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AEG35156 + sorafenib
- Development stage
- Phase 2
- Lead developer
- Pharmascience
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules
- Administration
- Intravenous, Oral
01
Overview
This is a **combination therapy** of AEG35156 and sorafenib, studied primarily for advanced hepatocellular carcinoma (HCC). - AEG35156 is a second-generation antisense oligonucleotide designed to target **X-linked inhibitor of apoptosis protein** (XIAP) mRNA, thereby lowering the apoptotic threshold in cancer cells and enhancing sensitivity to apoptosis. - Sorafenib is a small molecule multi-kinase inhibitor targeting kinases involved in tumor cell proliferation and angiogenesis (such as RAF kinases and VEGFR). AEG35156 in combination with sorafenib showed additional activity compared to sorafenib alone in terms of objective response rate (ORR) and was well tolerated in clinical studies, although progression-free survival (PFS) benefit was moderate[1][2][3][4][5].
02
Targets
BRAF (B-Raf proto-oncogene, serine/threonine kinase)RAF1 (c-Raf-1 (Y340D/Y341D))FGFR1 (Fibroblast growth factor receptor 1)FLT3 (Fms related receptor tyrosine kinase 3)PDGFRB (Platelet-derived growth factor receptor beta)VEGFR2 (Vascular endothelial growth factor receptor 2)VEGFR4 (Vascular endothelial growth factor Receptor-3)KIT (c-KIT proto-oncogene receptor tyrosine kinase)XIAP
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