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]

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]