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Phase I Study of Rogocekib in Patients with Advanced, Relapsed, or Refractory Malignant Solid Tumors

Jun Sato1, Yuki Katsuya1, Takafumi Koyama1

  • 1Department of Experimental Therapeutics, National Cancer Center Hospital, Tokyo, Japan.

Clinical Cancer Research : an Official Journal of the American Association for Cancer Research
|May 18, 2026
PubMed
Abstract

Insights

Rogocekib, an oral CLK inhibitor targeting RNA splicing, showed target engagement and preliminary antitumor activity in advanced solid tumors. Further investigation is warranted despite manageable toxicities and two treatment-related deaths.

Area of Science:

  • Oncology
  • Pharmacology
  • Molecular Biology

Background:

  • Aberrant RNA splicing is a key driver in cancer progression.
  • Targeted therapies for aberrant splicing, like CDC2-like kinase (CLK) inhibitors, are needed.
  • Rogocekib (CTX-712) is an oral CLK inhibitor designed to target RNA splicing.

Purpose of the Study:

  • Evaluate the safety, tolerability, and maximum tolerated dose (MTD) of rogocekib in advanced solid tumors.
  • Determine the recommended dose (RD) for further clinical trials.
  • Assess the pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of rogocekib.

Main Methods:

  • Phase I dose-escalation study using an accelerated titration followed by a 3+3 design.
  • Doses ranged from 10 mg to 175 mg, administered twice weekly (BIW) or once weekly (QW).
  • 46 patients with advanced solid tumors were enrolled.

Main Results:

  • The MTD was established at 140 mg BIW.
  • Common adverse events included nausea, vomiting, and diarrhea; two treatment-related deaths occurred.
  • Rogocekib demonstrated dose-dependent PK, target engagement (PD markers THAP9-AS1, S6K), and preliminary efficacy (6.5% partial response, primarily in ovarian cancer).

Conclusions:

  • Rogocekib shows target engagement and preliminary antitumor activity in solid tumors.
  • Most toxicities were manageable, but safety monitoring is crucial due to observed treatment-related deaths.
  • Further clinical investigation of rogocekib is warranted.

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