Phase 1 clinical trial of S64315, an intravenous MCL1 inhibitor, in patients with myeloid malignancies.
First human evidence shows an MCL1-blocking drug can be given safely to patients with hard-to-treat blood cancers, opening a new treatment pathway.
S64315 is a first-in-class intravenous inhibitor of MCL1, a key anti-apoptotic protein overexpressed in many hematologic cancers, evaluated in a Phase 1 dose-escalation study in patients with refractory myeloid malignancies. This trial provides the first human clinical evidence for MCL1 inhibition as a therapeutic strategy, addressing a major unmet need in AML and MDS where available salvage options are limited.
What the study was
- Study design
- Phase 1 dose-escalation clinical trial
- Population
- Patients with refractory/relapsed myeloid malignancies (AML, MDS, MPN)
- Category
- Drug Development
- Maturity
- Exploratory
- Journal
- Leukemia & lymphoma
Why it surfaced
Phase 1 first-in-human trial of MCL1 inhibitor, a long-validated but previously undruggable target. AML/MDS represents one of the highest unmet needs in hematology. Clinical Phase 1 data from a multicenter trial with leading hematology centers (Vey, Montesinos, Daver, Wei).
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