BOSTON, Nov. 6, 2019 /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), today announced completion of enrollment in the Company's
global, multicenter, randomized, placebo-controlled, 28-day, Phase
2 study evaluating its proprietary cystic fibrosis transmembrane
conductance regulator (CFTR) modulator combinations in F508del
homozygous and heterozygous CF subjects.
The trial is designed to assess the efficacy, safety and
tolerability of PTI's proprietary combinations over four weeks and
with higher doses of proprietary CFTR corrector and potentiator.
Dose selection (600 mg of PTI-801 and 300 mg of PTI-808, with or
without 10 mg of PTI-428) was based on the totality of dose range
finding data from approximately 250 CF subjects studied thus far.
Study endpoints include safety, changes in sweat chloride
concentration and changes in percent predicted FEV1
(ppFEV1). The study design targeted up to 30 F508del
homozygous and up to 30 F508del heterozygous subjects. Due to
rapid enrollment from centers in the
United States, Canada,
Western Europe, and New Zealand, data from the study are now
expected in the fourth quarter of 2019 instead of the first quarter
of 2020.
"We are working hard to introduce the power of choice to the CF
community and the completion of enrollment in our global Phase 2
study is an important step forward to completing that mission. We
heard loud and clear at our recently hosted Cystic Fibrosis Summit,
as well as at the last week's North American CF Conference, that
the community urgently demands options to address the inequity in
access, variability in clinical response and genetic disparity
amongst people with CF," said Geoffrey
Gilmartin, M.D., M.M.Sc., Chief Medical Officer of
Proteostasis. "Due to this strong demand for options and additional
trials from the patient and medical community, we're pleased to
announce that we now expect top-line data from the Phase 2 study
later this quarter."
The 28-day Phase 2 study follows the positive results of the
14-day Phase 1 clinical studies of PTI's proprietary doublet and
triplet combinations in F508del homozygous patients, including
those predisposed to rapid pulmonary decline based on their
bacterial colonization status. The previous studies demonstrated a
favorable safety and tolerability profile for the combinations, as
well as a statistically significant improvement in
ppFEV1 and sweat chloride concentration that was
superior to the available dual CFTR modulator standard of care.
About PTI-428, PTI-801, PTI-808
PTI-428 is an
investigational CFTR amplifier in development for the treatment of
CF in patients with at least one F508del mutation in the CFTR
gene, as part of PTI's proprietary triple combination regimen
that includes PTI-808, a novel potentiator, and PTI-801, a
third-generation CFTR corrector. PTI-801 received Fast Track
Designation from the U.S. Food and Drug Administration (FDA).
In May 2019, PTI-428 received
Orphan Drug Designation (ODD) from the European Commission (EC).
In addition to ODD from the EC, PTI-428 has ODD, Breakthrough
Therapy Designation and Fast Track Designation from the
FDA.
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. 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 the ongoing trials of our product
candidates, the expected timing for completion and reporting
of topline results of our Phase 2 clinical trial and our
expectations regarding expanding available therapeutic options for
CF patients. 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 potential of our proprietary combination
therapies for the treatment of CF, the potential benefit of our
proprietary combination therapies to patients, expected completion
of our clinical studies and cohorts for our clinical programs,
including our planned Phase 2 program and initiation of a pivotal
study, 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 or
other regulatory agency comments delay, change or do not permit
trial commencement, or intended label, or the FDA or other
regulatory agency 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),
in the commercialization and acceptance of new therapies, and those
set forth in our Annual Report on Form 10-K for the year ended
December 31, 2018, our Quarterly
Report on Form 10-Q for the quarter ended June 30, 2019 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
/ Claudia Styslinger
Argot Partners
212.600.1902
david@argotpartners.com / claudia@argotpartners.com
Media:
David Rosen
Argot Partners
212.600.1902
david.rosen@argotpartners.com
View original
content:http://www.prnewswire.com/news-releases/proteostasis-therapeutics-announces-completion-of-enrollment-in-global-phase-2-study-in-cystic-fibrosis-300952416.html
SOURCE Proteostasis Therapeutics, Inc.