PTI-428

Description: PTI-428 is a novel, potent CFTR amplifier being developed by Proteostasis Therapeutics, Inc. for the treatment of CF in patients who are homozygous for the F508del mutation in the CFTR gene as an add-on therapy to approved CFTR modulators or as part of PTI’s proprietary triple combination regimen that includes PTI-808, a potentiator, and PTI-801, a corrector. PTI-428 works early during CFTR biogenesis to increase levels of newly synthesized CFTR protein, suggesting potential therapeutic benefits in combination with CFTR correctors and potentiators. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for PTI-428, the Company’s cystic fibrosis transmembrane conductance regulator (CFTR) amplifier.

References: Proteostasis Therapeutics News Release