CLINICAL TRIALS PROFILE FOR ICLUSIG

All Clinical Trials for ICLUSIG

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00660920 ↗	Safety Study of AP24534 to Treat Chronic Myelogenous Leukemia (CML) and Other Hematological Malignancies	Completed	Ariad Pharmaceuticals	Phase 1	2008-06-01	The purpose of this study is to determine the maximum tolerated dose or a recommended dose of oral AP24534 in a defined schedule in patients with refractory or advanced chronic myelogenous leukemia and other refractory hematologic malignancies.
NCT01207440 ↗	Ponatinib for Chronic Myeloid Leukemia (CML) Evaluation and Ph+ Acute Lymphoblastic Leukemia (ALL)	Completed	Ariad Pharmaceuticals	Phase 2	2010-09-30	The purpose of this study is to determine the efficacy of ponatinib in patients with chronic myeloid leukemia (CML) in chronic phase (CP), accelerated phase (AP) or blast phase (BP) or with philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) who either are resistant or intolerant to either dasatinib or nilotinib, or have the (T)hreonine-315-(I)soleucine (T315I) mutation.
NCT01424982 ↗	Combination Chemotherapy and Ponatinib Hydrochloride in Treating Patients With Acute Lymphoblastic Leukemia	Recruiting	Ariad Pharmaceuticals	Phase 2	2011-10-05	This phase II trial studies the side effects and how well combination chemotherapy and ponatinib hydrochloride work in treating patients with acute lymphoblastic leukemia. Drugs used in chemotherapy, such as cyclophosphamide, vincristine sulfate, doxorubicin hydrochloride, and dexamethasone, 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. Ponatinib hydrochloride may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving combination chemotherapy and ponatinib hydrochloride may be an effective treatment for acute lymphoblastic leukemia.
NCT01424982 ↗	Combination Chemotherapy and Ponatinib Hydrochloride in Treating Patients With Acute Lymphoblastic Leukemia	Recruiting	M.D. Anderson Cancer Center	Phase 2	2011-10-05	This phase II trial studies the side effects and how well combination chemotherapy and ponatinib hydrochloride work in treating patients with acute lymphoblastic leukemia. Drugs used in chemotherapy, such as cyclophosphamide, vincristine sulfate, doxorubicin hydrochloride, and dexamethasone, 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. Ponatinib hydrochloride may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving combination chemotherapy and ponatinib hydrochloride may be an effective treatment for acute lymphoblastic leukemia.
NCT01620216 ↗	Targeted Therapy in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myelogenous Leukemia	Terminated	National Cancer Institute (NCI)	Phase 2	2012-05-11	This phase II trial studies how well targeted therapy works in treating patients with acute lymphoblastic leukemia or acute myelogenous leukemia that has come back after a period of improvement or does not respond to treatment. Testing patients' blood or bone marrow to find out if their type of cancer may be sensitive to a specific drug may help doctors choose more effective treatments. Dasatinib, sunitinib malate, sorafenib tosylate, ponatinib hydrochloride, pacritinib, ruxolitinib, and idelalisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving targeted therapy based on cancer type may be an effective treatment for acute lymphoblastic leukemia or acute myelogenous leukemia.
NCT01620216 ↗	Targeted Therapy in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myelogenous Leukemia	Terminated	Oregon Health and Science University	Phase 2	2012-05-11	This phase II trial studies how well targeted therapy works in treating patients with acute lymphoblastic leukemia or acute myelogenous leukemia that has come back after a period of improvement or does not respond to treatment. Testing patients' blood or bone marrow to find out if their type of cancer may be sensitive to a specific drug may help doctors choose more effective treatments. Dasatinib, sunitinib malate, sorafenib tosylate, ponatinib hydrochloride, pacritinib, ruxolitinib, and idelalisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving targeted therapy based on cancer type may be an effective treatment for acute lymphoblastic leukemia or acute myelogenous leukemia.

