Highlights from EHA 2025 – Updates in AML

June 25, 2025

Updates in AML from the European Hematology Association (EHA) congress 2025

What is venetoclax?

Venetoclax is a targeted cancer medicine used to treat acute myeloid leukemia (AML). It blocks a protein called BCL-2, which helps leukemia cells survive. By turning off this protein, venetoclax helps destroy cancer cells.

It’s often combined with other treatments like azacitidine or decitabine, especially for people who can’t have intensive chemotherapy. This combination can lead to remission (no signs of cancer in the blood or bone marrow) and is usually easier to tolerate than standard treatments.

Below are highlights from the EHA 2025 Annual Meeting, summarizing key findings about the safety and effectiveness of new treatment combinations for people with AML.

 

New tablet-only treatment combination of decitabine-cedazuridine with venetoclax1

What was the study about?

This early study (phase I/II) tested a new tablet-only treatment combination of decitabine-cedazuridine and venetoclax for people recently diagnosed with AML. The goal was to offer an effective treatment option that people could take at home—reducing the need for regular clinic visits.

Study overview

  • 189 people with AML took part in this study, average age 78 years
  • The people in this study were either aged 75 or older, or had other health conditions that meant they couldn’t safely receive intensive chemotherapy

 

 Key results

Results are from the phase IIB section of this study, which looks at how well the treatment works after earlier studies have looked at its possible risks and how it behaves in the body. This part of the study included 101 people.

  • 5% of people had little to no signs of cancer in their blood or bone marrow (complete remission)
  • 80% of people stayed in remission for at least 9 months
  • On average people lived for 15.5 months after treatment
  • Of those tested, 55.1% had no signs of cancer with very sensitive tests (called measurable residual disease negative)

 

Key safety information

The most common side effect from treatment was low levels of blood cells. This was an expected side effect from the treatment.

Other side effects included:

  • 19% of people felt sick
  • 4% had a reduced appetite
  • 7% had constipation

 

After 1 month, 3% of people died and this increased to 10% by month 2.

What does this study mean for people with AML?

The results of this study are encouraging. About half of people treated had no detectable signs of cancer in their blood or bone marrow after treatment (remission). The side effects were as expected and were considered manageable. While this treatment combination is still being tested and is not available yet, it may offer a more convenient, at-home option for people newly diagnosed with AML, helping to reduce how often they need to visit the clinic.

 

Bleximenib with venetoclax and azacitidine2

What was this study about?

This very early-stage study (phase IB) tested different doses of bleximenib, a type of medicine called a menin inhibitor, combined with venetoclax and azacitidine in people with AML. Menin inhibitors are a new type of treatment being studied for AML. They work by blocking a protein called menin, which helps certain leukemia cells grow. This study also looked at how people with specific genetic changes (called NPM1 mutations or KT2A rearrangements) would respond to treatment.

Study overview

  • 125 people with AML, average age 67 years old
  • 40 had newly-diagnosed AML
  • 85 had previously treated AML that had come back (relapsed) or hadn’t improved with other treatment (refractory)

 

Key results

  • The most effective dose of bleximenib was 100 mg twice a day
  • Newly diagnosed group: 75% of people had no signs of leukemia in their blood or bone marrow after treatment (complete remission)
  • Previously treated: 59% had no signs of leukemia in their blood or bone marrow after treatment (complete remission)
  • Treatment worked better in people who had never taken venetoclax before compared with those who had used it before (73% had remission, compared with 29% of those who had taken venetoclax previously)
  • The treatment also worked well in people with NPM1 mutations (m) and KMT2A rearrangements (r)
    • For the newly diagnosed group: 75% of both people with KMT2Ar, and NPM1m achieved complete remission (no signs of leukemia in the blood or bone marrow
    • For the previously treated group: 50% of people with KMT2Ar and 67% of people with NPM1m achieved complete remission

 

Key safety information

The 3 most common side effects with 100 mg dose were:

  • 65% felt sick
  • 61% had low platelets which are involved in blood clotting (thrombocytopenia)
  • 49% had low red blood cells (anemia)

Serious side effects such as changes to heart rhythm (3 cases) and a potentially severe condition called differentiation syndrome (2 cases) were rare.

What does this mean for people with AML?

The study results are positive, with 75% of people with newly diagnosed AML having no signs of cancer after treatment with bleximenib. People who had been treated before also saw a positive result with 60% having no signs of cancer after treatment. The side effects were consistent with what was expected for venetoclax and azacitidine together. As a result, this treatment combination is now moving to a larger phase III trial (called cAMeLot–2) for people who are newly diagnosed with AML but can’t have intensive chemotherapy.

 

Tuspentib with venetoclax and azacitidine3

What was this study about?

This early-stage study (phase I/II) tested different doses of a new medicine, tuspetinib, combined with venetoclax and azacitidine. Tuspetinib which is given as a tablet, blocks enzymes that help cancer grow. The study involved people with newly diagnosed AML who couldn’t have intensive chemotherapy.

Study overview

  • 10 people have been enrolled on this study so far, average age 75.5 years
  • Tuspetinib doses tested: 120 mg, 80 mg and 40 mg

 

Key results

  • Effectiveness data is available for 7 people who received the 40 mg and 80 mg doses
  • 80 mg group: All 3 people had no signs of cancer in their blood or bone marrow (complete remission)
  • 40 mg group: 3 out of 4 people had complete remission

 

Important safety information

The top 3 most common side effects from treatment were:

  • 71% of people had constipation
  • 71% had low platelets which are involved in blood clotting (thrombocytopenia)
  • 57% had low red blood cells/anemia

 

What does this mean for people with AML?

