Palvella Therapeutics (PVLA) H.C. Wainwright 28th Annual Global Investment Conference summary

Strategic vision and mission

• Focused on developing first-in-disease therapies for rare skin diseases and vascular anomalies with no approved treatments.

• Strategy leverages clear disease biology, existing human proof-of-concept data, and the proprietary QTORIN platform.

• Targeting large orphan markets with high unmet need and low competitive intensity, covering four diseases with multibillion-dollar addressable markets.

• Committed to building an enduring leadership position in rare disease therapeutics.

Pipeline progress and clinical milestones

• NDA for QTORIN rapamycin in microcystic lymphatic malformations submitted, with FDA rolling review underway.

• Phase III SELVA study met all primary and key secondary endpoints with strong efficacy and safety data; 95% of patients improved, 86% rated much or very much improved.

• Additional clinical programs: phase III for cutaneous venous malformations to start this year, phase II for angiokeratomas ongoing, and phase II for DSAP planned for Q4.

• Two new QTORIN program announcements expected by year-end.

Platform innovation and intellectual property

• QTORIN platform enables high drug loading, targeted delivery, and low systemic absorption for chronic use.

• QTORIN rapamycin granted FDA Breakthrough Therapy, Fast Track, and Orphan Designations.

• Six issued U.S. patents, broad claims, and additional filings could extend exclusivity into the mid-2040s.

• Innovations protected by patents, trade secrets, and regulatory exclusivities.