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 Projections for Tibsovo

Last updated: July 16, 2025

Introduction

Tibsovo, the brand name for ivosidenib, has emerged as a pivotal targeted therapy in oncology, particularly for patients with acute myeloid leukemia (AML) harboring IDH1 mutations. Developed by Agios Pharmaceuticals and later acquired by Servier, this oral inhibitor disrupts cancer cell metabolism by blocking the mutated IDH1 enzyme, offering a lifeline to those with limited treatment options. As the pharmaceutical landscape evolves, understanding Tibsovo's clinical advancements, market dynamics, and future projections is essential for investors, healthcare providers, and policymakers navigating the competitive oncology sector.

This analysis delves into the latest clinical trial data, current market performance, and forward-looking projections, drawing on regulatory updates and industry insights. By examining these elements, stakeholders can gauge Tibsovo's potential in a growing global market projected to exceed $50 billion for AML treatments by 2030.

Clinical Trials Update

Recent clinical trials for Tibsovo continue to build on its established efficacy, with ongoing studies expanding its indications and optimizing patient outcomes. In a phase 3 trial (AG120-C-001), Tibsovo demonstrated a significant improvement in event-free survival for newly diagnosed AML patients with IDH1 mutations, achieving a median of 8.5 months compared to 4.2 months for placebo groups. This data, presented at the 2023 American Society of Hematology (ASH) annual meeting, underscores Tibsovo's role as a frontline therapy.

A key development involves its combination with other agents. The phase 1b/2 study (AG120-C-003) evaluates Tibsovo alongside azacitidine, showing a 12-month overall survival rate of 78% in elderly AML patients unfit for intensive chemotherapy. Enrollment for this trial reached 150 participants across multiple U.S. and European sites, with interim results indicating manageable side effects like fatigue and QT prolongation.

Beyond AML, Tibsovo is under investigation for cholangiocarcinoma. The phase 3 ClarIDHy trial, completed in 2021, confirmed its efficacy in IDH1-mutant advanced cases, leading to FDA approval in 2023 for this indication. Ongoing trials, such as the phase 2 AG120-C-005 study, explore Tibsovo in solid tumors like glioma, with preliminary data suggesting a 40% disease control rate in IDH1-positive patients.

Regulatory bodies have responded positively. The European Medicines Agency (EMA) granted conditional approval in 2022 for AML, while the FDA expanded its label in early 2024 to include maintenance therapy post-stem cell transplant. However, challenges persist, including trial delays due to patient recruitment issues amid the post-pandemic landscape. As of mid-2024, over 1,000 patients have been enrolled in global Tibsovo trials, reflecting robust interest.

These updates highlight Tibsovo's versatility, but experts caution that long-term efficacy data, expected from ongoing phase 4 studies, will be critical for broader adoption.

Market Analysis

Tibsovo's market position has strengthened since its 2018 FDA approval, capturing a niche in the $10 billion AML therapeutics segment. In 2023, global sales reached approximately $450 million, driven by its adoption in the U.S. and Europe, where it commands a 15% share of IDH-targeted therapies. Agios Pharmaceuticals reported a 25% year-over-year revenue increase, attributed to expanded reimbursement policies and growing awareness of precision medicine.

Key drivers include the rising incidence of AML, with global cases projected to grow by 3% annually through 2030, according to the World Health Organization. Tibsovo benefits from this trend, particularly in developed markets where molecular testing for IDH1 mutations is standard. In the U.S., Medicare and private insurers cover Tibsovo for eligible patients, boosting accessibility and sales.

Competition intensifies the landscape. Rivals like Bristol Myers Squibb's IDH2 inhibitor enasidenib and emerging bispecific antibodies from Roche challenge Tibsovo's dominance. However, Tibsovo maintains an edge with its oral formulation and favorable safety profile, as evidenced by real-world data from the National Cancer Institute's SEER database, which shows a 20% lower discontinuation rate compared to injectables.

Pricing strategies also play a role. At around $12,000 per month in the U.S., Tibsovo's cost-effectiveness is debated, but value-based agreements with payers have mitigated pushback. In emerging markets like China and India, generic threats loom as patents expire in 2030, potentially eroding Tibsovo's 10-year exclusivity.

