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CTP-656 POTENTIATOR
- Question
- Hello
Could you tell us which company produces the activator CTP-656 and which patients will potentially benefit from this molecule?
Thank you for your reply
- Answer
- Hello
Concert Pharmaceuticals has just run a phase 1 with healthy volunteers in order to identify the optimal dose of CTP-656 for CF patients.
CTP-656 is a potentiator of the defective CFTR protein. The molecule has been developed from Ivacaftor (Kalydeco®) to which a deuterium has been added.
CTP-656 is expected to be used for CF patients carrying a gating mutation (class 3, like G551D).
During the phase 1 trial the pharmacokinetic of CTP-656 (this is what the molcule is turned to in the human body) was compared with that of Kalydeco® which is a treatment for patients with a gating mutation.
The company plans a phase 2 trial for CF patients carrying a gating mutation.
Best regards
Anna Ronayette
- 25.07.2016
