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.

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, Myeloid	12
Leukemia	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
UNKNOWN	6

Ivosidenib (Tibsovo): Clinical Trials Update, Market Analysis, and Projections

Last updated: May 2, 2026

Ivosidenib has maintained a sustained oncology footprint since launch, with the bulk of commercial value tied to relapsed or refractory (R/R) AML with a susceptible IDH1 mutation and expanding label usage across specific disease settings. The near-to-mid-term growth outlook depends on (1) uptake in AML line-of-therapy segments where testing identifies IDH1 mutation-positive patients, (2) competition-driven pricing pressure from other AML IDH1 and IDH2 agents, and (3) continued clinical differentiation from combination regimens that deepen response duration or improve overall survival in defined molecular subsets.

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What is the current clinical trial position for ivosidenib?

Core development theme

Ivosidenib’s clinical program concentrates on two paths: (1) IDH1-mutant AML in defined risk and treatment-history categories, and (2) combination strategies that pair IDH inhibition with standard-of-care backbones (including hypomethylating agents and intensive chemotherapy) or other targeted agents to enhance durability.

Trial update snapshot (functional status view)

The program structure is consistent across disease settings: pivotal and confirmatory AML studies established efficacy for R/R AML with IDH1 mutation; subsequent studies expand into combinations and earlier-line settings. Across the pipeline, endpoints typically include objective response rate (ORR), complete remission (CR) and CR with partial hematologic recovery (CRh), event-free survival (EFS), duration of response (DoR), and overall survival (OS), with mutation status and measurable residual disease (MRD) used to stratify depth of response.

Practical implication for “trial-driven value”

Market impact comes from confirmatory readouts that change treatment placement (for example, moving from “post-therapy” to “earlier lines”) and from trial data that strengthen claims around durability or MRD negativity. Where trials support additional line-of-therapy expansion, payer coverage patterns typically improve and pull-through rises with testing availability and clinician familiarity.

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Which indications anchor commercial uptake?

Launch indications and label scope (commercial anchors)

Ivosidenib is marketed as a precision oncology therapy for IDH1-mutant AML. The commercial engine is driven by the intersection of:

• IDH1 mutation prevalence in AML
• physician adoption of molecular testing
• treatment sequencing in R/R AML and other supported settings
• duration of therapy and discontinuation rates tied to response and tolerability

Competitive map by mechanism (high level)

Ivosidenib competes in the “IDH inhibitor” category and shares the same patient population defined by IDH mutation status, but not all competitors address the same mutation subtype. This matters because payers and formularies often consider the IDH inhibitor class as interchangeable at the mechanism level only when clinical profiles and outcomes align in the same line-of-therapy and regimen context.

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How is the market positioning shaped by IDH1 testing and sequencing?

Adoption dynamics

Commercial value for ivosidenib is sensitive to three system-level factors that influence whether the drug captures eligible patients:

1. Testing penetration: routine AML genomic profiling drives identification of IDH1 mutation-positive patients.
2. Sequencing behavior: clinicians choose targeted agents after or between standard regimens based on response kinetics, toxicity profile, and durability.
3. Treatment logistics: outpatient administration feasibility, lab monitoring intensity, and managing differentiation syndrome drive real-world persistence and discharge decisions.

Key “pull-through” lever: depth and durability

If clinical programs demonstrate:

• higher CR/CRh rates
• longer DoR
• improved MRD-negative conversion then uptake typically rises because these translate into stronger conversations with payers and clinicians on expected time on treatment and long-term outcomes.

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What do current market conditions imply for pricing and volume?

Pricing and access

Ivosidenib’s realized pricing depends on:

• formulary placement in AML specialized pathways
• managed entry agreements (where used)
• pharmacy benefit constraints tied to prior authorization and molecular test documentation
• competition from other IDH inhibitors and other AML targeted therapies

Volume sensitivity

Because eligibility is mutation-defined, volume grows primarily through:

• higher testing adoption
• earlier and broader placement within AML care pathways
• conversion of trial populations into real-world labeled populations

When payer access tightens, volume growth slows even if the clinical evidence expands.

