DALLAS — A dry powder formulation of treprostinil was safe and well tolerated at 2 months, with no drug-related serious adverse events, in patients with pulmonary arterial hypertension, according to new data from the phase 3 INSPIRE trial presented at the American Thoracic Society International Conference.
Treprostinil (Tyvaso, United Therapeutics Corp.) is a synthetic prostacyclin analogue that is approved for inhalation administration in patients with PAH via nebulized Tyvaso Inhalation Solution via nine breaths four times per day. The dry powder formulation of treprostinil (LIQ861, Liquidia Technologies) utilizes the company’s PRINT technology, which is designed to enhance deep-lung delivery and enables four-times-daily delivery of treprostinil doses in one to two breaths per capsule via a palm-sized dry powder inhaler, according to Liquidia Technologies.
The phase 3, open-label, multicenter INSPIRE study was designed to evaluate safety and tolerability of LIQ861 in cohorts of patients with PAH transitioned from nebulizer-delivered treprostinil (n = 44) or added on to prior background therapy consisting of no more than two non-prostacyclin oral PAH therapies (n = 65). Patients enrolled were mostly women and the mean age at screening was 55 years.
The new data were presented as a poster at the ATS International Conference by Nicholas Hill, MD, chief of the pulmonary, critical care and sleep division and professor of medicine at Tufts University School of Medicine.
No serious adverse events related to LIQ861 or dose-limiting toxicities occurred during 2 months. Treatment-emergent adverse events that occurred during the study period were mostly mild, according to the researchers. The most common treatment-emergent adverse event was cough in 33% of patients. Among six patients who have been treated for more than 12 months, no study drug-related serious adverse events have emerged during longer follow-up, according to the abstract.
Ninety-three percent of all patients completed 2 months of treatment — the primary endpoint of the study — and remained in the study beyond month 2. By cohort, the percentage of sustained treatment was 95.5% in the LIQ861 transition cohort and 90.8% in the add-on therapy cohort.
Additionally, the researchers evaluated activity benefits and quality of life by cohort based on baseline NYHA functional class. At screening, 66% of patients had NYHA class II PAH. In the transition cohort, patients with NYHA class II PAH maintained activity benefits based on 6-minute walk distance while improving quality of life based on Minnesota Living With Heart Failure Questionnaire score, and patients with class III PAH improved activity benefit while sustaining quality of life. In the add-on cohort, patients with class II PAH improved activity benefits while improving quality of life, and patients with class III PAH maintained activity benefits and quality of life, according to the findings.
“LIQ861 may provide functional and QOL benefits to functional class II and III PAH patients,” the researchers concluded in the abstract.
Hill NS, et al. Poster P1155. Presented at: American Thoracic Society International Conference; May 17-22, 2019; Dallas.
Disclosure: The study was funded by Liquidia Technologies. Hill reports he is a consultant for Liquidia Technologies and receives grant and/or research support to his institution from Actelion, Bayer, Gilead, Liquidia Technologies, Reata and United Therapeutics; and is scientific medical advisor to Liquidia Technologies.