Table 1.
Name | Clinical stage | Target | Mode of action | Company |
---|---|---|---|---|
Ivacaftor | Approved | CFTR | Potentiator | Vertex Pharmaceuticals |
Lumacaftor + ivacaftor | Approved | CFTR | Corrector + potentiator | Vertex Pharmaceuticals |
Tezacaftor + ivacaftor | Approved | CFTR | Corrector + potentiator | Vertex Pharmaceuticals |
VX-445 + tezacaftor + ivacaftor | Phase III | CFTR | VX-445 = new corrector | Vertex Pharmaceuticals |
QBW251 | Phase II | CFTR | Potentiator | Novartis Pharmaceuticals |
FDL169 | Phase II | CFTR | Corrector | Flatley Discovery Lab |
VX-152 + tezacaftor + ivacaftor | Phase III | CFTR | VX-152 = new corrector | Vertex Pharmaceuticals |
VX-440 + tezacaftor + ivacaftor | Phase III | CFTR | VX-440 = new corrector | Vertex Pharmaceuticals |
VX-561 (CTP-656) | Phase II | CFTR | Potentiator (deuterated ivacaftor) | Vertex Pharmaceuticals |
GLPG1837 | Phase II | CFTR | Potentiator | Galapagos NV/AbbVie |
GLPG2222 | Phase II | CFTR | C1 corrector | Galapagos NV/AbbVie |
GLPG2451 | Phase I | CFTR | Potentiator | Galapagos NV/AbbVie |
GLPG2737 | Phase I | CFTR | C2 corrector | Galapagos NV/AbbVie |
GLPG3067 | Phase I | CFTR | Potentiator | Galapagos NV/AbbVie |
GLPG3067 + GLPG2222 + GLPG2737 | Phase I | CFTR | Potentiator + C1 corrector + C2 corrector | Galapagos NV/AbbVie |
PTI-428 | Phase I | CFTR | Amplifier that increases amount of CFTR protein | Proteostasis Therapeutics |
QR-010 | Phase I | CFTR | Oligonucleotide that repairs CFTR mRNA | ProQR Therapeutics |
MRT5005 | Phase I | CFTR | Delivers CFTR mRNA | Translate Bio |
PTI-801 | Phase I | CFTR | Corrector | Proteostasis Therapeutics |
PTI-808 | Phase I | CFTR | Potentiator | Proteostasis Therapeutics |
QBW276 | Phase II | ENaC | Inhibits ENaC activity | Novartis Pharmaceuticals |
SPX-101 | Phase II | ENaC | Peptide that induces ENaC internalization | Spyryx Biosciences |
AZD5634 | Phase I | ENaC | Inhibits ENaC activity | AstraZeneca |
BI 443651 | Phase I | ENaC | Inhibits ENaC activity | Boehringer Ingelheim |
Notes: Summary of ion channel-targeting therapeutics according to their target, mode of action, and stage of clinical testing or approval. Adapted from Chest, 154(2), Gentzsch M, Mall MA, Ion Channel Modulators in Cystic Fibrosis, 383–393, Copyright (2018), with permission from Elsevier.23
Abbreviations: CFTR, cystic fibrosis transmembrane conductance regulator; ENaC, epithelial Na channel.