Palvella Therapeutics presented new Phase 2 TOIVA trial analyses showing that topical QTORIN™ rapamycin significantly reduced bleeding and improved lesion-related outcomes in patients with cutaneous venous malformations.
Written By: Fariha Sameen, PharmD
Reviewed By: Pharmacally Editorial Team
Palvella Therapeutics presented new analyses from the Phase 2 TOIVA trial (NCT06653842) showing that topical QTORIN™ rapamycin significantly reduced bleeding and improved lesion-related outcomes in patients with cutaneous venous malformations (VMs). The findings were presented at the 83rd Annual Meeting of the Society for Investigative Dermatology in Chicago.
The SID 2026 presentation expanded on topline results reported in December 2025, which showed statistically significant improvements across multiple clinician-reported and patient-reported endpoints. The new analyses focused on bleeding outcomes and patient quality-of-life burden.
TOIVA is a Phase 2, single-arm, open-label, baseline-controlled study evaluating once-daily topical QTORIN rapamycin in cutaneous VMs. The study includes a 12-week efficacy period followed by a 12-week extension phase, although extension-phase data were not presented at SID.
QTORIN rapamycin is Palvella’s proprietary topical formulation of rapamycin being developed for rare dermatologic conditions with no approved therapies.
Among patients with bleeding at baseline (n=4), 100% demonstrated statistically significant improvement on the Cutaneous Venous Malformations Investigator Global Assessment Bleeding scale (cVM-IGA Bleeding) at Week 12, with a mean effect size of +2.5 (p=0.003). Although the subgroup was small, all four patients were rated as either “Much Improved” or “Very Much Improved.”
In addition, 100% of patients with bleeding at baseline reported being “satisfied” or “very satisfied” with treatment on the Treatment Satisfaction Questionnaire for Medication at Week 12.
The company also presented findings from a pre-specified qualitative patient interview sub-study conducted under the FDA’s Patient-Focused Drug Development framework.
Patients reported that the burden of cutaneous VMs extends beyond lesion severity and includes pain, swelling, bluish discoloration, bleeding, emotional distress, and limitations in physical and social activities. Baseline interviews highlighted the substantial physical, functional, and psychosocial burden associated with the disease. Patients identified improvement in lesion appearance, pain reduction, and decreased lesion size as major treatment priorities.
Michael Kelly of the Cleveland Clinic said the study demonstrated meaningful reductions in bleeding and improvements in lesion appearance and height. He added that the correlation between clinical improvements and patient-reported outcomes suggests QTORIN rapamycin may help reduce the broader disease burden associated with cutaneous venous malformations.
Cutaneous venous malformations are rare vascular abnormalities that can cause chronic pain, bleeding, swelling, and functional impairment. According to the company, QTORIN rapamycin has the potential to become the first FDA-approved therapy and standard of care for the estimated more than 75,000 individuals living with cutaneous venous malformations in the United States.
In a separate program, Palvella Therapeutics previously reported positive Phase 3 SELVA trial results for QTORIN™ 3.9% rapamycin gel in microcystic lymphatic malformations, with the study meeting its primary endpoint and an NDA submission planned for the second half of 2026.
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About the Writer
Fariha Sameen, PharmD (LinkedIn), is a clinical pharmacy professional with hands-on experience in patient counselling, medication review, therapeutic monitoring, and clinical documentation across multiple departments. She has experience identifying and assessing drug-related problems and supporting medication safety practices. Her interests include pharmacovigilance, ADR reporting, clinical research, and medical writing focused on clear, evidence-based communication.