Drug Development Pipeline
Restore CFTR Protein
Posenacaftor is a type of CFTR modulator called a corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.
Phase 2 studies were conducted to test the safety and effectiveness of posenacaftor alone and in combination with two other CFTR modulators, nesolicaftor (PTI-428) and dirocaftor (PTI-808). No further studies of these compounds are planned at this time.
This program was sponsored by Proteostasis Therapeutics. It was conducted through the Therapeutics Development Network.
Recent Posenacaftor (PTI-801) Studies
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