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August 06, 2020
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Inhaled treprostinil improves QOL, other exploratory outcomes in patients with PAH

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New exploratory endpoint data from the INSPIRE study show improvements after 2 months of treatment with inhaled treprostinil in quality of life, 6-minute walk distance and New York Heart Association functional class.

The study evaluated LIQ861 (Liquidia Technologies), an investigational, inhaled dry powder formulation of treprostinil designed and engineered using Liquidia’s novel PRINT technology to enhance deep lung delivery of treprostinil in patients with pulmonary arterial hypertension.

Lungs and bronchi
Source: Shutterstock.

Healio previously reported data from the phase 3 INSPIRE trial that demonstrated LIQ861 was safe and well tolerated at 2 months, with no drug-related serious adverse events in patients with PAH. Final safety and tolerability results from the 2-month endpoint of the trial were presented recently at the International Society for Heart and Lung Transplantation Annual Meeting, demonstrating consistent findings from previous analyses, according to a company press release.

The new data were made available via a prerecorded poster session at the American Thoracic Society Virtual meeting and showed results from standard measures used to evaluate clinical symptoms and functional ability, which were exploratory endpoints and not subject to formal statistical analysis, according to the release.

The phase 3, open-label, multicenter INSPIRE study enrolled 121 patients with WHO Group 1 PAH (81.8% women; 79.3% white; mean age, 54 years; 66% New York Heart Association functional class 2; mean PAH duration, 5.9 years). The first patient group (n = 66) included stable patients with PAH on 2 or more non-prostacyclin add-on PAH therapies, who were assigned an initial dose of 25 g LIQ861 four times per day. The second patient group (n = 55) included patients who transitioned from treatment with treprostinil (Tyvaso, United Therapeutics Corp.) who received LIQ861 at an initial dose comparable to their Tyvaso dose at the time of transition. In both groups, titration to a higher dose of LIQ861 was permitted, based on symptom relief at the discretion of the treating physician.

At 2 months, results of the exploratory endpoints showed an improvement in 6-minute walk distance, with an overall median increase of 10.1 m. The majority of all patients in both groups (75.9%) maintained New York Heart Association functional status, and functional class improved in 20.5% from baseline to month 2. The Minnesota Living with Heart Failure Questionnaire showed a meaningful improvement in total quality of life score, defined as a decrease in score of 5 points or more, in addition to emotional and physical dimensions. In addition, more patients met two or three PAH low-risk criteria, based on European guideline-specified criteria, from baseline to month 2, with a greater change observed in the patients in the add-on group.

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The researchers observed no change in NT-proBNP from baseline to 2 months.

In other results, more than 70% of patients were able to titrate to a LIQ861 dose of 79.5 µg or greater. An additional exploratory endpoint showed that 85.5% of patients in the transition cohort preferred LIQ861 to their former treprostinil treatment.

“LIQ861, after 2 months, proved to be a convenient, safe, well tolerated option for inhaled prostacyclin therapy,” Nicholas S. Hill, MD, chief of the pulmonary, critical care and sleep division at Tufts Medical Center and professor of medicine at Tufts University School of Medicine, said during his prerecorded presentation.

Liquidia announced in April that the FDA accepted its New Drug Application for review of LIQ861 and was provided a Prescription Drug User Fee Act goal date of Nov. 24, 2020, according to the release.

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