Phase 1 study of rogocekib in patients with relapsed or refractory hematologic malignancies.

ABSTRACT: Rogocekib (development name CTX-712) is a first-in-class, orally available, highly potent, and selective small-molecule inhibitor of CDC2-like kinase, a key regulator of the RNA splicing process. Preclinical studies demonstrated antiproliferative activity on various in vitro and in vivo models of hematologic malignancies. Based on these findings, a phase 1 study of rogocekib was conducted to evaluate the safety and preliminary efficacy in patients with relapsed or refractory acute myeloid leukemia (AML) and higher-risk myelodysplastic syndrome (MDS). Using a 3+3 design informed by previous solid tumor safety data, patients received 70 mg or 105 mg twice a week in capsule form. One dose-limiting toxicity of grade 4 pneumonia was observed in the 105 mg twice a week cohort. Among 12 patients

SUBMITTER: Yokoyama H 

PROVIDER: S-EPMC12848337 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature