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Tuesday, December 12, 2017

Proteostasis Therapeutics (PTI) : positive study results across all three of the company's CF pipeline programs

Headquarters: Massachusetts
Founded: 2006


Proteostasis Therapeutics commences 7 mln common stock offering 

Proteostasis Therapeutics announced positive study results across all three of the company's CF pipeline programs;  
  • Results of PTI-428 Meet Efficacy Endpoint in 28-day Study in CF Patients on Background Orkambi - Proteostasis announced that it has completed its Phase 2 study designed to evaluate the efficacy, safety, and tolerability of 50 mg once-a-day of PTI-428 over a 28-day treatment of CF patients on background Orkambi (lumacaftor/ivacaftor). The addition of PTI-428 to Orkambi demonstrated mean absolute improvements in ppFEV1 of 5.2 percentage points from baseline compared to placebo 
  • The two registrational phase 3 studies of Orkambi, TRAFFIC and TRANSPORT, showed that magnitude of response to Orkambi varied according to patient lung function at screening, suggesting that the overall efficacy was mainly driven by the subgroup with baseline ppFEV1 below 70% (+3.3 percentage points) while the changes in the group with FEV1 =70% were not statistically significant.
  • Preliminary Ad Hoc Analysis of PTI-801 in Ongoing 14-day Study in CF Patients on Background Orkambi - The Company has also shared initial data from the first five subjects (four PTI-801 treated and one placebo) of the first dose level tested in the 14-day dosing study of PTI-801 in CF patients on background Orkambi therapy.


Proteostasis Therapeutics, Inc. is a biopharmaceutical company. The Company is engaged in discovery and development of therapeutics that treat diseases caused by an imbalance in the proteostasis network, a set of pathways that control protein biosynthesis, folding, trafficking and clearance. It has developed the Disease Relevant Translation (DRT) technology platform, a drug screening approach for identifying highly translatable therapeutics based on predictive and functionally pertinent phenotypic assays and disease relevant models. Using this platform, it has identified a new class of small molecules, amplifiers that modulate proteins in the proteostasis network. It is developing and intend to commercialize its lead amplifier of CFTR protein, PTI-428, to improve CFTR protein function. It also focuses on developing PTI-NC-733, PTI-130, Usp14 program and unfolded protein response (UPR) program. PTI-130 is a small molecule amplifier.

Key stats and ratios

Q3 (Sep '17)2016
Net profit margin-899.68%-444.08%
Operating margin-908.06%-446.74%
EBITD margin--443.09%
Return on average assets-91.17%-67.80%
Return on average equity-125.11%-

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