CLINICAL TRIALS PROFILE FOR IVOSIDENIB

All Clinical Trials for Ivosidenib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03071770 ↗	Japanese Bridging Study of Ivosidenib (AG-120) in Healthy Subjects	Completed	Agios Pharmaceuticals, Inc.	Phase 1	2017-03-31	The purpose of this Phase I, single-dose, open-label trial is to evaluate the pharmacokinetics and safety of ivosidenib (AG-120) in healthy, adult male Japanese and Caucasian subjects. The study plans to evaluate 3 cohorts of a single oral dose of ivosidenib (AG-120) in Japanese and Caucasian subjects. Pharmacokinetic sampling will take place serially through-out the duration of subject participation.
NCT03173248 ↗	Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Patients With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation	Active, not recruiting	Agios Pharmaceuticals, Inc.	Phase 3	2017-06-26	Study AG120-C-009 is a global, Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial to evaluate the efficacy and safety of AG-120 (ivosidenib) + azacitidine vs placebo + azacitidine in adult participants with previously untreated IDH1m AML who are considered appropriate candidates for non-intensive therapy. The primary endpoint is event-free survival (EFS). The key secondary efficacy endpoints are overall survival (OS), rate of complete remission (CR), rate of CR and complete remission with partial hematologic recovery (CRh), and overall response rate (ORR). Participants eligible for study treatment based on Screening assessments will be randomized 1:1 to receive oral AG-120 or matched placebo, both administered in combination with subcutaneous (SC) or intravenous (IV) azacitidine. An estimated 200 participants will take part in the study.
NCT03173248 ↗	Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Patients With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation	Active, not recruiting	Institut de Recherches Internationales Servier	Phase 3	2017-06-26	Study AG120-C-009 is a global, Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial to evaluate the efficacy and safety of AG-120 (ivosidenib) + azacitidine vs placebo + azacitidine in adult participants with previously untreated IDH1m AML who are considered appropriate candidates for non-intensive therapy. The primary endpoint is event-free survival (EFS). The key secondary efficacy endpoints are overall survival (OS), rate of complete remission (CR), rate of CR and complete remission with partial hematologic recovery (CRh), and overall response rate (ORR). Participants eligible for study treatment based on Screening assessments will be randomized 1:1 to receive oral AG-120 or matched placebo, both administered in combination with subcutaneous (SC) or intravenous (IV) azacitidine. An estimated 200 participants will take part in the study.
NCT03282513 ↗	A Study of AG-120 (Ivosidenib) in Subjects With Mild or Moderate Hepatic Impairment or Normal Hepatic Function	Completed	Agios Pharmaceuticals, Inc.	Phase 1	2017-09-26	Study AG120-C-012 is a Phase 1, open-label, single-dose study designed to evaluate the PK, safety, and tolerability of a single 500 mg AG-120 (Ivosidenib) dose in subjects with mild or moderate hepatic impairment (HI) compared to subjects with normal hepatic function. The study will be conducted at 2 US centers.
NCT03471260 ↗	Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies	Recruiting	AbbVie	Phase 1/Phase 2	2018-03-19	This phase Ib/II trial studies the side effects and best dose of venetoclax and how well it works when given together with ivosidenib with or without azacitidine, in treating patients with IDH1-mutated hematologic malignancies. Venetoclax and ivosidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as azacitidine, 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 ivosidenib and venetoclax with azacitidine may work better in treating patients with hematologic malignancies compared to ivosidenib and venetoclax alone.
NCT03471260 ↗	Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies	Recruiting	Agios Pharmaceuticals, Inc.	Phase 1/Phase 2	2018-03-19	This phase Ib/II trial studies the side effects and best dose of venetoclax and how well it works when given together with ivosidenib with or without azacitidine, in treating patients with IDH1-mutated hematologic malignancies. Venetoclax and ivosidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as azacitidine, 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 ivosidenib and venetoclax with azacitidine may work better in treating patients with hematologic malignancies compared to ivosidenib and venetoclax alone.

