WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) announced it has completed TOIVA, a Phase 2 proof-of-concept trial of QTORIN™ 3.9% rapamycin anhydrous gel, for the treatment of cutaneous venous malformations (cutaneous VMs). The trial enrolled 16 patients across leading vascular anomaly centers, meeting its recruitment target.
Cutaneous VMs are rare genetic disorders caused by mutations that overactivate the PI3K/mTOR signaling pathway, resulting in dysfunctional veins within the skin. The condition can lead to pain, disfigurement, and functional impairment, yet no FDA-approved therapies currently exist.
The TOIVA study evaluated once-daily, topical administration of QTORIN rapamycin. The trial is designed to assess safety and tolerability, alongside efficacy measures including clinician and patient global assessments and symptom-specific improvements over 12 weeks.
“Cutaneous venous malformations can severely impact quality of life, and patients currently have no approved treatment options,” said Dr. Megha Tollefson, pediatric dermatologist at Mayo Clinic and Principal Investigator of TOIVA. “These results will be important for understanding the potential role of QTORIN rapamycin in addressing this unmet need.”
Palvella CEO Wes Kaupinen emphasized the urgency of advancing a targeted, localized therapy. “The insights gained from this study will guide future trial design and help us move closer to delivering the first FDA-approved treatment for more than 75,000 U.S. patients living with cutaneous VMs,” he said.
Top-line results from the Phase 2 trial are expected in mid-December 2025.
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