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CTP-656 (Deuterated ivacaftor)

Phase Two

Phase Two

Therapeutic Approach

Restore CFTR Function

CTP-656 is an altered form of the potentiator ivacaftor (Kalydeco®). Potentiators are drugs that facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell. CTP-656 may be more stable in the body than regular ivacaftor, which would allow it to be taken once a day.

Status

A Phase 2 trial for people with CF who have two copies of the F508del mutation was completed January 2015. Several Phase 3 trials are underway in people who have one or two copies of the F508del mutation.

Sponsor

This program is sponsored by CoNCERT Pharmaceuticals. It is being conducted within the Cystic Fibrosis Foundation's Therapeutics Development Network.

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