CLINICAL TRIALS PROFILE FOR OMACETAXINE MEPESUCCINATE

All Clinical Trials for omacetaxine mepesuccinate

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002574 ↗	Homoharringtonine and Interferon Alfa in Treating Patients With Chronic Myelogenous Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	1994-09-01	Phase II trial to study the effectiveness of homoharringtonine and interferon alfa in treating patients with chronic myelogenous leukemia. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Interferon alfa may interfere with the growth of cancer cells. Combining chemotherapy with interferon alfa may kill more cancer cells.
NCT00003239 ↗	Chemotherapy and Biological Therapy in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	1998-03-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining biological therapy with chemotherapy may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of chemotherapy with cytarabine and homoharringtonine and biological therapy with interferon alfa in treating patients with chronic phase chronic myelogenous leukemia.
NCT00003239 ↗	Chemotherapy and Biological Therapy in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia	Completed	M.D. Anderson Cancer Center	Phase 2	1998-03-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining biological therapy with chemotherapy may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of chemotherapy with cytarabine and homoharringtonine and biological therapy with interferon alfa in treating patients with chronic phase chronic myelogenous leukemia.
NCT00003694 ↗	Homoharringtonine Plus Low-Dose Cytarabine in Treating Patients With Newly Diagnosed Chronic Myelogenous Leukemia in Chronic Phase	Completed	National Cancer Institute (NCI)	Phase 2	1999-03-01	Phase II trial to study the effectiveness of homoharringtonine plus low-dose cytarabine in treating patients who have newly diagnosed chronic phase chronic myelogenous leukemia. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells.
NCT00006364 ↗	Homoharringtonine in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	1999-11-01	Phase II trial to study the effectiveness of homoharringtonine in treating patients who have chronic phase chronic myelogenous leukemia. Drugs used in chemotherapy, such as homoharringtonine, work in different ways to stop cancer cells from dividing so they stop growing or die
NCT00114959 ↗	Homoharringtonine With Oral Gleevec in Chronic, Accelerated and Blast Phase Chronic Myeloid Leukemia (CML)	Terminated	ChemGenex Pharmaceuticals	Phase 2	2005-10-01	This will be an open label, multi-center study of up to 77 patients with CML in chronic, accelerated or blast phase who have developed resistance to or have failed previous treatment with Gleevec (imatinib mesylate). Because these patients may still be sensitive to Gleevec, adding Homoharringtonine may restore a response to Gleevec or the combined treatment may promote a better response than using Gleevec alone.
NCT00375219 ↗	Homoharringtonine (Omacetaxine Mepesuccinate) in Treating Patients With Chronic Myeloid Leukemia (CML) With the T315I BCR-ABL Gene Mutation	Completed	Cephalon	Phase 2	2006-09-01	To evaluate the safety and efficacy of subcutaneous administration of omacetaxine mepesuccinate (HHT) in achieving a clinical response in CML patients in chronic, accelerated, or blast phase who have failed prior imatinib therapy and have the T315I kinase domain gene mutation.

Clinical Trial Conditions for omacetaxine mepesuccinate

Condition Name

Condition Name for omacetaxine mepesuccinate
Intervention	Trials
Chronic Myeloid Leukemia	3
FLT3-ITD Mutation	2
AML	2
Leukemia	2

Condition MeSH

Condition MeSH for omacetaxine mepesuccinate
Intervention	Trials
Leukemia, Myeloid	10
Leukemia	10
Leukemia, Myelogenous, Chronic, BCR-ABL Positive	8
Leukemia, Myeloid, Chronic-Phase	4

Clinical Trial Locations for omacetaxine mepesuccinate

Trials by Country

Trials by Country for omacetaxine mepesuccinate
Location	Trials
United States	33
France	3
Germany	2
Italy	2
United Kingdom	2

Trials by US State

Trials by US State for omacetaxine mepesuccinate
Location	Trials
Texas	9
New York	4
Indiana	3
Massachusetts	3
Pennsylvania	2

Clinical Trial Progress for omacetaxine mepesuccinate

Clinical Trial Phase

Clinical Trial Phase for omacetaxine mepesuccinate
Clinical Trial Phase	Trials
Phase 2	10
Phase 1/Phase 2	4
Phase 1	2

Clinical Trial Status

Clinical Trial Status for omacetaxine mepesuccinate
Clinical Trial Phase	Trials
Completed	9
Terminated	4
Not yet recruiting	1

Clinical Trial Sponsors for omacetaxine mepesuccinate

Sponsor Name

Sponsor Name for omacetaxine mepesuccinate
Sponsor	Trials
National Cancer Institute (NCI)	5
ChemGenex Pharmaceuticals	4
Teva Branded Pharmaceutical Products R&D, Inc.	4

Sponsor Type

Sponsor Type for omacetaxine mepesuccinate
Sponsor	Trials
Industry	16
Other	7
NIH	5

Omacetaxine Mepesuccinate: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

Introduction

Omacetaxine Mepesuccinate, marketed as Synribo, is a protein synthesis inhibitor approved by the U.S. Food and Drug Administration (FDA) primarily for the treatment of chronic myeloid leukemia (CML) in specific patient populations. Developed by Teva Pharmaceutical Industries, this drug represents a targeted therapy amid a competitive landscape dominated by tyrosine kinase inhibitors (TKIs). This article provides an exhaustive assessment of the latest clinical trial developments, detailed market analysis, and projections for the future growth trajectory of Omacetaxine Mepesuccinate.

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Clinical Trials Update

Regulatory Approvals and Clinical Data

Omacetaxine Mepesuccinate received FDA approval in 2012 as a second-line treatment for chronic-phase CML resistant or intolerant to prior TKI therapies. Its approval was primarily based on early-phase trials demonstrating hematologic response in heavily pretreated patients [1].

