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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-approved Therapies
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™ rapa
About this update from Palvella Therapeutics, Inc.
[{"type":"image","alt":"Palvella Therapeutics Inc.","displaySize":"","headline":null,"caption":"Palvella Therapeutics Inc.","className":"","disableSlideshowImg":false,"size":{"original":{"width":300,"height":300,"url":"https://media.zenfs.com/en/globenewswire.com/4b273ae029a5e8cb06a3771e7b41e04d"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/IZ4VZ0Qdm50kE5TTbiIBBg--/YXBwaWQ9aGlnaGxhbmRlcjt3PTQyMDtoPTQyMDtjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/4b273ae029a5e8cb06a3771e7b41e04d","width":300,"height":300}},"lazy":false},{"type":"text","content":"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","length":302,"tagName":"p"},{"type":"text","content":"Achieved statistical significance on multiple pre-specified clinician-reported and patient-reported efficacy endpoints, including dynamic change endpoints and static severity endpoints","length":184,"tagName":"p"},{"type":"text","content":"QTORIN™ rapamycin was generally well-tolerated, with no drug-related serious adverse events reported","length":100,"tagName":"p"},{"type":"text","content":"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","length":257,"tagName":"p"},{"type":"text","content":"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.","length":192,"tagName":"p"},{"type":"text","content":"Company to host conference call at 8:30am ET today","length":50,"tagName":"p"},{"type":"text","content":"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 resul...