Proteostasis Therapeutics (NASDAQ:PTI) slumps 34% premarket on robust volume on the heels of topline results from a Phase 2 clinical trial evaluating its CFTR modulator combinations in cystic fibrosis (CF) patients.
The study assessed dirocaftor (PTI-808) and posenacaftor (PTI-801) with or without nesolicaftor (PTI-428) in 28 F508del homozygous and 40 F508del heterozygous subjects.
On the plus side, homozygous patients receiving the triple combo (n=11) experienced a mean absolute 8% relative improvement in lung function compared to pooled placebo at day 28 (p<=0.01). The treatment effect on lung function and sweat chloride concentration was higher for the triplet therapy versus the doublet (dirocafter + posenacaftor) but specific values are not provided.
In the heterozygous group, the results were mixed. The mean relative change in lung function for the triplet therapy was not statistically significant, but the change in sweat chloride concentration was.
The company plans to launch a Phase 3 study, MORE, in homozygous CF patients in 2020.
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