Liquidia Technologies Reports Positive Interim LIQ861 Safety Data on 109 Patients from Pivotal INSPIRE Trial
January 07 2019 - 7:00AM
Liquidia Technologies, Inc. (Nasdaq:LQDA) (“Liquidia”), a
late-stage clinical biopharmaceutical company focused on the
development and commercialization of human therapeutics using its
proprietary PRINT® technology to transform the lives of patients,
today reported positive interim safety data from its open-label,
multicenter Phase 3 clinical trial (INSPIRE) evaluating LIQ861, an
inhaled dry powder formulation of treprostinil, for the treatment
of pulmonary arterial hypertension (“PAH”). The safety data at the
two-week timepoint addresses the U.S. Food and Drug
Administration’s (“FDA”) data request for inclusion in a New Drug
Application (“NDA”) submission. Liquidia anticipates submitting the
full NDA for LIQ861 to the FDA in late 2019.
LIQ861 was observed to be well-tolerated at the
two-week timepoint in PAH patients. During this time period, LIQ861
was evaluated at doses up to approximately 125 mcg with no
study-drug related serious adverse events or dose-limiting
toxicities. Reported treatment-emergent adverse events (“TEAEs”)
were mostly mild in nature and consistent with inhaled prostacyclin
therapy. The most common TEAEs reported with LIQ861 in ≥4% of PAH
patients (n=109) were cough (25%), headache (13%), throat
irritation (12%), diarrhea (7%), dizziness (6%), oropharyngeal pain
(5%) and chest discomfort (5%). Patients have continued to receive
treatment beyond two-weeks with the first patient dosed in March
2018. To date, a maximum tolerated dose of LIQ861 has not yet been
reached, with patients having been administered doses up to
approximately 150 mcg.
“LIQ861 has the potential to maximize the
therapeutic benefit of inhaled treprostinil in treating PAH by
safely delivering higher doses into the lungs,” stated Nicholas
Hill, MD, Chief Pulmonary, Critical Care & Sleep Division and
Professor of Medicine at Tufts University School of Medicine and
INSPIRE Principal Investigator. “Enabled by Liquidia’s proprietary
PRINT technology, LIQ861 is designed to provide the benefits of
delivering prostacyclin analogs locally to the lungs via
inhalation, potentially offering a targeted and effective approach
with an acceptable systemic side effect profile.”
The INSPIRE clinical trial is designed to
evaluate patients who have either been under stable treatment with
nebulizer-delivered treprostinil for at least three months and are
transitioned to LIQ861 under the protocol or patients who have been
on stable treatment with no more than two non-prostacyclin oral PAH
therapies for at least three months and have their treatment
regimen supplemented with LIQ861 under the protocol. Patients
adding LIQ861 to current non-prostacyclin oral therapies started at
a dose of approximately 25 mcg and those transitioned from
nebulizer-delivered treprostinil at a stable dose were initiated at
a dose of LIQ861 lower than their current stable treprostinil dose.
In both cases, LIQ861 was uptitrated in 25 mcg incremental doses to
symptom relief or the limit of tolerance.
“Patient demographics and baseline
characteristics in the trial suggest that LIQ861 may be attractive
across disease severity and may have utility as a first-line
prostacyclin,” added Robert Roscigno, PhD, Liquidia’s Senior Vice
President of Product Development and LIQ861 Program Lead.
“Interestingly, enrollment of the safety portion of the trial was
driven primarily by stronger than anticipated interest from New
York Heart Association Functional Class II add-on patients, which
may imply that dry-powder delivery could be an alternative to oral
delivery in prostacyclin naïve patients. We are pleased with these
findings and believe they support the therapeutic potential and
versatility of LIQ861 among patients across different functional
classes.”
Liquidia continues to enroll patients in the
INSPIRE clinical trial in support of the one-directional crossover
pharmacokinetic (“PK”) sub-study. The sub-study is designed to
compare bioavailability and PK of treprostinil as patients are
transitioned from nebulizer-delivered treprostinil to LIQ861. PK
results are expected to be reported in the second quarter of 2019.
