Proteostasis Therapeutics shares surge on positive Phase 1 trial data for cystic fibrosis therapy

Shares of Proteostasis Therapeutics (NASDAQ:PTI) are soaring in Thursday’s pre-market session thanks to news of optimistic preliminary data from a Phase 1 trial evaluating its cystic fibrosis treatment.

The studies were evaluating PTI-801, a third-generation cystic fibrosis transmembrane conductance regulator corrector (CFTR), PTI-808, a CFTR potentiator, and PTI-428, a CFTR amplifier, compared to a placebo over a 14-day dosing period in patients with cystic fibrosis.

Patients with cystic fibrosis who were given the double combination therapy of 300mg of both PTI-801 and PTI-808 showed an increase in their lung function at day seven and day 14 from the baseline and when measured against a placebo. A decrease in sweat chloride concentration was also observed at day seven and day 14.  

READ: Proteostasis surges as FDA grants Breakthrough Therapy Designation for its cystic fibrosis treatment

In response to the results, investors sent Proteostasis shares up 71% to $3.23 before Thursday’s opening bell.

“Doublet combinations, the current standard of care for many cystic fibrosis patients, are the benchmark for the current treatment in cystic fibrosis,” noted Dr Carsten Schwarz, head of the Adult Cystic Fibrosis Centre at the Berlin University Medical Center, in a statement.

“These data are the first results seen using an entirely new Cystic Fibrosis doublet and compare favorably to standard of care, and demonstrate the potential of next-generation CFTR modulators to further improve outcomes in this disease,” he further explained.

Additional preliminary data from the study of the triple combination of PTI-801, PTI-808 and PTI-428 is set to be delivered in the fourth quarter of this year. But complete data for all double and triple combinations of the cystic fibrosis treatments won’t be available until early next year.

Headquartered in Boston, Proteostasis Therapeutics is a clinical-stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing.

Contact Ellen Kelleher at [email protected]

Story by ProactiveInvestors