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.

Clinical Trial Conditions for ELZONRIS

Condition Name

Condition Name for ELZONRIS
Intervention	Trials
Blastic Plasmacytoid Dendritic Cell Neoplasm	1
Mixed Phenotype Acute Leukemia	1
BPDCN	1
Myelodysplastic/Myeloproliferative Neoplasm	1

Condition MeSH

Condition MeSH for ELZONRIS
Intervention	Trials
Leukemia	4
Lymphoma	2
Neoplasms	2
Precursor Cell Lymphoblastic Leukemia-Lymphoma	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
Stemline Therapeutics, Inc.	1
Jonsson Comprehensive Cancer Center	1

Sponsor Type

Sponsor Type for ELZONRIS
Sponsor	Trials
Other	6
NIH	1

ELZONRIS (tagraxofusp): Clinical Trial Update, Market Analysis, and Projections

Last updated: February 16, 2026

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What is the current status of clinical trials for ELZONRIS?

ELZONRIS (tagraxofusp) is a CD123-targeted therapy approved in the U.S. for blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare hematologic malignancy. The drug is under investigation in multiple clinical trials to expand its indications and optimize its usage.

As of December 2022, the drug has completed several pivotal studies. The primary trial, a Phase 2 open-label study (NCT02101357), demonstrated an objective response rate (ORR) of 60% in BPDCN, leading to FDA approval in 2018. No ongoing Phase 3 trials are actively recruiting for BPDCN, but additional studies are exploring ELZONRIS in other hematologic malignancies.

Current trials include:

• NCT04544275: A Phase 1 study evaluating ELZONRIS in combination with other agents for acute myeloid leukemia (AML). Estimated primary completion date: August 2024.
• NCT04698301: A study assessing ELZONRIS in children and young adults with relapsed or refractory hematologic cancers. Expected completion: December 2023.
• NCT04509650: An exploratory trial testing ELZONRIS in myelodysplastic syndromes (MDS). Objectives focus on safety, tolerability, and preliminary efficacy.

Key Point: ELZONRIS’s pipeline remains active, with multiple early-phase studies, primarily targeting additional indications beyond BPDCN.

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What is the current market landscape for ELZONRIS?

Market authorization and sales:

• Approved by the FDA in July 2018 for BPDCN.
• Revenue growth peaked in 2020, reaching approximately $85 million.
• Estimated 2022 sales approximately $90 million, with year-over-year growth less than 2%.

Market size:

• BPDCN's prevalence in the U.S. is estimated at fewer than 500 cases annually.
• The small patient population constrains sales volume, even with high per-treatment prices.

Pricing and reimbursement:

• List price: approximately $275,000 per infusion course (generally 2-4 infusions per patient).
• Payer coverage is robust due to FDA approval and Orphan Drug designation.
• The high price limits affordability outside the U.S., especially in emerging markets.

Competitive landscape:

• No direct FDA-approved alternatives for BPDCN.
• Allogeneic stem cell transplant remains a potential curative option but limited by patient fitness.
• Off-label use of other chemotherapies is rare due to limited efficacy.

Pipeline competition:

• Ligand-based therapies and immunotherapies in early-stage trials for hematologic malignancies could become competitors if successful, but none are directly comparable at this stage.

Market challenges:

• Orphan drug status provides seven years of market exclusivity in the U.S.
• Manufacturing complexity due to biologics limits supply scalability.
• The ultra-rare indication means revenue remains capped without expanded labels.

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What are projections for ELZONRIS’s future sales and market presence?

Short-term outlook (next 2 years):

• Sales growth likely marginal due to flat BPDCN incidence, with incremental gains from increased diagnosis and physician familiarity.
• Pediatric and MDS trials engaging could lead to label expansions, opening new revenue streams.

Mid-term outlook (3-5 years):

• Possible approval in AML if ongoing trials demonstrate efficacy.
• Expansion into NHL (non-Hodgkin’s lymphoma) or other myeloid malignancies remains speculative but plausible with suitable trial results.

Long-term outlook (beyond 5 years):

• If approved for additional indications and commercialized broadly, annual revenues could reach $200 million to $300 million.
• Market growth depends on successful development, approval timelines, and competitive landscape evolution.

Factors influencing projections:

• Regulatory developments and trial outcomes are critical.
• Reimbursement policies and manufacturer capacity.
• Potential with biosimilars or alternative CD123-targeted therapies entering the market.

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Summary and Key Takeaways

• ELZONRIS is FDA-approved for BPDCN, with ongoing trials to expand indications such as AML, MDS, and pediatric hematologic malignancies.
• The drug’s market remains small, constrained by the rarity of BPDCN, limiting revenue growth despite high treatment costs.
• Future success hinges on positive trial results, potential label extensions, and competition developments.
• Current sales are around $90 million, with projections suggesting modest growth unless broader indications are approved.
• Structural challenges include manufacturing complexity and the limited patient population restricting total market size.

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FAQs

1. What other indications is ELZONRIS being tested for?
   Trials are exploring its use in AML, MDS, and pediatric hematologic cancers.

2. When might ELZONRIS be approved for additional indications?
   Approval depends on trial outcomes, expected by 2024 for AML and possibly later for other indications.

3. What challenges could limit ELZONRIS’s market expansion?
   Its focus on rare diseases, high drug cost, manufacturing complexities, and potential emergence of competitors.

4. How does the pricing influence market adoption?
   The high price limits accessibility, especially outside the U.S., and raises reimbursement barriers.

5. What is the long-term revenue potential for ELZONRIS?
   If expanded to other indications, revenues could reach hundreds of millions annually; otherwise, growth remains limited.

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Sources

1. ClinicalTrials.gov https://clinicaltrials.gov/
2. FDA Label and Approval Documents (2018)
3. IQVIA Sales Data (2022)
4. MarketWatch, 2022
5. Company Reports and Press Releases