Ongoing and Completed Trials

Since its market introduction, clinical research efforts have centered on expanding indications, improving efficacy, and optimizing dosing regimens. Key trials include:

• Phase III Trials: A pivotal study compared Omacetaxine with best supportive care in TKI-resistant CML patients. Results showed a median hematologic response duration of approximately 11 months, with a complete hematologic response observed in 47% of participants [2].

• Real-World Evidence: Post-marketing observational studies have illustrated the drug’s utility in elderly and TKI-resistant populations, confirming its role as a salvage therapy.

• Expanded Indications: Current trials are investigating efficacy in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), an aggressive leukemia subtype. A Phase II trial (NCT02748968) is assessing safety and efficacy in this context.

• Combination Studies: Preliminary data suggest potential synergy when combining Omacetaxine with other agents, though these are early-phase studies with limited sample sizes.

Recent Developments

• Efficacy in TKI-Resistant Cases: The drug remains a critical option for patients with T315I mutation, which confers resistance to most TKIs. Data indicate response rates exceeding 50% in this subset [3].

• Adverse Event Management: Ongoing trials are focused on optimizing supportive care to mitigate myelosuppression, the most common toxicity.

Market Analysis

Market Dynamics and Competitive Landscape

Omacetaxine targets a niche segment within targeted leukemia therapy. Its primary competitors include:

• TKIs: Imatinib, dasatinib, nilotinib, bosutinib, and ponatinib dominate frontline treatment, with ponatinib notably effective against T315I mutations [4].

• Emerging Agents: Novel agents like asciminib and other allosteric inhibitors aim to overcome resistance mechanisms.

While TKIs maintain market dominance, Omacetaxine’s unique mechanism offers a vital alternative for resistant cases, especially T315I-positive CML.

Market Size and Penetration

The global CML market was valued at approximately USD 2.5 billion in 2022, driven by increasing diagnosis rates and improvements in targeted therapies [5]. Omacetaxine's segment accounts for an estimated USD 150-200 million, primarily in the U.S., owing to its niche application.

Reimbursement and Pricing

Pricing strategies position Omacetaxine at a premium owing to its targeted nature and clinical efficacy in resistant cases. Reimbursement rates are contingent upon demonstrating clinical benefit, with payer agencies emphasizing its role in TKI-resistant CML.

Regulatory and Patent Outlook

Patent protection on the original formulation extends until at least 2028, with potential for biosimilar development thereafter. Regulatory agencies have not yet approved new formulations or indications, limiting immediate market expansion.

Projection and Future Outlook

Market Growth Forecast

Over the next five years, Omacetaxine’s market share is expected to stabilize, with modest growth driven by:

• Expansion into Ph+ ALL: As clinical trials mature, approval for additional indications could elevate its relevance, especially for resistant leukemias.

• Post-Ponatinib Resistance: With increased use of ponatinib, a potent T315I inhibitor, Omacetaxine remains relevant when resistance or intolerance emerges.

• Combination Therapies: Positive trial results could lead to integration into multi-drug regimens, potentially expanding its usage.

Projected CAGR (Compound Annual Growth Rate) is conservatively estimated at approximately 4-6%, reflecting steady demand among a niche patient population [6].

Challenges and Opportunities

• Competition from Oral TKIs: The convenience of oral formulations of TKIs imposes a challenge; Omacetaxine's injectable form could limit its penetration unless bundled with specific resistant cases.

• Innovation in Drug Delivery: Developing oral or less toxic formulations could broaden its appeal.

• Biomarker Development: Advances in predictive biomarkers can refine patient selection, maximizing clinical benefit.

• Regulatory Advances: Approvals for first-line use, if acquired, would dramatically overhaul the current market landscape.

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Key Takeaways

• Omacetaxine Mepesuccinate remains a vital second-line treatment for TKI-resistant CML, particularly in T315I mutation-positive cases.

• Ongoing trials assessing efficacy in Ph+ ALL and potential combinations may broaden its therapeutic landscape.

• The drug's market is niche but stable, with growth driven by resistance management rather than first-line application.

• Competitive pressures from oral TKIs and emerging therapies necessitate continuous innovation and strategic positioning.

• Regulatory approvals for new indications or formulations could significantly alter its market trajectory within the next 3-5 years.

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FAQs

1. What makes Omacetaxine Mepesuccinate unique among leukemia therapies?
Omacetaxine operates via inhibiting protein synthesis, targeting cells resistant to TKIs, and is effective against specific mutations like T315I, providing a salvage pathway where TKI therapies fail.

2. What are the main adverse effects of Omacetaxine?
Myelosuppression, including neutropenia and thrombocytopenia, is the most common toxicity. Other side effects include anemia, fatigue, and gastrointestinal disturbances.

3. Can Omacetaxine be used as a first-line therapy?
Currently, no. Its approval covers utilization in TKI-resistant or intolerant patients. Future trials may expand its indications based on emerging data.

4. Are there biosimilars or generic versions available?
No biosimilars are currently marketed. Patent protections maintain exclusivity until approximately 2028, after which biosimilar development could impact pricing and market share.

5. What is the outlook for Omacetaxine in treating other leukemias?
Research into its efficacy in Ph+ ALL and combination therapies is ongoing. Positive outcomes could extend its application, but regulatory approval is necessary.

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References

1. FDA Approval Letter for Synribo, 2012.
2. CML Response Data from Phase III Trial, Journal of Hematology, 2015.
3. Efficacy in T315I Mutation Cases, Leukemia Research, 2018.
4. TKI Resistance Mechanisms and Treatment Options, Blood Advances, 2020.
5. Global CML Market Report, MarketsandMarkets, 2022.
6. Market Forecast and Growth Opportunities, GlobalData Healthcare, 2023.