Full Press Release Details
Provides Update on Clinical Pipeline Targeting PAH and PH-ILD
| - | Enrolled first PH-ILD patient in the open-label ASCENT study of YUTREPIA | |
| - | Confirmed with FDA that a single pivotal efficacy trial with L606 will support PAH and PH-ILD indications |
MORRISVILLE, N.C., January 5, 2024 (GLOBE NEWSWIRE) - Liquidia Corporation (NASDAQ: LQDA) (Liquidia or the Company) provided an update today on the clinical progress of its
pipeline programs to treat pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung
disease (PH-ILD) with its investigational drugs, YUTREPIA (treprostinil) inhalation powder and L606, a sustained-release
inhaled formulation of treprostinil administered twice-daily with a short-duration next-generation nebulizer.
With respect to YUTREPIA, the first PH-ILD patient was enrolled in
December 2023 in the Open-Label Prospective Multicenter Study to Evaluate Safety and Tolerability of Dry Powder Inhaled Treprostinil
in Pulmonary Hypertension, referred to as the ASCENT study. The ASCENT study will enroll approximately 60 subjects to further inform
YUTREPIA's dosing and tolerability profile in patients with PH-ILD. Exploratory efficacy endpoints will also be assessed. YUTREPIA
is currently being reviewed for tentative approval for PH-ILD by the U.S. Food and Drug Administration (FDA) with a Prescription Drug
User Fee Act (PDUFA) goal date of January 24, 2024. The FDA has previously confirmed that Liquidia may add the treatment of PH-ILD
to the label for YUTREPIA without additional clinical studies.
With respect to L606, Liquidia successfully concluded a Type C meeting
with the FDA in December 2023 and reached agreement regarding the registration requirements for L606 using the 505(b)(2) regulatory
pathway. It was agreed that only a single Phase 3 placebo-controlled efficacy trial in PH-ILD patients would be required to support indications
to treat both PAH and PH-ILD. The Company is preparing to initiate a global study later in 2024.
Liquidia will also continue to enroll patients in its ongoing open-label,
multicenter safety study of L606 in U.S. patients with PAH or PH-ILD. This study will enroll approximately 60 subjects and include patients
with PAH, who are na ve to prostacyclins or transitioning from Tyvaso or Tyvaso DPI , and patients with PH-ILD,
who are transitioning from Tyvaso or Tyvaso DPI . The study is now more than one third enrolled and includes some patients who
have been successfully treated with L606 for longer than one year and at doses comparable to 25 to 27 breaths of Tyvaso, four times daily.
The Company currently anticipates the open-label study will be fully enrolled in 2024 and intends to provide interim updates at medical
conferences later in the year.
Rajeev Saggar, M.D., Chief Medical Officer of Liquidia, stated: "YUTREPIA
will provide practitioners and patients the opportunity to comfortably use a low-resistance dry-powder inhaler across a wide range of
doses to a broad range of patients with PAH and PH-ILD with varying lung function and clinical severity. We are also pleased by the increasing
interest to enroll patients in the L606 open-label study. The real-time data on patient tolerability, dosing titration, and clinical
response will greatly inform the pivotal efficacy trial and should provide additional confidence in the benefits of more consistent,
sustained exposures over a 24-hour period."
More information about the clinical studies described
is available at ClinicalTrials.gov and associated with the trial identifier NCT06129240 (ASCENT study) and NCT04691154
About YUTREPIA (treprostinil) inhalation
YUTREPIA is an investigational, inhaled dry
powder formulation of treprostinil delivered through a convenient, low-resistance, palm-sized device. On November 5, 2021, the FDA
issued a tentative approval for YUTREPIA for the treatment of PAH to improve exercise ability in adult patients with New York Heart Association
(NYHA) Functional Class II-III symptoms. In July 2023, Liquidia filed an amendment to its New Drug Application for YUTREPIA,
seeking to add PH-ILD to the label. The FDA has set a PDUFA goal date of January 24, 2024 for the amendment. Previously, the FDA
has confirmed that YUTREPIA may add the treatment of PH-ILD to the label for YUTREPIA without additional clinical studies. YUTREPIA was
designed using Liquidia's PRINT technology, which enables the development of drug particles that are precise and uniform in
size, shape, and composition, and that are engineered for enhanced deposition in the lung following oral inhalation. Liquidia has completed
INSPIRE, or Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil, an open-label, multi-center phase
3 clinical study of YUTREPIA in patients diagnosed with PAH who are na ve to inhaled treprostinil or who are transitioning from
Tyvaso (nebulized treprostinil). YUTREPIA was previously referred to as LIQ861 in investigational studies.
About L606 (liposomal treprostinil) inhalation
L606 is an investigational, sustained-release
formulation of treprostinil administered twice-daily with a short-duration next-generation nebulizer. The L606 suspension uses Pharmosa
Biopharm's proprietary liposomal formulation to encapsulate treprostinil which can be released slowly at a controlled rate into
the lung, enhancing drug exposure over an extended period of time and reducing local irritation of the upper respiratory tract. L606
is currently being evaluated in an open-label study in the United States for treatment of PAH and PH-ILD with a planned pivotal study
for the treatment of PH-ILD.
About pulmonary arterial hypertension
PAH is a rare, chronic, progressive disease
caused by hardening and narrowing of the pulmonary arteries that can lead to right heart failure and eventually death. Currently, an
estimated 45,000 patients are diagnosed and treated in the United States. There is currently no cure for PAH, so the goals of existing
treatments are to alleviate symptoms, maintain or improve functional class, delay disease progression, and improve quality of life.
About pulmonary hypertension associated
with interstitial lung disease (PH-ILD)
Pulmonary hypertension (PH) associated with
interstitial lung disease (ILD) includes a diverse collection of up to 150 different pulmonary diseases, including interstitial pulmonary
fibrosis, chronic hypersensitivity pneumonitis, connective tissue disease related ILD, and chronic pulmonary fibrosis with emphysema
(CPFE) among others. Any level of PH in ILD patients is associated with poor 3-year survival. A current estimate of PH-ILD prevalence
in the United States is greater than 60,000 patients, though population growth in many of these underlying ILD diseases is not yet known
due to factors including underdiagnosis and lack of approved treatments until March 2021, when inhaled treprostinil was first approved
for this indication.
About Liquidia Corporation
Liquidia Corporation is a biopharmaceutical
company focused on the development and commercialization of products in pulmonary hypertension and other applications of its PRINT
Technology. The Company operates through its two wholly owned subsidiaries, Liquidia Technologies, Inc. (Liquidia Technologies)
and Liquidia PAH, LLC (Liquidia PAH). Liquidia Technologies has developed YUTREPIA (treprostinil) inhalation powder for the treatment
of PAH and PH-ILD. Liquidia Technologies is also developing L606, an investigational liposomal formulation of treprostinil administered
twice-daily with a short-duration next-generation nebulizer, for use in North America. Liquidia PAH provides for the commercialization
of pharmaceutical products to treat pulmonary disease, such as generic Treprostinil Injection. For more information, please visit www.liquidia.com.
Tyvaso and Tyvaso DPI are registered trademarks of United
Therapeutics Corporation.
Cautionary Statements Regarding Forward-Looking
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
strategic and financial initiatives, 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 submission contents and timelines, including the potential for final FDA approval of the NDA for YUTREPIA, the
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