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
41st European Cystic Fibrosis Society (ECFS) Conference on June 6-9, 2018 in
Belgrade, Serbia.
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Details of the presentations are as follows:
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Date & Time:
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Thursday, June 7, 2018, 10:30 AM to 12:00 PM CEST
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Session Title:
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Mix and Match! Combination Cystic Fibrosis Transmembrane
Conductance
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Regulator (CFTR) Modulation Therapies and New Trials
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Session Type:
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Symposium
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Presenter:
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Geoffrey S. Gilmartin, M.D., M.M.Sc., Chief Development Officer of
Proteostasis
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Moderators:
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Dr. Pavel Drevinek, Charles University, Prague, Czech Republic; Dr. Silke
van
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Koningsbruggen-Rietschel, Cystic Fibrosis Study Center, University
Hospital, Cologne, Germany
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Date & Time:
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Thursday, June 7, 2018, 3:00 PM to 4:30 PM CEST
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Session Title:
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Exciting News From CFTR Modulator Clinical Trials
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Session Type:
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Panel Presentation
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Presenter:
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Patrick Flume, M.D., Professor of Medicine and Pediatrics at
the Medical University of
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South Carolina
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Moderators:
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Dr. Silke van Koningsbruggen-Rietschel, Cystic Fibrosis Study Center,
University Hospital, Cologne, Germany; Dr. Lieven Dupont, Univeritair Ziekenhuis Leuven, Belgium
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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, www.proteostasis.com.
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
www.proteostasis.com.
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.
CONTACTS:
Investors:
David Pitts
Argot Partners
212.600.1902
david@argotpartners.com
Media:
David Rosen
Argot Partners
212.600.1902
david.rosen@argotpartners.com
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SOURCE Proteostasis Therapeutics, Inc.