What type of study is this?

This is a Pre-Registered Trial study.

What question did this study set out to answer?

The study aims to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of cln-049 in patients with relapsed/refractory AML and MDS.

December 8, 2025Blood

Preliminary anti-leukemia activity from A phase 1 study of cln-049, a novel anti-FLT3 x anti-CD3 bispecific T-cell engager, in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS)

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The study aims to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of cln-049 in patients with relapsed/refractory AML and MDS.
Conducted a phase 1 multiple ascending dose study with a 3+3 design.
Enrolled patients aged ≥ 18 years with R/R AML or MDS.
Evaluated pharmacokinetics and pharmacodynamics following intravenous dosing of cln-049.
Adverse events assessed by CTCAE v5 and specific criteria for CRS and ICANS.
Measured response rates using ELN 2022 and IWG 2023 criteria.
40 patients enrolled, demonstrating a safety profile with treatment-emergent adverse events in 95% of patients.
Achieved a complete response rate of 9% and a composite complete response rate of 30% in evaluated patients.
Notable anti-leukemic activity observed at a target dose of ≥ 6 µg/kg.
Responses noted in patients with TP53-mutated AML, highlighting effectiveness in a poor prognosis group.
Pharmacokinetics showed a dose-dependent increase in exposure without significant accumulation.

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Implication

Phase 1 study shows promising complete response and safety profile in patients with R/R AML and MDS, indicating potential as a treatment option.

Cite This Study

Vale et al. (2025) studied this question.

synapsesocial.com/papers/693624d74fa91c937236d092 https://doi.org/10.1182/blood-2025-768

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Implication

Phase 1 study shows promising complete response and safety profile in patients with R/R AML and MDS, indicating potential as a treatment option.

Preliminary anti-leukemia activity from A phase 1 study of cln-049, a novel anti-FLT3 x anti-CD3 bispecific T-cell engager, in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS)

Key Points

Discussion

Implication

Cite This Study

Preliminary anti-leukemia activity from A phase 1 study of cln-049, a novel anti-FLT3 x anti-CD3 bispecific T-cell engager, in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS)

Key Points

Discussion

Implication

Cite This Study

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