REFOCUS: A First-in-Human Study of Highly Selective FGFR2 Inhibitor, RLY-4008, in Patients With ICC and Other Advanced Solid Tumors

Summary

This is a Phase 1/2, open-label, FIH study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDy), and antineoplastic activity of RLY-4008, a potent and highly selective FGFR2 inhibitor, in patients with unresectable or metastatic cholangiocarcinoma (CCA) and other solid tumors. The study consists of 4 parts: a dose escalation (Part 1), a dose expansion (Part 2), and an extension (Part 3) and a rollover (Part 4).

Timeline

Start

2020-09-02

Primary completion

2025-09-05

Completion

2025-09-05

Publications

• Ellis H, Balasooriya ER, Varkaris A, Hajian B, Wan J, Shekhar M, Gritti I, Vijay V, Albertelli L, Piot S, Lau K, Kehlmann A, Chevalier N, Nugent FW, Zhen Y, Silveira VS, Sellers WR, Corcoran RB, Juric D, Bardeesy N. Mechanisms of clinical resistance to selective FGFR2 inhibition by lirafugratinib. Ann Oncol. 2026 Jun;37(6):798-812. doi: 10.1016/j.annonc.2026.01.004. Epub 2026 Jan 20.

Drugs

Indications