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Palvella Therapeutics Announces Positive Topline Results from Phase 2 TOIVA Clinical Trial of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Cutaneous Venous Malformations, a Serious, Rare Genetic Disease with No FDA-app...
Palvella Therapeutics Announces Positive Topline Results from Phase 2 TOIVA Clinical Trial of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the
About this update from Palvella Therapeutics, Inc.
[{"type":"text","content":"Palvella Therapeutics Announces Positive Topline Results from Phase 2 TOIVA Clinical Trial of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Cutaneous Venous Malformations, a Serious, Rare Genetic Disease with No FDA-approved Therapies \n 73% of trial participants (11/15 participants) improved on the Overall Cutaneous Venous Malformations Investigator Global Assessment (Overall cVM-IGA) at Week 12; 67% of trial participants (10/15 participants) rated as “Much Improved” (+2) or “Very Much Improved” (+3) on the Overall cVM-IGA at Week 12 Achieved statistical significance on multiple pre-specified clinician-reported and patient-reported efficacy endpoints, including dynamic change endpoints and static severity endpoints QTORIN™ rapamycin was generally well-tolerated, with no drug-related serious adverse events reported Based on Phase 2 results, Palvella to pursue near-term discussions with FDA regarding the potential for Breakthrough Therapy Designation and a Phase 3 pivotal study; FDA previously granted Fast Track Designation to QTORIN™ rapamycin for venous malformations QTORIN™ rapamycin has the potential to become the first FDA-approved therapy and standard of care for the estimated more than 75,000 individuals with cutaneous venous malformations in the U.S. Company to host conference call at 8:30am ET today WAYNE, Pa., Dec. 15, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or “the Company”), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced positive topline results from the Company’s Phase 2 TOIVA study of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of cutaneous venous malformations (cutaneous VMs). “Based on the large magnitude of the treatment effect observed in the majority of patients in the Phase 2 TOIVA study, QTORIN™ rapamycin has potential to become first-line therapy and to establish a much-needed standard of care for individuals living with cutaneous venous malformations,” said Megha Tollefson, M.D., pediatric dermatologist at Mayo Clinic and Principal Investigator of the Phase 2 TOIVA study. “Cutaneous venous malforma...