Novel Cystic Fibrosis Treatment Granted Fast Track Status

A triple combination program to treat cystic fibrosis has been granted Fast Track Designation by the Food and Drug Administration (FDA). 

Proteostasis Therapeutics is developing the combination treatment which includes a novel transmembrane conductance regulator (CFTR) amplifier (PTI-428), a third generation corrector (PTI-801) and a potentiator (PTI-808). The three candidates were discovered internally and are currently being assessed in clinical trials in the U.S. and Europe. 

Results from a safety and pharmacokinetics trial of PTI-428 last year found the treatment to be well tolerated. A study of the triple combination – which has received endorsement from the Therapeutics Development Network, the Clinical Trial Network, and the Cystic Fibrosis Foundation – is expected to begin this quarter. 

“We believe combinations of CFTR modulators hold the promise of improving treatment efficacy for different segments of the CF population,” said Meenu Chhabra, CEO of Proteostasis Therapeutics. “Including F508del heterozygotes and F508del homozygotes, where patients have limited access to treatment, are underserved by existing treatments or see a declining benefit in lung function over time.”

For more information visit Proteostasis.com.