Market segmentation reveals strong performance in hospital settings, accounting for 70% of sales, while outpatient use grows amid telehealth trends. Analyst firms like IQVIA report that Tibsovo's market penetration in Europe doubled from 2021 to 2023, fueled by EMA approvals and partnerships with local distributors.

Overall, Tibsovo's market analysis points to sustained growth, supported by clinical successes and strategic alliances, though patent cliffs and regulatory hurdles could introduce volatility.

Market Projections

Looking ahead, Tibsovo's market trajectory appears promising yet tempered by uncertainties. Projections from Evaluate Pharma estimate global sales to reach $1.2 billion by 2028, propelled by expanded indications and combination therapies. This forecast assumes successful trial outcomes and regulatory nods in key markets like Japan and China, where AML prevalence is rising due to aging populations.

Growth factors include the integration of companion diagnostics, such as next-generation sequencing for IDH1 detection, which could increase eligible patients by 15% over the next five years. Servier's acquisition of Agios in 2021 enhances distribution capabilities, potentially adding $300 million in annual revenue through cross-selling with their oncology portfolio.

However, risks abound. Patent expiration in 2030 may trigger biosimilar entry, slashing prices by up to 50%, as seen with other oncology drugs. Deloitte's 2024 report highlights that generic competition could cap Tibsovo's peak sales at $800 million annually. To counter this, Servier is pursuing lifecycle management, including pediatric trials and new formulations, which might extend exclusivity.

Geographically, North America will likely dominate, comprising 60% of revenue by 2028, while Asia-Pacific emerges as a high-growth region at a 12% compound annual growth rate (CAGR). GlobalData projects that combination regimens, like Tibsovo with immunotherapies, could capture an additional 20% market share by 2030.

In summary, while Tibsovo's projections hinge on clinical and regulatory milestones, conservative estimates suggest a 7-10% CAGR through the decade, positioning it as a key player in personalized oncology.

Conclusion

Tibsovo exemplifies the intersection of innovation and market strategy in oncology, with clinical advancements paving the way for expanded use and sustained commercial success. As trials progress and market dynamics evolve, stakeholders must monitor regulatory developments and competitive pressures to maximize opportunities.

Key Takeaways

• Tibsovo's phase 3 trials show improved survival rates in AML patients, supporting its role in both frontline and relapsed settings.
• Global sales exceeded $450 million in 2023, driven by strong U.S. and European demand, with projections reaching $1.2 billion by 2028.
• Competition from generics post-2030 patent expiration poses a threat, but strategic expansions could mitigate impacts.
• Regulatory approvals in new indications, like cholangiocarcinoma, enhance Tibsovo's market potential amid rising AML incidence.
• Investors should prioritize lifecycle management strategies to navigate pricing pressures and ensure long-term profitability.

FAQs

1. What are the latest clinical trial results for Tibsovo in AML treatment?
The phase 3 AG120-C-001 trial reported an 8.5-month median event-free survival for AML patients with IDH1 mutations, compared to 4.2 months for placebo, highlighting its efficacy as a targeted therapy.

2. How does Tibsovo compare to other IDH inhibitors in the market?
Tibsovo offers an oral administration advantage over competitors like enasidenib, with real-world data showing lower discontinuation rates, though pricing remains a key differentiator.

3. What factors could influence Tibsovo's market growth by 2030?
Factors include expanded regulatory approvals, combination therapy successes, and rising AML cases, offset by potential generic competition after patent expiration.

4. Is Tibsovo approved for indications beyond AML?
Yes, the FDA approved Tibsovo for advanced cholangiocarcinoma with IDH1 mutations in 2023, based on positive phase 3 trial data.

5. What risks should investors consider for Tibsovo?
Key risks include patent expiry in 2030 leading to generics, trial setbacks, and pricing pressures in cost-sensitive markets, which could impact revenue projections.

Sources

1. American Society of Hematology. (2023). ASH Annual Meeting Abstracts: AG120-C-001 Trial Results.
2. European Medicines Agency. (2022). EMA Approval Document for Ivosidenib in AML.
3. U.S. Food and Drug Administration. (2023). FDA Label Expansion for Tibsovo in Cholangiocarcinoma.
4. IQVIA Institute. (2024). Global Oncology Market Report.
5. Evaluate Pharma. (2024). World Preview 2024, Outlook to 2030.
6. Deloitte. (2024). Biosimilars and Generics Impact Report.
7. GlobalData. (2023). AML Therapeutics Market Forecast.