Proteostasis Therapeutics Announces Scientific Presentations on New Data at 30th Annual North American Cystic Fibrosis Confer...
September 22 2016 - 6:30AM
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 that company scientists will present data
from its ongoing Phase 1 clinical trials studying PTI-428 in
subjects with CF and healthy volunteers at the 30th Annual North
American Cystic Fibrosis Conference (NACFC). In addition,
Proteostasis Therapeutics scientists will present preclinical data
on its planned triple combination therapy, as well as a new class
of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)
modulators with characteristics distinct from known potentiators,
correctors and amplifiers. The meeting will take place Oct. 27 – 29
at the Orange County Convention Center in Orlando, Florida.
“We expect that the data being presented at NACFC will
illustrate the potential of our groundbreaking approach to treat
people living with cystic fibrosis, a devastating disease with few
effective therapies,” said Meenu Chhabra, President and Chief
Executive Officer of Proteostasis Therapeutics. “In addition to
unveiling the interim results of our Phase I trial of PTI-428,
we’re excited to discuss findings from our preclinical studies
demonstrating the advantages of our planned triple combination
therapy over existing treatments including Orkambi and Kalydeco, as
well as the discovery of a new class of CFTR modulators to benefit
patients with the F508del- mutation – the most common mutation
associated with cystic fibrosis.”
Following is a schedule of the Company’s presentations at NACFC,
including links to the abstracts:
Title: Phase 1 Initial Results Evaluating
Safety, Tolerability, PK and Biomarker Data Using PTI-428, a Novel
CFTR Modulator, in Patients with Cystic
FibrosisTime/Date: Saturday, Oct. 29, 10:30-11:50
a.m. ETDescription: Preliminary data will be
presented from the single ascending dose (SAD) and multiple
ascending dose (MAD) cohorts of the Phase 1 trials of PTI-428 – a
novel genotype-agnostic amplifier – including safety, tolerability,
pharmacokinetics (PK) and biomarker evaluation.
Title: A Novel Modulator of CFTR Chloride Ion
Mobility with a Distinct Mutation-Specific Profile to Existing CFTR
ModulatorsTime/Date: Thursday, Oct. 27, 11:15
a.m.-1:45 p.m.Description: Proteostasis
Therapeutics scientists will report a new class of CFTR modulators
with characteristics distinct from known potentiators, correctors
and amplifiers, which may represent a novel mechanism for improving
mutant CFTR function. Investigation of these modulators shows that
on F508del- CFTR, they work cooperatively with known correctors and
amplifiers, but do not synergize with known potentiators. In
CFTR-mediated chloride transport assays, these compounds
demonstrate a mutation-dependent efficacy that is distinct from
that of known CFTR potentiators.
Title: Novel CFTR Modulator Combination of
Amplifier, Corrector and Potentiator Provides Advantages Over Two
Corrector-Based CombinationsTime/Date: Thursday,
Oct. 27, 11:15 a.m.-1:45 p.m.Description: Building
on the foundation of the novel amplifier class of CFTR modulator,
Proteostasis Therapeutics is developing a novel triple-combination
therapy using CFTR amplifiers, correctors and potentiators. In
human bronchial epithelial (HBE) cells electrophysiology
measurements, the triple combination therapy restores the activity
of mutant F508del-CFTR protein to near-normal CFTR activity. In
addition, the amplifier-based triple combination provides potential
advantages through its ability to modulate non-F508del CFTR and by
overcoming substrate limitations for correctors and potentiators.
Based on the data generated in the HBE cells, the combined use of
these three molecules has the potential to restore mutant CFTR
function in CF patients homozygous for the F508del mutation to
clinically beneficial levels.
Analyst and Investor Event
InformationProteostasis Therapeutics will host an analyst
and investor event onsite at NACFC that will be webcast live at
8:15 p.m. ET on Thursday, Oct. 27 to discuss data and provide a
brief overview of the science and clinical development plans
surrounding the Company’s CF program. The live webcast and
accompanying slide presentation can be accessed on the Event
Calendar page in the Investors & Media section of the Company's
website at www.proteostasis.com. The webcast will be available for
replay on the company website. Alternatively, analysts and
investors may listen to the event by dialing 1-877-626-4740 in the
United States or 1-281-973-6278 from outside the United States.
Please refer to conference ID number 85250799.
About Cystic FibrosisCystic fibrosis (CF) is a
life-threatening, progressive genetic disease affecting an
estimated 70,000-100,000 people worldwide. The disease is caused by
defects in the cystic fibrosis transmembrane conductance regulator
(CFTR) protein, which acts as a passageway to promote the transport
of salt and water to tissues such as the lungs, skin and pancreas.
The defect disrupts healthy ion flow and causes a buildup of thick
mucus and bacteria in several organs, resulting in persistent lung
infections and the inability for the body to break down food and
absorb vital nutrients. While advancements in research and
treatments have extended the life expectancy for those living with
the disease, CF remains a serious, life-limiting condition with no
known cure.
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, a unique CFTR modulator, called a CFTR
amplifier, that when used in combination with existing treatments
and therapies has shown a consistent positive effect on CFTR
protein activity in pre-clinical studies. 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 Statements This 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. These risks
and uncertainties include, but are not limited to, the risk of
cessation or delay of any of the ongoing or planned clinical
studies or development activities for our product candidates, the
risk of a delay in the enrollment of patients in the Company’s
clinical studies, the risk that the results of previously conducted
studies involving similar product candidates will not be repeated
or observed in ongoing or future studies involving current product
candidates, and the risk that any one or more of our product
candidates will not be successfully developed and commercialized.
For a discussion of other 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 annual report on Form
10-K for the year ended December 31, 2015, as well as discussions
of potential risks, uncertainties, and other important factors in
our subsequent 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
Proteostasis Therapeutics (NASDAQ:PTI)
Historical Stock Chart
From Aug 2024 to Sep 2024
Proteostasis Therapeutics (NASDAQ:PTI)
Historical Stock Chart
From Sep 2023 to Sep 2024