Palvella Therapeutics (PVLA) investor relations material

Program Expansion and Disease Background

• QTORIN rapamycin 3.9% anhydrous gel is expanding to target clinically significant angiokeratomas, a rare lymphatic skin disease with no FDA-approved therapies, affecting over 50,000 diagnosed patients in the U.S.

• Angiokeratomas have been reclassified as isolated lymphatic malformations by the ISSVA in 2025, sharing features and pathophysiology with microcystic lymphatic malformations.

• These lesions are chronic, debilitating, prone to bleeding, pain, and infection, and significantly impact quality of life.

• Current treatments are invasive, procedural, and often inadequate, with high recurrence and no approved pharmacotherapies.

• Scientific evidence supports targeting VEGF signaling and mTOR inhibition, with real-world and case study data suggesting rapamycin's therapeutic potential.

Market Opportunity and Physician Interest

• Over 50,000 U.S. patients are affected by angiokeratomas, with the addressable market for QTORIN rapamycin across all indications exceeding 150,000 patients and multibillion-dollar potential.

• Market research shows 96% of surveyed physicians would adopt a topical rapamycin gel, and 85% see a clear unmet need.

• Drug pricing is expected to align with other QTORIN indications due to disease severity and lack of alternatives.

Development Strategy and Clinical Milestones

• QTORIN rapamycin 3.9% gel will use the same anhydrous formulation, leveraging existing manufacturing, regulatory assets, and an open IND.

• A Phase 2 trial for angiokeratomas is planned for the second half of 2026, with an FDA meeting on study design in the first half of 2026; the trial will enroll 10–20 patients and mirror microcystic LM endpoints.

• The approach offers significant time and cost advantages over traditional de novo development, with expedited regulatory pathways and potential for supplemental NDA submission.

• Additional pipeline expansion and new program announcements are expected later in the year.