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Enasidenib Mesilate

Enasidenib Mesylate, also known as Idhifa, is being studied in several clinical trials as a potential treatment for acute myeloid leukemia (AML), particularly in cases with IDH2 gene mutations. These trials are exploring its use alone and in combination with other drugs for both newly diagnosed and relapsed/refractory AML patients. The studies aim to evaluate the safety, efficacy, and optimal dosing of enasidenib-based regimens in various AML patient populations.

Table of Contents

What is Enasidenib?

Enasidenib Mesylate, also known by its brand name Idhifa, is a medication used in the treatment of certain types of blood cancers[1]. It belongs to a class of drugs called targeted therapies, which are designed to attack specific cancer cells while minimizing damage to healthy cells. Enasidenib is also referred to by several other names, including AG-221 Mesylate, CC-90007, and Enasidenib Methanesulfonate[2].

How Does Enasidenib Work?

Enasidenib works by targeting a specific genetic mutation found in some cancer cells. This mutation affects a protein called IDH2 (isocitrate dehydrogenase-2). In patients with this mutation, the IDH2 protein doesn’t function correctly, leading to the growth of cancer cells. Enasidenib blocks the abnormal IDH2 protein, which may help stop or slow down the growth of cancer cells[1].

What Conditions Does Enasidenib Treat?

Enasidenib is primarily used to treat a type of blood cancer called Acute Myeloid Leukemia (AML) in patients who have a specific IDH2 mutation. It’s particularly useful in the following situations:

  • Relapsed or Refractory AML: This refers to AML that has either come back after treatment (relapsed) or hasn’t responded well to initial treatments (refractory)[2].
  • AML in patients who have undergone stem cell transplantation: Enasidenib is being studied as a maintenance therapy for patients who have received a donor stem cell transplant[1].
  • Newly diagnosed AML: Some studies are exploring the use of Enasidenib in combination with other drugs for patients newly diagnosed with AML[3].

Current Clinical Trials

Several clinical trials are currently underway to further investigate the effectiveness of Enasidenib in various scenarios:

  • As a maintenance therapy after stem cell transplantation[1].
  • In combination with other drugs like Cobimetinib for relapsed or refractory AML[2].
  • Combined with Azacitidine for relapsed or refractory AML[3].
  • As part of a combination therapy with CPX-351 (a chemotherapy drug) for relapsed AML[4].
  • In a study comparing different treatment combinations for relapsed AML after stem cell transplantation[5].

How is Enasidenib Administered?

Enasidenib is typically taken orally (by mouth) once daily. The exact dosage and duration of treatment can vary depending on the specific condition being treated and the individual patient’s response. In most clinical trials, Enasidenib is given in cycles, with each cycle lasting 28 days[1][2].

Potential Side Effects

As with any medication, Enasidenib can cause side effects. The most common and serious side effects are still being studied in clinical trials. One particular side effect that doctors watch for is called IDH-inhibitor related differentiation syndrome (IDH-DS), which can cause fever, difficulty breathing, and other symptoms[3]. It’s important for patients to report any unusual symptoms to their healthcare provider promptly.

Combination Therapies

Researchers are exploring the use of Enasidenib in combination with other cancer treatments to potentially improve outcomes for patients. Some of these combinations include:

  • Enasidenib with Cobimetinib (another targeted therapy)[2].
  • Enasidenib with Azacitidine (a chemotherapy drug)[3].
  • Enasidenib with CPX-351 (a combination chemotherapy drug)[4].
  • Enasidenib with Glasdegib (another targeted therapy)[5].

These combination therapies are being studied to determine if they can provide better results than using Enasidenib alone.

