Liquidia (LQDA) R&D Day 2025 summary

Pipeline overview and program updates

• Yutrepia, an inhaled treprostinil, is positioned as a best-in-class therapy for PAH and PH-ILD, with strong real-world uptake and positive tolerability feedback after five months on the market.

• Yutrepia, a dry-powder treprostinil, received FDA approval in May 2025 for PAH and PH-ILD.

• L606, a next-generation liposomal sustained-release inhaled treprostinil, aims to improve dosing convenience and tolerability, targeting twice-daily administration.

• The RESPIRE global phase III trial for L606 in PH-ILD is set to enroll 344 patients across 120+ sites in 20+ countries, with robust design and stratification.

• Focus on optimizing inhaled drug delivery for respiratory and vascular diseases, with Yutrepia and L606 as lead programs.

Clinical trial data and development milestones

• ASCENT study in PH-ILD showed most patients titrated to higher doses with improved 6MWD (+41m at 24 weeks) and stable tolerability.

• ASCENT study showed Yutrepia enables rapid titration to high doses with minimal discontinuations and mild, stable cough rates.

• L606 open-label study (48 weeks) demonstrated high tolerability, low cough incidence, and maintenance or improvement in six-minute walk distance, even in heavily pretreated patients.

• Phase I data for L606 revealed a seven-fold lower Cmax than Tyvaso, supporting improved systemic tolerability.

• Phase 1 data for L606 showed similar bioavailability to Tyvaso with lower peak plasma levels and reduced cough.

R&D strategy and innovation priorities

• Focus on optimizing drug delivery to the lungs, reducing systemic side effects, and enhancing patient compliance through device innovation.

• Emphasis on proprietary PRINT and liposomal technologies to enhance lung targeting, dose flexibility, and patient compliance.

• Innovation in device design (e.g., FOX vibrating mesh nebulizer) to improve portability and ease of use.

• Emphasis on achieving higher, sustained drug exposures to replicate the benefits of continuous infusion prostacyclins in a non-parenteral format.