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 focus on targeted lung delivery, portability, tolerability, and titratability.

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

• L606, a next-generation liposomal sustained-release formulation, aims to improve dosing frequency to twice daily, enhancing patient convenience and compliance.

• L606, a liposomal treprostinil inhalation suspension, is advancing through clinical development with a pivotal Phase 3 trial planned across 20+ countries.

• 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.

• L606 open-label study (48 weeks) demonstrated high tolerability, minimal cough (14%), and no severe treatment-related adverse events.

• Both Yutrepia and L606 showed improvements in six-minute walk distance, with a significant proportion of patients surpassing the clinically relevant 30-meter threshold.

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

• ASCENT study showed Yutrepia enabled rapid titration to high doses with minimal discontinuations and mild, stable cough as the most common side effect.

R&D strategy and innovation priorities

• Focus on optimizing drug delivery to the lungs using proprietary PRINT and liposomal technologies to maximize efficacy and minimize side effects.

• Emphasis on developing therapies that provide sustained exposure, reduce dosing frequency, and improve patient adherence.

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