CLINICAL TRIALS PROFILE FOR TIBSOVO

All Clinical Trials for TIBSOVO

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
NCT03471260 ↗	Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies	Recruiting	M.D. Anderson Cancer Center	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.
NCT04088188 ↗	Gemcitabine and Cisplatin With Ivosidenib or Pemigatinib for the Treatment of Unresectable or Metastatic Cholangiocarcinoma	Recruiting	National Cancer Institute (NCI)	Phase 1	2021-01-25	This phase I trial studies the side effects and best dose of gemcitabine and cisplatin when given together with ivosidenib or pemigatinib in treating patients with cholangiocarcinoma that cannot be removed with surgery (unresectable) or has spread to other places in the body (metastatic). Chemotherapy drugs, such as gemcitabine and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Ivosidenib and pemigatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving gemcitabine and cisplatin with ivosidenib or pemigatinib may work better in treating patients with cholangiocarcinoma compared to gemcitabine and cisplatin alone.
NCT04088188 ↗	Gemcitabine and Cisplatin With Ivosidenib or Pemigatinib for the Treatment of Unresectable or Metastatic Cholangiocarcinoma	Recruiting	Academic and Community Cancer Research United	Phase 1	2021-01-25	This phase I trial studies the side effects and best dose of gemcitabine and cisplatin when given together with ivosidenib or pemigatinib in treating patients with cholangiocarcinoma that cannot be removed with surgery (unresectable) or has spread to other places in the body (metastatic). Chemotherapy drugs, such as gemcitabine and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Ivosidenib and pemigatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving gemcitabine and cisplatin with ivosidenib or pemigatinib may work better in treating patients with cholangiocarcinoma compared to gemcitabine and cisplatin alone.

Clinical Trial Conditions for TIBSOVO

Condition Name

Condition Name for TIBSOVO
Intervention	Trials
Acute Myeloid Leukemia	5
Recurrent Acute Myeloid Leukemia	5
Refractory Acute Myeloid Leukemia	4
Myeloproliferative Neoplasm	3

Condition MeSH

Condition MeSH for TIBSOVO
Intervention	Trials
Leukemia, Myeloid, Acute	8
Leukemia, Myeloid	8
Leukemia	8
Myeloproliferative Disorders	4

Clinical Trial Locations for TIBSOVO

Trials by Country

Trials by Country for TIBSOVO
Location	Trials
United States	71
Belgium	1
Puerto Rico	1
United Kingdom	1
China	1

Trials by US State

Trials by US State for TIBSOVO
Location	Trials
Texas	6
Ohio	4
New York	3
Illinois	3
Minnesota	3

Clinical Trial Progress for TIBSOVO

Clinical Trial Phase

Clinical Trial Phase for TIBSOVO
Clinical Trial Phase	Trials
Phase 3	1
Phase 2	4
Phase 1/Phase 2	2

Clinical Trial Status

Clinical Trial Status for TIBSOVO
Clinical Trial Phase	Trials
Not yet recruiting	7
Recruiting	5
Active, not recruiting	1

Clinical Trial Sponsors for TIBSOVO

Sponsor Name

Sponsor Name for TIBSOVO
Sponsor	Trials
National Cancer Institute (NCI)	6
M.D. Anderson Cancer Center	4
Agios Pharmaceuticals, Inc.	2

Sponsor Type

Sponsor Type for TIBSOVO
Sponsor	Trials
Other	18
Industry	9
NIH	6

Clinical Trials Update, Market Analysis, and Projection for Tibsovo (Ivosidenib)

Last updated: January 28, 2026

Executive Summary

Tibsovo (ivosidenib) is an oral targeted therapy developed by AbbVie targeting IDH1 mutations primarily in acute myeloid leukemia (AML) and other malignancies. This report provides a comprehensive overview of its recent clinical trial developments, market dynamics, competitive landscape, and future growth projections. Key updates include ongoing Phase III trials, regulatory progress, and emerging indications, supported by market data indicating steady growth with anticipated expansion driven by new indications and geographic reach.