Clinical Trial Conditions for ICLUSIG

Condition Name

Condition Name for ICLUSIG
Intervention	Trials
Acute Lymphoblastic Leukemia	7
Refractory Acute Lymphoblastic Leukemia	4
Philadelphia Chromosome Positive	4
Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive	4

Condition MeSH

Condition MeSH for ICLUSIG
Intervention	Trials
Leukemia	19
Leukemia, Myeloid	16
Precursor Cell Lymphoblastic Leukemia-Lymphoma	13
Leukemia, Myelogenous, Chronic, BCR-ABL Positive	12

Clinical Trial Locations for ICLUSIG

Trials by Country

Trials by Country for ICLUSIG
Location	Trials
United States	95
France	19
China	18
Japan	12
Poland	12

Trials by US State

Trials by US State for ICLUSIG
Location	Trials
Texas	12
Oregon	8
Maryland	5
Michigan	5
California	5

Clinical Trial Progress for ICLUSIG

Clinical Trial Phase

Clinical Trial Phase for ICLUSIG
Clinical Trial Phase	Trials
PHASE2	1
Phase 3	3
Phase 2	18

Clinical Trial Status

Clinical Trial Status for ICLUSIG
Clinical Trial Phase	Trials
Recruiting	12
Completed	6
Withdrawn	2

Clinical Trial Sponsors for ICLUSIG

Sponsor Name

Sponsor Name for ICLUSIG
Sponsor	Trials
Ariad Pharmaceuticals	11
National Cancer Institute (NCI)	7
M.D. Anderson Cancer Center	6

Sponsor Type

Sponsor Type for ICLUSIG
Sponsor	Trials
Other	20
Industry	19
NIH	7

Clinical Trials Update, Market Analysis, and Projection for Iclusig (Ponatinib)

Last updated: October 28, 2025

---

Introduction

Iclusig (ponatinib) is a potent tyrosine kinase inhibitor (TKI) developed by ARIAD Pharmaceuticals, now part of Pfizer, primarily approved for treating certain types of chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Approved by the FDA in 2012 under accelerated approval and later fully licensed, Iclusig has established itself as a pivotal option for patients intolerant or resistant to previous TKIs. This strategic analysis explores recent clinical trial developments, analyzes market dynamics, and projects future growth trajectories for Iclusig.

---

Clinical Trials Update

Recent and Ongoing Clinical Trials

Since its initial approval, Iclusig has undergone continuous clinical evaluation for expanded indications and optimized safety profiles. Recent regulatory filings and trial data are indicative of ongoing efforts to reinforce its positioning in hematologic malignancies.

• Phase III Studies:
  Notably, Iclusig was evaluated in the PACE (Ponatinib Ph+ ALL and CML Evaluation) trial, which remains a cornerstone for its efficacy profile. The trial outcomes showed high response rates in resistant or intolerant CML cases, reaffirming its role in heavily pretreated populations. Follow-up results, published in recent conferences (e.g., ASH 2022), demonstrate durable responses with manageable safety risks.

• Expanded Indication Trials:
  Trials like the EPIC (Efficacy and Safety of Ponatinib in Newly Diagnosed Chronic Myeloid Leukemia) focus on evaluating Iclusig as a first-line therapy. Preliminary data suggest promising efficacy with acceptable safety, potentially broadening its market scope.

• Safety and Resistance Monitoring:
  Updated safety profiles, emphasizing vascular occlusive events previously associated with Iclusig, are integral to ongoing studies. The latest data indicate improved risk mitigation protocols, with dose adjustments correlating to reduced adverse events.

Regulatory Developments

• The FDA's post-marketing commitments include confirmatory trials addressing long-term safety and efficacy, especially concerning arterial thrombosis risks.
• Regulatory agencies in Europe and Japan are reviewing similar data, with some regions considering expanding approvals based on recent trial outcomes.

---

Market Analysis

Current Market Landscape

Iclusig’s market hinges on its status as a second- and third-line therapy in CML and Ph+ ALL. Historically, the tyrosine kinase inhibitor landscape has been dominated by drugs such as imatinib, dasatinib, nilotinib, and bosutinib. Iclusig’s unique position is as a potent inhibitor effective in resistant cases, especially when other TKIs fail.

• Market Penetration:
  Despite its efficacy, Iclusig’s adoption remains tempered by safety concerns, predominantly vascular adverse events. Physicians tend to reserve it for patients who have exhausted other options, curbing its initial market potential.

• Competitive Dynamics:
  The market features a robust pipeline of newer agents offering improved safety profiles, such as asciminib. Nevertheless, Iclusig continues to attract niche indications due to its efficacy in resistant cases, especially in complex mutation profiles like T315I.

