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ataluren + pembrolizumab
- Development stage
- Phase 2
- Lead developer
- PTC Therapeutics
- Modality
- Monoclonal Antibodies → Antibody-Based Therapeutics, Small Molecules
- Administration
- Oral, Intravenous
01
Overview
Ataluren + pembrolizumab is an investigational drug combination being studied primarily for the treatment of metastatic mismatch repair deficient (dMMR) and proficient (pMMR) colorectal adenocarcinoma, as well as metastatic dMMR endometrial carcinoma. Ataluren is a small molecule that enables ribosomes to read through premature stop codons in mRNA, potentially restoring the production of full-length proteins in cells with nonsense mutations. This mechanism may generate novel peptides in dMMR tumors, increasing their immunogenicity. Pembrolizumab is a humanized monoclonal antibody that targets and blocks the programmed cell death protein 1 (PD-1) receptor on T-cells, thereby preventing tumor cells from evading immune detection via PD-L1/PD-L2 ligands. The rationale for combining these agents is that Ataluren-induced neoantigens may enhance the anti-tumor immune response elicited by PD-1 blockade with pembrolizumab[1][4][6].
- Brand names
- Translarna (for ataluren)Keytruda (for pembrolizumab)
02
Targets
PDCD1 (Programmed cell death protein 1 receptor)80S ribosome (Ribosome and ribosomal components)PTC (50S ribosomal peptidyl transferase center)
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