Palvella Therapeutics (PVLA) Canaccord Genuity’s 45th Annual Growth Conference summary

Mission, strategy, and vision

• Focused on serving patients with serious, rare genetic diseases by developing and commercializing first-approved therapies.

• Targets rare skin diseases with high unmet need and low competition, aiming to become a leader in this area.

• Only pursues diseases with no FDA-approved therapies, seeking transformative impact for patients and shareholders.

Lead indication: microcystic lymphatic malformations (MLM)

• MLM is a lifelong, serious genetic disease caused by PI3K pathway mutations, leading to skin lesions and risk of infection.

• No approved therapies exist; current treatments include surgery, sclerotherapy, and off-label rapamycin, all with limited efficacy.

• QTORIN™ rapamycin, a topical formulation, is in phase 3 trials aiming to be the first FDA-approved therapy for MLM.

Clinical development and data

• Phase 2 study showed all patients were much or very much improved at 12 weeks, leading to FDA breakthrough therapy designation.

• Phase 3 study uses an enhanced clinician global impression of change endpoint, with 51 patients enrolled and 24-week duration.

• Study design is single-arm due to lack of spontaneous disease regression and strong FDA alignment on endpoints.