BOSTON, July 25, 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 that the first patient has been dosed in the
Company's 28-day, Phase 2 study evaluating its proprietary cystic
fibrosis transmembrane conductance regulator (CFTR) modulator
combinations in F508del homozygous and heterozygous CF
subjects.
Initiation of the 28-day Phase 2 study follows the positive
results of the 14-day Phase 1 clinical studies of PTI's proprietary
doublet (PTI-808 and PTI-801) and triplet (PTI-808, PTI-801 and
PTI-428) combinations. The previous studies demonstrated a
favorable safety and tolerability profile for the combinations, as
well as statistically significant improvement in percent predicted
FEV1 (ppFEV1) and sweat chloride
concentration that was superior to the current CFTR modulator
standard of care for F508del homozygous patients. The ongoing Phase
2 trial will explore efficacy over a longer duration and in
additional genotypes including subjects heterozygous for the
F508del mutation with PTI's doublet and triplet combinations. Dose
selection (600 mg of PTI-801 and 300 mg or PTI-808, with or without
10 mg PTI-428) was based on the totality of dose range finding data
from approximately 250 CF subjects studied thus
far.
"Despite the inclusion of CF subjects colonized with bacteria
associated with a more rapid decline in lung function, our doublet
and triplet studies have delivered a compelling signal of
ppFEV1 improvement," said Geoffrey Gilmartin, M.D., M.M.Sc., Chief Medical
Officer of Proteostasis. "By focusing on European centers, our next
stage in development will target the recruitment of patients
comparable to those used in other CFTR modulator combination
studies which, together with optimal dose levels and longer
treatment duration, could potentially further enhance the magnitude
of pulmonary benefit."
Proteostasis' Phase 2, global, multicenter, randomized,
placebo-controlled study is expected to enroll up to 30 F508del
homozygous patients and up to 30 F508del heterozygous patients.
Study endpoints include safety, changes in sweat chloride
concentration and changes in ppFEV1. Data from the
study are expected in the first quarter of 2020.
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 has 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 potential of our proprietary combination
therapies for the treatment of CF, the potential benefit to
patients of our proprietary combination therapies, expected timing
of patient enrollment in, data from, the completion of, our
clinical studies and cohorts for our clinical programs, including
our planned Phase 2 program and initiation of a pivotal
study. 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 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),
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 March 31, 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
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SOURCE Proteostasis Therapeutics, Inc.