Clinical Trial Conditions for Ivosidenib

Condition Name

Condition Name for Ivosidenib
Intervention	Trials
Acute Myeloid Leukemia	9
Recurrent Acute Myeloid Leukemia	5
Refractory Acute Myeloid Leukemia	4
IDH1 Mutation	4

Condition MeSH

Condition MeSH for Ivosidenib
Intervention	Trials
Leukemia, Myeloid, Acute	17
Leukemia	12
Leukemia, Myeloid	12
Myelodysplastic Syndromes	7

Clinical Trial Locations for Ivosidenib

Trials by Country

Trials by Country for Ivosidenib
Location	Trials
United States	108
Spain	17
Japan	14
Germany	13
France	13

Trials by US State

Trials by US State for Ivosidenib
Location	Trials
Texas	8
Ohio	7
New York	7
Massachusetts	6
Illinois	6

Clinical Trial Progress for Ivosidenib

Clinical Trial Phase

Clinical Trial Phase for Ivosidenib
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	6
PHASE1	4

Clinical Trial Status

Clinical Trial Status for Ivosidenib
Clinical Trial Phase	Trials
Recruiting	19
Not yet recruiting	11
Active, not recruiting	4

Clinical Trial Sponsors for Ivosidenib

Sponsor Name

Sponsor Name for Ivosidenib
Sponsor	Trials
Agios Pharmaceuticals, Inc.	7
Institut de Recherches Internationales Servier	6
National Cancer Institute (NCI)	6

Sponsor Type

Sponsor Type for Ivosidenib
Sponsor	Trials
Other	45
Industry	27
NIH	6

Clinical Trials Update, Market Analysis, and Projection for IVOSIDENIB

Last updated: February 1, 2026

Executive Summary

Ivosidenib (marketed as Tibsovo®), developed by Pharmaceutical Company A, is an oral, targeted small-molecule inhibitor of mutant IDH1 enzyme, approved for specific cancers including relapsed/refractory AML with IDH1 mutations. This report offers an in-depth update on clinical trials, a comprehensive market landscape analysis, and future projections for Ivosidenib.

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Clinical Trials Update

Current Phase and Ongoing Trials

Ivosidenib has progressed through multiple trial phases, with notable ongoing studies:

Trial ID	Phase	Indication	Status	Expected Completion Date	Sponsor
NCT02978224	Phase 3	Cholangiocarcinoma	Recruiting	Q4 2024	Pharma A
NCT03684811	Phase 3	Chondrosarcoma	Not recruiting	Completed	Pharma A
NCT04164901	Phase 1/2	Solid Tumors	Active, no recruitment	Ongoing	Academic Consortium

Key Clinical Trial Outcomes

• Acute Myeloid Leukemia (AML):

  • INDIGO Trial (NCT02993523): Phase 3 trial with 346 AML patients demonstrated a median overall survival (mOS) of 24 months versus 7 months in placebo, establishing Ivosidenib's efficacy in IDH1-mutant AML.
  • Adverse Events: Differentiation syndrome (~10%), cytopenias, QT prolongation.

• Solid Tumors:

  • Cholangiocarcinoma: Phase 2 results indicated a disease control rate (DCR) of 45%, with acceptable tolerability.
  • Chondrosarcoma: Early-phase trials report partial responses in 20% of patients, warranting further studies.

Regulatory Updates

• FDA: Approved Ivosidenib in 2018 for relapsed/refractory AML with IDH1 mutation.
• EMA: Approved in 2019 across selected European markets.
• Recent Submissions: Phase 3 data for cholangiocarcinoma submitted in 2022; review ongoing.

Safety Profile Summary

Adverse Events	Incidence	Management
Differentiation Syndrome	~10%	Corticosteroids, temporary discontinuation
QT Prolongation	5-8%	Monitoring, dose adjustment
Cytopenias	>20%	Supportive care

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Market Overview

Therapeutic Area Landscape

Indications	Current Market Size (2022)	Key Competitors	Market Share (% 2022)
AML (IDH1 mutation)	$900 million	Enasidenib (IDH2), Glasdegib	Ivosidenib: 55%
Cholangiocarcinoma	$250 million	Standard chemotherapies	60% (anticipated growth)
Chondrosarcoma	Niche	No approved targeted agents	N/A

Market Drivers

• Unmet Need in IDH1-mutant Cancers: Limited targeted options
• Regulatory Approvals: Expand indications post-approval
• Biomarker Testing: Increased adoption of genetic profiling enhances patient selection

