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Last Updated: January 20, 2025

Investigational Drug Information for AG-120


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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 June 26th 2017.

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 twenty US patents protecting this investigational drug and two hundred and ninety-eight international patents.

Recent Clinical Trials for AG-120
TitleSponsorPhase
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 StudyAlice MimsPhase 2
Decitabine Alone or in Combination With Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib as Maintenance Therapy for the Treatment of Acute Myeloid Leukemia in RemissionNational 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 RemissionM.D. Anderson Cancer CenterPhase 1

See all AG-120 clinical trials

Clinical Trial Summary for AG-120

Top disease conditions for AG-120
trials0246810121416Leukemia, Myeloid, AcuteLeukemia, MyeloidLeukemiaMyelodysplastic Syndromes[disabled in preview]
Top clinical trial sponsors for AG-120
trials0246810121416Agios Pharmaceuticals, Inc.National Cancer Institute (NCI)Institut de Recherches Internationales Servier[disabled in preview]

See all AG-120 clinical trials

US Patents for AG-120

DrugnamePatent NumberPatent TitlePatent AssigneeEstimated Expiration
AG-120 ⤷  Subscribe Methods of treatment of malignancies Celgene Corporation (Summit, NJ) Agios Pharmaceuticals, Inc. (Cambridge, MA) ⤷  Subscribe
AG-120 ⤷  Subscribe Controlled release dosage form TRIASTEK, INC. (Nanjing, CN) ⤷  Subscribe
AG-120 ⤷  Subscribe Methods of treatment of malignancies Agios Pharmaceuticals, Inc. (Cambridge, MA) ⤷  Subscribe
AG-120 ⤷  Subscribe Pharmaceutical compositions of therapeutically active compounds Agios Pharmaceuticals, Inc. (Cambridge, MA) ⤷  Subscribe
>Drugname>Patent Number>Patent Title>Patent Assignee>Estimated Expiration
Showing 1 to 4 of 4 entries

International Patents for AG-120

DrugnameCountryDocument NumberEstimated ExpirationRelated US Patent
AG-120 Australia AU2016393869 2036-02-26 ⤷  Subscribe
AG-120 Australia AU2016393870 2036-02-26 ⤷  Subscribe
AG-120 Canada CA3015753 2036-02-26 ⤷  Subscribe
AG-120 Canada CA3015757 2036-02-26 ⤷  Subscribe
>Drugname>Country>Document Number>Estimated Expiration>Related US Patent
Showing 1 to 4 of 4 entries

AG-120 (Ivosidenib): A Groundbreaking IDH1 Mutant Inhibitor in Cancer Therapy

Introduction to AG-120

AG-120, also known as ivosidenib, is a pioneering oral small molecule inhibitor developed by Agios Pharmaceuticals, specifically targeting the mutated IDH1 enzyme. This drug has revolutionized the treatment landscape for patients with IDH1-mutant positive cancers, particularly those with acute myeloid leukemia (AML) and other hematologic malignancies.

Mechanism of Action and Clinical Significance

AG-120 works by inhibiting the mutated IDH1 enzyme, which is implicated in the production of the oncometabolite 2-hydroxyglutarate (2-HG). This oncometabolite interferes with cellular metabolism and epigenetics, leading to the proliferation of cancer cells. By blocking this enzyme, AG-120 restores normal cellular function and induces differentiation of cancer cells into mature blood cells[4].

Clinical Trials and Efficacy Data

The efficacy of AG-120 has been extensively evaluated in clinical trials. In an ongoing Phase 1 trial, AG-120 demonstrated durable clinical activity in patients with advanced hematologic malignancies, including AML. The trial reported an overall response rate of 31% among response-evaluable patients, with responses lasting up to 11 months. This included complete remissions, complete remissions with incomplete platelet recovery, marrow complete remissions, and partial remissions[1].

In more detailed clinical trials, AG-120 showed an overall response rate of 42% and a complete response rate of 22% in patients with IDH1-mutant AML. The recommended dose of ivosidenib is 500 mg per day, and it has been approved by the US FDA for the treatment of relapsed or refractory AML with an IDH1 mutation[4].

Safety and Tolerability

The safety profile of AG-120 has been favorable. In the Phase 1 trial, the majority of adverse events were mild to moderate, with common side effects including fatigue, diarrhea, pyrexia, and nausea. Serious adverse events were mostly disease-related, and no maximum tolerated dose was reached. There were 13 deaths reported, but all were considered unrelated to AG-120[1].