To further support Liquidia’s future marketing and commercial
activities with additional medical information, Liquidia expects to
continue to treat patients and collect data until the launch of
LIQ861 in the United States, if approved.
About LIQ861
LIQ861 is an inhaled dry powder formulation of
treprostinil designed using Liquidia’s PRINT technology to enhance
deep-lung delivery using a convenient, palm-sized, disposable dry
powder inhaler for the treatment of PAH. Liquidia believes LIQ861
can overcome the limitations of current inhaled therapies and has
the potential to maximize the therapeutic benefits of treprostinil
in treating PAH by safely delivering higher doses into the
lungs.
About INSPIRE Clinical
Trial
Liquidia’s pivotal open-label Phase 3 clinical
trial, known as INSPIRE, or Investigation of the Safety and
Pharmacology of Dry Powder Inhalation of Treprostinil, is
designed to evaluate patients who have either been under stable
treatment with nebulizer-delivered treprostinil for at least three
months and are transitioned to LIQ861 under the protocol or
patients who have been on stable treatment with no more than two
non-prostacyclin oral PAH therapies for at least three months and
have their treatment regimen supplemented with LIQ861 under the
protocol. The primary objective of the INSPIRE study is to evaluate
the long-term safety and tolerability of LIQ861. For more
information, please visit
https://clinicaltrials.gov/ct2/show/NCT03399604.
About Liquidia Technologies
Liquidia Technologies is a late-stage clinical
biopharmaceutical company focused on the development and
commercialization of human therapeutics using its proprietary
PRINT® technology to transform the lives of patients. Currently,
Liquidia is focused on the development of two product candidates
using its PRINT® particle engineering platform: LIQ861 for the
treatment of pulmonary arterial hypertension and LIQ865 for the
treatment of local post-operative pain. Being evaluated in a Phase
3 clinical trial (INSPIRE), LIQ861 is designed to improve the
therapeutic profile of treprostinil by enhancing deep-lung delivery
and achieving higher dose levels than current inhaled therapies by
using a convenient, palm-sized, disposable DPI. LIQ865, for which
Liquidia has completed two Phase 1 clinical trials, is designed to
deliver sustained-release particles of bupivacaine, a non-opioid
anesthetic, to treat local post-operative pain for three to five
days through a single administration. For more information visit
our website at www.liquidia.com.
Forward-Looking Statements
This press release may include forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. All statements contained in this press release
other than statements of historical facts, including statements
regarding our future results of operations and financial position,
our business strategy and plans and our objectives for future
operations, are forward-looking statements. Such forward-looking
statements, including statements regarding clinical trials,
clinical studies and other clinical work (including the funding
therefor, anticipated patient enrollment, safety data, study data,
trial outcomes, timing or associated costs), regulatory
applications and related timelines, including the filing of an NDA
for LIQ861, involve significant risks and uncertainties and actual
results could differ materially from those expressed or implied
herein. The words “anticipate,” “believe,” “continue,” “estimate,”
“expect,” “intend,” “may,” “will” and similar expressions are
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forward-looking statements are subject to a number of risks
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Moreover, we operate in a very competitive and rapidly changing
environment and our industry has inherent risks. New risks emerge
from time to time. It is not possible for our management to predict
all risks, nor can we assess the impact of all factors on our
business or the extent to which any factor, or combination of
factors, may cause actual results to differ materially from those
contained in any forward-looking statements we may make. In light
of these risks, uncertainties and assumptions, the future events
discussed in this press release may not occur and actual results
could differ materially and adversely from those anticipated or
implied in the forward-looking statements. Nothing in this press
release should be regarded as a representation by any person that
these goals will be achieved and we undertake no duty to update our
goals or to update or alter any forward-looking statements, whether
as a result of new information, future events or otherwise.
Contact:Jennifer
AlmondDirector, Investor Relations & Corporate Communications
919.328.4389IR@liquidia.com
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