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What is the competitive landscape and how does it affect ivosidenib forecasts?

Mechanism adjacency

Within AML targeted therapy, the competitive set includes:

• other IDH inhibitors (IDH1 and IDH2 classes)
• BCL-2 inhibition and FLT3/other targeted AML pathways in patients where those alterations are dominant
• combination regimens that may shift the standard-of-care away from monotherapy placement

Competitive effect on ivosidenib’s expected trajectory

Competition typically affects:

• Net price (discount pressure, preferred formulary status shifts)
• Share (movement of targeted-first sequencing)
• Duration of peak sales (as incremental utility claims converge)

The most durable advantage usually comes from trials that show differentiated OS or durable MRD outcomes in earlier lines.

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Market analysis and projection framework for ivosidenib

Base case drivers

A defensible forecast requires mapping sales to the patient funnel:

• AML incidence and IDH1 mutation-positive fraction
• eligible line-of-therapy share for labeled settings
• treatment persistence through response and tolerability
• conversion from real-world patient identification to therapy initiation

Forecast outcomes (directional)

• Bull case: label expansion or stronger durability data moves ivosidenib into earlier treatment lines, improving capture rate and persistence; payer access stays broad.
• Base case: steady capture in labeled R/R settings with modest share gains from testing growth; price erosion from within-class competition offsets some volume growth.
• Bear case: payer restrictions tighten; competition and combination standards reduce monotherapy share; clinical differentiation does not translate into durable switching behavior.

Key uncertainty that is economically determinative

Forecast sensitivity is highest to (1) label expansion timing and (2) durability claims that influence both clinician treatment sequencing and payer coverage.

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What is the projection time window and how should investors read it?

The value chain for ivosidenib is best understood in event-driven cycles:

• Clinical readout cycle: trial outcomes shift perceived utility and thus adoption.
• Regulatory cycle: label expansions open reimbursement breadth.
• Market cycle: competitor behavior (pricing, access, preferred formulary arrangements) changes net sales trajectories.

In practice, the highest valuation impact comes from confirmed label expansions or practice-changing results rather than early-phase or non-comparative findings.

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Key Takeaways

• Ivosidenib’s commercial footprint is anchored in IDH1-mutant AML, with growth dependent on testing penetration and how clinicians sequence targeted therapy in specific line-of-therapy settings.
• The clinical program’s economic impact is tied to durability and MRD-depth evidence that changes treatment placement and payer access.
• Market projections are most sensitive to label expansion and durability-driven adoption, with net pricing and within-class competition shaping the net sales curve.

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FAQs

1) What categories of endpoints most directly influence ivosidenib commercial uptake?

Endpoints linked to durable response and long-term outcomes such as CR/CRh, DoR, EFS, and OS, plus MRD-related depth of response.

2) What drives volume for ivosidenib more than new patient incidence?

Mutation detection and treatment sequencing, meaning molecular testing penetration and clinicians’ positioning of ivosidenib within AML line-of-therapy.

3) How does competition within targeted AML therapies impact forecasts?

It primarily affects net price through formulary preference and discounts, and it can reduce share if combination regimens shift standard sequencing away from monotherapy placement.

4) Why do label expansions matter more than incremental study results?

Label expansions change reimbursement scope and expand the eligible patient funnel, which translates into measurable volume capture.

5) What is the single most forecast-relevant clinical attribute?

Durability: evidence that improves DoR and long-term survival signals stronger expected benefit, which drives adoption and payer acceptance.

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References

[1] FDA. “Tibsovo (ivosidenib) Prescribing Information.” U.S. Food and Drug Administration.
[2] ClinicalTrials.gov. “Ivosidenib” trials listings and study records.
[3] EMA. “Tibsovo (ivosidenib) EPAR.” European Medicines Agency.
[4] National Cancer Institute (NCI). Targeted therapy resources and IDH1 inhibitor background (as applicable to ivosidenib context).