Palvella Therapeutics (PVLA) investor relations material

Platform and strategy

• Targets serious rare skin diseases and vascular anomalies spanning close to 700 diseases; over 98% lack an approved treatment, with strategy focused on first-in-disease therapies in lower-competition markets.

• QTORIN combines topical formulation expertise, manufacturing know-how and fit-for-purpose IP; the established platform is intended to advance new programs from concept to Phase II with less capital than de novo development.

• Development philosophy emphasizes resolving program risk with the smallest capital outlays.

Microcystic lymphatic malformation launch

• NDA received rolling review and six-month priority review; PDUFA date is February 28, 2027, with commercial and medical infrastructure being scaled ahead of a potential launch.

• Phase III showed 95% of patients improving on the primary endpoint; Phase II showed 100% improving on clinical global impression of change.

• Field teams have met over 200 of ~400 US vascular anomaly centers, which collectively represent ~15,000 patients; the goal is to reach all centers before the February 2027 FDA decision.

• Survey of ~150 US physicians found over 70% would prescribe within three months of approval and over three-quarters would prescribe for more than one patient.

• Estimated diagnosed population is >30,000; published estimates and claims analyses suggest a larger potential population, with claims data indicating 45,000–95,000 patients.

Market access and pricing

• Pricing range remains $100,000–$200,000 per patient per year; additional payer research is planned ahead of launch, with an update expected around February 2027.

• Payer research indicated no expected step-through requirement to interventional procedures or off-label pharmacologic therapies; coverage policies may take one to three quarters to establish after launch.