Liquidia Corporation provided an update on the clinical progress of its pipeline programs to treat pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) with its investigational drugs, YUTREPIA (treprostinil) inhalation powder and L606, a sustained-release inhaled formulation of treprostinil administered twice-daily with a short-duration next-generation nebulizer. With respect to YUTREPIA, the first PH-ILD patient was enrolled in December 2023 in the Open-Label Prospective Multicenter Study to Evaluate Safety and Tolerability of Dry Powder Inhaled Treprostinil in Pulmonary Hypertension, referred to as the ASCENT study. The ASCENT study will enroll approximately 60 subjects to further inform YUTREPIA?s dosing and tolerability profile in patients with PH-ILD.

Exploratory efficacy endpoints will also be assessed. YUTREPIA is currently being reviewed for tentative approval for PH-ILD by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) goal date of January 24, 2024. The FDA has previously confirmed that Liquidia may add the treatment of PH-ILD to the label for YUTREPIA without additional clinical studies.

With respect to L606, Liquidia successfully concluded a Type C meeting with the FDA in December 2023 and reached agreement regarding the registration requirements for L606 using the 505(b)(2) regulatory pathway. It was agreed that only a single Phase 3 placebo-controlled efficacy trial in PH-ILD patients would be required to support indications to treat both PAH and PH-ILD. The Company is preparing to initiate a global study later in 2024.

Liquidia will also continue to enroll patients in its ongoing open-label, multicenter safety study of L606 in U.S. patients with PAH or PH-ILD. This study will enroll approximately 60 subjects and include patients with PAH, who are naïve to prostacyclins or transitioning from Tyvaso or Tyvaso DPI, and patients with PH-ILD, who are transitioning from Tyvaso or Tyvaso DPI. The study is now more than one third enrolled and includes some patients who have been successfully treated with L606 for longer than one year and at doses comparable to 25 to 27 breaths of Tyvaso, four times daily.

The Company currently anticipates the open-label study will be fully enrolled in 2024 and intends to provide interim updates at medical conferences later in the year.