Acute Myeloid Leukemia (AML)

DE-RISK YOUR AML PROGRAM IN PRIMARY PATIENT CELLS

From venetoclax/HMA combination studies to resistance profiling and biomarker-stratified response analysis, xCellSense® measures ex vivo drug response directly in primary AML cells to support candidate, combination, and translational development decisions.

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The AML DRUG development challenge

AML is molecularly heterogeneous and increasingly treated with combination regimens. Venetoclax/HMA backbones, emerging triplets, and resistance after venetoclax exposure create a need for functional data that complement genomic profiling.

What we test in AML

1

Combination design
Test your asset alone and in combination with venetoclax, HMAs, or other standard-of-care agents—with quantitative dose-response, synergy analysis, and ranked combination performance.

2

Resistance profiling
Benchmark ex vivo activity across treatment-naïve and previously treated AML samples, including patients previously exposed to venetoclax or other standard-of-care regimens, subject to availability at study scoping.

3

Modality-specific readouts
Assess target expression and mechanism-specific pharmacodynamic readouts — receptor occupancy and target-protein degradation — for ADC, bispecific, and targeted-degrader programs, where supported by qualified reagents.

4

Biomarker correlation
Correlate ex vivo drug response with molecular features from the same specimen—including AML alterations such as FLT3, NPM1, IDH1/2, and TP53—to identify response-associated candidate biomarkers and generate biomarker-stratification hypotheses.

Evidence in Acute Myeloid Leukemia

  • ASH 2025 oral: 21-agent ex vivo profiling across 31 AML samples; drug sensitivity correlated with overall survival in newly diagnosed AML (ROC-AUC up to 0.87).
  • ASH 2025 poster: 5 drug pairs across 15 AML bone marrow aspirates; marked inter- and intra-patient variability in combination response. Published in Biotechnology and Bioprocess Engineering (2026).
  • ASH 2023 (Blood 142:1523): 21-agent ex vivo panel in 45 AML patients stratified early-death risk (ROC-AUC 0.825 venetoclax/HMA; 0.833 anthracycline/cytarabine).
  • Haematologica (2024): Machine-learning integration of ELN 2022 criteria with genomic clusters improved prognostic stratification in 279 older adults with AML.
Scientific Publications
Haematologica

Prognostic value of European LeukemiaNet 2022 criteria and genomic clusters using machine learning in older adults with acute myeloid leukemia

Haematologica
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Scientific Publications
Blood Research Journal Logo

Recent advances in and applications of ex vivo drug sensitivity analysis for blood cancers

Blood Research
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Scientific Publications
Biotechnology and Bioprocess Engineering

Quantitative ex vivo synergy profiling uncovers heterogeneous combination responses in acute myeloid leukemia

Biotechnology and Bioprocess Engineering
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Presentations
ASH

Quantitative ex vivo synergy profiling uncovers heterogeneous combination responses in AML primary samples

67th ASH Annual Meeting and Exposition
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Presentations
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Ex vivo drug sensitivity testing in Korean AML patients: Integration of functional and genomic profiles for predicting clinical response and survival

67th ASH Annual Meeting and Exposition
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Presentations
ASH

Prognostic Utility of the Patient-Derived AML Cells' Ex Vivo Drug Sensitivity Results

65th ASH Annual Meeting and Exposition
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Why it de-risks development

Generate functional evidence to prioritize combinations, characterize response in treatment-exposed AML, and refine response-associated biomarker hypotheses — before committing to early clinical development.

Key Deliverables

01
Combination synergy and ranking
Quantitative synergy matrices are generated using established models such as Bliss, Loewe, ZIP, and HSA, enabling the ranking of combination performance.
02
Resistance-focused activity benchmarking
Ex vivo activity is benchmarked across both treatment-naïve and previously treated AML samples, including those exposed to venetoclax and standard-of-care therapies.
03
Modality-specific pharmacodynamic readouts
Target expression and mechanism-specific pharmacodynamic (PD) endpoints, such as receptor occupancy or target-protein degradation.
04
Molecular–functional correlation
Ex vivo drug response is correlated with genomic and transcriptomic features from the same specimen to identify hypotheses regarding response-associated biomarkers.
05
Responder profiling and integrated reporting
Functional responder profiles are generated, providing decision-ready drug-response, biomarker, and analytical outputs within weeks.
Let's Talk

Scope an AML Study on primary patient cells

A scientist — not a sales desk — will scope a tailored AML study and return a design and timeline.