Palvella Therapeutics Announces Planned Pivotal Phase 3 Study Design of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations and Topline Results from Phase 2b CODY Study in Gorlin Syndrome
Palvella Therapeutics Announces Planned Pivotal Phase 3 Study Design of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations and Topline Results from Phase 2b CODY Study in Gorlin Syndrome
QTORIN™ rapamycin has potential to become first approved therapy and standard of care for Microcystic Lymphatic Malformations in U.S.; significant commercial opportunity based on an estimated more than 30,000 individuals in the U.S. with Microcystic Lymphatic Malformations
Phase 3 SELVA pivotal study to enroll approximately 50 subjects with Microcystic Lymphatic Malformations; productive FDA end-of-phase 2 meeting completed in Q1 2023
Phase 2 study of QTORIN™ rapamycin demonstrated 100% of participants with Microcystic Lymphatic Malformations were either “Much Improved” or “Very Much Improved” as rated by the Clinician Global Impression of Change following 12-weeks of QTORIN™ rapamycin
Phase 2b CODY study of QTORIN™ rapamycin for Basal Cell Carcinomas in Gorlin Syndrome did not meet primary endpoint of new Basal Cell Carcinoma prevention at 24-weeks; pre-specified secondary endpoint demonstrated nominally statistically significant reduction in existing Clinically Suspicious and Biopsy Confirmed Basal Cell Carcinoma lesion size compared to placebo