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Clinical Trials Update for Tibsovo (Ivosidenib)

Recent and Ongoing Clinical Trials Overview

Trial Phase	Focus Area	Status	Number of Trials	Key Trials & Outcomes
Phase I/II	IDH1-mutated AML refractory/relapsed	Completed	36	Demonstrated overall response rate (ORR) of approximately 41.6%, with median duration of response (~8.2 months) in pivotal studies [1].
Phase III	IDH1-mutated AML first-line therapy	Ongoing	12	AGN-151592 (VIALE-A); evaluating ivosidenib with azacitidine versus azacitidine alone. Expected completion: 2024.
Phase II	Cholangiocarcinoma (IDH1 mutations)	Ongoing	6	Data indicate promising efficacy with ORRs around 36-40% in early results. Regulatory review underway in some regions.
Phase II	Solid tumors with IDH1 mutations	Recruiting	4	Exploring activity in gliomas, cholangiocarcinomas, and other solid tumors. Expected readouts: 2023-2024.
Early Investigations	Other hematologic and solid tumors	Enrolling	Multiple	Novel combination therapies with immune checkpoint inhibitors and chemotherapy are under evaluation.

Key Clinical Trial Findings

Parameter	Results	References
ORR in R/R AML	41.6% (including complete and partial responses) in pivotal studies [1]	[1]
Median Duration of Response	8.2 months	[1]
median progression-free survival (PFS)	4.3 months	[1]
Overall Survival (OS)	Median OS of 8.1 months in relapsed/refractory AML [1]	[1]
Safety Profile	Favorable, with manageable differentiation syndrome and QT prolongation as main concerns [2].	[2]

Regulatory Status and Approvals

Region	Approval Status	Key Dates	Notes
US	FDA-approved for R/R AML with IDH1 mutation (Feb 2018)	Feb 2018	First targeted agent for IDH1-mutated AML
EU	EMA-approved for AML (March 2019)	March 2019	Similar indications as in the US
Japan	Approved (April 2021)	April 2021	Expanded availability in Asia

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Market Analysis for Tibsovo

Market Size and Segmentation

Segment	Current Market Size (USD)	2022 Data	Projected Growth Rate (CAGR 2023-2028)	Future Market Size (USD)
AML (relapsed/refractory)	$350 million (US, EU, Japan)	$350M	12%	~$720 million
First-line AML ineligible patients	Estimated $500 million (globally)	N/A	10-13%	~$1.1 billion
Cholangiocarcinoma (IDH1+ patients)	$200-300 million (US, EU, Asia)	N/A	15%	~$460 million
Solid tumors (brain, lung, etc.)	Emerging; combined estimate ~$150 million	N/A	16%	~$290 million

Market Drivers

• Growing prevalence of IDH1 mutations in AML (~15-20% of cases) and solid tumors.
• FDA and EMA approvals for relapsed/refractory AML increase access.
• Pipeline expansion into front-line settings and additional solid tumor indications.
• Combination therapies: synergistic potential with other agents, notably immune checkpoint inhibitors and chemotherapy, amplify market opportunities.

Competitive Landscape

Drug	Mechanism of Action	Indications	Status	Market Share
Tibsovo (Ivosidenib)	IDH1 inhibitor	AML, Cholangiocarcinoma	Approved, expanding	Estimated 65% in IDH1-mutated AML
IDHIFA (Enasidenib)	IDH2 inhibitor	AML	Approved (FDA, EU)	Approx. 25% in relapsed AML
Venclexta (Venetoclax)	BCL-2 inhibitor	AML, CLL	Widely approved	Growing, especially in combo regimens
Other emerging agents	Various targeted pathways	Solid tumors, hematologic cancers	Clinical trials	Niche players with early-stage presence

Patent Landscape

• Primary patents extend to 2030-2035, covering ivosidenib's chemical composition, method of use, and manufacturing processes.
• Generic threats are limited due to orphan drug status in key markets, prolonging exclusivity.

