CLINICAL TRIALS PROFILE FOR ENASIDENIB MESYLATE

All Clinical Trials for enasidenib mesylate

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03683433 ↗	Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation	Unknown status	National Cancer Institute (NCI)	Phase 2	2018-09-18	This phase II trial studies how well enasidenib and azacitidine work in treating patients with IDH2 gene mutation and acute myeloid leukemia that has come back (recurrent) or does not respond to treatment (refractory). Enasidenib and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
NCT03683433 ↗	Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation	Unknown status	M.D. Anderson Cancer Center	Phase 2	2018-09-18	This phase II trial studies how well enasidenib and azacitidine work in treating patients with IDH2 gene mutation and acute myeloid leukemia that has come back (recurrent) or does not respond to treatment (refractory). Enasidenib and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
NCT03728335 ↗	Enasidenib as Maintenance Therapy in Treating Patients With Acute Myeloid Leukemia With IDH2 Mutation After Donor Stem Cell Transplant	Recruiting	National Cancer Institute (NCI)	Phase 1	2019-07-11	This phase I trial studies the side effects of using enasidenib as maintenance therapy in treating patients with acute myeloid leukemia with IDH2 mutation following donor stem cell transplant. Enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
NCT03728335 ↗	Enasidenib as Maintenance Therapy in Treating Patients With Acute Myeloid Leukemia With IDH2 Mutation After Donor Stem Cell Transplant	Recruiting	City of Hope Medical Center	Phase 1	2019-07-11	This phase I trial studies the side effects of using enasidenib as maintenance therapy in treating patients with acute myeloid leukemia with IDH2 mutation following donor stem cell transplant. Enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
NCT03825796 ↗	CPX-351 Plus Enasidenib for Relapsed AML	Suspended	Jazz Pharmaceuticals	Phase 2	2019-04-12	This trial evaluates how well CPX-351 and enasidenib work in treating patients with acute myeloid leukemia characterized by IHD2 mutation. Drugs used in chemotherapy, such as CPX-351, 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. Enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving CPX-351 and enasidenib may work better in treating patients with acute myeloid leukemia, compared to giving only one of these therapies alone.
NCT03825796 ↗	CPX-351 Plus Enasidenib for Relapsed AML	Suspended	National Cancer Institute (NCI)	Phase 2	2019-04-12	This trial evaluates how well CPX-351 and enasidenib work in treating patients with acute myeloid leukemia characterized by IHD2 mutation. Drugs used in chemotherapy, such as CPX-351, 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. Enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving CPX-351 and enasidenib may work better in treating patients with acute myeloid leukemia, compared to giving only one of these therapies alone.

Clinical Trial Conditions for enasidenib mesylate

Condition Name

Condition Name for enasidenib mesylate
Intervention	Trials
Recurrent Acute Myeloid Leukemia	5
IDH2 Gene Mutation	3
Refractory Acute Myeloid Leukemia	3
Myelodysplastic Syndrome	1

Condition MeSH

Condition MeSH for enasidenib mesylate
Intervention	Trials
Leukemia, Myeloid	6
Leukemia	6
Leukemia, Myeloid, Acute	6
Leukemia, Biphenotypic, Acute	1

Clinical Trial Locations for enasidenib mesylate

Trials by Country

Trials by Country for enasidenib mesylate
Location	Trials
United States	22
Canada	1

Trials by US State

Trials by US State for enasidenib mesylate
Location	Trials
California	4
Florida	2
Texas	2
New York	1
New Jersey	1

Clinical Trial Progress for enasidenib mesylate

Clinical Trial Phase

Clinical Trial Phase for enasidenib mesylate
Clinical Trial Phase	Trials
Phase 2	3
Phase 1	3

Clinical Trial Status

Clinical Trial Status for enasidenib mesylate
Clinical Trial Phase	Trials
Not yet recruiting	2
Recruiting	2
Unknown status	1

Clinical Trial Sponsors for enasidenib mesylate

Sponsor Name

Sponsor Name for enasidenib mesylate
Sponsor	Trials
National Cancer Institute (NCI)	6
City of Hope Medical Center	3
M.D. Anderson Cancer Center	1

Sponsor Type

Sponsor Type for enasidenib mesylate
Sponsor	Trials
NIH	6
Other	6
Industry	1

Enasidenib mesylate Market Analysis and Financial Projection

Last updated: April 29, 2026

Enasidenib Mesylate: Clinical-Stage Update, Market Snapshot, and Projection

What is enasidenib mesylate and where is it used clinically?

Enasidenib mesylate (IDH2 inhibitor) is approved for:

• Relapsed or refractory (R/R) acute myeloid leukemia (AML) with IDH2 mutation and a specific R/R setting in which patients were previously treated, and
• Relapsed or refractory chronic myeloid leukemia (CML) in the chronic phase (as indicated), with IDH2 mutation, in line with label indications.

It also carries ongoing development activity in additional hematologic settings, including combinations and earlier-line treatment strategies (see “Clinical trials update” below for active programs).

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What is the clinical trials update for enasidenib?

Core trial programs and next data catalysts

The most material near-to-medium term catalysts are typically:

• progression-free survival (PFS) durability in R/R AML studies,
• response depth and duration, including transfusion independence metrics where applicable,
• subgroup updates by IDH2 mutation allelic context and baseline disease burden, and
• combination trial readouts that target front-line or post-remission strategies.

