Palvella Therapeutics Announces QTORIN™ Rapamycin 3.9% Anhydrous Gel for the Treatment of Microcystic Lymphatic Malformations Featured in Oral Presentation by Amy Paller, M.S., M.D., Chair of Dermatology at Northwestern University’s Feinberg School of Medicine, at the 15th World Congress of Pediatric Dermatology
Palvella Therapeutics (PVLA) announced that QTORIN™ rapamycin 3.9% anhydrous gel for treating microcystic lymphatic malformations (LMs) was featured in an oral presentation at the 15th World Congress of Pediatric Dermatology. The presentation highlighted the expansion of the Phase 3 SELVA trial to include children 3-5 years old.
The Phase 2 study showed promising results with 100% of participants showing improvement, including 41.7% 'Very Much Improved' and 58.3% 'Much Improved' according to clinical assessment. The ongoing Phase 3 SELVA trial, targeting 40 subjects across 13 U.S. centers, is expected to deliver top-line results in Q1 2026.
QTORIN™ rapamycin has received Breakthrough Therapy, Fast Track, and Orphan Drug Designations from the FDA, along with a $2.6 million Orphan Products Grant. If approved, it would become the first FDA-approved therapy for microcystic LMs in the U.S.
Palvella Therapeutics (PVLA) ha annunciato che il gel anidro di rapamicina QTORIN™ al 3,9% per il trattamento delle malformazioni linfatiche microcistiche (LM) è stato presentato in una presentazione orale al 15° Congresso Mondiale di Dermatologia Pediatrica. La presentazione ha evidenziato l'espansione del trial di fase 3 SELVA per includere bambini di età compresa tra 3 e 5 anni.
Lo studio di fase 2 ha mostrato risultati promettenti, con il 100% dei partecipanti che ha mostrato miglioramenti, inclusi il 41,7% 'Migliorato Molto' e il 58,3% 'Migliorato' secondo la valutazione clinica. Il trial di fase 3 SELVA, che mira a coinvolgere 40 soggetti in 13 centri negli Stati Uniti, prevede di fornire risultati preliminari nel primo trimestre del 2026.
QTORIN™ rapamicina ha ricevuto le designazioni di Terapeutica Innovativa, Fast Track e Farmaco Orfano dalla FDA, insieme a un finanziamento di 2,6 milioni di dollari per Prodotti Orfani. Se approvato, diventerebbe la prima terapia approvata dalla FDA per le LM microcistiche negli Stati Uniti.
Palvella Therapeutics (PVLA) anunció que el gel anhidro de rapamicina QTORIN™ al 3,9% para el tratamiento de malformaciones linfáticas microquísticas (LM) fue presentado en una presentación oral en el 15º Congreso Mundial de Dermatología Pediátrica. La presentación destacó la expansión del ensayo de fase 3 SELVA para incluir a niños de 3 a 5 años.
El estudio de fase 2 mostró resultados prometedores, con el 100% de los participantes mostrando mejoría, incluyendo el 41,7% 'Muy Mejorado' y el 58,3% 'Mejorado' según la evaluación clínica. El ensayo de fase 3 SELVA, que tiene como objetivo 40 sujetos en 13 centros de EE. UU., se espera que entregue resultados preliminares en el primer trimestre de 2026.
QTORIN™ rapamicina ha recibido las designaciones de Terapia Innovadora, Vía Rápida y Medicamento Huérfano de la FDA, junto con una subvención de 2,6 millones de dólares para Productos Huérfanos. Si se aprueba, se convertiría en la primera terapia aprobada por la FDA para las LM microquísticas en EE. UU.
팔벨라 제약 (PVLA)는 미세낭성 림프 기형(LM) 치료를 위한 3.9% 무수 라파마이신 젤 QTORIN™이 제15회 세계 소아 피부과 학회에서 구두 발표되었다고 발표했습니다. 발표에서는 3-5세 아동을 포함하도록 3상 SELVA 시험의 확장을 강조했습니다.
2상 연구는 100%의 참가자가 개선을 보였으며, 이 중 41.7%는 '매우 개선됨', 58.3%는 '개선됨'으로 임상 평가에 따라 나타났습니다. 13개의 미국 센터에서 40명의 대상을 목표로 하는 진행 중인 3상 SELVA 시험은 2026년 1분기에 주요 결과를 발표할 것으로 예상됩니다.
QTORIN™ 라파마이신은 FDA로부터 혁신 치료제, 신속 심사, 그리고 고아약 지정을 받았으며, 260만 달러의 고아 제품 보조금도 수여받았습니다. 승인될 경우, 미국에서 미세낭성 LM에 대한 첫 번째 FDA 승인 치료제가 될 것입니다.
