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,
announced today that its triple combination clinical study protocol
has received endorsement and a high strategic fit score from the
Therapeutics Development Network (TDN) and the Clinical Trial
Network (CTN). The TDN and CTN are the drug development arms of the
Cystic Fibrosis Foundation (CFF) and the European CF Society
(ECFS), respectively. The study is designed to investigate
the safety, tolerability and pharmacokinetics (PK) of the
coadministration of PTI’s cystic fibrosis transmembrane conductance
regulator (CFTR) modulators, PTI-428, PTI-801 and PTI-808, and will
also assess lung function (FEV1). Both patient advocacy
organizations reviewed the clinical data from healthy volunteers
and CF subjects, including the recent proof-of-concept study with
PTI-428 as add-on to Orkambi®1, as well as the healthy volunteer
data from the coadministration study of all three of PTI’s CFTR
modulators.
Meenu Chhabra, Proteostasis Therapeutics’ President and Chief
Executive Officer, said: “We are humbled that the two preeminent
global CF patient advocacy organizations share our view on the
positive potential clinical impact of our CFTR modulators. Our
proprietary triple combination study is on track to initiate dosing
of CF patients in the first half of this year and we anticipate
initial results in the second half of 2018. We believe that
PTI-428, PTI-801 and PTI-808 have the potential to be a novel, best
in class treatment for CF patients.”
The CFF and ECFS are the most impactful CF patient advocacy
organizations and shepherd the execution of clinical trials in the
U.S. and Europe, respectively. Both organizations have well
established processes to evaluate submitted study protocols based
on scientific merit, study design, feasibility and the overall
clinical research priorities of the CF community. Protocols
are reviewed by a selected group of experienced CF physician
investigators, research coordinators, biostatisticians, people with
CF and other specialists.
In the U.S., upon the protocol endorsement, the TDN provides
access to 89 accredited care centers with demonstrated expertise in
clinical research, and supports study participant recruitment and
execution of studies. Since its founding in 1998, the TDN has
conducted more than 130 clinical studies for CF, including studies
of CFTR modulators. Similarly, in Europe, the CTN provides access
for endorsed studies to 43 large and experienced CF centers located
in 15 different countries, including the U.K. The TDN and the CTN
have established a strong partnership for endorsed studies
conducted in both the U.S. and Europe.
Patrick A. Flume, MD, Professor of Medicine and Pediatrics and
Director of the Cystic Fibrosis Center at the Medical University of
South Carolina, and Lead Principal Investigator for PTI’s triple
combination program said: “We are experiencing an exciting time in
CF drug development and research, with an unprecedented number of
ongoing clinical trials. At the same time, our CF patient
population is a valuable and limited resource. The TDN’s protocol
review and scoring process creates a framework that helps the
investigator and patient community maintain alignment on clinical
research priorities.”
Co-administration of PTI-428, PTI-808 and PTI-801 in CF
subjects on track to initiate in 1H 2018 with initial clinical data
in 2H 2018
PTI recently completed a healthy volunteer co-administration
safety and tolerability study of its three proprietary CFTR
modulators. Safety and PK profiles achieved with seven days of
once-a-day oral dosing of PTI-428, PTI-801 and PTI-808 indicate
these compounds were generally well-tolerated and are potentially
amenable for once a day dosing.
PTI-801, a third generation CFTR corrector, and PTI-428, a CFTR
amplifier, have shown synergy in vitro with Orkambi® and have been
or are being investigated in clinical studies in CF subjects as
add-on modulators in patients on background Orkambi® therapy.
Preliminary safety and PK data with the first dose of PTI-801 from
the ongoing in 14-day dose-range finding study in CF subjects on
background Orkambi® supports its further development as part of
PTI’s proprietary triple combination therapy. PTI recently
announced Phase 2 data for PTI-428 in CF subjects on background
Orkambi®, which showed an improvement in lung function in this
patient population and helped to identify the initial dose for
further combination development.
PTI plans to initiate dosing of CF patients with its proprietary
triple combination in the first half of this year in U.S. and
European CF centers within the TDN and CTN networks, with initial
clinical data expected in the second half of 2018.
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 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 dual 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 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.
CONTACTS:
Investors:David PittsArgot
Partners212.600.1902david@argotpartners.com
Media:David RosenArgot
Partners212.600.1902david.rosen@argotpartners.com
1 Orkambi® is a registered trademark of Vertex Pharmaceuticals,
Inc.
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