Palvella Therapeutics Inc. (PVLA) announced that the first patients have been dosed in its Phase 2 LOTU clinical trial evaluating QTORIN rapamycin gel for treating clinically significant angiokeratomas, a rare lymphatic malformation affecting an estimated 50,000 patients in the United States.
The single-arm, baseline-controlled trial will enroll up to 15 subjects aged six and older at vascular anomaly centers and dermatology centers across the U.S. The study evaluates the safety and efficacy of QTORIN 3.9% rapamycin anhydrous gel administered topically once daily for 12 weeks.
Angiokeratomas are characterized by hyperkeratotic vascular lesions that can bleed with minor trauma and are susceptible to infection. Current treatment relies on invasive procedures including laser therapy and electrocautery, which can result in pain, scarring, and recurrence. No FDA-approved therapies currently exist for the condition.
The International Society for the Study of Vascular Anomalies classified angiokeratomas as isolated lymphatic malformations in 2025, placing them in the same category as microcystic lymphatic malformations, which represents Palvella’s lead indication for QTORIN rapamycin.
QTORIN rapamycin received Fast Track Designation from the FDA for treating angiokeratomas. The topical gel is designed to deliver rapamycin, an mTOR inhibitor, to affected skin tissue while minimizing systemic exposure.
The trial will assess safety based on adverse events and evaluate efficacy through clinician and patient global impression assessments, measuring changes from baseline to Week 12. Topline results are expected in the second half of 2027.
Wayne, Pennsylvania-based Palvella focuses on developing therapies for rare skin diseases and vascular malformations. The company’s pipeline is based on its QTORIN platform technology.