Palvella Therapeutics Provides Corporate Update and 2026 Outlook: Advancing a Late Clinical-Stage Pipeline and Platform to Address Multiple Serious, Rare Skin Diseases and Vascular Malformations with No FDA-Approved Therapies

Core Insights - The Phase 3 SELVA study for QTORIN™ rapamycin is on track, with topline results expected in March 2026, and a potential NDA submission planned for the second half of 2026 if results are positive [1][4] - QTORIN™ rapamycin aims to be the first FDA-approved therapy for microcystic lymphatic malformations, a serious condition affecting over 30,000 patients in the U.S. [1][4] - The company is expanding its QTORIN pipeline, with plans to announce a new product candidate and a fourth clinical indication for QTORIN™ rapamycin in the second half of 2026 [2][9] Clinical Development - The SELVA study has enrolled 51 subjects, exceeding the target of 40, and includes an 8-week observational period followed by 24 weeks of active treatment [4] - Positive Phase 2 results for QTORIN™ rapamycin in cutaneous venous malformations were announced in December 2025, leading to a request for a Preliminary Breakthrough Therapy Designation meeting with the FDA [2][5] - A Phase 2 study for QTORIN™ pitavastatin in disseminated superficial actinic porokeratosis and QTORIN™ rapamycin in clinically significant angiokeratomas is expected to start in the second half of 2026 [2][5] Market Potential - Microcystic lymphatic malformations are characterized by malformed lymphatic vessels causing significant morbidity, with no FDA-approved therapies currently available [4][5] - Cutaneous venous malformations affect over 130,000 diagnosed patients in the U.S., with no existing FDA-approved treatments [5] - Clinically significant angiokeratomas are estimated to affect more than 50,000 patients in the U.S., also lacking FDA-approved therapies [5] Intellectual Property and Leadership - The company has strengthened its intellectual property portfolio with new patents for QTORIN™ rapamycin and QTORIN™ pitavastatin, extending protection through 2038 and 2046 respectively [9] - Key leadership hires have been made to enhance commercial, R&D, and medical affairs capabilities, supporting the anticipated U.S. launch of QTORIN™ rapamycin [9]