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 New Drug Application (NDA) submission planned for the second half of 2026, pending positive results [1][4] - QTORIN™ rapamycin has the potential 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][13] QTORIN™ Rapamycin for Microcystic Lymphatic Malformations - Microcystic lymphatic malformations are a rare genetic disease with no FDA-approved therapies, characterized by malformed lymphatic vessels causing severe complications [4][5] - The SELVA study enrolled 51 subjects, exceeding the target of 40, and is designed to evaluate the efficacy of QTORIN™ rapamycin over a 24-week treatment period [4][5] - The FDA has granted multiple designations to QTORIN™ rapamycin, including Breakthrough Therapy and Orphan Designation [5] QTORIN™ Rapamycin for Cutaneous Venous Malformations - Positive Phase 2 results for QTORIN™ rapamycin in treating cutaneous venous malformations were announced in December 2025, with a significant improvement seen in 73% of trial participants [9] - The company plans to meet with the FDA for a Preliminary Breakthrough Therapy Designation Advice meeting in Q1 2026 and initiate a Phase 3 study in the second half of 2026 [9] QTORIN™ Rapamycin for Clinically Significant Angiokeratomas - The company has expanded the development of QTORIN™ rapamycin to include clinically significant angiokeratomas, with an estimated 50,000 diagnosed patients in the U.S. [9] - Fast Track Designation has been granted for this indication, and a Phase 2 study is expected to begin in the second half of 2026 [9] QTORIN™ Pitavastatin for Disseminated Superficial Actinic Porokeratosis - QTORIN™ pitavastatin is being developed for a premalignant skin disease with no FDA-approved therapies, targeting over 50,000 diagnosed patients in the U.S. [9][14] - The company plans to meet with the FDA in the first half of 2026 to discuss a Phase 2 clinical trial design, with initiation expected in the second half of 2026 [9] Corporate Update and Leadership - Palvella Therapeutics is focused on developing therapies for serious, rare skin diseases and vascular malformations, leveraging its QTORIN™ platform [12] - The leadership team has been strengthened with key hires in commercial, research and development, and regulatory affairs to support the anticipated U.S. launch of QTORIN™ rapamycin [13]