Proteostasis Announces Scientific Presentations On New Data At 30th Annual North American Cystic Fibrosis Conference

CAMBRIDGE, Mass., Sept. 22, 2016 (GLOBE NEWSWIRE) -- Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a biopharmaceutical company developing small molecule therapeutics to treat diseases caused by dysfunctional protein processing, such as cystic fibrosis (CF), today announced that company scientists will present data from its ongoing Phase 1 clinical trials studying PTI-428 in subjects with CF and healthy volunteers at the 30th Annual North American Cystic Fibrosis Conference (NACFC). In addition, Proteostasis Therapeutics scientists will present preclinical data on its planned triple combination therapy, as well as a new class of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators with characteristics distinct from known potentiators, correctors and amplifiers. The meeting will take place Oct. 27 – 29 at the Orange County Convention Center in Orlando, Florida.

“We expect that the data being presented at NACFC will illustrate the potential of our groundbreaking approach to treat people living with cystic fibrosis, a devastating disease with few effective therapies,” said Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics. “In addition to unveiling the interim results of our Phase I trial of PTI-428, we’re excited to discuss findings from our preclinical studies demonstrating the advantages of our planned triple combination therapy over existing treatments including Orkambi and Kalydeco, as well as the discovery of a new class of CFTR modulators to benefit patients with the F508del- mutation – the most common mutation associated with cystic fibrosis.”

Following is a schedule of the Company’s presentations at NACFC, including links to the abstracts:

Title: Phase 1 Initial Results Evaluating Safety, Tolerability, PK and Biomarker Data Using PTI-428, a Novel CFTR Modulator, in Patients with Cystic Fibrosis
Time/Date: Saturday, Oct. 29, 10:30-11:50 a.m. ET
Description: Preliminary data will be presented from the single ascending dose (SAD) and multiple ascending dose (MAD) cohorts of the Phase 1 trials of PTI-428 – a novel genotype-agnostic amplifier – including safety, tolerability, pharmacokinetics (PK) and biomarker evaluation.

Title: A Novel Modulator of CFTR Chloride Ion Mobility with a Distinct Mutation-Specific Profile to Existing CFTR Modulators
Time/Date: Thursday, Oct. 27, 11:15 a.m.-1:45 p.m.
Description: Proteostasis Therapeutics scientists will report a new class of CFTR modulators with characteristics distinct from known potentiators, correctors and amplifiers, which may represent a novel mechanism for improving mutant CFTR function. Investigation of these modulators shows that on F508del- CFTR, they work cooperatively with known correctors and amplifiers, but do not synergize with known potentiators. In CFTR-mediated chloride transport assays, these compounds demonstrate a mutation-dependent efficacy that is distinct from that of known CFTR potentiators.

Title: Novel CFTR Modulator Combination of Amplifier, Corrector and Potentiator Provides Advantages Over Two Corrector-Based Combinations
Time/Date: Thursday, Oct. 27, 11:15 a.m.-1:45 p.m.
Description: Building on the foundation of the novel amplifier class of CFTR modulator, Proteostasis Therapeutics is developing a novel triple-combination therapy using CFTR amplifiers, correctors and potentiators. In human bronchial epithelial (HBE) cells electrophysiology measurements, the triple combination therapy restores the activity of mutant F508del-CFTR protein to near-normal CFTR activity. In addition, the amplifier-based triple combination provides potential advantages through its ability to modulate non-F508del CFTR and by overcoming substrate limitations for correctors and potentiators. Based on the data generated in the HBE cells, the combined use of these three molecules has the potential to restore mutant CFTR function in CF patients homozygous for the F508del mutation to clinically beneficial levels.

Analyst and Investor Event Information
Proteostasis Therapeutics will host an analyst and investor event onsite at NACFC that will be webcast live at 8:15 p.m. ET on Thursday, Oct. 27 to discuss data and provide a brief overview of the science and clinical development plans surrounding the Company’s CF program. The live webcast and accompanying slide presentation can be accessed on the Event Calendar page in the Investors & Media section of the Company's website at The webcast will be available for replay on the company website. Alternatively, analysts and investors may listen to the event by dialing 1-877-626-4740 in the United States or 1-281-973-6278 from outside the United States. Please refer to conference ID number 85250799.

About Cystic Fibrosis
Cystic fibrosis (CF) is a life-threatening, progressive genetic disease affecting an estimated 70,000-100,000 people worldwide. The disease is caused by defects in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which acts as a passageway to promote the transport of salt and water to tissues such as the lungs, skin and pancreas. The defect disrupts healthy ion flow and causes a buildup of thick mucus and bacteria in several organs, resulting in persistent lung infections and the inability for the body to break down food and absorb vital nutrients. While advancements in research and treatments have extended the life expectancy for those living with the disease, CF remains a serious, life-limiting condition with no known cure.

About Proteostasis Therapeutics, Inc.
Proteostasis Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery of groundbreaking therapies to treat diseases caused by dysfunctional protein processing, such as cystic fibrosis (CF). Headquartered in Cambridge, MA, the Proteostasis Therapeutics team focuses on identifying therapies that modulate the proteostasis imbalance in cells and restore protein function. Proteostasis Therapeutics is currently enrolling eligible adults with CF to participate in its Phase 1 clinical trials of PTI-428, a unique CFTR modulator, called a CFTR amplifier, that when used in combination with existing treatments and therapies has shown a consistent positive effect on CFTR protein activity in pre-clinical studies. In addition to its multiple programs in cystic fibrosis, Proteostasis Therapeutics has formed collaborations with Biogen to research and identify therapeutic candidates for neurodegenerative disease and with Astellas Pharma, Inc. to research and identify therapies targeting the Unfolded Protein Response (UPR) pathway. For more information visit

Forward-Looking Statements
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the advancement of, and anticipated development plans related to, the Company’s product candidates and preclinical and clinical studies. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk of cessation or delay of any of the ongoing or planned clinical studies or development activities for our product candidates, the risk of a delay in the enrollment of patients in the Company’s clinical studies, the risk that the results of previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, and the risk that any one or more of our product candidates will not be successfully developed and commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our annual report on Form 10-K for the year ended December 31, 2015, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Proteostasis Therapeutics undertakes no duty to update this information unless required by law.

Media Contact: 
Katie Engleman, Pure Communications, Inc.?
(910) 509-3977

Investor Contact:
Luke Heagle, Pure Communications, Inc.
(910) 726-1372

Back to news