Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a biopharmaceutical
company developing small molecule therapeutics to treat diseases
caused by dysfunctional protein processing such as cystic fibrosis
(CF), today announced updates across the Company’s later stage
development programs in CF, including PTI-428, a cystic fibrosis
transmembrane conductance regulator (CFTR) amplifier, PTI-801, a
new generation CFTR corrector, and PTI-808, a CFTR
potentiator. The updates were announced during the 40th
European Cystic Fibrosis Society conference in Seville, Spain,
where the Company is presenting detailed results from the single
ascending dose (SAD) cohort of its Phase 1 trial of PTI-428 in
healthy volunteer and CF subjects, as well as its biobank and
discovery of read-through molecules initiatives. A copy of
the detailed results will be made available on www.proteostasis.com
at the start of the presentation on Friday, June 9, at 3:15 pm CET.
“With renewed momentum behind our three lead
CFTR modulator programs, we look forward to meaningful clinical
results later this month,” said Meenu Chhabra, president and chief
executive officer of Proteostasis Therapeutics. “For our lead
program, PTI-428, we anticipate safety and pharmacokinetic data
from at least eight CF subjects receiving PTI-428 with Orkambi®
background therapy for seven days, as well as up to eight subjects
receiving PTI-428 as their sole CFTR modulator therapy for seven
days. The results will also include secondary endpoints, such as
lung function, sweat chloride and CFTR expression. Results
from these cohorts, and others we expect in the third quarter, will
provide a solid foundation for understanding PTI-428’s role in
triple combination therapy. We believe that the treatment paradigm
for the vast majority of CF patients could be based on combination
therapies of CFTR modulators anchored by PTI-428. By year end, we
expect to advance into the clinic with our proprietary triple
combination therapy, which we believe has the potential to be best
in class.”
PTI-428 Amplifier Clinical Data
in Three Separate Populations of CF Patients on Track for
Mid-Year
By the end of June, Proteostasis expects to
report preliminary data, including lung function, from multiple
ascending dose (MAD) cohorts of two different CF patient
populations: at least eight CF subjects receiving PTI-428 or
placebo in addition to Orkambi® as their background for seven days
and up to eight CF subjects receiving PTI-428 as their sole CFTR
modulator therapy or placebo for seven days. Lung function,
as measured by forced expiratory volume in one second, or FEV1, is
being assessed, as well as changes in sweat chloride. Underscoring
rising momentum in the program, expanded data from four additional
subjects in each arm is anticipated next month.
After a washout period, patients on Orkambi®
background therapy who were enrolled in the 7-day dosing MAD cohort
are eligible to enroll in a 28-day clinical study for PTI-428,
which is expected to start at the end of June. This longer duration
study of CF subjects already on standard Orkambi® background
therapy is expected to generate safety and efficacy data from up to
20 subjects, with the goal of identifying a dose level for the
triple combination proof of concept (POC) combining PTI-428,
PTI-801 and PTI-808 (known as PTI-NC-733).
Proteostasis is studying PTI-428 in multiple CF
populations because in vitro studies indicate it is agnostic to CF
genotype. Results from a separate study in a third patient
population, where CF patients will receive PTI-428 or placebo in
addition to Kalydeco® as background therapy for 14 days, is
expected in the third quarter.
PTI-801 New Generation Corrector
Phase 1 Healthy Volunteer SAD Study Complete; MAD Study
Underway
Proteostasis announced today completion of the
healthy volunteer, SAD portion of its Phase 1 study to assess the
safety and pharmacokinetics of PTI-801. A 14-day MAD portion in
healthy volunteers is underway in the U.S., with results from both
study portions to be submitted for presentation at an upcoming
medical meeting. Safety, pharmacokinetics and efficacy data
from CF subjects on background Orkambi® therapy is expected in the
second half of 2017. The goal of this study is to identify a
dose level for the triple combination POC combining PTI-428,
PTI-801 and PTI-808.
PTI-808 Potentiator Advancing to
IND; DRF Study Intended to be POC for Triple Combination by Year
End 2017
As previously announced, Proteostasis expects to
submit an Investigational New Drug (IND) application for PTI-808,
its CFTR potentiator, to the Food and Drug Administration (FDA) by
the end of this month; the Company plans to initiate a Phase 1
study in healthy volunteers in July.
If the Company receives positive results from
its PTI-428 and PTI-801 programs, the Company intends to initiate a
dose range finding (DRF) study at the end of 2017 in an F508del
homozygous population who are not taking Orkambi®. The study design
is expected to include DRF of PTI-808 with fixed dose combination
of PTI-428 and PTI-801. The DRF study with PTI-808 is also intended
to be considered a POC study for the triple combination.
About Proteostasis Therapeutics,
Inc.
Proteostasis Therapeutics, Inc. is a
biopharmaceutical company dedicated to the discovery of
groundbreaking therapies to treat diseases caused by dysfunctional
protein processing, such as cystic fibrosis (CF). 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. 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 expected timing of the
initiation of, patient enrollment in, data from, and our completion
of, our clinical studies and cohorts for PTI-428, PTI-801, PTI-808
and our triple combination therapy candidate, PTI-NC-733, and the
timing of our expected IND filing for PTI-808.
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, uncertainties inherent in the execution and completion
of clinical trials (including, without limitation, the possibility
FDA requires us to run cohorts sequentially or additional cohorts
or 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 the actions of regulatory agencies, in
endorsement, if any, by therapeutic development arms of CF patient
advocacy groups, and those set forth in our Annual Report on Form
10-K for the year ended December 31, 2016, 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
Argot Partners
212.600.1902
david@argotpartners.com
Media:
Eliza Schleifstein
Argot Partners
973.361.1546
eliza@argotpartners.com
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