Market Drivers

• Unmet Medical Need:
  Resistance and intolerance to earlier TKIs create persistent demand for Iclusig in treatment-resistant populations.
• Regulatory Approvals:
  Potential expansion of approved indications, notably as first-line treatment in specific subpopulations, could significantly enhance its market share.
• Physician Adoption:
  Growing familiarity and experience with managing its risks could foster broader acceptance, particularly as safety mitigation strategies improve.

Market Challenges

• Safety Concerns:
  Adverse vascular events remain a critical barrier, necessitating careful patient selection and vigilant monitoring.
• Competition:
  Emergence of newer TKIs and novel agents, including all-oral therapies and mutation-specific inhibitors, threaten Iclusig’s market share.
• Pricing and Reimbursement:
  Cost considerations and complex reimbursement landscapes limit rapid adoption, especially in emerging markets.

---

Market Projection and Future Outlook

Near-term (2023–2025)

Market analysts project a steady but restrained growth trajectory. With ongoing clinical trials potentially supporting label expansion, Iclusig could see increased usage in resistant or intolerant patient populations. Strategically, Pfizer’s real-world evidence generation and risk mitigation initiatives will be pivotal in expanding physician confidence.

Medium to Long-term (2026–2030)

The future of Iclusig depends heavily on regulatory decisions regarding expanded labeling and its positioning relative to competitor agents. If the drug secures approval for first-line indications, particularly in patients with high-risk mutations or resistant disease, the market could compound significantly.

Furthermore, advances in combination therapies—integrating Iclusig with immunomodulatory agents—may unlock new treatment paradigms, pushing sales upward. The global hematology drug market’s CAGR (compound annual growth rate) anticipates a 7-9% increase through 2028[1], with targeted therapies like Iclusig contributing to this trend.

Geographical Market Expansion

Currently concentrated in North America and Europe, emerging markets in Asia Pacific, Latin America, and the Middle East present substantial growth opportunities. Patent expiration, generic competition, and local approval processes will influence how aggressively Pfizer can penetrate these regions.

---

Strategic Considerations

To capitalize on its potential, Pfizer must:

• Improve Safety Profile:
  Continual refinement of dosing and monitoring protocols enhances tolerability.
• Broaden Indications:
  Accelerate registration trials for first-line use and additional hematologic malignancies.
• Real-World Evidence:
  Leverage post-marketing data to demonstrate effective risk management strategies and patient benefits.
• Partnership and Licensing:
  Collaborate with regional authorities and health organizations to facilitate access and reimbursement.

---

Key Takeaways

• Clinical Trials:
  Ongoing studies demonstrate promising efficacy in resistant populations and potential for expanded use, with safety management as a focal point.

• Market Position:
  Iclusig remains a critical option for resistant CML and Ph+ ALL patients, distinguished by its potency but constrained by safety concerns.

• Growth Projections:
  Modest near-term growth anticipated, with significant upside if regulatory approvals for first-line therapy materialize and safety profiles improve.

• Competitive Landscape:
  Emerging TKIs and mutation-specific agents pose threats but also opportunities for differentiation through safety and tailored therapy.

• Strategic Outlook:
  Success hinges on regulatory agility, safety management, and expanding indications, especially in underserved markets.

---

FAQs

1. What is the primary indication of Iclusig?
Iclusig (ponatinib) is approved for treating chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), especially in patients resistant or intolerant to prior TKIs.

2. How does Iclusig differ from other TKIs?
Iclusig is a maximal potent inhibitor targeting multiple BCR-ABL mutations including T315I, making it effective in resistant cases where other TKIs fail.

3. What safety concerns are associated with Iclusig?
The main risks involve arterial occlusive events, including vascular thrombosis, necessitating careful patient selection and dose management.

4. Is there ongoing research to expand Iclusig’s indications?
Yes, ongoing trials are evaluating its use as a first-line therapy and in combination regimens, with preliminary results suggesting potential for broader application.

5. How does market competition impact Iclusig’s prospects?
Emerging TKIs with improved safety profiles and mutation-specific options pose competitive threats, although Iclusig’s unique efficacy in resistant cases preserves its niche.

---

References

[1] Market Research Future, “Hematology Drugs Market – Global Forecast to 2028,” 2022.