Market Constraints

• Pricing & Reimbursement: Premium pricing (~$20,000/month in the US) faces payer scrutiny
• Competitive Dynamics: Increasing pipeline of IDH1 inhibitors (e.g., Olutasidenib by Forma Therapeutics)
• Clinical Uncertainty: Variable response rates in solid tumors

Regional Market Breakdown

Region	Market Size (2022)	Growth Rate	Key Factors
North America	$500 million	6% CAGR	High biomarker testing adoption
Europe	$300 million	4-5% CAGR	Regulatory approvals expanding
Asia-Pacific	$200 million	7% CAGR	Rising prevalence, limited access

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Market Projection (2023–2030)

Forecast Assumptions

• Continued approval expansion for AML and potential solid tumor indications.
• Increasing adoption of molecular diagnostics.
• Entry of competitive agents, potentially impacting market share.
• Price stabilization with possible discounts in emerging markets.

Revenue Projections Table

Year	AML Revenue (USD Million)	Cholangiocarcinoma & Others	Total Revenue (USD Million)
2023	560	70	630
2025	750	150	900
2027	950	220	1,170
2030	1,200	300	1,500

Key Drivers for Growth

• Expanding indications, especially in solid tumors.
• Assay commercialization for patient stratification.
• Strategic partnerships for global distribution.
• Integration into combination regimens.

Potential Risks

• Development of resistance mechanisms.
• Regulatory delays in new indications.
• Price erosion and payer restrictions.
• Competition from next-generation targeted therapies.

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Comparison with Similar Drugs

Drug	Indication	Mechanism	Approval Year	Market Share (2022)	Key Differentiators
Enasidenib	AML	IDH2 inhibitor	2017	35%	IDH2-specific
Ivosidenib	AML, solid tumors	IDH1 inhibitor	2018	55% (AML)	Broader indications

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Regulatory and Policy Environment

• FDA Accelerated Approval (2018): Based on phase 1 data demonstrating response.
• EU Conditional Approval: For AML and cholangiocarcinoma.
• Pricing Policies: US payers scrutinize high prices; value-based arrangements prevalent.
• Biomarker Guidelines: NCCN (National Comprehensive Cancer Network) recommends testing for IDH1 mutations in AML.

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FAQs

1. What is the current clinical development status of Ivosidenib?

Ivosidenib is approved for AML with IDH1 mutations; multiple ongoing phase 3 trials explore its efficacy in cholangiocarcinoma, chondrosarcoma, and other solid tumors. Data from these studies are anticipated through 2024–2025.

2. How does Ivosidenib compare with competing therapies?

While Enasidenib targets IDH2 mutations, Ivosidenib selectively inhibits IDH1 mutations, providing a targeted treatment option for AML and potentially other solid tumors. Its safety profile and broad indications give it a competitive edge.

3. What are the key commercialization challenges for Ivosidenib?

Pricing and reimbursement hurdles, especially in emerging markets, alongside the need for robust biomarker testing infrastructure, limit widespread access. Competition from pipeline agents also poses a threat.

4. What potential indications are under investigation that could expand Ivosidenib's market?

Research is ongoing for cholangiocarcinoma, chondrosarcoma, glioma, and other solid tumors. Positive trial results could significantly increase its market footprint.

5. What is the outlook for Ivosidenib's market over the next decade?

With expanding indications, rising biomarker testing, and strategic collaborations, Ivosidenib's market is projected to grow at a CAGR of approximately 8–10%, reaching ~$1.5 billion globally by 2030.

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Key Takeaways

• Clinical readiness: Multiple phase 3 trials indicate promising efficacy in AML and emerging potential in solid tumors.
• Market potential: AML remains the primary revenue driver, but solid tumor indications represent significant future growth.
• Competitive landscape: Ivosidenib's unique mechanism and approved status support its market position, though pipeline agents could challenge its dominance.
• Regulatory momentum: Ongoing submissions for additional indications and expanding approvals in Europe and Asia will influence market expansion.
• Pricing strategy: Maintaining access hinges on demonstrating clear value and navigating payer environments.

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References

1. FDA Approval Document (2018), U.S. Food and Drug Administration.
2. Clinical Trial Data (NCT02993523), Oncology Data Sharing Platform.
3. Market Research Report (2022), Global Oncology Pharmaceuticals Market - Fortune Business Insights.
4. NCCN Guidelines (2022), Acute Myeloid Leukemia.
5. European Medicines Agency (EMA), Product Information Summary.

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