Market Projections and Growth

The market for IDH inhibitors, led by AG-120, is projected to experience significant growth. The global IDH inhibitors market is expected to expand from $1.7 billion in 2022 to $39.16 billion by 2033, with a compound annual growth rate (CAGR) of 33% from 2023 to 2033. This growth is driven by the development of more potent and selective IDH inhibitors and their expanding use into other cancer types[2].

Key Market Indicators

  • Market Size: Expected to reach $39.16 billion by 2033.
  • CAGR: 33% from 2023 to 2033.
  • Regional Dominance: North America is expected to hold 46% of the market share, followed by Europe with 43% in 2023[2].

Upcoming Milestones and Expansion

Agios Pharmaceuticals and its collaborators, including Bayer, are planning several key milestones for AG-120:

  • Expansion Cohorts: Initiating three expansion cohorts to evaluate AG-120 in 175 patients with IDH1-mutated advanced hematologic malignancies.
  • Global Registration-Enabling Study: Planning to initiate a global registration-enabling Phase 3 study in AML patients with an IDH1 mutation in the first half of 2016.
  • Combination Trials: Beginning combination trials to evaluate AG-120 as a potential frontline treatment for IDH1-mutated AML and other hematologic malignancies[1].

Collaboration and Development

AG-120 is part of a global strategic collaboration between Agios Pharmaceuticals and Celgene. This collaboration includes the development of other IDH inhibitors such as AG-221 and AG-881. The joint development and commercialization agreement with Bayer for ivosidenib further strengthens the market position of AG-120[1].

Future Perspectives

The future of AG-120 looks promising, with ongoing and planned trials in various solid tumors, including glioma, cholangiocarcinoma, and chondrosarcomas. The drug's potential to treat glioma, given its brain penetration capabilities, is particularly noteworthy[3].

Quote from Industry Expert

"The durable clinical activity observed with AG-120 in such a refractory patient population is impressive," said Stéphane de Botton, M.D., principal investigator at the Institut de Cancérologie. "These encouraging data represent the tremendous progress to date in our AG-120 program, as this therapy is proving to be well tolerated and effective"[1].

Key Takeaways

  • Efficacy: AG-120 has shown a significant response rate in patients with IDH1-mutant AML and other hematologic malignancies.
  • Safety: The drug has a favorable safety profile with mostly mild to moderate adverse events.
  • Market Growth: The IDH inhibitors market, led by AG-120, is projected to grow significantly, reaching $39.16 billion by 2033.
  • Upcoming Milestones: Expansion cohorts, global registration-enabling studies, and combination trials are planned.
  • Collaboration: Agios Pharmaceuticals is collaborating with Celgene and Bayer for the development and commercialization of AG-120.

FAQs

What is AG-120 and how does it work?

AG-120, or ivosidenib, is an oral small molecule inhibitor that targets the mutated IDH1 enzyme, preventing the production of the oncometabolite 2-HG and restoring normal cellular function.

What are the common side effects of AG-120?

Common side effects include fatigue, diarrhea, pyrexia, and nausea, with most adverse events being mild to moderate.

What is the current market projection for IDH inhibitors?

The global IDH inhibitors market is expected to grow from $1.7 billion in 2022 to $39.16 billion by 2033, with a CAGR of 33% from 2023 to 2033.

Which companies are involved in the development and commercialization of AG-120?

Agios Pharmaceuticals is collaborating with Celgene and Bayer for the development and commercialization of AG-120.

What are the upcoming milestones for AG-120?

Initiation of three expansion cohorts, a global registration-enabling Phase 3 study, and combination trials are planned for AG-120.

Is AG-120 approved by regulatory authorities?

Yes, AG-120 (ivosidenib) has been approved by the US FDA for the treatment of relapsed or refractory AML with an IDH1 mutation.

Sources

  1. Agios Pharmaceuticals, Inc. - "Agios Announces New Data from Ongoing Phase 1 Trial of AG-120 Showing Durable Clinical Activity in Patients with Advanced Hematologic Malignancies"[1].
  2. PharmiWeb - "Isocitrate Dehydrogenase (IDH) Inhibitors Market Secures Remarkable Valuation of US$39.16 Billion in 2033"[2].
  3. Flagship Pioneering - "Agios Presents Phase 1 Data from Dose Escalation and Expansion Cohorts of AG-120 (Ivosidenib) in Patients with Previously Treated IDH1 Mutant Positive Cholangiocarcinoma"[3].
  4. Frontiers in Pharmacology - "Recent advances of IDH1 mutant inhibitor in cancer therapy"[4].
  5. PR Newswire - "Isocitrate Dehydrogenase (IDH) Inhibitors Market Study 2020 with Sales Forecast to 2030"[5].

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