China-based SciClone Pharmaceuticals Inc. (Nasdaq: SCLN) announced that the first patient has been dosed in the Phase III regulatory REPLATINUM study in China for its RRx-001, a next-generation small-molecule immunotherapy targeting CD47/SIRPα (signal-regulatory protein alpha). The drug, in-licensed from US firm EpicentRx Inc. in July 2020, is being evaluated as a third-line treatment for small-cell lung cancer (SCLC).
Study Design and Progress
The controlled, open, global randomized Phase III study is being conducted concurrently in China and the US. It aims to assess the efficacy and safety of sequential administration of RRx-001 and platinum-containing doublet therapy compared with platinum-containing doublet therapy alone as a third-line or subsequent treatment for SCLC. The study has enrolled 292 patients, with primary endpoints being progression-free survival (PFS) and overall survival (OS), and secondary endpoints being overall response rate (ORR) and duration of response (DOR).
Mechanism of Action
RRx-001 is designed to target the CD47-SIRPα axis to repolarize tumor-associated macrophages (TAM) and other immunosuppressive cells in the tumor microenvironment into immunostimulant phenotypes. The goal is to improve tumor blood flow to enhance oxygen supply and drug delivery capabilities. The drug can be used as a monotherapy or in combination with chemotherapy, other immunotherapies, radiotherapy, or targeted drugs. Phase II clinical trials have shown that RRx-001 demonstrated good efficacy and safety in the treatment of solid tumors such as SCLC.-Fineline Info & Tech
