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.

Clinical Trial Conditions for Ivosidenib

Condition Name

Condition Name for Ivosidenib
Intervention	Trials
Acute Myeloid Leukemia	7
Recurrent Acute Myeloid Leukemia	5
Refractory Acute Myeloid Leukemia	4
Myeloproliferative Neoplasm	3

Condition MeSH

Condition MeSH for Ivosidenib
Intervention	Trials
Leukemia, Myeloid, Acute	13
Leukemia, Myeloid	12
Leukemia	12
Myelodysplastic Syndromes	6

Clinical Trial Locations for Ivosidenib

Trials by Country

Trials by Country for Ivosidenib
Location	Trials
United States	92
Spain	14
Italy	11
Japan	10
France	10

Trials by US State

Trials by US State for Ivosidenib
Location	Trials
Texas	7
Ohio	6
New York	5
Minnesota	5
Massachusetts	4

Clinical Trial Progress for Ivosidenib

Clinical Trial Phase

Clinical Trial Phase for Ivosidenib
Clinical Trial Phase	Trials
Phase 3	6
Phase 2	8
Phase 1/Phase 2	3

Clinical Trial Status

Clinical Trial Status for Ivosidenib
Clinical Trial Phase	Trials
Not yet recruiting	11
Recruiting	10
Active, not recruiting	4

Clinical Trial Sponsors for Ivosidenib

Sponsor Name

Sponsor Name for Ivosidenib
Sponsor	Trials
Agios Pharmaceuticals, Inc.	7
National Cancer Institute (NCI)	6
M.D. Anderson Cancer Center	4

Sponsor Type

Sponsor Type for Ivosidenib
Sponsor	Trials
Other	31
Industry	22
NIH	6

Ivosidenib: Clinical Trials, Market Analysis, and Projections

Introduction to Ivosidenib

Ivosidenib, also known by its brand name TIBSOVO, is a first-in-class mutant isocitrate dehydrogenase 1 (mIDH1) inhibitor. It has been approved for use in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) who have the IDH1 mutation. Here, we will delve into the latest clinical trial updates, market analysis, and projections for this promising drug.

Clinical Trials Update

Phase 1 Substudy for MDS Patients

A recent phase 1 single-arm substudy focused on the use of ivosidenib in patients with relapsed/refractory (R/R) mIDH1 myelodysplastic syndrome (MDS) after failure of standard-of-care therapies. The study enrolled 19 patients, with 18 included in the efficacy analysis. The primary objectives were to determine safety, tolerability, and clinical activity. The results showed a complete remission (CR) + partial remission (PR) rate of 38.9% and an objective response rate (CR + PR + marrow CR) of 83.3%[1].

IDIOME Trial for MDS Patients

The IDIOME trial presented at the European Hematology Association 2024 conference provided updates on ivosidenib's efficacy in MDS patients. The trial included 48 patients with different risk levels of MDS. High-risk MDS patients who did not respond to previous azacitidine treatment achieved a response rate of 63.6%, while those who received ivosidenib as a first-line treatment had a response rate of 78.3%. For low-risk MDS patients who did not respond to previous erythropoietin treatment, 60% achieved complete remission[4].

Safety and Tolerability

Both studies highlighted that ivosidenib was generally well tolerated. Common side effects included differentiation syndrome, which was reversible in all cases. The drug demonstrated a manageable safety profile, with treatment-related adverse events occurring in approximately 42% of patients in the phase 1 substudy[1][4].

Market Analysis

Global Market Size and Growth

As of 2024, the global ivosidenib market size was estimated to be USD 18.5 million. This market is projected to expand at a compound annual growth rate (CAGR) of 4.20% from 2024 to 2031[2].

Regional Market Insights

North America dominated the market in 2024, accounting for around 40% of the global revenue, with a market size of USD 7.40 million. The region's focus on precision medicine and better treatment alternatives for AML, particularly for FLT3-mutated cases, drives this growth. The Middle East and Africa also show promising growth, with a CAGR of 3.9% from 2024 to 2031, due to increasing recognition of the drug's importance in treating FLT3-mutated AML[2].

