CAMBRIDGE, Mass., Jan. 08, 2018 (GLOBE NEWSWIRE) -- 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 today that its triple combination clinical study protocol has received endorsement and a high strategic fit score from the Therapeutics Development Network (TDN) and the Clinical Trial Network (CTN). The TDN and CTN are the drug development arms of the Cystic Fibrosis Foundation (CFF) and the European CF Society (ECFS), respectively.  The study is designed to investigate the safety, tolerability and pharmacokinetics (PK) of the coadministration of PTI's cystic fibrosis transmembrane conductance regulator (CFTR) modulators, PTI-428, PTI-801 and PTI-808, and will also assess lung function (FEV₁). Both patient advocacy organizations reviewed the clinical data from healthy volunteers and CF subjects, including the recent proof-of-concept study with PTI-428 as add-on to Orkambi®¹, as well as the healthy volunteer data from the coadministration study of all three of PTI's CFTR modulators.   

Meenu Chhabra, Proteostasis Therapeutics' President and Chief Executive Officer, said: "We are humbled that the two preeminent global CF patient advocacy organizations share our view on the positive potential clinical impact of our CFTR modulators. Our proprietary triple combination study is on track to initiate dosing of CF patients in the first half of this year and we anticipate initial results in the second half of 2018. We believe that PTI-428, PTI-801 and PTI-808 have the potential to be a novel, best in class treatment for CF patients." 

The CFF and ECFS are the most impactful CF patient advocacy organizations and shepherd the execution of clinical trials in the U.S. and Europe, respectively.  Both organizations have well established processes to evaluate submitted study protocols based on scientific merit, study design, feasibility and the overall clinical research priorities of the CF community.  Protocols are reviewed by a selected group of experienced CF physician investigators, research coordinators, biostatisticians, people with CF and other specialists.

In the U.S., upon the protocol endorsement, the TDN provides access to 89 accredited care centers with demonstrated expertise in clinical research, and supports study participant recruitment and execution of studies.  Since its founding in 1998, the TDN has conducted more than 130 clinical studies for CF, including studies of CFTR modulators. Similarly, in Europe, the CTN provides access for endorsed studies to 43 large and experienced CF centers located in 15 different countries, including the U.K. The TDN and the CTN have established a strong partnership for endorsed studies conducted in both the U.S. and Europe.

Patrick A. Flume, MD, Professor of Medicine and Pediatrics and Director of the Cystic Fibrosis Center at the Medical University of South Carolina, and Lead Principal Investigator for PTI's triple combination program said: "We are experiencing an exciting time in CF drug development and research, with an unprecedented number of ongoing clinical trials.  At the same time, our CF patient population is a valuable and limited resource. The TDN's protocol review and scoring process creates a framework that helps the investigator and patient community maintain alignment on clinical research priorities."

Co-administration of PTI-428, PTI-808 and PTI-801 in CF subjects on track to initiate in 1H 2018 with initial clinical data in 2H 2018

PTI recently completed a healthy volunteer co-administration safety and tolerability study of its three proprietary CFTR modulators. Safety and PK profiles achieved with seven days of once-a-day oral dosing of PTI-428, PTI-801 and PTI-808 indicate these compounds were generally well-tolerated and are potentially amenable for once a day dosing.

PTI plans to initiate dosing of CF patients with its proprietary triple combination in the first half of this year in U.S. and European CF centers within the TDN and CTN networks, with initial clinical data expected in the second half of 2018.

Safe Harbor

CONTACTS:

Investors:
David Pitts
Argot Partners
212.600.1902
[email protected]

Media:
David Rosen
Argot Partners
212.600.1902
[email protected]

¹ Orkambi® is a registered trademark of Vertex Pharmaceuticals, Inc.