WAYNE, PA — Palvella Therapeutics (Nasdaq: PVLA) recently dosed the first patients in a Phase 2 study evaluating QTORIN rapamycin gel for angiokeratomas, a rare vascular-related skin disorder with no FDA-approved treatments, as the biotechnology company expands development of its topical mTOR inhibitor platform into additional rare disease indications.
The LOTU trial will evaluate the safety and efficacy of the once-daily topical therapy in patients with clinically significant angiokeratomas, which can cause bleeding, infection risk, pain, and functional impairment.
Palvella expects topline data from the study in the second half of 2027.
The single-arm, baseline-controlled trial is expected to enroll up to 15 patients age 6 and older at vascular anomaly and dermatology centers across the United States.
Angiokeratomas were classified in 2025 by the International Society for the Study of Vascular Anomalies as isolated lymphatic malformations, aligning the condition with diseases involving dysregulated lymphatic signaling pathways that Palvella is targeting through mTOR inhibition.
The company believes the classification strengthens the rationale for expanding QTORIN rapamycin beyond its lead indication in microcystic lymphatic malformations.
“Current management relies on invasive, often destructive procedures, including laser therapy and electrocautery, which can be associated with pain, scarring, recurrence, and incomplete disease control,” Maria Buethe, division chief of dermatology at Rady Children’s Hospital of Orange County and principal investigator for the study, said in a statement.
Palvella estimates more than 50,000 patients in the United States have been diagnosed with clinically significant angiokeratomas.
QTORIN rapamycin previously received Fast Track designation from the U.S. Food and Drug Administration for the indication.
The therapy is designed to deliver rapamycin directly to affected skin tissue while limiting systemic drug exposure associated with oral mTOR inhibitors.
“We continue to see broad potential for QTORIN rapamycin across serious, rare diseases characterized by dysregulated mTOR signaling,” Chief Executive Officer Wes Kaupinen said in a statement.
The study will measure changes in clinician and patient assessments of disease severity and symptom burden over a 12-week treatment period.
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