Business
Palvella Therapeutics Announces Publication of Results from Phase 2 Clinical Trial of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations in the Journal of Vascular Anomalies
Publication reports 100% of participants were either “Much Improved” or “Very Much Improved” as rated by the Clinician Global Impression of Change following
About this update from Palvella Therapeutics, Inc.
[{"type":"text","content":"Publication reports 100% of participants were either “Much Improved” or “Very Much Improved” as rated by the Clinician Global Impression of Change following 12-weeks of QTORIN™ rapamycin FDA previously granted Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation to QTORIN™ rapamycin for microcystic lymphatic malformations (microcystic LMs) Ongoing Phase 3 single-arm, baseline-controlled trial evaluating QTORIN™ rapamycin for the treatment of microcystic LMs with topline data expected in Q1 2026 QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for microcystic LMs WAYNE, Pa., Jan. 10, 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 results from the Phase 2 study of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs) were published in the Journal of Vascular Anomalies (JoVA). JoVA, which is the official journal of the International Society for the Study of Vascular Anomalies (ISSVA), is an international peer reviewed journal dedicated to the discovery and report of the scientific investigation, diagnosis, and treatment of congenital and acquired human vascular lesions. \"The Phase 2 results highlight QTORIN™ rapamycin’s potential to be the first targeted therapy for children and adults living with microcystic lymphatic malformations, a serious, rare genetic disease,\" said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. \"We look forward to further evaluating the potential of QTORIN™ rapamycin in the ongoing Phase 3 SELVA trial and to expediting this potential first-in-disease therapy to patients.” As previously reported by Palvella, the publication presents results demonstrating nominal statistical significance across several of the efficacy endpoints assessing the change from pre-treatment baseline to end of treatment (Week 12) with once daily QTORIN™ rapamycin (n=12), including clinician and patient global impression assessments as well as assessments of individual clinical manifestations th...