Proteostasis Therapeutics Receives FDA Fast Track Designation for Triple Combination Program in Patients with Cystic Fibrosis

CAMBRIDGE, Mass., April 4, 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, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company's triple combination program for the treatment of cystic fibrosis. The Company's proprietary triple combination includes a novel cystic fibrosis transmembrane conductance regulator (CFTR) amplifier, third generation corrector and potentiator, known as PTI-428, PTI-801 and PTI-808, respectively. The Company announced in January that the protocol for its triple combination clinical study, which the Company plans to initiate in the current quarter, has received endorsement and a high strategic fit score from the Therapeutics Development Network (TDN) and the Clinical Trial Network (CTN), the drug development arms of the Cystic Fibrosis Foundation (CFF) and the European CF Society (ECFS), respectively. 

"Fast Track designation represents another positive step for the development of our triple combination therapy and underscores the serious unmet need that remains for the vast majority of CF patients," said Meenu Chhabra, president and chief executive officer of Proteostasis Therapeutics. "We believe this designation, together with other recent designations from regulators and CF organizations following review of our results with PTI-428, PTI-801 and PTI-808, is recognition of the potential of these programs in this disease.  We believe combinations of CFTR modulators hold the promise of improving treatment efficacy for different segments of the CF population, including F508del heterozygotes and F508del homozygotes, where patients have limited access to treatment, are underserved by existing treatments or see a declining benefit in lung function over time."

The FDA's Fast Track program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. An investigational drug that receives Fast Track program designation is eligible for more frequent communications between the FDA and the company relating to the development plan and clinical trial design, and may be eligible for priority review if certain criteria are met.

About Triple Combination Therapy

Proteostasis Therapeutics is developing a combination treatment for CF through co-administration of three separate CFTR modulators: PTI-428, an amplifier, PTI-801, a corrector, and PTI-808, a potentiator.  All three drug candidates were discovered internally at the Company and are designed to act synergistically through complementary mechanisms of action which address specific dysfunctions of the CFTR protein, the underlying cause of the disease.  PTI-428, PTI-801, and PTI-808 have active INDs under the FDA and are currently in clinical trials in patients in the U.S. and Europe.

About Proteostasis Therapeutics, Inc.

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

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 the expected benefits of fast track designation.   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 we may not achieve expedited clinical development or review as a result of the fast track designation, the FDA rescinds such designation if our development program does not continue to meet the criteria for fast track designation, 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 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 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 endorsement, if any, by therapeutic development arms of CF patient advocacy groups, and those set forth in our Annual Report on Form 10-K for the year ended December 31, 2017 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.

CONTACTS:

Investors:
David Pitts 
Argot Partners
212.600.1902 
david@argotpartners.com  

Media: 
David Rosen
Argot Partners 
212.600.1902
david.rosen@argotpartners.com  

View original content with multimedia:http://www.prnewswire.com/news-releases/proteostasis-therapeutics-receives-fda-fast-track-designation-for-triple-combination-program-in-patients-with-cystic-fibrosis-300624037.html

SOURCE Proteostasis Therapeutics, Inc.