Press Releases

Proteostasis Announces Presentations at the 41st European Cystic Fibrosis Society Conference

June 1, 2018 at 7:00 AM EDT

BOSTON, June 1, 2018 /PRNewswire/ -- Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, announced that data from the Company's CF clinical development programs will be presented during two panel presentations at the 41stEuropean Cystic Fibrosis Society (ECFS) Conference on June 6-9, 2018 in Belgrade, Serbia.

Details of the presentations are as follows:

Date & Time:

Thursday, June 7, 2018, 10:30 AM to 12:00 PM CEST

Session Title: 

Mix and Match! Combination Cystic Fibrosis Transmembrane Conductance

Regulator (CFTR) Modulation Therapies and New Trials

Session Type: 



Geoffrey S. Gilmartin, M.D., M.M.Sc., Chief Development Officer of Proteostasis


Dr. Pavel Drevinek, Charles University, Prague, Czech Republic; Dr. Silke van

Koningsbruggen-Rietschel, Cystic Fibrosis Study Center, University Hospital, Cologne, Germany

Date & Time: 

Thursday, June 7, 2018, 3:00 PM to 4:30 PM CEST

Session Title: 

Exciting News From CFTR Modulator Clinical Trials

Session Type: 

Panel Presentation


Patrick Flume, M.D., Professor of Medicine and Pediatrics at the Medical University of

South Carolina


Dr. Silke van Koningsbruggen-Rietschel, Cystic Fibrosis Study Center, University Hospital, Cologne, Germany; Dr. Lieven Dupont, Univeritair Ziekenhuis Leuven, Belgium


Following the presentations at ECFS, the presentations will be available on the Investor Events page in the Investors & Media section of the Company's website,

About PTI-428

PTI-428 is a first in class investigational CFTR amplifier for the treatment of CF in patients who are homozygous for the F508del mutation. PTI-428 is designed to increase the amount of newly synthesized cystic fibrosis transmembrane conductance regulator (CFTR) protein and to act synergistically with other CFTR modulators, such as correctors and potentiators. PTI-428 has received Fast Track, Orphan Drug and Breakthrough Therapy designations by the FDA.

PTI-428 is in development as an add-on therapy to approved CFTR modulator products such as Orkambi® and Symdeko™, and as part of PTI's proprietary once-a-day dosing triple combination regimen, which includes PTI-808, the Company's potentiator, and PTI-801, its corrector. The ongoing study of the triple combination received endorsement from the Therapeutics Development Network (TDN), the drug development arm of the Cystic Fibrosis Foundation (CFF), and PTI expects to report preliminary data from this study in the second half of 2018. In April, the TDN also endorsed Proteostasis' study protocol to investigate PTI-428 in CF patients on background Symdeko therapy. Proteostasis plans to initiate this Phase 2 study in the third quarter of 2018. Previously, PTI announced the results of a 28-day Phase 2 study in CF subjects on background Orkambi therapy, in which PTI-428 demonstrated mean absolute improvements in ppFEV1 of 5.2 percentage points from baseline compared to placebo (p<0.05).  

About PTI-801

PTI-801 is a third generation CFTR corrector, which is currently being studied in a 14-day trial in CF patients on background Orkambi treatment. PTI expects to report additional data from this study in the second quarter of 2018. In December, Proteostasis announced initial data from the first five subjects (four PTI-801 treated and one placebo) of the first dose level, once-a-day 100 mg of PTI-801. PTI-801 is also being studied in a double combination trial with PTI-808 and as part of PTI's proprietary triple combination.

About PTI-808

PTI-808 is the Company's novel CFTR potentiator, which is currently being evaluated as part of PTI's proprietary double (PTI-801 and PTI-808) and triple combination treatments. Proteostasis announced in December that it had completed PTI-808 safety and PK profiles from its SAD and MAD study in healthy volunteers. A total of 48 healthy volunteers had participated and completed the study of up to 300 mg of once-a-day, orally dosed PTI-808 in single and multiple dose cohorts. PTI-808 was found to be generally well tolerated.           

About Proteostasis Therapeutics, Inc.

Proteostasis Therapeutics, Inc. is a clinical stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing. Headquartered in Boston, MA, the Proteostasis Therapeutics team focuses on identifying therapies that restore protein function. In addition to its multiple programs in cystic fibrosis, Proteostasis Therapeutics has formed a collaboration with Astellas Pharma, Inc. to research and identify therapies targeting the Unfolded Protein Response (UPR) pathway. For more information, visit

Safe Harbor

To the extent that statements in this release are not historical facts, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.  Words such as "aim," "may," "will," "expect," "anticipate," "estimate," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements.  Examples of forward-looking statements made in this release include, without limitation, statements regarding expected presentations and expected timing of the initiation of, patient enrollment in, data from, and the completion of, our clinical studies and cohorts for our clinical programs.  Forward-looking statements made in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved.  Such risks and uncertainties include, without limitation, the possibility final or future results from our drug candidate trials (including, without limitation, longer duration studies) do not achieve positive results or are materially and negatively different from or not indicative of the preliminary results reported by the Company (noting that these results are based on a small number of patients and small data set), uncertainties inherent in the execution and completion of clinical trials (including, without limitation, the possibility that FDA comments delay, change or do not permit trial commencement, or intended label, or the FDA requires us to run cohorts sequentially or conduct additional cohorts or pre-clinical or clinical studies), in the enrollment of CF patients in our clinical trials in a competitive clinical environment, in the timing of availability of trial data, in the results of the clinical trials, in possible adverse events from our trials, in the actions of regulatory agencies, in the endorsement, if any, by therapeutic development arms of CF patient advocacy groups (and the maintenance thereof), and those set forth in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2018 and our other SEC filings.  We assume no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Orkambi®, Kalydeco® and Symdeko™ are trademarks of Vertex Pharmaceuticals Incorporated.



David Pitts
Argot Partners

David Rosen
Argot Partners

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