Patient Segmentation

The market is segmented by patient type, with adults holding a prominent market share due to the high prevalence of AML in this demographic. Ivosidenib's effectiveness in treating relapsed or refractory AML with an IDH1 mutation is a key driver of this segment. The pediatric segment is emerging as a significant growth area due to the drug's potential efficacy in treating children with relapsed or refractory AML and IDH1 mutations[2].

Market Projections

Forecasted Market Size

By 2030, the global ivosidenib market is forecasted to reach USD 238.5 million, growing at a CAGR of 4.8% during the period from 2024 to 2030. This growth is driven by increasing awareness and diagnosis of FLT3-mutated AML, as well as the drug's efficacy in treating patients with IDH1 mutations[5].

Key Drivers

The market growth is driven by several factors, including:

• Precision Medicine: The increasing focus on precision medicine, especially in regions like North America, is driving the adoption of targeted therapies like ivosidenib.
• Genetic Awareness: Growing awareness and diagnosis of genetic mutations such as FLT3 and IDH1 in AML patients are fueling the demand for ivosidenib.
• Clinical Efficacy: The drug's demonstrated clinical efficacy in achieving remission and extending overall survival in patients with MDS and AML is a significant driver of market growth[2][4][5].

Conclusion

Ivosidenib has shown promising results in clinical trials for patients with MDS and AML, particularly those with IDH1 mutations. The drug's manageable safety profile and significant clinical activity make it a favorable treatment option. The global market for ivosidenib is expected to grow substantially, driven by increasing awareness of genetic mutations, the adoption of precision medicine, and the drug's efficacy in treating these conditions.

Key Takeaways

• Clinical Efficacy: Ivosidenib has demonstrated high response rates and durable remissions in patients with MDS and AML.
• Market Growth: The global ivosidenib market is projected to grow at a CAGR of 4.20% from 2024 to 2031.
• Regional Dominance: North America dominates the market, driven by a focus on precision medicine.
• Patient Segmentation: Adults are the primary market segment, but the pediatric segment is emerging as a significant growth area.
• Safety and Tolerability: The drug is generally well tolerated, with manageable side effects.

FAQs

What is ivosidenib used for?

Ivosidenib is used to treat patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) who have the IDH1 mutation.

What are the key findings from the phase 1 substudy for MDS patients?

The phase 1 substudy showed a complete remission (CR) + partial remission (PR) rate of 38.9% and an objective response rate of 83.3% in patients with R/R mIDH1 MDS[1].

How is the global ivosidenib market projected to grow?

The global ivosidenib market is projected to grow at a CAGR of 4.20% from 2024 to 2031, reaching USD 238.5 million by 2030[2][5].

What are the common side effects of ivosidenib?

Common side effects include differentiation syndrome, which is reversible in all cases. Treatment-related adverse events occurred in approximately 42% of patients[1][4].

Which region dominates the ivosidenib market?

North America dominates the market, accounting for around 40% of the global revenue, driven by a focus on precision medicine and better treatment alternatives for AML[2].

What is the significance of the IDIOME trial for ivosidenib?

The IDIOME trial showed high response rates and prolonged overall survival in MDS patients, particularly those who received ivosidenib as a first-line treatment, making it a potential first-line treatment option[4].

Sources

1. PubMed: Final phase 1 substudy results of ivosidenib for patients with mutant IDH1 myelodysplastic syndrome.
2. Cognitive Market Research: Global Ivosidenib Market Report 2024.
3. DelveInsight: TIBSOVO Market Forecast TIBSOVO drug Insight.
4. HealthTree: EHA 2024: Ivosidenib for High-Risk MDS Patients Updated Results.
5. Valuates Reports: Ivosidenib Drugs - Market, Report Size, Worth, Revenue, Growth.