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 Viewpoint - Palvella Therapeutics is expanding its QTORIN™ rapamycin development program to include clinically significant angiokeratomas, a rare skin disease with no FDA-approved therapies available for over 50,000 diagnosed patients in the U.S. [1][2] Company Overview - Palvella Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapies for serious, rare skin diseases without FDA-approved treatments [5] - The company is led by veterans in rare disease drug development and is developing a pipeline based on its patented QTORIN™ platform [5] Product Development - The company plans to initiate a Phase 2 trial for QTORIN™ rapamycin targeting clinically significant angiokeratomas in the second half of 2026, following a meeting with the FDA in the first half of 2026 [3] - QTORIN™ rapamycin is also being evaluated in ongoing clinical trials for microcystic lymphatic malformations (Phase 3 SELVA study) and cutaneous venous malformations (Phase 2 TOIVA study) [3] Disease Context - Clinically significant angiokeratomas are characterized by lymphatic-derived skin lesions that can cause bleeding, pain, and functional impairment, with no tendency for spontaneous regression [2] - Current treatment options are limited and involve potentially destructive procedures that carry significant risks [2]