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PTI-428 + lumacaftor + ivacaftor
- Development stage
- Phase 2
- Lead developer
- TuHURA Biosciences
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
This is an investigational **triple combination therapy** for cystic fibrosis (CF), consisting of: - **PTI-428** (nesolicaftor): a CFTR amplifier that increases the expression of the CFTR protein, - **Lumacaftor**: a CFTR corrector that promotes proper folding and trafficking of the F508del-CFTR protein to the cell surface, - **Ivacaftor**: a CFTR potentiator that enhances chloride channel activity of CFTR at the cell surface. Preclinical and early clinical studies suggest that adding PTI-428 to lumacaftor and ivacaftor may further boost CFTR function beyond the standard doublet, especially in people with F508del mutations. The triple therapy is under development for CF, primarily in patients who are homozygous or heterozygous for the F508del mutation in CFTR[2][3][4][1].
- Other names
- PTI-428 + lumacaftor + ivacaftor
02
Targets
CFTR (Cystic fibrosis transmembrane conductance regulator)
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