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 new preclinical data on the company’s
first-in-class genotype-agnostic amplifier and novel corrector at
the 13th Annual European Cystic Fibrosis Society (ECFS) Basic
Science Conference near Pisa, Italy from March 30-April 2. The
company will hold a conference call and webcast today at 4:00 p.m.
ET to discuss the data being presented at the conference and the
company’s outlook for 2016.
“We are strongly encouraged by our early findings and the
potential implications for improving the lives of people with
cystic fibrosis,” said Meenu Chhabra, President and Chief Executive
Officer of Proteostasis Therapeutics. "Patients treated with
currently available therapies still struggle to achieve full lung
function, while many are not receiving treatment at all due to rare
mutations of the disease. The results from our preclinical studies
show the potential of our CFTR amplifier to restore lung function
to near-normal levels not only in patients with the most common
gene mutation found in cystic fibrosis, but across multiple gene
mutations.”
In the company’s poster presentation, “Characterization of CFTR
amplifiers, mutation-agnostic modulators that increase protein
levels and complement other CF therapeutic modalities,” company
researchers describe a new class of modulator – an amplifier –
which increases the levels of immature CFTR protein available for
other modulators, such as correctors and potentiators, to act
upon.
The study found that amplifiers
- increase CFTR immature protein and stabilize CFTR mRNA;
- increase substrate for additional modulators;
- work across CFTR genotypes; and,
- demonstrate activity in non-lung tissues and in vivo.
The early findings suggest the potential for the novel class of
amplifiers to enhance CFTR translation efficiency across CFTR
mutations and be used in combinations to boost the activity of
additional modulators in treating CF.
In a second poster presentation, company scientists have
identified a novel corrector with properties that differentiate it
from clinical-stage correctors such as lumacaftor or VX-661. The
study, “A novel corrector for F508del-CFTR that complements
existing CFTR modulators in vitro,” demonstrated that exposure to
Proteostasis Therapeutics’ corrector significantly increases the
proportion of mature CFTR protein. Moreover, in vitro CFTR function
is increased accordingly when the company’s corrector is dosed with
other modulators, including lumacaftor, potentiators and its
amplifier.
"We believe we have the potential to dramatically improve
therapeutic outcomes for patients with cystic fibrosis," said
Po-Shun Lee, M.D., Executive Vice President and Chief Medical
Officer of Proteostasis Therapeutics. “The data not only validate
the use of our novel CFTR amplifiers in combination with existing
therapies such as Orkambi and Kalydeco, but they also represent an
important early milestone in developing a new and possibly
game-changing triple combination therapy for those living with the
disease.”
Following is a schedule of the company’s poster presentations
and conference call:
Poster Presentations
Thursday, March
31Time: 2:30-3:30 p.m. CEST
Title: Characterization of CFTR amplifiers,
mutation-agnostic modulators that increase protein levels and
complement other CF therapeutic modalities
Friday, April
1Time: 6:30-7:30 p.m. CEST
Title: A novel corrector for F508del-CFTR
that complements existing CFTR modulators in vitro
Conference Call and Webcast
Wednesday, March
30Time: 4:00 p.m. ETThe conference
call can be accessed by dialing 1-877-626-4740 (toll-free domestic)
or 1-281-973-6278 (international) and referring to conference ID
78792593. A live webcast and accompanying slide presentation will
be available on the Event Calendar page in the Investors &
Media section of the company’s website, www.proteostasis.com. A
replay of the webcast will be available on the company’s website
shortly after the conclusion of the conference call.
About Cystic Fibrosis
Cystic 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
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 the proteostasis network and
identifying therapies that modulate the proteostasis imbalance in
cells and restore protein function. The company is currently
enrolling eligible adults with CF to participate in its Phase 1
clinical trial of PTI-428, a unique modulator called an 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 CF, the company 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 registration statement
on Form S-1 that we filed in connection with our initial public
offering, 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.
Availability of other information about Proteostasis
Therapeutics, Inc. Investors and others should note that
we communicate with our investors and the public using our company
website (www.proteostasis.com), including but not limited to
investor presentations and FAQs, Securities and Exchange
Commission filings, press releases, public conference calls
and webcasts. We encourage investors, the media, and others
interested in Proteostasis Therapeutics to review the information
that we post on our investor relations website on a regular basis.
The contents of our website, or any other website that may be
accessed from our website, shall not be deemed incorporated by
reference in any filing under the Securities Act of 1933 or
Securities Exchange Act of 1934.
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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