Palvella Therapeutics Announces Presentations at the 12th Pediatric Dermatology Research Alliance (PeDRA) Annual Conference

Company Overview - Palvella Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare genetic skin diseases without FDA-approved treatments [1][7] - The company is developing a pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases [7] Product Development - The lead product candidate, QTORIN™ rapamycin, is a 3.9% anhydrous gel designed to treat microcystic lymphatic malformations (microcystic LMs) and other serious skin diseases [5][7] - QTORIN rapamycin has received FDA Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation for microcystic LMs, and Fast Track Designation for venous malformations [5] - The product aims to minimize systemic exposure and potential adverse reactions associated with systemic therapy [5][4] Clinical Trials - Palvella initiated the SELVA study, a 24-week, Phase 3, single-arm, baseline-controlled clinical trial of QTORIN rapamycin for microcystic LMs, expected to enroll 40 subjects [6] - The study's primary and key secondary endpoints are clinician-reported outcomes [6] Upcoming Presentations - Palvella will present data at the 12th Pediatric Dermatology Research Alliance (PeDRA) Annual Conference on October 24-26, 2024, in Phoenix, AZ [1][2] - Presentations will include data supporting QTORIN rapamycin as a potential targeted therapy for microcystic LMs and a review of the Phase 3 SELVA study [2] Market Context - Microcystic LMs is a rare genetic disease affecting over 30,000 diagnosed patients in the U.S., characterized by malformed lymphatic vessels and associated complications [3] - There are currently no FDA-approved treatments for microcystic LMs, highlighting a significant unmet medical need [3]