INSPIRE: Final Results from a Phase 3, Open-Label, Pivotal Study to Evaluate the Safety and Tolerability of LIQ861 in Pulmonary Arterial Hypertension

OF OBJECTIVES: Significant advantages are associated with inhaled therapies for pulmonary arterial hypertension (PAH), including enhanced delivery of drug to the lungs at a lower overall dose, improved ventilation-perfusion matching, and decreased risk of systemic vasodilation and other systemic adverse effects. LIQ861 is an investigational, novel, dry-powder formulation of treprostinil (TRE) designed using PRINT® technology to enhance deep-lung drug deposition and enable delivery of TRE doses in 1 to 2 breaths per capsule 4 times per day while using a convenient dry-powder inhaler. The primary objective of the INSPIRE study is to evaluate the safety and tolerability of LIQ861 in PAH patients transitioning to LIQ861 from a stable dose of Tyvaso® (Transition Group) and those adding LIQ861 to therapy with ≤2 non-prostacyclin (PGI) oral agents (Add-on Group). Final results from INSPIRE will be presented. METHODS: INSPIRE is a phase 3, open-label, multicenter study that enrolled WHO Group I PAH patients classified as New York Heart Association (NYHA) functional class (FC) II-IV. Patients in the Transition Group received an initial dose of LIQ861 that was believed to be comparable to their Tyvaso® dose, while those in the Add-on Group initiated LIQ861 at a dose of 25 mcg capsule strength QID. LIQ861 dose increases in both groups were titrated in 25 mcg capsule strength QID increments to tolerance and symptom relief. ENDPOINTS: The primary endpoint is the incidence of treatment-emergent adverse events and serious adverse events at Month 2. Exploratory endpoints are changes from baseline through Month 2 in physician assessment of stability; six-minute walk distance; NYHA FC, N-terminal B-type natriuretic peptide levels; and patient-reported quality of life. An additional endpoint for the Transition Group is the proportion of patients maintaining a sustained transition through Month 2, with sustained transition defined as continued LIQ861 therapy after discontinuation of Tyvaso®, no interruptions in LIQ861 therapy for ≥7 days; and no treatment with any other PGI analog or PGI receptor agonist.