WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) has completed TOIVA, its Phase 2 clinical trial evaluating QTORIN™ 3.9% rapamycin anhydrous gel as a potential treatment for cutaneous venous malformations (VMs), a rare genetic skin disorder with no FDA-approved therapies.
The trial enrolled 16 patients at leading vascular anomaly centers, meeting its target recruitment goal. Conducted as a single-arm, open-label study, TOIVA is assessing the safety, tolerability, and early efficacy of QTORIN rapamycin, which is applied topically once a day. Unlike systemic rapamycin treatments, which can cause adverse side effects, QTORIN is designed to deliver localized therapy while limiting systemic exposure.
Cutaneous VMs are caused by gene mutations that overactivate the PI3K/mTOR signaling pathway, leading to malformed veins in the skin. The condition can cause chronic pain, disfigurement, bleeding, and functional impairment. Estimates suggest more than 75,000 people in the U.S. live with cutaneous VMs.
“Cutaneous venous malformations can have a severe impact on quality of life, leading to substantial morbidity and functional impairment of the skin, and yet there are currently no FDA-approved therapies to address this high unmet need,” said Dr. Megha Tollefson, pediatric dermatologist at the Mayo Clinic and principal investigator of the study.
Palvella CEO Wes Kaupinen said the Phase 2 proof-of-concept data will play a key role in designing future clinical trials. “An urgent need exists for an FDA-approved, targeted, localized therapy to treat cutaneous VMs,” he said. “The insights gained from this study will be critical in advancing Palvella’s mission to bring the first FDA-approved therapy to patients.”
Top-line results from the trial are expected in mid-December 2025.
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