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Palvella Therapeutics Announces First Patients Dosed in Phase 2 TOIVA Clinical Trial of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Cutaneous Venous Malformations
Phase 2 single-arm, baseline-controlled trial evaluating QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of cutaneous venous
About this update from Palvella Therapeutics, Inc.
[{"type":"text","content":"Phase 2 single-arm, baseline-controlled trial evaluating QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of cutaneous venous malformations (cutaneous VMs) to enroll approximately 15 subjects at leading vascular anomaly centers across the U.S. Cutaneous VMs are a serious and lifelong genetic disease that can result in substantial morbidity and functional impairment of the skin affecting an estimated more than 75,000 diagnosed patients in the U.S. QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for cutaneous VMs WAYNE, Pa., Jan. 08, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies, today announced the first patients have recently been dosed in TOIVA, a multicenter, Phase 2 clinical trial designed to evaluate the safety and efficacy of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of cutaneous venous malformations (cutaneous VMs). “Cutaneous VMs are a serious, lifelong disease which leads to significant disease burden for children and adults living with the disease, including risk of serious complications such as bleeding, ulceration, thrombosis, and pain leading to significant impact on quality of life and daily function,” said Megha M. Tollefson, M.D., Pediatric Dermatologist and Medical Director of Mayo Clinic Vascular Malformation Clinic. “We’re excited to have the first patients dosed in the landmark Phase 2 TOIVA study evaluating QTORIN rapamycin, a targeted topical therapy with potential to inhibit the mammalian target of rapamycin (mTOR) pathway which is a causative driver of this disease. A potential new treatment option would be transformative for children and adults living with this disease, as no FDA-approved therapies currently exist.” Cutaneous VMs are a rare genetic disease caused by mutations in genes that cause overactivation of the PI3K/mTOR signaling pathway, leading to dysfunctional veins within the skin. These malformations can cause substantial morbidity and functional impairment, significantly impact quality of life, and are associated with severe bleeding, u...