Proteostasis Therapeutics Skyrocket on Optimistic Cystic Fibrosis Trial Results

Proteostasis Therapeutics

Proteostasis Therapeutics, Inc. (NASDAQ: PTI)


Proteostasis Therapeutics stock is flying high after the company announced positive preliminary data from its early-stage cystic fibrosis study.

The Phase 1 trial evaluated the effect of combining PTI-808, a CFTR potentiator, and PTI-801, which is a third-generation cystic fibrosis transmembrane conductance regulator corrector.

Cystic fibrosis patients who took the double combination therapy of 300 mg of the PTI-808 and PTI 801 combination, exhibited an increase in their lung function when measured against a placebo and at day 7 and day 14 from the baseline.

Researchers working on the study also observed a reduction in sweat chloride concentration at day 7 and day 14. Carsten Schwarz, head of the Adult Cystic Fibrosis Centre at Berlin University, commented,

“Doublet combinations, the current standard of care for many CF patients, are the benchmark for current treatment in cystic fibrosis.  These data are the first results seen using an entirely new CF doublet, compare favorably to the standard of care, and demonstrate the potential of next-generation CFTR modulators to further improve outcomes in this disease. I look forward to results from the fourth dosing cohort and to understanding the potential of another novel combination, PTI-801, PTI-808, and PTI-428.”

According to Proteostasis, complete data for the first cohort of the triplet combination will be available by the first quarter of next year, with preliminary data scheduled later this year.

Shares of the company skyrocketed recorded a four-fold gain of $320% in afternoon trading session Thursday in New York. Volume reached more than 44 million shares, up from the full-day average of 152,000 shares.

The stock had lost more than 65% this year through Wednesday, while the Standard & Poor’s 500 was 5%.

Proteostasis Therapeutics, Inc. Company Profile

Proteostasis Therapeutics, Inc., a clinical stage biopharmaceutical company, discovers and develops novel therapeutics to treat cystic fibrosis and other diseases caused by an imbalance in the proteostasis network.

Its lead product candidate is PTI-428, an orally bioavailable cystic fibrosis transmembrane conductance regulator modulator belonging to the amplifier class, which is in Phase II study.

The company is also involved in developing PTI-801, a corrector molecule; and PTI-808, a potentiator molecule that have been completed Phase I Study. It has a collaboration agreement with Astellas Pharma Inc. Astellas Pharma, Inc. to research and identify therapies targeting the UPR pathway.

The company was formerly known as Proteoguard, Inc. and changed its name to Proteostasis Therapeutics, Inc. in September 2007. Proteostasis Therapeutics, Inc. was founded in 2006 and is headquartered in Cambridge, Massachusetts. – Yahoo Finance