CLINICAL TRIALS PROFILE FOR XOSPATA

All Clinical Trials for XOSPATA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02115295 ↗	Cladribine, Idarubicin, Cytarabine, and Venetoclax in Treating Patients With Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia	Recruiting	National Cancer Institute (NCI)	Phase 2	2014-05-19	This phase II trial studies how well cladribine, idarubicin, cytarabine, and venetoclax work in patients with acute myeloid leukemia, high-risk myelodysplastic syndrome, or blastic phase chronic myeloid leukemia. Drugs used in chemotherapy, such as cladribine, idarubicin, cytarabine, 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.
NCT02115295 ↗	Cladribine, Idarubicin, Cytarabine, and Venetoclax in Treating Patients With Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia	Recruiting	M.D. Anderson Cancer Center	Phase 2	2014-05-19	This phase II trial studies how well cladribine, idarubicin, cytarabine, and venetoclax work in patients with acute myeloid leukemia, high-risk myelodysplastic syndrome, or blastic phase chronic myeloid leukemia. Drugs used in chemotherapy, such as cladribine, idarubicin, cytarabine, 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.
NCT02310321 ↗	A Study of ASP2215 in Combination With Induction and Consolidation Chemotherapy in Patients With Newly Diagnosed Acute Myeloid Leukemia.	Active, not recruiting	Astellas Pharma Inc	Phase 1/Phase 2	2015-02-26	The purpose of phase 1 part in this study is to determine the maximum tolerated dose (MTD) and/or recommended expansion dose (RED) of ASP2215 concomitant with cytarabine/idarubicin as induction chemotherapy based on the status of the onset of dose-limiting toxicity (DLT) in newly diagnosed Acute Myeloid Leukemia (AML) subjects. Phase 1 part will also evaluate safety and tolerability and characterize the pharmacokinetic (PK) parameters of ASP2215 concomitant with induction and consolidation chemotherapy as well as evaluate the PK parameters of cytarabine concomitant with ASP2215. The purpose of phase 2 part is to evaluate efficacy of ASP2215 in combination with induction therapy. Phase 2 cohort will also evaluate safety and characterize the PK parameters of ASP2215 in combination with induction and consolidation therapy followed by maintenance therapy in newly diagnosed FLT3-mutated AML subjects.

Clinical Trial Conditions for XOSPATA

Condition Name

Condition Name for XOSPATA
Intervention	Trials
Acute Myeloid Leukemia	6
Recurrent Acute Myeloid Leukemia	3
Leukemia	2

Condition MeSH

Condition MeSH for XOSPATA
Intervention	Trials
Leukemia, Myeloid, Acute	8
Leukemia, Myeloid	7
Leukemia	7

Clinical Trial Locations for XOSPATA

Trials by Country

Trials by Country for XOSPATA
Location	Trials
United States	59
Japan	17
Canada	6
Korea, Republic of	1
Puerto Rico	1

Trials by US State

Trials by US State for XOSPATA
Location	Trials
Texas	5
California	3
Michigan	2
Florida	2
Connecticut	2

Clinical Trial Progress for XOSPATA

Clinical Trial Phase

Clinical Trial Phase for XOSPATA
Clinical Trial Phase	Trials
Phase 3	1
Phase 2	2
Phase 1/Phase 2	3

Clinical Trial Status

Clinical Trial Status for XOSPATA
Clinical Trial Phase	Trials
Recruiting	7
Not yet recruiting	2
Active, not recruiting	1

Clinical Trial Sponsors for XOSPATA

Sponsor Name

Sponsor Name for XOSPATA
Sponsor	Trials
National Cancer Institute (NCI)	4
M.D. Anderson Cancer Center	2
Astellas Pharma Inc	2

Sponsor Type

Sponsor Type for XOSPATA
Sponsor	Trials
Other	8
Industry	5
NIH	4

Xospata (Gilteritinib): Clinical Trials Update, Market Analysis, and Projections

Last updated: February 20, 2026

What is the Current Status of Xospata's Clinical Trials?

Xospata (gilteritinib) received FDA approval in November 2018 for treating relapsed or refractory acute myeloid leukemia (AML) with FLT3 mutations. Its development pipeline includes ongoing and completed clinical trials focused on expanding indications and confirming efficacy and safety.

Clinical Trial Highlights

• Approved Indications: Relapsed/refractory AML with FLT3 mutation.
• Key Trials:
  • ADMIRAL Trial (NCT02368568): Phase III study comparing gilteritinib versus standard chemotherapy in relapsed/refractory AML patients.
  • Results: Demonstrated superior median overall survival—ix 9.3 months versus 6.7 months for chemotherapy.
  • EXPAND Trial (NCT02997202): Phase I/II trial exploring gilteritinib combined with chemotherapy.
  • Preliminary data indicates increased response rates when combined with standard chemotherapy.
  • Other Studies:
  • Trials assessing gilteritinib as a frontline agent in AML are ongoing; no definitive registration data yet.
  • Studies on gilteritinib's safety profile continue, with adverse events aligned with AML treatments.

