Positive Results Announced from Study of Olutasidenib for AML

Positive results have been announced from a Phase 2 trial of olutasidenib, an investigational selective mIDH1 inhibitor used in combination with azacitidine for the treatment of the mIDH1 form of acute myeloid leukemia (AML). These results were announced on December 13 at the 63rd American Society of Hematology (ASH) Annual Meeting, which you can learn more about here.

About AML

Firstly, it’s important to understand what exactly AML is. It is a rare form of cancer of the blood and bone marrow. AML causes the bone marrow to produce abnormal blood cells, which then crowd out healthy cells and causes the following symptoms:

  • Easy bruising
  • Lethargy
  • Fatigue
  • Fever
  • Pale skin
  • Shortness of breath
  • Bone pain
  • Unusual bleeding
  • Frequent infections

Medical professionals are unsure of the underlying causes of this cancer, but they do know of a few risk factors. These include radiation, chemotherapy, and exposure to certain chemicals. In terms of treatment, it varies based on a patient’s overall health, cancer stage, and patient preferences. Options include remission induction therapy, stem cell transplant, chemotherapy, and drug therapies.

About the Results

This trial aimed to evaluate olutasidenib in combination with azacitidine for patients with AML who have not experienced a durable response using previous therapies. If its results continue to be positive, olutasidenib could become a first or second-line treatment when used with a standard therapy.

Results were presented in both an oral and poster presentation over two days, and they include:

  • Olutasidenib plus azacitidine treatment resulted in complete remission (CR) or CR with partial hematologic recovery (CRh)
  • Olutasidenib was well-tolerated when used in combination with azacitidine
    • The safety profile remained consistent with that of olutasidenib’s alone
  • 25% or more of participants experienced treatment-emergent adverse events (TEAEs)
    • These included nausea, neutropenia, vomiting, thrombocytopenia, diarrhea, and constipation
  • Responses were recorded in all subtypes of IDH1 mutations
    • Lower baseline IDH1 expression patients were more likely to respond to olutasidenib alone than patients with higher expression

As AML patients are in need of viable treatment options that yield durable responses, the positive results of this trial are exciting so far. Hopefully, more will follow.

You can find the source article here.

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