Proteostasis Therapeutics Announces FDA Grants Breakthrough Therapy Designation for PTI-428 in Cystic Fibrosis
March 12 2018 - 4:05PM
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 U.S. Food and Drug
Administration (FDA) has granted Breakthrough Therapy
Designation for PTI-428, the Company’s cystic fibrosis
transmembrane conductance regulator (CFTR) amplifier.
FDA Breakthrough Therapy Designation is intended to expedite the
development and review of a drug aimed at treating a serious or
life-threatening disease where there is a significant unmet need
and preliminary clinical evidence indicates that the drug may offer
substantial improvement over existing therapies. Less than
half of the drugs submitted for Breakthrough Therapy under the Food
and Drug Administration Safety and Innovation Act have been granted
the designation since the inception of the program. In the CFTR
modulator category, Breakthrough Therapy Designation was granted to
Kalydeco® and Orkambi®.
FDA granted the Breakthrough Therapy Designation for PTI-428 for
the treatment of CF in homozygous patients for the F508del mutation
who are receiving Orkambi® as background therapy. The
designation was based on the results from a recent Phase 2,
randomized, placebo controlled study of PTI-428, in 24 CF subjects
on background treatment with Orkambi® and who were treated with
either 50 mg PTI-428 once daily or placebo for 28 consecutive days.
The study results showed that treatment with PTI-428 led to mean
absolute improvement in percent predicted forced expiratory volume
in 1 second (ppFEV1) of 5.2 percentage points from baseline through
Day 28 compared to placebo (p<0.05). Additionally,
consistent with the CFTR amplifier mechanism of action, a positive
increase in nasal mucosal CFTR protein was observed in PTI-428
treated subjects and the magnitude of change compared to baseline
was consistent with the changes in CFTR protein levels observed in
the in vitro human bronchial cell model, whereas the placebo
subjects had no significant increase in CFTR protein during the
treatment period.
“We believe the Breakthrough Therapy Designation for PTI-428
reflects the strength of the recent Phase 2 study results for our
amplifier, a novel and proprietary class of CFTR modulators,”
said Meenu Chhabra, president and chief executive officer of
Proteostasis. “PTI-428 can potentially be added to current
and future standards of care, offering the potential for
improvement in pulmonary function for patients with cystic
fibrosis. We look forward to working closely with
the FDA as we advance our clinical programs for PTI-428,
including as part of our proprietary triple combination with
PTI-801 and PTI-808, our third generation corrector and
potentiator, respectively.”
About PTI-428
PTI-428 is an investigational CFTR amplifier in development for
the treatment of CF in patients who are homozygous for the F508del
mutation in the CFTR gene as an add-on therapy to approved CFTR
modulators or as part of PTI’s proprietary triple combination
regimen that includes PTI-808, a potentiator, and PTI-801, a
corrector. PTI-428 works early during CFTR biogenesis to increase
levels of newly synthesized CFTR protein, suggesting potential
therapeutic benefits in combination with CFTR correctors and
potentiators.
About Proteostasis Therapeutics, Inc.
Proteostasis Therapeutics, Inc. is a clinical stage
biopharmaceutical company developing small molecule therapeutics to
treat cystic fibrosis (CF) and other diseases caused by
dysfunctional protein processing. Headquartered in Cambridge,
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.
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 expected benefits of breakthrough therapy
designation and possible add-on therapy indications for our drug
candidates. 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 we may not achieve
expedited clinical development or review as a result of the
breakthrough therapy designation, the FDA rescinds such designation
if our development program does not continue to meet the criteria
for breakthrough therapy designation, 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 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 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 endorsement, if any, by
therapeutic development arms of CF patient advocacy groups, and
those set forth in our Quarterly Report on Form 10-Q for the
quarter ended September 30, 2017 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 is a registered trademark of Vertex Pharmaceuticals,
Inc.
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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