Palvella Therapeutics Announces Expansion of QTORIN™ Rapamycin's Development into Clinically Significant Angiokeratomas, a Rare, Chronically Debilitating Lymphatic Disease with No FDA-approved Therapies

Core Insights - Palvella Therapeutics is expanding its QTORIN™ rapamycin development program to include clinically significant angiokeratomas, a rare skin disease with no FDA-approved therapies available [1][2] - The company plans to initiate a Phase 2 trial for QTORIN™ rapamycin in the second half of 2026, following a meeting with the FDA in the first half of 2026 [3] Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare skin diseases without FDA-approved treatments [5] - The company is led by veterans in rare disease drug development and is working on a pipeline of product candidates based on its patented QTORIN™ platform [5] Clinical Development Plans - QTORIN™ rapamycin is currently being evaluated in two other clinical trials: the Phase 3 SELVA study for microcystic lymphatic malformations and the Phase 2 TOIVA study for cutaneous venous malformations [3] - Top-line results for the SELVA study are expected in the first quarter of 2026, while results for the TOIVA study are anticipated in mid-December 2025 [3] Market Context - Clinically significant angiokeratomas are characterized by persistent bleeding and significant quality-of-life impacts, affecting an estimated 50,000 patients in the U.S. [1][2] - Current treatment options for angiokeratomas are limited and involve potentially destructive procedures with significant risks [2]