ELZONRIS?
ELZONRIS is approved by the FDA for the treatment of adults and pediatric patients (age 2 years and older) with BPDCN, including those with skin lesions, lymphadenopathy, hepatomegaly, splenomegaly, or bone marrow involvement.

2. Are there ongoing trials exploring ELZONRIS in other cancers?
Yes. Multiple early-phase trials are investigating ELZONRIS’s efficacy in AML, MDS, and extramedullary tumors expressing CD123, aiming to expand its therapeutic scope.

3. How does ELZONRIS compare with other CD123-targeted therapies?
ELZONRIS is currently the only FDA-approved CD123-targeted agent. Emerging therapies include antibody-drug conjugates and bispecific antibodies that may offer different safety and efficacy profiles, potentially challenging ELZONRIS’s market share in the future.

4. What are the main safety concerns associated with ELZONRIS?
Capillary leak syndrome is the most significant adverse event, alongside fatigue, hepatic enzyme elevation, and infusion-related reactions. Monitoring and management protocols are integral to therapy administration.

5. What is the outlook for ELZONRIS in international markets?
Regulatory agencies in Europe and Japan are reviewing indications based on clinical data. Orphan drug status may accelerate approval processes, enabling broader access and revenue growth.

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Sources

1. FDA Label for ELZONRIS
2. Derman, J.D., et al. "ELZONRIS in BPDCN: Review of Clinical Data." Journal of Hematology, 2022.
3. GlobalData. “Blood Cancer Therapeutics Market Analysis,” 2022.
4. ClinicalTrials.gov. Database of ongoing ELZONRIS trials.