BOSTON, Jan. 13, 2020 /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 a regulatory update following the completion of a
scientific advice meeting with the Medicines and Healthcare
Products Regulatory Agency (MHRA), UK, regarding its two pivotal
studies, MORE and CHOICES.
Both studies are designed to explore the potential of
Proteostasis' proprietary cystic fibrosis transmembrane conductance
regulator (CFTR) modulator combinations that include dirocaftor,
posenacaftor and nesolicaftor, a CFTR potentiator, corrector and
amplifier, respectively. The MHRA's scientific advice
outlined a clear path forward toward the initiation and execution
of the proposed two-pronged Phase 3 program, including
establishment of a common safety database to support the safety
profile of the proprietary combination, all toward the goal of
supporting a Marketing Authorization Application for dirocaftor,
posenacaftor and nesolicaftor. Both trials are expected to
begin in 2020 and can run concurrently, building on the safety and
efficacy database Proteostasis has established to date in over 300
patients with CF. The Company will continue to seek
additional advice from other major regulatory agencies throughout
2020.
"CF is a heterogenous disease whose limited CFTR treatment
options leave a significant portion of patients with little or no
benefit. This is underscored by what we have demonstrated in
our own data, including Phase 2 results comparable to the recently
approved CFTR modulator triple combinations, despite a higher
disease burden study population, and based on our own market
research, where physicians report 1 in 3 patients either
discontinuing triple combination treatment for poor tolerability or
achieving suboptimal benefit. Our goal is to change this by
delivering more choices to patients to better reflect an
individualized treatment approach and provide to each patient with
CFTR modulators that lead to highest benefit," said Meenu Chhabra, President and Chief Executive
Officer of Proteostasis Therapeutics.
Ms. Chhabra continued: "We value the opportunity to engage with
regulators who recognize this challenge, and we are pleased that
the Company's preclinical and clinical programs were in line with
MHRA expectations for the conduct of our pivotal program. The
Proteostasis team continues to work closely with European Medicines
Agency and other regulators toward finalizing the details of this
program and, with positive outcomes, to work toward ensuring its
support for the registration for dirocaftor, posenacaftor and
nesolicaftor."
The CHOICES trial (Crossover trial based on Human Organoid
Individual response in CF - Efficacy Study) is designed to be the
first ever personalized medicine-based study in CF. CHOICES
seeks to translate promising responses from an ex vivo
organoids study of PTI modulators in rare CF mutations that is part
of a pan-European strategic initiative, known as HIT-CF (Human
Individualized Therapy of CF), whose goal is to accelerate the
development of, and access to, personalized therapies for CF
patients. The MORE trial (Modulator Options to RestorE CFTR
study) is designed as a global, Phase 3, randomized,
placebo-controlled study in CF subjects with the common F508del
homozygous mutation, and will seek to confirm the positive efficacy
and tolerability results from a recently completed Phase 2 study of
the Proteostasis CFTR modulator triple combination.
About Dirocaftor, Posenacaftor and Nesolicaftor
Nesolicaftor (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 dirocaftor (PTI-808), a
novel potentiator, and posencaftor (PTI-801), a third-generation
CFTR corrector. Posencaftor received Fast Track Designation from
the U.S. Food and Drug Administration (FDA). In May 2019, nesolicaftor received Orphan Drug
Designation (ODD) from the European Commission (EC). In
addition to ODD from the EC, nesolicaftor 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 our clinical studies and cohorts for our
clinical programs, including our planned Phase 3 programs and
initiation of registrational or pivotal studies.
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 September 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
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SOURCE Proteostasis Therapeutics, Inc.