Drug intelligence / Profile preview

regorafenib + trifluridine + tipiracil

Development stage
Unknown
Lead developer
TRF Pharma
Modality
Small Molecules
Administration
Oral
01

Overview

regorafenib + TAS-102 is a combination therapy being developed by TRF Pharma (Therapeutic Repurposing Foundation) for the treatment of sickle cell disease (SCD). This therapeutic strategy involves the repurposing of two established oncology treatments: regorafenib (TRF-1101), a multi-kinase inhibitor, and TAS-102 (TRF-1201), which consists of the nucleoside analog trifluridine and the thymidine phosphorylase inhibitor tipiracil. The combination is designed to induce the production of fetal hemoglobin (HbF), which prevents the polymerization of sickle hemoglobin (HbS) and the subsequent sickling of red blood cells. By increasing HbF levels and improving microvascular blood flow, the therapy aims to reduce vaso-occlusive crises and associated pain. TRF-1101 is currently being evaluated in Phase 1/2 clinical trials for SCD, while the combination with TAS-102 is part of a broader program to optimize HbF induction through synergistic mechanisms.

Brand names
StivargaLonsurf
Other names
regorafenib + TAS-102
02

Targets

BRAF (B-Raf proto-oncogene, serine/threonine kinase)TS (Thymidylate synthase)KIT (c-KIT proto-oncogene receptor tyrosine kinase)TEK (Tie2)PDGFRB (Platelet-derived growth factor receptor beta)TYMP (Thymidine phosphorylase)RAF1 (c-Raf-1 (Y340D/Y341D))DNAVEGFR3 (Vascular endothelial growth factor receptor 3)VEGFR-1 (Vascular endothelial growth factor receptor 1)VEGFR2 (Vascular endothelial growth factor receptor 2)FGFR1 (Fibroblast growth factor receptor 1)RET (Rearranged during transfection receptor tyrosine kinase)

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