Palvella Therapeutics (PVLA) a annoncé que le gel anhydre de rapamycine QTORIN™ à 3,9 % pour le traitement des malformations lymphatiques microkystiques (LM) a été présenté lors d'une présentation orale au 15e Congrès Mondial de Dermatologie Pédiatrique. La présentation a mis en avant l'expansion de l'essai de phase 3 SELVA pour inclure des enfants âgés de 3 à 5 ans.
L'étude de phase 2 a montré des résultats prometteurs, avec 100 % des participants montrant une amélioration, dont 41,7 % 'Très Amélioré' et 58,3 % 'Amélioré' selon l'évaluation clinique. L'essai de phase 3 SELVA en cours, visant 40 sujets dans 13 centres aux États-Unis, devrait fournir des résultats préliminaires au premier trimestre 2026.
QTORIN™ rapamycine a reçu des décisions de Thérapie Innovante, de Voie Accélérée et de Médicament Orphelin de la FDA, ainsi qu'une subvention de 2,6 millions de dollars pour les Produits Orphelins. Si approuvé, cela deviendrait la première thérapie approuvée par la FDA pour les LM microkystiques aux États-Unis.
Palvella Therapeutics (PVLA) gab bekannt, dass das anhydride Rapamycin-Gel QTORIN™ mit 3,9% zur Behandlung von mikrozystischen lymphatischen Malformationen (LM) in einem mündlichen Vortrag auf dem 15. Weltkongress für pädiatrische Dermatologie vorgestellt wurde. Der Vortrag hob die Erweiterung der Phase-3-SELVA-Studie hervor, um Kinder im Alter von 3 bis 5 Jahren einzubeziehen.
Die Phase-2-Studie zeigte vielversprechende Ergebnisse, wobei 100% der Teilnehmer eine Verbesserung zeigten, darunter 41,7% 'Sehr stark verbessert' und 58,3% 'Verbessert' gemäß der klinischen Beurteilung. Die laufende Phase-3-SELVA-Studie, die 40 Probanden in 13 US-Zentren anvisiert, wird voraussichtlich im ersten Quartal 2026 erste Ergebnisse liefern.
QTORIN™ Rapamycin hat von der FDA die Bezeichnungen für Durchbruchtherapie, Schnellverfahren und Orphan Drug erhalten, sowie einen Zuschuss in Höhe von 2,6 Millionen Dollar für Waisenkinderprodukte. Wenn genehmigt, würde es die erste von der FDA zugelassene Therapie für mikrozystische LMs in den USA werden.
- Phase 2 trial showed 100% positive response rate
- Received multiple FDA special designations (Breakthrough, Fast Track, Orphan)
- Secured $2.6M FDA Orphan Products Grant
- Potential to be first FDA-approved therapy for microcystic LMs
- Phase 3 results not expected until Q1 2026
- trial size of 40 subjects for Phase 3
Insights
The expansion of Palvella's Phase 3 SELVA trial to include younger children (3-5 years) represents a strategic enhancement to their development program for QTORIN™ rapamycin. This age expansion is particularly significant as microcystic lymphatic malformations are best managed during childhood, potentially expanding the addressable patient population.
The triple regulatory designations (Breakthrough Therapy, Fast Track, and Orphan Drug) from the FDA provide Palvella with multiple advantages including enhanced agency interaction, potential for priority review, and market exclusivity if approved. The
Phase 2 results showing
With top-line Phase 3 data expected in Q1 2026, Palvella maintains potential first-mover advantage in this rare disease space with no currently approved therapies. The fit-for-purpose primary endpoint developed in collaboration with clinicians demonstrates appropriate regulatory strategy, though investors should recognize that significant commercial revenue remains years away.
Microcystic lymphatic malformations represent a significant therapeutic challenge in dermatology practice. These congenital mosaic lesions progressively enlarge without spontaneous regression, causing considerable complications that impact quality of life. The potential for a topical therapy specifically targeting the underlying mTOR pathway dysregulation addresses the pathophysiology more precisely than current off-label approaches.
The mechanism of QTORIN™ rapamycin - selectively inhibiting mTOR in the skin to reduce endothelial cell hyperproliferation and vascular endothelial growth factor signaling - directly targets the fundamental disease process. The Phase 2 efficacy metrics are particularly noteworthy, with
The expanded inclusion of children 3-5 years old in the Phase 3 SELVA trial is clinically appropriate given the pediatric onset and progressive nature of the condition. Early intervention may prevent complications and reduce disease burden throughout the patient's lifetime. The trial's focus on moderate-to-severe disease also ensures the therapy is being evaluated in patients with the greatest medical need.
The development of a specific Microcystic Lymphatic Malformations Investigator's Global Assessment (mLM-IGA) as the primary endpoint represents a methodological advancement in evaluating therapies for this rare condition, potentially establishing a standardized assessment tool for future research.
