Palvella Therapeutics said new phase 2 data for Qtorin rapamycin showed a sharp improvement in bleeding among patients with cutaneous venous malformations, alongside strong patient satisfaction scores.
In the TOIVA trial, 4 patients entered with bleeding at baseline. By week 12, all 4 patients — 100% — showed improvement on the cutaneous venous malformations investigator global assessment bleeding scale. The mean change was +2.5 points, with a p-value of 0.003. On the 7-point scale, every patient was rated either “much improved” or “very much improved” at week 12.
Patient-reported results moved in the same direction. All 4 patients with baseline bleeding said they were either “satisfied” or “very satisfied” with Qtorin rapamycin on the overall satisfaction item of the treatment satisfaction questionnaire at week 12.
Palvella also said baseline interviews showed the disease burden went well beyond visible lesions. Patients described bluish discoloration, pain, swelling, protrusions, bleeding or leakage, plus limits on physical activity, social participation, and work or school functioning. The company said treatment priorities centered on improving appearance, reducing pain, and shrinking lesion size.
The company said the findings add to previously announced results from December 2025, when TOIVA met statistical significance on multiple pre-specified clinician-reported and patient-reported endpoints. Today the company's shares have moved -4.1% to a price of $114.095. If you want to know more, read the company's complete 8-K report here.