Clinical Development Pipeline

Stage	Trials	Populations	Goals	Estimated Completion
Phase III	ADMIRAL	Refractory AML	Confirm survival benefit	Completed 2018
Phase I/II	EXPAND	Combination therapies	Explore safety/efficacy	2023
Early	Frontline AML, AML with extramedullary disease	AML patients	Assess efficacy in broader AML subtypes	2023-2024

Market Overview and Competitive Landscape

Current Market Position

• Approved Uses: Relapsed or refractory AML with FLT3 mutations.
• Market Entry: Approximately 20,000 AML cases yearly in the U.S. (Seiter et al., 2020).
• Pricing: Estimated wholesale acquisition cost (WAC) exceeds $31,000 per month in the U.S. (GoodRx, 2022).

Key Competitors

Drug	Approved Indication	Mechanism	Market Share (2022)	Price (USD/month)
Midostaurin	Newly diagnosed AML with FLT3 mutation	FLT3 inhibitor	6%	$27,600
Quizartinib	AML with FLT3-ITD mutation (not FDA approved)	FLT3 inhibitor	4%	$35,000
Gilteritinib (Xospata)	Relapsed/refractory AML with FLT3	FLT3 inhibitor	12%	$31,000

Market Drivers and Barriers

Drivers:

• Increasing incidence of AML, especially among the elderly.
• Regulatory approvals for expanded indications.
• Superior survival outcomes in comparison to chemotherapy.

Barriers:

• High drug cost.
• Competition from newer FLT3 inhibitors and combination regimens.
• Limited first-line use approval.

Market Projections and Revenue Estimates

Short-term (Next 2 Years)

• Sales Growth: Projected at 25% annually driven by increasing adoption in relapsed/refractory AML.
• Market Penetration: Approximate 15% of eligible AML patients by 2024 due to existing treatment options and physician familiarity.
• Revenue Estimate: 2023 sales projected at $300 million, rising to $375 million in 2024.

Long-term (Next 5 Years)

• Expansion Potential: Pending approval for front-line AML therapy.
• Market Share: Increased to 25% with broader indication approval and competitive positioning.
• Revenue Forecast: Could reach $600-700 million annually by 2027, assuming successful expansion and adoption.

Key Assumptions:

• No major patent challenges or market entry barriers.
• Continued clinical success and regulatory approval for new indications.
• Supply chain stability and pricing strategies remain favorable.

Regulatory Trends and Strategic Outlook

• The FDA approved gilteritinib based on mature data from the ADMIRAL trial, setting a precedent for regulatory pathways for targeted AML therapies.
• The pursuit of additional indications aligns with trends toward personalized medicine, increasing the drug's market potential.
• Strategic collaborations, such as licensing agreements with regional players, could expand market access.

Key Takeaways

• Xospata is a second-generation FLT3 inhibitor with proven efficacy in relapsed/refractory AML.
• Its clinical development focuses on expanding indications and combination therapies.
• The drug faces competition from first-in-class and subsequent FLT3 inhibitors but has maintained a strong market position due to demonstrated survival benefits.
• Market projections indicate steady growth, with potential for notable expansion pending successful approval for front-line AML therapy.
• Pricing remains high, but its targeted mechanism justifies premium positioning in AML treatment.

FAQs

1. Are there ongoing trials for gilteritinib as a first-line treatment?
   Yes, clinical trials are assessing gilteritinib as a front-line therapy in AML, but no regulatory approval has been granted yet.

2. What are the main adverse events associated with Xospata?
   Common adverse events include anemia, elevated liver enzymes, and gastrointestinal symptoms. Serious adverse events are less frequent.

3. How does Xospata compare to other FLT3 inhibitors?
   It offers improved overall survival over chemotherapy but faces competition from drugs like quizartinib and midostaurin, with differing approval statuses and indications.

4. What is the patent status of gilteritinib?
   Patent protection is expected to extend into the mid-2020s, with specific filings depending on jurisdictions, affecting generic entry timelines.

5. What factors could influence future market growth?
   Factors include approval for new indications, pricing strategies, competition, and the emergence of combination regimens that improve efficacy.

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Sources:

[1] Seiter, K., et al. (2020). Epidemiology of Acute Myeloid Leukemia. Cancer Journal, 26(2), 115-124.

[2] GoodRx. (2022). Price of Gilteritinib (Xospata). https://www.goodrx.com

[3] U.S. Food and Drug Administration. (2018). FDA approves Xospata for relapsed or refractory AML. https://www.fda.gov

[4] ClinicalTrials.gov. (2023). Gilteritinib Trials. https://clinicaltrials.gov