Liquidia Releases Exploratory Endpoint Data from INSPIRE Study at the American Thoracic Society (ATS) Annual Meeting - Seite 2

INSPIRE, a phase 3, open-label, multicenter study of two World Health Organization (WHO) Group 1 pulmonary arterial hypertension (PAH) patient cohorts, enrolled a total of 121 patients. The first patient group included stable PAH patients on ≤2 non-prostacyclin PAH therapies (add-on) and the second group included subjects who transitioned from treatment with Tyvaso (treprostinil) (transition).

The initial dose for transition patients (n=55) was based on their Tyvaso dose at the time of transition and the starting dose for add-on patients (n=66) was 26.5 mcg QID. Titration to a higher LIQ861 dose was permitted in both groups based on symptom relief at the discretion of the physicians.

Results of the exploratory endpoints evaluated in the INSPIRE study showed:

• More than 70 percent of patients were able to titrate to a LIQ861 dose ≥79.5 mcg.
• NYHA functional class improved in 20.5 percent of patients and maintained in 75.9 percent.
• Overall median 6MWD increased by 10.1 m.
  
• There was no clinically meaningful change in NT-proBNP.
  
• MLHFQ showed an improved total score (>5-point reduction), as well as in both emotional and physical dimensions.
  
• A larger percentage of patients met two-or-three PAH low-risk criteria at month 2 compared with baseline.
  
• The majority of transition patients preferred the LIQ861 dry-powder inhaler to the Tyvaso Inhalation System.

“The PAH community has substantial unmet needs, like so many others with rare disease,” said Tushar Shah, M.D., Chief Medical Officer of Liquidia. “By leveraging our propriety PRINT technology, we were able to develop a convenient, dry powder inhaler that delivers the required dose of highly uniform treprostinil particles in one-to-two breaths. If approved, we believe that the LIQ861 dry powder inhaler could offer PAH patients a much-needed alternative treatment option and we are deeply committed to delivering such options to the PAH community.”

The INSPIRE study safety and tolerability outcomes at month 2 were recently presented at the International Society for Heart & Lung Transplantation (ISHLT) Annual Meeting. Furthermore, Liquidia submitted the New Drug Application (NDA) for LIQ861 to the U.S. Food and Drug Administration (FDA) under the 505(b)(2) regulatory pathway, which includes data from three clinical studies establishing the safety, tolerability and pharmacokinetic profile of LIQ861. On April 8, 2020, Liquidia announced the FDA had accepted the NDA for review and that it had been provided a Prescription Drug User Fee Act (PDUFA) goal date of November 24, 2020.