Palvella Therapeutics Announces Positive Topline Results from Phase 2 Study of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations, a Serious, Rare Genetic Skin Disease with No FDA-approved Therapies
– QTORIN™ rapamycin generally well-tolerated; no drug related severe adverse events and no observed rapamycin in systemic circulation
– 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
– End of Phase 2 meeting completed with U.S. Food and Drug Administration in February 2023; pending additional interactions with FDA, anticipate potential initiation of pivotal Phase 3 study in second half of 2023
– FDA previously granted Fast Track Designation and Orphan Drug Designation to QTORIN™ rapamycin for Microcystic Lymphatic Malformations
– QTORIN™ rapamycin has potential to become first therapy and standard of care for the estimated more than 30,000 individuals with Microcystic Lymphatic Malformations in U.S., if approved