Drug Development Pipeline
PTI-428 (alone and in combination)
Restore CFTR Function
PTI-428 is a type of CFTR modulator called an amplifier. Amplifiers increase the amount of CFTR protein in the cell. This makes more CFTR protein available for other therapies, such as ivacaftor and lumacaftor, to work on. When combined with other therapies, PTI-428 may increase CFTR function across multiple gene mutations.
PTI-428 is currently being studied both alone as well as in combination with two other CFTR modulators, PTI-801 and PTI-808.
A phase 2 study of PTI-428 in people with two copies of the F508del CFTR mutation is underway.
This program is sponsored by Proteostasis Therapeutics. The early discovery phase was partially funded by the Cystic Fibrosis Foundation.
Recent PTI-428 (alone and in combination) Studies
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