Investigational Drug Information for AG-120

What is the development status for investigational drug AG-120?

AG-120 is an investigational drug.

There have been 28 clinical trials for AG-120. The most recent clinical trial was a Phase 3 trial, which was initiated on March 1^st 2019.

The most common disease conditions in clinical trials are Leukemia, Myeloid, Acute, Leukemia, Myeloid, and Leukemia. The leading clinical trial sponsors are Agios Pharmaceuticals, Inc., National Cancer Institute (NCI), and Institut de Recherches Internationales Servier.

There are two hundred and twenty-nine US patents protecting this investigational drug and three hundred and nine international patents.

Summary for AG-120

US Patents	229
International Patents	309
US Patent Applications	763
WIPO Patent Applications	502
Japanese Patent Applications	21
Clinical Trial Progress	Phase 3 (2019-03-01)
Vendors	54

Recent Clinical Trials for AG-120

Title	Sponsor	Phase
Ivosidenib or Enasidenib Followed by Azacitidine Plus Venetoclax or Azacitidine Plus Venetoclax Followed by Ivosidenib or Enasidenib for the Treatment of IDH Mutated Acute Myeloid Leukemia in Older Patients. The I-DATA Study	Alice Mims	Phase 2
Decitabine Alone or in Combination With Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib as Maintenance Therapy for the Treatment of Acute Myeloid Leukemia in Remission	National Cancer Institute (NCI)	Phase 1
Decitabine Alone or in Combination With Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib as Maintenance Therapy for the Treatment of Acute Myeloid Leukemia in Remission	M.D. Anderson Cancer Center	Phase 1

See all AG-120 clinical trials

Clinical Trial Summary for AG-120

Top disease conditions for AG-120

Top clinical trial sponsors for AG-120

See all AG-120 clinical trials

US Patents for AG-120

Drugname	Patent Number	Patent Title	Patent Assignee	Estimated Expiration
AG-120		Methods of treatment of malignancies	Celgene Corporation (Summit, NJ) Agios Pharmaceuticals, Inc. (Cambridge, MA)
AG-120		Controlled release dosage form	TRIASTEK, INC. (Nanjing, CN)
AG-120		Methods of treatment of malignancies	Agios Pharmaceuticals, Inc. (Cambridge, MA)

International Patents for AG-120

Drugname	Country	Document Number	Estimated Expiration	Related US Patent
AG-120	Australia	AU2016393869	2036-02-26
AG-120	Australia	AU2016393870	2036-02-26
AG-120	Canada	CA3015753	2036-02-26

AG-120 (Ivosidenib): Development Status and Market Outlook

Last updated: February 16, 2026

Development Update

AG-120, marketed as Ivosidenib, is a targeted therapy developed by Agios Pharmaceuticals. It selectively inhibits mutant isocitrate dehydrogenase 1 (IDH1), an enzyme involved in abnormal cellular metabolism in certain cancers.

• FDA Approval: Ivosidenib received FDA approval in 2018 for treating IDH1-mutant acute myeloid leukemia (AML) in adults who are unfit for induction chemotherapy. The approval is based on Phase 1 and Phase 2 trial data demonstrating response rates of approximately 30-40% in relapsed/refractory AML patients with IDH1 mutations.[1]

• Complementary Indications: Recently, Ivosidenib gained approval for newly diagnosed AML in adult patients who are not candidates for intensive chemotherapy, reflecting its expanded use.

• Ongoing Trials: Multiple Phase 3 studies are underway:

  • NCT03530966: Evaluating Ivosidenib in combination with azacitidine for newly diagnosed AML.
  • NCT03744810: Testing Ivosidenib alongside chemotherapy in untreated AML.
  • NCT02975249: Investigating Ivosidenib in cholangiocarcinoma with IDH1 mutations.

• Pipeline Expansion: Ivosidenib is under investigation for additional cancers with IDH1 mutations, including gliomas, chondrosarcomas, and solid tumors. Trials for glioma (NCT03684811) show promise, though not yet approved for these indications.

• Pricing and Sales: Since launch, Ivosidenib’s annual revenue exceeded $250 million (2022), driven by AML indications and expanding labels in oncology.

Market Projection

• The global AML treatment market size was valued at $1.2 billion in 2022 and is projected to grow at a compound annual growth rate (CAGR) of 7% through 2030.[2]

• Target Population: Approximately 20,000 AML cases annually in the U.S., with an estimated 40% harboring IDH1 mutations (around 8,000 patients), making Ivosidenib applicable for roughly 6,400 patients annually in the U.S. alone.

• Market Penetration: Current share includes frontline approval and off-label use for relapsed/refractory AML. As clinical trials confirm efficacy in broader populations, including combination therapies, sales could surpass $1 billion globally by 2030.

• Competitive Landscape:

  • IDH2 inhibitors like Enasidenib hold similar markets for IDH2-mutant AML.
  • Other targeted therapies include FLT3 inhibitors (e.g., Midostaurin), and emerging combination regimens could diminish Ivosidenib’s market share.

• Pricing Trends: Approximate wholesale acquisition cost (WAC) per month is $27,000. Contracting, rebates, and insurance impact net revenue.

• Market Strategies:

  • Expansion into solid tumors.
  • Combination regimens with existing chemotherapies.
  • Biomarker-driven patient selection to optimize efficacy.

Regulatory Outlook & Future Developments

• Continued approval extensions for different AML subpopulations.
• Trials in solid tumors aim to expand indications.
• Potential combination therapies could increase overall market share.

Citations

[1] FDA. (2018). "Ivosidenib for AML."
[2] Grand View Research. (2022). "AML Treatment Market Size & Trends."

Key Takeaways

• Ivosidenib remains a targeted therapy for IDH1-mutant AML with ongoing trials expanding its label.
• The AML market is growing, driven by new approvals and combination therapies.
• Estimated annual sales could reach $1 billion by 2030, contingent on expanding indications and clinical success.
• Competition from other IDH inhibitors and combination regimens could influence market share.
• The pipeline expansion into solid tumors offers upside, but regulatory approvals are pending.

FAQs

1. What is the approval status of Ivosidenib outside AML?
2. How does Ivosidenib compare to IDH2 inhibitors in efficacy?
3. What combination therapies are being tested with Ivosidenib?
4. What are the main adverse effects associated with Ivosidenib?
5. How will trial outcomes impact future market potential?