Trial Aspect Details
Patient Populations Newly diagnosed AML, Relapsed/Refractory AML, Post-transplant AML
Enasidenib Combinations Monotherapy, With azacitidine, With venetoclax and decitabine, With glasdegib
Key Outcomes Measured Safety, Efficacy, Remission rates, Overall survival, Pharmacokinetics
Biomarker Assessments IDH2 mutation clearance, Minimal residual disease, Leukemia stem cell burden
Administration Route Oral, daily dosing
Trial Phases Phase 1b, Phase 2

FAQ

  • What is Enasidenib Mesylate? Enasidenib Mesylate, also known as Idhifa, is a medication that targets and inhibits mutated IDH2 enzymes in cancer cells. It is being studied as a treatment for acute myeloid leukemia (AML) patients who have specific genetic mutations.
  • How does Enasidenib work in treating AML? Enasidenib works by blocking mutated IDH2 enzymes, which are involved in the growth of some AML cells. By inhibiting these enzymes, enasidenib may stop or slow down the growth of cancer cells in patients with IDH2 mutations.
  • What types of AML patients are being studied in these clinical trials? The clinical trials are studying enasidenib in various AML patient groups, including those with newly diagnosed AML, relapsed or refractory AML, and AML patients who have undergone stem cell transplantation. Some trials focus specifically on patients with IDH2 gene mutations.
  • Is Enasidenib being studied alone or in combination with other drugs? Enasidenib is being studied both as a single agent and in combination with other drugs. Some trials are exploring its use with chemotherapy drugs like azacitidine, while others are combining it with targeted therapies like venetoclax or glasdegib.
  • What are the main goals of these clinical trials? The main goals of these clinical trials include assessing the safety and effectiveness of enasidenib, determining the best dosing regimens, evaluating its impact on patient survival and remission rates, and studying how it affects specific biomarkers related to AML and IDH2 mutations.

Ongoing Clinical Trials on Enasidenib Mesilate

  • Study on Everolimus and Drug Combination for Children with Relapsed or Refractory Pediatric Cancer

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Denmark France Italy The Netherlands Spain

  • Study on the Effectiveness of Enasidenib Mesilate in Patients with Myelodysplastic Syndrome with IDH2 Mutation

    Not recruiting

    1 1 1
    Investigated drugs:
    France Germany

  • Study on the Safety and Effectiveness of Ivosidenib or Enasidenib with Azacitidine for Patients with Newly Diagnosed Acute Myeloid Leukemia with IDH1 or IDH2 Mutations

    Not recruiting

    1 1 1
    Investigated drugs:
    France Germany Italy The Netherlands

  • Study of Ivosidenib or Enasidenib with Chemotherapy for Patients with Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with IDH1 or IDH2 Mutation

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Estonia Finland France Germany +6

Glossary

  • Acute Myeloid Leukemia (AML): A type of cancer that affects the blood and bone marrow, characterized by the rapid growth of abnormal white blood cells that interfere with the production of normal blood cells.
  • IDH2 Mutation: A genetic change in the IDH2 gene, which is involved in cellular metabolism. This mutation is found in some cases of AML and is the target of enasidenib treatment.
  • Relapsed AML: AML that has returned after a period of improvement or remission following initial treatment.
  • Refractory AML: AML that does not respond to standard treatments or stops responding after initial improvement.
  • Hematopoietic Cell Transplantation (HCT): A procedure where healthy blood-forming stem cells are transplanted to replace diseased or damaged bone marrow. Also known as stem cell transplant.
  • Complete Remission (CR): A state where there is no evidence of disease and blood cell counts have returned to normal levels.
  • Minimal Residual Disease (MRD): Small numbers of cancer cells that remain in the body during or after treatment, often detectable only by very sensitive tests.
  • Pharmacokinetics: The study of how a drug is absorbed, distributed, metabolized, and eliminated by the body.
  • Dose-Limiting Toxicity: Side effects of a treatment that are severe enough to prevent an increase in dosage or require a reduction in dosage.
  • Overall Response Rate (ORR): The proportion of patients whose cancer shrinks or disappears after treatment.

References

  1. https://clinicaltrials.gov/study/NCT03728335
  2. https://clinicaltrials.gov/study/NCT05441514
  3. https://clinicaltrials.gov/study/NCT03683433
  4. https://clinicaltrials.gov/study/NCT03825796
  5. https://clinicaltrials.gov/study/NCT04655391