Japan’s first investigator-initiated phase I clinical trials of a tankyrase inhibitor launched

Toward regulatory approval of the tankyrase inhibitor RK-582

A collaborative research group of Japanese Foundation for Cancer Research (JFCR), the RIKEN CSRS and the RIKEN Center for Biosystems Dynamics Research has initiated Japan’s first investigator-initiated phase I clinical trials of the tankyrase inhibitor RK-582. The first patient was dosed on March 18, 2025, at the Cancer Institute Hospital of JFCR.

The investigational drug RK-582 is a novel tankyrase inhibitor developed at RIKEN, based on a seed compound selected from the University of Tokyo's compound library. Its structure was optimized through derivatization, followed by pharmacological evaluation at the enzymatic and cellular levels, as well as pharmacokinetic evaluation.

This clinical trial targets patients with unresectable, advanced, or recurrent colorectal cancer and aims to evaluate the safety, tolerability, and exploratory efficacy of RK-582. The treatment is designed to suppress tumor growth by blocking hyperactivated Wnt/β-catenin signaling, driven by loss-of-function mutations in the tumor suppressor gene APC, found in approximately 80% of colorectal cancers. This clinical trial will proceed with careful evaluation through close monitoring of patient outcomes following administration.

 

Contact

Minoru Yoshida: Group Director (at the time)
Chemical Genomics Research Group (at the time)
Drug Discovery Seed Compounds Exploratory Unit (at the time)

Yoko Yashiroda; Senior Research Scientist (at the time)
Chemical Genomics Research Group (at the time)

Hiroo Koyama; Unit Leader
Fumiyuki Shirai; Senior Research Scientist
Drug Discovery Chemistry Platform Unit