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December 8, 2025BloodOpen Access

Kpg-818, a novel cereblon (CRBN) modulator, in patients with hematological malignancies: Results of a Phase 1, open-label, multiple ascending dose study

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Authors

AMAhmad MattourHenry Ford HospitalJWJ. WangSino Biopharmaceutical (China)YWYao WangShanxi Provincial Cancer Hospital

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Overview

Phase 1 trial found KPG-818 with dexamethasone shows safety and potential efficacy in hematological malignancies, indicating further development is warranted.

Key Points

  • This research aims to evaluate the safety and efficacy of KPG-818 in patients with hematological malignancies.
  • Conducted an open-label, multicenter, multiple-dose escalation study.
  • Enrolled adults with relapsed or refractory multiple myeloma and other hematological malignancies.
  • Administered KPG-818 daily in combination with dexamethasone or as monotherapy over several cycles.
  • 18 patients received KPG-818, with 94.4% experiencing at least one treatment emergent adverse event.
  • Maximum tolerated dose was exceeded with neutrophil count decrease noted as a common dose-limiting toxicity.
  • Overall response rate was 50% in heavily treated multiple myeloma patients at certain dosing schedules.

Cite This Study

Mattour et al. (2025) studied this question.

synapsesocial.com/papers/69362f634fa91c937236de2dhttps://doi.org/10.1182/blood-2025-5809
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