Palvella Therapeutics Announces Publication of Results from Phase 2 Clinical Trial of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations in the Journal of Vascular Anomalies
Palvella Therapeutics has announced the publication of results from its Phase 2 clinical trial of QTORIN™ 3.9% Rapamycin Anhydrous Gel for treating microcystic lymphatic malformations (microcystic LMs) in the Journal of Vascular Anomalies. The study reported that 100% of participants were rated as either “Much Improved” or “Very Much Improved” by the Clinician Global Impression of Change after 12 weeks of treatment. The FDA has previously granted QTORIN™ rapamycin Breakthrough Therapy, Fast Track, and Orphan Drug Designations for this indication.
The ongoing Phase 3 SELVA trial, which is single-arm and baseline-controlled, aims to further evaluate QTORIN™ rapamycin for microcystic LMs, with topline data expected in Q1 2026. If successful, QTORIN™ rapamycin could become the first approved therapy and standard of care in the U.S. for microcystic LMs. The Phase 2 study demonstrated nominal statistical significance across several efficacy endpoints, including clinician and patient global impression assessments. No drug-related serious adverse events were reported, indicating that QTORIN™ rapamycin was generally well-tolerated.
The publication can be accessed in the Journal of Vascular Anomalies, the official journal of the International Society for the Study of Vascular Anomalies (ISSVA). Palvella is currently enrolling approximately 40 subjects in the SELVA trial, which is supported by an Orphan Products Grant from the FDA's Office of Orphan Products Development.
Palvella Therapeutics ha annunciato la pubblicazione dei risultati del suo studio clinico di Fase 2 relativo al gel anidride rapamicina QTORIN™ 3,9% per il trattamento delle malformazioni linfatiche microcistiche (microcystic LMs) nella rivista Journal of Vascular Anomalies. Lo studio ha riportato che il 100% dei partecipanti è stato classificato come "Notevolmente Migliorato" o "Moltissimo Migliorato" secondo il Clinician Global Impression of Change dopo 12 settimane di trattamento. La FDA ha precedentemente conferito a QTORIN™ rapamicina le designazioni di Breakthrough Therapy, Fast Track e Orphan Drug per questa indicazione.
La trial di Fase 3 SELVA, che è a braccio singolo e controllata basale, mira a valutare ulteriormente la rapamicina QTORIN™ per le LMs microcistiche, con dati preliminari attesi nel primo trimestre del 2026. Se avrà successo, la rapamicina QTORIN™ potrebbe diventare la prima terapia approvata e standard di cura negli Stati Uniti per le LMs microcistiche. Lo studio di Fase 2 ha dimostrato una significativa rilevanza statistica nominale su diversi endpoint di efficacia, tra cui le valutazioni globali di clinici e pazienti. Non sono stati riportati eventi avversi gravi correlati al farmaco, indicando che la rapamicina QTORIN™ è stata generalmente ben tollerata.
La pubblicazione può essere consultata nel Journal of Vascular Anomalies, la rivista ufficiale della International Society for the Study of Vascular Anomalies (ISSVA). Palvella sta attualmente arruolando circa 40 soggetti nello studio SELVA, che è supportato da una sovvenzione per Prodotti Orfani dall'Ufficio della FDA per lo Sviluppo di Prodotti Orfani.
Palvella Therapeutics ha anunciado la publicación de los resultados de su ensayo clínico de Fase 2 sobre el gel anhidro de rapamicina QTORIN™ 3.9% para el tratamiento de malformaciones linfáticas microquísticas (microcystic LMs) en la revista Journal of Vascular Anomalies. El estudio reportó que el 100% de los participantes fueron calificados como "Mucho Mejor" o "Muy Mejorado" por el Clinician Global Impression of Change tras 12 semanas de tratamiento. La FDA ha otorgado previamente a QTORIN™ rapamicina las designaciones de Terapia Innovadora, Fast Track y Medicamento Huérfano para esta indicación.
El ensayo de Fase 3 SELVA, que es de un solo brazo y controlado por línea de base, tiene como objetivo evaluar más a fondo la rapamicina QTORIN™ para las LMs microquísticas, con datos preliminares esperados en el primer trimestre de 2026. Si tiene éxito, la rapamicina QTORIN™ podría convertirse en la primera terapia aprobada y estándar de atención en EE. UU. para las LMs microquísticas. El estudio de Fase 2 demostró una significación estadística nominal en varios puntos finales de eficacia, incluidas las evaluaciones globales de clínicos y pacientes. No se reportaron eventos adversos graves relacionados con el fármaco, lo que indica que la rapamicina QTORIN™ fue generalmente bien tolerada.
La publicación se puede acceder en el Journal of Vascular Anomalies, la revista oficial de la International Society for the Study of Vascular Anomalies (ISSVA). Palvella está actualmente reclutando aproximadamente 40 sujetos para el ensayo SELVA, el cual cuenta con el apoyo de una subvención de Productos Huérfanos de la Oficina de Desarrollo de Productos Huérfanos de la FDA.
