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]

Core Insights - Palvella Therapeutics has appointed Dr. Vimal Patel as Senior Vice President of Medical Affairs, bringing over 25 years of experience in dermatology and immunology to the role [1][2][3] - Dr. Patel will lead the Medical Affairs organization to enhance scientific engagement, KOL collaboration, and disease state awareness for the QTORIN™ programs targeting serious, rare skin diseases [1][2] Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for serious, rare skin diseases without FDA-approved treatments [5][6] - The company is developing a pipeline based on its patented QTORIN™ platform, with lead candidates including QTORIN™ 3.9% rapamycin anhydrous gel and QTORIN™ pitavastatin [6][7] Leadership Experience - Dr. Patel previously served at Incyte as Dermatology Therapeutic Area Head, where he led medical strategy for various dermatological conditions and contributed to the launch of therapies like OPZELURA and povorcitinib [2][3] - His prior experience includes senior roles at Sun Pharma and Johnson & Johnson, where he contributed to the success of multiple high-impact therapies in dermatology and immunology [3] Strategic Vision - Dr. Patel emphasizes the importance of rigorous science and strong clinical evidence in advancing therapies for rare skin diseases, aligning with Palvella's mission-driven approach [4] - The QTORIN™ platform is seen as a pioneering solution with the potential to transform treatment paradigms for rare skin diseases by addressing their underlying pathobiology [4]

Core Insights - The FDA has granted Fast Track designation to QTORIN™ rapamycin for the treatment of angiokeratomas, highlighting its potential to address a significant unmet medical need [1][2][3] - Palvella Therapeutics plans to initiate a Phase 2 trial for QTORIN™ rapamycin in the second half of 2026, following discussions with the FDA [1][4] Company Overview - Palvella Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare skin diseases without FDA-approved treatments [5] - The company is developing a pipeline of product candidates based on its QTORIN™ platform, with QTORIN™ rapamycin as the lead candidate targeting microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas [5] Industry Context - Angiokeratomas are chronic skin lesions with no FDA-approved therapies, affecting an estimated 50,000 diagnosed patients in the U.S. [1][2] - The FDA's Fast Track program aims to expedite the development and review of drugs for serious conditions, facilitating earlier access for patients [3]

Core Insights - The FDA has granted Fast Track designation to QTORIN™ rapamycin for the treatment of angiokeratomas, highlighting its potential to address a significant unmet medical need [1][2][3] - Palvella Therapeutics plans to initiate a Phase 2 trial for QTORIN™ rapamycin in the second half of 2026, following discussions with the FDA [1][4] Company Overview - Palvella Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare skin diseases without FDA-approved treatments [5] - The company is developing a pipeline of product candidates based on its QTORIN™ platform, with QTORIN™ rapamycin as the lead candidate targeting microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas [5][6] Industry Context - Angiokeratomas are chronic skin lesions with no FDA-approved therapies, affecting an estimated 50,000 diagnosed patients in the U.S. [1][2] - The FDA's Fast Track program aims to expedite the development and review of drugs for serious conditions, facilitating earlier access for patients [3]

Core Insights - Palvella Therapeutics is advancing its QTORIN™-derived therapies for four serious, rare skin diseases, each lacking FDA-approved treatments, with the potential to be first in these markets [2][14] - The company reported financial results for Q3 2025, highlighting a net loss of $11.3 million, or $1.03 per share, compared to a net loss of $7.0 million, or $3.94 per share, in Q3 2024 [17] Recent Developments - The Phase 2 TOIVA trial for QTORIN™ rapamycin in cutaneous venous malformations is fully enrolled, with top-line results expected in mid-December 2025 [1][11] - The Phase 3 SELVA trial for QTORIN™ rapamycin in microcystic lymphatic malformations is on track for top-line results in Q1 2026 [1][6] - Palvella is expanding QTORIN™ rapamycin's development into clinically significant angiokeratomas, with a Phase 2 study anticipated to start in the second half of 2026 [1][11] - A new product candidate, QTORIN™ pitavastatin, is being developed for disseminated superficial actinic porokeratosis, with a Phase 2 study also expected to begin in the second half of 2026 [1][11] Financial Overview - As of September 30, 2025, the company had cash and cash equivalents of $63.6 million, expected to fund operations into the second half of 2027 [1][11] - Research and development expenses for Q3 2025 were $6.5 million, up from $3.2 million in Q3 2024, primarily due to increased spending on clinical trials [10][12] - General and administrative expenses rose to $3.6 million in Q3 2025 from $1.9 million in Q3 2024, driven by higher employee compensation and costs associated with being publicly traded [12] Leadership and Innovation - David W. Osborne, Ph.D., has been appointed as Chief Innovation Officer to lead early-stage R&D efforts, focusing on maximizing the QTORIN™ platform [5] - Jeffrey Martini, Ph.D., presented at a dermatological conference, emphasizing the potential of the QTORIN™ platform and its lead candidate [5]

Core Insights - Palvella Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare skin diseases without FDA-approved treatments [1][3] - The company will present at the Stifel 2025 Healthcare Conference on November 12, 2025, at 4:00 p.m. ET [1] - Palvella's lead product candidate is QTORIN™ 3.9% rapamycin anhydrous gel, targeting microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas [3] - The second product candidate, QTORIN™ pitavastatin, is being developed for disseminated superficial actinic porokeratosis [3] Company Overview - Palvella Therapeutics was founded by veterans in rare disease drug development and is dedicated to creating novel therapies for patients with serious, rare skin diseases [3] - The company utilizes its patented QTORIN™ platform to develop a broad pipeline of product candidates [3] - Both QTORIN™ rapamycin and QTORIN™ pitavastatin are currently for investigational use only and have not received FDA approval [4]

Core Insights - Palvella Therapeutics has announced QTORIN™ pitavastatin as a new product candidate for the treatment of disseminated superficial actinic porokeratosis (DSAP), a serious skin disease with no FDA-approved therapies available [1][3] - The company plans to initiate a Phase 2 clinical trial for QTORIN™ pitavastatin in the second half of 2026, following discussions with the FDA [1][3] Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare skin diseases without FDA-approved treatments [5][6] - The company utilizes its patented QTORIN™ platform to develop novel topical product candidates, with QTORIN™ pitavastatin being the second product candidate in its pipeline [5][6] Disease Context - DSAP is characterized by persistent lesions that can lead to chronic skin integrity loss and has the potential to transform into squamous cell carcinoma [2] - There are over 50,000 diagnosed patients in the U.S. suffering from DSAP, highlighting a significant unmet medical need [2]