BOSTON, June 4, 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) and other
diseases caused by dysfunctional protein processing, today
announced that the European Commission (EC) has granted orphan drug
designation (ODD) to PTI-428 for the treatment of cystic fibrosis.
PTI-428 is the Company's proprietary cystic fibrosis transmembrane
conductance regulator (CFTR) amplifier that is currently in
clinical development. In addition to ODD from the EC, PTI-428 has
ODD, Breakthrough Therapy Designation and Fast Track Designation
from the U.S. Food and Drug Administration.
ODD in the European Union (EU) is based upon a positive
opinion from the European Medicines Agency's (EMA) Committee for
Orphan Medicinal Products (COMP) and provides regulatory and
financial incentives for companies to develop and market therapies
to treat serious disorders affecting no more than five in 10,000
persons in the EU. Companies that obtain ODD benefit from a
number of incentives, including ten-year marketing exclusivity in
the EU upon approval, as well as eligibility for protocol
assistance, reduced fees and access to the EU's centralized
marketing authorization procedure. Currently, over half of the
global CF population lives in Europe with limited access to approved CFTR
modulator therapies.
"This designation by the EC is further validation of both
PTI-428's potential and PTI's mission to offer additional disease
modifying treatment options for CF," said Meenu Chhabra, President and Chief Executive
Officer of Proteostasis Therapeutics. "Orphan drug status is
granted to development-stage drugs that make a major contribution
to patients' care, either by demonstrating a significant clinical
benefit over existing therapies or by providing a treatment for
patients for whom existing therapies do not work. We look forward
to advancing PTI-428 in the clinic later this year, as part of the
planned 28-day Phase 2 studies of our proprietary combination CFTR
modulator treatments."
About PTI-428
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 corrector. PTI-428 has been shown to
work early during CFTR biogenesis to increase levels of newly
synthesized CFTR protein, suggesting potential therapeutic benefits
in combination with CFTR correctors and potentiators.
In March, PTI announced results from the Company's 14-day
clinical studies of its proprietary combination CFTR
modulators in CF subjects. Later this year, the Company is
planning to initiate Phase 2, placebo-controlled,
28-day studies in CF subjects with at least
one F508del mutation.
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 presentations and expected timing of the initiation 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 the potential benefits
associated with orphan drug 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 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, including maintaining orphan drug
designation, 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.