WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) presented new Phase 2 data showing statistically significant reductions in lesion bleeding among patients treated with QTORIN rapamycin for cutaneous venous malformations, as the company advances a therapy that currently faces no FDA-approved competition in the rare disease indication.
The data, presented at the Society for Investigative Dermatology annual meeting in Chicago, came from the company’s TOIVA trial evaluating once-daily topical QTORIN rapamycin in patients with cutaneous venous malformations, or VMs.
Patients with bleeding at baseline demonstrated a mean 2.5-point improvement on the Cutaneous Venous Malformations Investigator Global Assessment Bleeding scale at Week 12, achieving statistical significance with a p-value of 0.003, according to the company.
All four patients in the bleeding subgroup were rated either “Much Improved” or “Very Much Improved” at Week 12 on the clinician-assessed bleeding scale.
In addition, 100% of patients with bleeding at baseline reported being either “satisfied” or “very satisfied” with treatment on the Treatment Satisfaction Questionnaire for Medication.
“Cutaneous venous malformations affect many aspects of a patient’s life, resulting in pain, functional limitations, and mental health issues,” Dr. Michael Kelly of the Cleveland Clinic’s Vascular Anomalies Program stated.
Kelly added that the study demonstrated “a strong correlation between improvements in clinical measures and patient-reported outcomes.”
The TOIVA study is a Phase 2 open-label, baseline-controlled trial evaluating QTORIN rapamycin over a 12-week treatment period followed by a 12-week extension phase.
Palvella previously reported that the study met multiple clinician-reported and patient-reported endpoints in results disclosed in December 2025.
New qualitative patient interview data presented alongside the clinical findings highlighted the broader physical and psychosocial burden associated with cutaneous venous malformations.
Patients described pain, swelling, bleeding, visible skin discoloration and protruding lesions that affected physical activity, work, school participation and social interactions.
The company indicated patient priorities included reducing lesion size, improving appearance and relieving pain — outcomes that may not be fully captured through conventional clinical severity measurements.
Palvella is developing QTORIN rapamycin as a potential treatment for an estimated 75,000 patients in the United States living with cutaneous venous malformations. The condition currently has no FDA-approved therapies.
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