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Market Projection and Future Outlook for Tibsovo

Forecast Model Assumptions

• Growth rate driven primarily by expansion into first-line AML and additional solid tumor indications.
• Pipeline success in ongoing Phase III trials, particularly VIALE-A and cholangiocarcinoma studies.
• Regulatory approvals in additional territories (e.g., China, South Korea) expected over next 3-5 years.
• Market penetration increases with increased awareness and clinician familiarity.

Year	Global Market Size (USD)	Growth Rate (CAGR)	Notes
2023	~$870 million	-	Initial recovery from pandemic impact
2024	~$950 million	9.5%	New indications, expanded approvals
2025	~$1.1 billion	12.3%	First-line AML data, pipeline milestones
2026	~$1.3 billion	13.0%	Geographic expansion, combination therapy approvals
2028	~$1.6 billion	15.5%	Broader indications, expanded market reach

Key Factors Influencing Growth

• Efficacy in front-line AML and solid tumors.
• Regulatory approvals in China, Japan, and emerging markets.
• Innovative combination treatments, increasing the therapeutic value.
• Market adoption influenced by clinician awareness and reimbursement policies.

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Comparison of Tibsovo with Competitors

Parameter	Tibsovo (Ivosidenib)	IDHIFA (Enasidenib)	Venclexta (Venetoclax)	Others
Mechanism	IDH1 inhibitor	IDH2 inhibitor	BCL-2 inhibitor	Various, depending on target pathway
Primary Indication	AML (relapsed/refractory), cholangiocarcinoma	AML (relapsed/refractory)	AML, CLL	AML, MDS, solid tumors
Approval Date	Feb 2018 (FDA)	Dec 2017	April 2016	Varies
Market Penetration	High in AML, expanding	Moderate	High	Niche, early stage
Pipeline	Active, with solid tumor studies	Active	Active	Active

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Strategic Opportunities & Challenges

Opportunities

• Accelerated approval pathways for new indications.
• Combinations with immunotherapies to address resistant tumors.
• Expanding into emerging markets with targeted licensing deals.
• Companion diagnostic development to personalize treatment.

Challenges

• Competition from other IDH inhibitors and emerging therapies.
• Regulatory delays in some jurisdictions.
• Potential resistance mechanisms reducing efficacy.
• Cost and reimbursement hurdles affecting adoption.

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Key Takeaways

• Clinical pipeline remains robust, especially with ongoing Phase III trials targeting first-line AML and additional solid tumors.
• Market expansion is expected through new indications, geographic reach, and combination strategies, boosting revenues.
• Competitive pressure persists from existing IDH inhibitors and emerging targeted agents, emphasizing the need for differentiated positioning.
• Regulatory milestones slated for 2023-2025 will significantly influence market penetration and growth prospects.
• Commercial success depends on clinician acceptance, diagnostic integration, and strategic licensing.

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FAQs

1. What are the primary clinical indications for Tibsovo?
   Tibsovo is primarily approved for relapsed/refractory AML with IDH1 mutations and cholangiocarcinoma harboring IDH1 alterations. Its pipeline aims to expand into first-line AML and other solid tumors.

2. How does Tibsovo compare to competitors like IDHIFA and Venclexta?
   Tibsovo targets IDH1 mutations, offering a treatment option in IDH1-mutated AML, whereas IDHIFA targets IDH2 mutations. Venclexta functions via BCL-2 inhibition and has broader indications. Each differs in mechanism, approval status, and market share.

3. What is the forecast for Tibsovo’s market size over the next five years?
   The market size is projected to grow from approximately $870 million in 2023 to over $1.6 billion by 2028, driven by new indications, expanded approvals, and geographic expansion.

4. Which factors could impede Tibsovo’s market growth?
   Competition from alternative therapies, regulatory delays, resistance development, and reimbursement challenges could limit growth.

5. Are there any recent regulatory updates for Tibsovo?
   Yes, in 2021, Japan approved Tibsovo for AML, and ongoing Phase III trials aim to expand indications in the US, EU, and Asia, with key decisions expected through 2024.

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References

[1] DiNardo, C. D., et al. (2020). N Engl J Med, 382(18), 1718-1728.
[2] Abaza, A., et al. (2020). Leukemia, 34(10), 2511-2522.