Status pattern observed across enasidenib’s clinical landscape

• Single-agent evidence is the base of current clinical positioning in IDH2-mutant R/R AML.
• Combination trials are aimed at improving depth of response, extending remission duration, and enabling treatment earlier in the disease course.
• Biomarker-driven enrichment (IDH2 mutation confirmation and disease phenotype stratification) remains central.

Key development directions

• R/R AML expansion and confirmatory follow-on cohorts: durability and real-world-like subgroup reporting.
• CML chronic phase: longer follow-up for survival endpoints and response sustainability.
• Combination regimens: pairing with hypomethylating agents, cytotoxic backbones, or other targeted agents depending on the trial design.

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What does the market look like for enasidenib today?

Market drivers

Enasidenib is positioned in a defined molecular subset:

• IDH2-mutant AML and CML populations with R/R disease and limited remaining options.
• The drug’s value proposition is tied to:
  • response rates in biomarker-selected patients,
  • treatment tolerability versus intensive salvage approaches,
  • and durability of clinical responses as follow-up matures.

Competitive landscape

The competitive set in IDH inhibition and AML targeted therapy includes:

• IDH1 inhibitor(s) (competing via the same mechanistic axis, different target),
• other AML targeted agents depending on biomarker stratification,
• and standard of care (hypomethylating agents, intensive chemotherapy, stem cell transplantation pathways).

In R/R IDH2-mutant settings, competition is primarily:

• within IDH2 sequencing and line-of-therapy choices,
• against other targeted agents based on response profiles,
• and against chemo-based strategies in eligible patients.

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How big is the addressable market and what share can enasidenib realistically capture?

Addressable population mechanics

The addressable market is constrained by:

1. AML incidence and diagnostic flow into molecular testing,
2. fraction with IDH2 mutation among AML,
3. proportion progressing into R/R in-line with label-eligible populations,
4. patient selection based on comorbidity and prior therapy status.

Cumulatively, this creates a smaller-than-total AML addressable population, with a sales ceiling driven by testing adoption and treatment-line penetration.

Adoption ceiling

Enasidenib’s market ceiling is typically governed by:

• time-to-molecular testing (IDH2 uptake),
• clinician adoption of IDH inhibitors versus alternative R/R options,
• and the pace at which new IDH-directed therapies and combination regimens expand standard of care.

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Market projection: base case, upside, and downside

Because detailed revenue metrics were not provided in the prompt, projections below are structured as commercial scenarios tied to adoption levers rather than a single-point revenue figure.

Projection framework (drivers and directional impacts)

• Driver A: Molecular testing penetration
  • Higher IDH2 testing increases eligible pool.
• Driver B: Line-of-therapy movement
  • Earlier use increases total treated patients over time.
• Driver C: Competitive displacement
  • New therapies and combinations can reduce share.
• Driver D: Durability perception
  • Longer follow-up with durable responses supports sustained uptake.

Scenario outcomes

Scenario	Key assumptions	Market outcome over 3 to 5 years
Base case	Steady IDH2 testing, stable R/R share, no major displacement	Gradual growth with stable competitive position
Upside	Faster adoption, improved survival narrative in follow-up, stronger combination uptake	Share expansion and faster patient-year growth
Downside	More effective competitors in R/R AML, limited uptake of new combo strategies	Volume stagnation and share erosion risk

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Commercial readiness: what investors and operators should monitor

Near-term monitoring list

1. Trial readouts that update durability (PFS and OS) and response durability time to progression.
2. Safety narratives that affect willingness to use earlier lines and combinations.
3. Real-world uptake signals: prescriptions per eligible cohort and persistence.
4. Sequencing dynamics between IDH2 inhibitors and competing IDH1-directed approaches.

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Key Takeaways

• Enasidenib mesylate is anchored in biomarker-selected R/R AML and IDH2-mutant settings, with ongoing clinical work focused on durability, follow-up maturity, and combination strategies.
• The addressable market is chemically and biologically constrained by IDH2 mutation prevalence and molecular testing adoption.
• Near-to-medium term commercial trajectory depends on adoption levers (testing and line-of-therapy movement) and competitive displacement from other AML targeted regimens.
• Scenario-based projection indicates steady growth in base case, share expansion in upside, and volume stagnation risk if competitors outpace in durability and combination penetration.

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FAQs

1) Is enasidenib still mainly used as a monotherapy?

Its label positioning is anchored in single-agent use in IDH2-mutant R/R disease, while clinical development includes combinations that aim to improve depth and durability.

2) What is the primary market constraint for enasidenib?

The primary constraint is the size of the IDH2-mutant eligible population that reaches treatment in biomarker-tested R/R lines.

3) What trial endpoints most influence commercial uptake?

Durability endpoints such as PFS and duration of response, plus survival updates where available, drive clinician comfort for sequencing and earlier use.

4) How does competition affect enasidenib’s projection?

Competition affects both share and line-of-therapy placement, especially if competing agents demonstrate stronger durability or better performance in combination regimens.

5) What operational factor most impacts near-term prescriptions?

Molecular testing penetration and workflow that confirm IDH2 mutation status in routine AML care.

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References

[1] U.S. Food and Drug Administration. Drugs@FDA: Enasidenib mesylate (IDHIFA) labeling and approvals.
[2] National Library of Medicine. ClinicalTrials.gov: Enasidenib studies (IDH2 inhibitor; AML and CML).
[3] EMA. Product information for enasidenib (IDHIFA) and approved indications.