Proteostasis Therapeutics Issues Mid-Year Corporate Update on Cystic Fibrosis Pipeline Programs
August 11 2016 - 07:45AM
Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a biopharmaceutical
company developing small molecule therapeutics to treat diseases
caused by dysfunctional protein processing such as cystic fibrosis,
today announced a mid-year corporate update highlighting the
company’s continued progress and expansion of clinical programs and
path forward to reach anticipated milestones.
Proteostasis Therapeutics’ lead drug candidate is PTI-428. It is
the first genotype-agnostic, disease-modifying agent to be tested
in the clinic for cystic fibrosis. PTI-428 is a unique modulator
(amplifier) of the Cystic Fibrosis Transmembrane Conductance
Regulator (CFTR) protein that has shown a consistent positive
effect on CFTR mRNA and protein activity in vitro and in
pre-clinical studies when used in combination with existing
treatments.
Proteostasis Therapeutics is currently enrolling eligible adults
in two ongoing Phase 1 clinical trials with PTI-428. The first
trial is in cystic fibrosis patients with any CFTR mutation, and
the second trial is in normal healthy volunteers.
Preliminary data in healthy volunteers suggest a dose-linear
pharmacokinetic drug profile that supports once-a-day dosing. Data
from 24 healthy volunteers confirmed the expected and
dose-dependent relationship between PTI-428 exposure and the
magnitude of CFTR mRNA increase. A single dose of PTI-428 can
increase CFTR mRNA expression by approximately two-fold in those
healthy volunteers. No safety concerns have been identified to date
based on reviews of vital signs, clinical exams and hematology lab
values. In vitro and in vivo animal studies with PTI-428 have shown
that a two-fold increase in CFTR mRNA may lead to a potential
doubling of lung function improvement on top of the existing
standard-of-care therapies.
“This is an incredibly exciting and unprecedented time in CF
drug development. Never before have there been 59 cystic fibrosis
clinical studies ongoing simultaneously in the U.S. Given this
effective traffic jam at the cystic fibrosis clinical centers, we
have experienced slightly longer than anticipated site start-up
times. Now, with a critical mass of sites up and running, coupled
with the high level of enthusiasm from our investigators about the
unique profile of our drug, we remain on track with our original
schedule to generate topline efficacy by year end,” said Meenu
Chhabra, president and chief executive officer of Proteostasis
Therapeutics. “We have made significant progress across our cystic
fibrosis pipeline where we have collected initial clinical data
with PTI-428. In addition, we have declared two new clinical
candidates: PTI-801, a “turbocorrector,” and PTI-808, a
potentiator, as part of our planned triple combination therapy. We
expect to file INDs for these two programs by year end.”
Additional Highlights
CFTR Nasal BiomarkerMeasurements in nasal
brushing samples confirm up-regulation of the CFTR mRNA upon the
exposure of subject to PTI-428. Preliminary data supports expected
target engagement and further enables the projection of the
clinically efficacious dose for the subsequent proof-of-concept
study in cystic fibrosis patients.
PTI-801 and PTI-808We have further advanced
preclinical development of PTI-801 (corrector) and PTI-808
(potentiator) molecules which, when combined with PTI-428, are the
active components of the proprietary triple combination therapy
PTI-NC-733. In the Ussing chamber assay, PTI-NC-733 achieves
approximately 100 percent of normal CFTR levels, which could
translate into a more clinically meaningful benefit to a broader
set of mutation classes than current or pipeline therapies.
North American Cystic Fibrosis Conference
(NACFC)Proteostasis Therapeutics has been selected to
present results of several studies at the 30th Annual NACFC from
Oct. 27-29, 2016 in Orlando, Florida. Planned presentations include
data from clinical studies on PTI-428 as well as preclinical data
on PTI-NC-733 and additional novel compounds aimed at restoration
of CFTR activity.
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 modulate the proteostasis imbalance in cells and restore
protein function. Proteostasis Therapeutics is currently enrolling
eligible adults with CF to participate in its Phase 1 clinical
trials of PTI-428. In addition to its multiple programs in cystic
fibrosis, Proteostasis Therapeutics has formed collaborations with
Biogen to research and identify therapeutic candidates for
neurodegenerative disease and with Astellas Pharma, Inc. to
research and identify therapies targeting the Unfolded Protein
Response (UPR) pathway. For more information, visit
www.proteostasis.com.
Forward-Looking StatementsThis release contains
"forward-looking statements" within the meaning of the Private
Securities Litigation Reform Act of 1995, including statements
regarding the advancement of, and anticipated development plans
related to, the Company's product candidates and preclinical and
clinical studies. Any forward-looking statements are based on
management's current expectations of future events and are subject
to a number of risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in
or implied by such forward-looking statements. For a discussion of
risks and uncertainties, and other important factors, any of which
could cause our actual results to differ from those contained in
the forward-looking statements, see the section entitled "Risk
Factors" in our most recent Quarterly Report on Form 10-Q on file
with the Securities and Exchange Commission, as well as discussions
of potential risks, uncertainties, and other important factors in
our subsequent and future filings with the Securities and Exchange
Commission. All information in this press release is as of the date
of the release, and Proteostasis Therapeutics undertakes no duty to
update this information unless required by law.
Media Contact:
Katie Engleman, Pure Communications, Inc.
(910) 509-3977
katie@purecommunicationsinc.com
Investor Contact:
Luke Heagle, Pure Communications, Inc.
(910) 726-1372
luke@purecommunicationsinc.com
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