Proteostasis Therapeutics Announces FDA Grants Orphan Drug Designation for PTI-428 in Cystic Fibrosis
March 14 2018 - 4:30PM
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 Orphan Drug Designation for
PTI-428, the Company’s cystic fibrosis transmembrane conductance
regulator (CFTR) amplifier drug candidate.
“This is the second important regulatory
designation PTI-428 has been granted from the FDA this week,
highlighting our amplifier’s potential to provide clinical benefit
in the treatment of CF,” said Meenu Chhabra, president and chief
executive officer of Proteostasis Therapeutics."
The FDA Office of Orphan Products Development grants Orphan Drug
Designation to novel drugs or biologics that are intended for the
treatment of rare diseases or conditions affecting fewer than
200,000 patients in the United States. The designation allows
the sponsor of the drug to be eligible for various incentives,
including a seven-year period of U.S. marketing exclusivity upon
regulatory approval of the drug, as well as tax credits for
clinical research costs, annual grant funding, clinical trial
design assistance, and the waiver of Prescription Drug User Fee Act
(PDUFA) filing fees.
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 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 addition to Orphan Drug
Designation, PTI-428 has been granted Breakthrough Therapy
Designation as well as Fast Track Designation from the FDA.
In December, Proteostasis announced the results from a Phase 2,
randomized, placebo controlled study of PTI-428, in 24 CF subjects
on background Orkambi therapy which 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).
Proteostasis plans to initiate a triple combination CF Study of
PTI-428 with PTI-801, its third generation corrector, and PTI-808,
its potentiator, in the second quarter of 2018, with preliminary
results anticipated in the second half of 2018. The Company is also
planning an additional study of PTI-428 in CF Subjects on Symdeko™,
with initial data anticipated in early 2019.
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 benefits of Orphan Drug
designation and the expected timing of the initiation of, patient
enrollment in, data from, and the completion of, our clinical
studies and cohorts for PTI-428, PTI-801, PTI-808 and our double
and triple combination therapy 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 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 in
the possibility that we do not maintain Orphan Drug status and
therefore lose the benefits thereof, and those set forth in our
Quarterly Report on Form 10-Q for fiscal 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® and Symdeko™ are trademarks of Vertex
Pharmaceuticals Incorporated.
CONTACTS:
Investors:David PittsArgot
Partners212.600.1902david@argotpartners.com
Media:David RosenArgot
Partners212.600.1902david.rosen@argotpartners.com
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