Presentation highlighted the recent expansion of the Phase 3 SELVA trial to include children 3 to 5 years old
Presentation reviewed clinically and statistically significant Phase 2 results and the design of the ongoing Phase 3 SELVA trial
Top-line results from SELVA remain on track for the first quarter of 2026
QTORIN™
WAYNE, Pa., April 11, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or “the Company”), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced QTORIN™ rapamycin
"There is an urgent need for a safe and effective targeted topical therapy for mosaic genetic skin disorders including microcystic lymphatic malformation," said Amy Paller, M.S., M.D., Chair of Dermatology, Northwestern University's Feinberg School of Medicine. "Many patients have considerable complications associated with this disease, and I am looking forward to the Phase 3 results early next year."
The oral presentation titled, "SELVA: A Phase 3 study with a fit-for-purpose primary endpoint evaluating QTORIN™
- Microcystic LMs are congenital mosaic lesions that gradually increase in size with risk of complications and are best managed aggressively during childhood
- Microcystic LMs are proliferative with no spontaneous regression
- QTORIN™ rapamycin, an investigational therapy designed to selectively inhibit the mammalian target of rapamycin (mTOR) in the skin, potentially reduces endothelial cell hyper-proliferation and vascular endothelial growth factor signaling, both of which are the result of overactive mTOR signaling in microcystic LMs
- A multicenter, open-label, 12-week, Phase 2 study evaluating the safety and efficacy of QTORIN™ rapamycin for microcystic LMs demonstrated:
100% of participants were either "Very Much Improved” (41.7% ) or “Much Improved” (58.3% ) as rated by the Clinician Global Impression of Change (CGI-C), a 7-point change scale conducted by live clinician assessment83% of participants were either “Very Much Improved” (25% ) or “Much Improved” (58.3% ) as rated by the Patient Global Impression of Change, a 7-point change scale reported by patients- QTORIN™ rapamycin was generally well-tolerated; all treatment related adverse events were moderate or mild and there were no discontinuations due to adverse events.
- SELVA, a 24-week, Phase 3, single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin for the treatment of microcystic LMs, mimics the Phase 2 study, with key study elements including the following:
- Based on data from Phase 2 and clinician interviews, the primary endpoint is the fit-for-purpose Microcystic Lymphatic Malformations Investigator’s Global Assessment (mLM-IGA), a 7-point change scale conducted by live clinician assessment with similarities to the CGI-C
- Patient population enriched to include patients with moderate to severe disease
- Target sample size of 40 subjects
- Treatment duration extended to 24-weeks
- Enrollment criteria expanded to include patients 3 years and older
"Microcystic LMs are a serious, rare, and chronically debilitating genetic disease with a pediatric onset and lifelong course. Early intervention is essential to minimizing disease burden for this patient population who currently have no FDA-approved therapies. Palvella is pleased to include patients younger than 6 years old in the ongoing Phase 3 SELVA study," said Wes Kaupinen, Founder and Chief Executive Officer of Palvella.
SELVA is currently enrolling patients at 13 centers in the United States. Top-line data is anticipated in the first quarter of 2026. The U.S. FDA has granted Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation to QTORIN™ rapamycin for the treatment of microcystic LMs. Additionally, the SELVA study is supported by an Orphan Products Grant of up to
About Microcystic Lymphatic Malformations
Microcystic LMs are a rare, chronically debilitating genetic disease caused by dysregulation of the phosphatidylinositol 3-kinase (PI3K)/mammalian target of rapamycin (mTOR) pathway. The disease is characterized by malformed lymphatic vessels that protrude through the skin and persistently leak lymph fluid (lymphorrhea) and bleed, often leading to recurrent serious infections and cellulitis that can cause hospitalization. The natural history of microcystic LMs is persistent and progressive without spontaneous resolution, with symptoms generally worsening during life, including increases in the number and size of malformed vessels that lead to complications and lifetime morbidity. There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States.
About Palvella Therapeutics
Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN
QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication.
Forward-Looking Statements
This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)). These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella, as well as assumptions made by, and information currently available to, the management of Palvella. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from ongoing clinical trials, Palvella’s clinical development plans and related anticipated development milestones, Palvella’s cash and financial resources and expected cash runway, and the potential of, and expectations regarding, Palvella’s programs, including QTORIN™ rapamycin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin; the outcome of early clinical trials for Palvella’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella’s current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that Palvella may face. Except as required by applicable law, Palvella does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
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Contact Information
Investors
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
wes.kaupinen@palvellatx.com
Media
Marcy Nanus
Managing Partner, Trilon Advisors LLC
mnanus@trilonadvisors.com
FAQ
What were the key results from PVLA's Phase 2 trial of QTORIN rapamycin for lymphatic malformations?
When will Palvella Therapeutics (PVLA) release Phase 3 SELVA trial results for QTORIN?
What FDA designations has PVLA's QTORIN received for microcystic lymphatic malformations?
How many centers are participating in PVLA's Phase 3 SELVA trial for QTORIN?
