Palvella Therapeutics Releases New Data from Qtorin Clinical Trial

Palvella Therapeutics announced new data from the Phase 2 TOIVA trial of Qtorin rapamycin in patients with cutaneous venous malformations were presented at the 83rd Annual Meeting of the Society for Investigative Dermatology in Chicago. TOIVA is a Phase 2, single-arm, open-label, baseline-controlled clinical trial of Qtorin rapamycin administered topically once daily for a 12-week efficacy evaluation period followed by a 12-week treatment extension period, for cutaneous VMs. Patients with bleeding at baseline demonstrated a statistically significant improvement in change in cVM-IGA Bleeding scores at Week 12 with a mean effect size of +2.5. cVM-IGA Bleeding is a seven-point clinician-assessed dynamic scale measuring change in bleeding from baseline to Week 12, ranging from "Very Much Worse" to "Very Much Improved". At week 12, 100% of patients were either "Much Improved" or "Very Much Improved" on the cVM-IGA Bleeding at Week 12. In total, 100% of patients with bleeding at baseline reported being "satisfied" or "very satisfied" with Qtorin rapamycin on the overall satisfaction item of the Treatment Satisfaction Questionnaire for Medication at Week 12. A pre-specified patient qualitative interview sub-study was incorporated to systematically capture the patient experience, including the symptoms, functional impacts, and treatment-related changes most meaningful to patients, consistent with FDA's Patient-Focused Drug Development framework. QoL burden extends beyond clinical lesion severity, including bluish discoloration, pain/discomfort, swelling, protrusions, and bleeding or leakage. Patients reported limitations in physical activity, social participation, and work/school functioning, as well as emotional distress related to lesion visibility. Treatment priorities included improving appearance, reducing pain, and decreasing lesion size, highlighting patient-important domains not fully captured by traditional clinical severity assessments. Findings underscore the patient-experienced burden of moderate-to-severe cutaneous VMs and reinforce the value of patient-reported outcomes in clinical development.