LIQ861, an inhaled dry power formulation of the prostacyclin treprostinil, was well tolerated in patients with pulmonary arterial hypertension (PAH) at a 2-week time point in the INSPIRE phase 3 clinical trial (Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil; ClinicalTrials.gov Identifier: NCT03399604), according to interim results.
Participants in the ongoing open-label multicenter clinical trial have stable PAH and have received nebulizer-delivered treprostinil for ≥3 months and are then transitioned to LIQ861 under protocol or have received ≤2 non-prostacyclin oral therapies for ≥3 months after which their treatment is supplemented with LIQ861 under protocol.
Those receiving supplemental therapy with LIQ861 receive an initial dose of approximately 25 mcg and those being transitioned from nebulizer therapy receive an initial dose of LIQ861 that is lower than their current treprostinil dose. In both sets of patients, LIQ861 is titrated up in 25-mcg incremental doses to symptom relief or to the limit of tolerance.
LIQ861 was administered in doses up to approximately 125 mcg without serious adverse events related to the drug and without dose-limiting toxicities.
The most common treatment-emergent adverse events were cough, headache, throat irritation, diarrhea, dizziness, oropharyngeal pain, and chest discomfort, but these were mostly mild and consistent with inhaled prostacyclin therapy.
Currently, a maximum tolerated dose of the study drug has not yet been reached, with some patients tolerating 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,” the study’s principal investigator, Nicholas Hill, MD, chief Pulmonary, Critical Care & Sleep Division and professor of medicine at Tufts University School of Medicine, stated in a press release.
“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,” Dr Hill added.
The 2-week time point safety evaluation was conducted to address the US Food and Drug Administration’s data request for inclusion in Liquidia’s New Drug Application submission. Liquidia Technologies expects to submit the full application for LIQ861 to the US Food and Drug Administration in late 2019.
Liquidia Technologies. Liquidia Technologies reports positive interim LIQ861 safety data on 109 patients from pivotal INSPIRE trial [press release]. Research Triangle Park, NC: Liquidia Technologies; January 7, 2019. Accessed January 9, 2019.