LIQ861 was well-tolerated in PAH patients at two-weeks of treatment, the safety endpoint requested by U.S. FDA
NDA submission targeted for late 2019
RESEARCH TRIANGLE PARK, N.C., Jan. 07, 2019 (GLOBE NEWSWIRE) — 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.
