Proteostasis Therapeutics Files Investigational New Drug Application for PTI-808, Cystic Fibrosis Potentiator
June 23 2017 - 7:00AM
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 the Company has filed an Investigational
New Drug (IND) application with the U.S. Food and Drug
Administration (FDA) for PTI-808, its cystic fibrosis transmembrane
conductance regulator (CFTR) potentiator. Proteostasis plans to
initiate a Phase 1 study of PTI-808 in healthy volunteers in July.
“While potentiators are an established part of CF therapy, there
remains significant room for improvement in this critical component
of therapy, and we believe PTI-808, with potential once a day
dosing regimen, offers the potential to fill this gap,”
said Meenu Chhabra, president and chief executive officer
of Proteostasis Therapeutics. “Each component of our potential
triple combination therapy is now clinical stage, with active and
ongoing clinical development programs for both PTI-428, an
amplifier, and PTI-801, a new generation corrector. With the
filing of an IND for the potentiator PTI-808, the third and final
piece of our triple combination, we have reached an important
milestone in assembling PTI-NC-733. Pending positive results
from ongoing clinical trials with PTI-428 and PTI-801, we expect to
initiate a trial combining all three proprietary CFTR modulators
before the end of 2017.”
By the end of June, Proteostasis expects to report preliminary
data from multiple ascending dose (MAD) cohorts of two different CF
patient populations: CF subjects receiving PTI-428 or placebo in
addition to Orkambi® as their background therapy for seven days and
CF subjects receiving PTI-428 as their sole CFTR modulator therapy
or placebo for seven
days. 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.
Proteostasis is currently conducting a 14-day
MAD Phase 1 study of PTI-801 in healthy volunteers in the U.S., to
be followed by dosing in CF patients. If positive results are
achieved in the Company’s PTI-428 and PTI-801 programs,
Proteostasis 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 in a
triple combination also known as PTI-NC-733.
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.
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 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 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
Proteostasis Therapeutics (NASDAQ:PTI)
Historical Stock Chart
From Mar 2024 to Apr 2024
Proteostasis Therapeutics (NASDAQ:PTI)
Historical Stock Chart
From Apr 2023 to Apr 2024