While promising, these results are from a very early stage of testing in a small group of people. More time is needed to see how people treated with the highest dose of tuspetinib will respond. This will allow a decision to be made over which dose is best for treating people with AML. This treatment combination could provide another option for people who are not able to have intensive chemotherapy.

 

3-year data on treatment of AML with gilteritinib4

What was the study about?

This study evaluated how well gilteritinib works compared to standard chemotherapy in adults with a specific subtype of AML characterized by an FLT3 mutation. FLT3 AML can be harder to treat. The people in the study had previously treated AML that had come back or hadn’t improved with treatment (relapsed/refractory).

Study overview

  • 276 people took part in the study
  • 88% were Asian
  • People were followed for up to 3 years

 

Key results

  • People who took gilteritinib lived longer than those who got chemotherapy:
    • 3 months on average with gilteritinib vs. 5.4 months for those on chemotherapy (median overall survival)
  • People on gilteritinib also had better responses:
    • 20% on gilteritinib had no signs of cancer in their blood or bone marrow (complete remission) compared with 11.5% on chemotherapy
    • 68% on gilteritinib had their cancer shrink or had no signs of cancer compared with 27% on chemotherapy
  • More people who took gilteritinib (23%) were able to have bone marrow transplants compared with chemotherapy (8%)
  • Nearly half of the people on gilteritinib were able to stop needing blood transfusions

 

Important safety information

  • Side effects were similar to what was seen previously with giltertinib:
    • Low red blood cells/ anemia
    • Low platelets which are involved in blood clotting (thrombocytopenia)
    • Fever5
  • Gilteritinib was generally well tolerated

 

What does this mean for people with AML?

In this study of mostly Asian people with FLT3-mutated AML, those treated with gilteritinib lived longer than those who had standard chemotherapy. More people treated with gilteritinib had no signs of cancer in their blood or bone marrow compared with the chemotherapy group. The side effects from treatment with gilteritinib were thought to be manageable. These results support using gilteritinib as a treatment option in people with FLT3-mutated AML that have previously not responded to treatment or whose cancer has returned.

 

References

  1. Roboz, G et al. Presentation at the EHA 2025 Congress, Milan, June, 2025. Abstract s135.
  2. Wei, A et al. Presentation at the EHA 2025 Congress, Milan, June, 2025. Abstract s137.
  3. Mannis, G et al. Presentation at the EHA 2025 Congress, Milan, June, 2025. Abstract s139.
  4. Wang, J et al. Presentation at the EHA 2025 Congress, Milan, June, 2025. Abstract s143.
  5. Wang J et al. Leukemia 2024;38(11):2410-2418.

 

Additional data published

Gemtuzumab:

  • Sperr et al., Abstract PB2491; GO-FIRST: Real-World Data of Front Line GO in Intermediate and Favorable Risk AML Patients in a Multicenter Retrospective Chart Review (link)

 

Bleximinib:

  • Wei et al.; Abstract S137; RP2D DETERMINATION OF BLEXIMENIB IN COMBINATION WITH VEN+AZA: PHASE 1B STUDY IN ND & R/R AML WITH KMT2A/NPM1 ALTERATIONS (link)

 

Revumenib:

  • Arellano et al:, Abstract  PS1467; PATIENTS WITH RELAPSED OR REFRACTORY (R/R) NUCLEOPHOSMIN 1-MUTATED (NPM1M) ACUTE MYELOID LEUKEMIA (AML): UPDATED RESULTS FROM THE PHASE 2 AUGMENT-101 STUDY (Link)
  • Aldoss et al.; Abstract PS1473; UPDATED RESULTS AND LONGER FOLLOW-UP FROM THE AUGMENT-101 PHASE 2 STUDY OF REVUMENIB IN PATIENTS WITH RELAPSED OR REFRACTORY (R/R) KMT2AR ACUTE LEUKEMIA (Link)
  • Issa et al.; Abstract PS1501; REVUMENIB ACTIVITY IN PATIENTS WITH ACUTE LEUKEMIA WITH NUP98R: RESULTS FROM THE AUGMENT-101 PHASE 1 STUDY (Link)

 

Quizartinib:

  • The Combination of a FLT3-ITD, NPM1mut and an Epigenetic Regulatory Gene Mutation Confers Unique Sensitivity to Quizartinib: Analysis From the QuANTUM-First Trial (Oral)
  • Trial in Progress: The Phase 3, Randomized, Double-Blind, Placebo[1]Controlled Quantum-Wild Study of Quizartinib in Combination with Chemotherapy and As Single-Agent Maintenance in Newly Diagnosed, FLT3-ITD–Negative Acute Myeloid Leukemia (Publication)
  • Final results of VEN A QUI TRIAL: A phase I-II trial comparing Venetoclax with Low Dose Cytarabine or Azacitidine combined with Quizatinib in non-fit patients with Acute Myeloid Leukemia (Poster)
  • Measurable Minimal Residual Disease by an ultra-sensitive NGS method to evaluate the response in Acute Myeloid leukemia, the VENAQUI clinical trial (Poster)
  • Prognostic significance of clonal monitoring assessed by VAF in unfit newly diagnosed AML: insights from the PETHEMA VEN-A-QUI trial (Poster)
  • Phase I/II Study of Quizartinib, Venetoclax, and Decitabine Triplet Combination in FLT3-ITD Mutated AML (Oral)
  • Multidrug resistance and metabolic reprogramming in FLT3 mutation AML following prolonged FLT3 inhibitor treatment  (Publication only)

 

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