Palvella Therapeutics는 미세 낭포 림프 변형(microcystic LMs) 치료를 위한 QTORIN™ 3.9% 라파마이신 무수 젤의 2상 임상 시험 결과를 Journal of Vascular Anomalies에 발표했다고 전했습니다. 이 연구에 따르면, 100%의 참가자가 치료 12주 후에 임상의가 평가한 변화의 전반적인 인상에서 "상당히 개선됨" 또는 "매우 개선됨"으로 평가되었습니다. FDA는 이 적응증에 대해 QTORIN™ 라파마이신에 대해 혁신 치료, 패스트트랙 및 고아 의약품 지정을 부여했습니다.
현재 진행 중인 3상 SELVA 시험은 단일 팔 및 기준선 통제 방식으로, QTORIN™ 라파마이신의 미세 낭포 LMs에 대한 추가 평가를 목표로 하며, topline 데이터는 2026년 1분기에 예상됩니다. 성공할 경우, QTORIN™ 라파마이신은 미국에서 미세 낭포 LMs에 대한 첫 번째 승인 치료제가자 및 표준 치료가 될 수 있습니다. 2상 연구는 임상 및 환자의 전반적인 인상 평가를 포함하여 여러 효능 지표에서 명목상의 통계적 유의성을 보였습니다. 약물과 관련된 심각한 부작용은 보고되지 않았으며, QTORIN™ 라파마이신이 전반적으로 잘 견뎌졌음을 나타냅니다.
출판물은 국제 혈관 이상 연구 협회(ISSVA)의 공식 저널인 Journal of Vascular Anomalies에서 확인할 수 있습니다. Palvella는 현재 SELVA 시험에 약 40명의 피험자를 등록하고 있으며, 이는 FDA의 고아 의약품 개발 사무소로부터 고아 제품 보조금으로 지원받고 있습니다.
Palvella Therapeutics a annoncé la publication des résultats de son essai clinique de Phase 2 concernant le gel anhydre de rapamycine QTORIN™ 3,9% pour le traitement des malformations lymphatiques microkystiques (microcystic LMs) dans le Journal des Anomalies Vasculaires. L'étude a rapporté que 100 % des participants avaient été classés comme "Considérablement Améliorés" ou "Très Améliorés" par l'impression globale de changement du clinicien après 12 semaines de traitement. La FDA a précédemment accordé à QTORIN™ rapamycine les désignations de thérapie innovante, de parcours accéléré et de médicament orphelin pour cette indication.
L'essai de Phase 3 SELVA, qui est à bras unique et contrôlé par ligne de base, vise à évaluer davantage la rapamycine QTORIN™ pour les LMs microkystiques, avec des données préliminaires attendues au premier trimestre 2026. En cas de succès, QTORIN™ rapamycine pourrait devenir la première thérapie approuvée et standard de soins aux États-Unis pour les LMs microkystiques. L'étude de Phase 2 a montré une signification statistique nominale sur plusieurs critères d'efficacité, y compris les évaluations globales des cliniciens et des patients. Aucun événement indésirable grave lié au médicament n'a été signalé, indiquant que QTORIN™ rapamycine a été généralement bien toléré.
La publication peut être consultée dans le Journal des Anomalies Vasculaires, la revue officielle de la Société Internationale pour l'Étude des Anomalies Vasculaires (ISSVA). Palvella recrute actuellement environ 40 sujets pour l'essai SELVA, qui est soutenu par une subvention pour produits orphelins du bureau de la FDA pour le développement de produits orphelins.
Palvella Therapeutics hat die Veröffentlichung der Ergebnisse seiner Phase-2-Studie zum QTORIN™ 3,9% Rapamycin-Anhydrogelen zur Behandlung von mikrozystischen lymphatischen Malformationen (mikrozystische LMs) im Journal of Vascular Anomalies angekündigt. Die Studie berichtete, dass 100% der Teilnehmer von den klinischen Beurteilungen des Globalen Eindrucks der Veränderung nach 12 Wochen Behandlung als "Deutlich verbessert" oder "Sehr stark verbessert" eingestuft wurden. Die FDA hat QTORIN™ Rapamycin zuvor die Bezeichnungen der Durchbruchtherapie, des Fast Track und des Orphan Drug für diese Indikation verliehen.
Die laufende Phase-3-SELVA-Studie, die einarmig und baseline-kontrolliert ist, zielt darauf ab, QTORIN™ Rapamycin für mikrozystische LMs weiter zu bewerten, wobei die ersten Daten im ersten Quartal 2026 erwartet werden. Im Falle eines Erfolges könnte QTORIN™ Rapamycin die erste zugelassene Therapie und Behandlungsstandard in den USA für mikrozystische LMs werden. Die Phase-2-Studie zeigte unter mehreren Wirksamkeits-Endpunkten eine nominale statistische Signifikanz, einschließlich der globalen Einschätzungen von Kliniken und Patienten. Es wurden keine schwerwiegenden Nebenwirkungen im Zusammenhang mit dem Medikament gemeldet, was darauf hindeutet, dass QTORIN™ Rapamycin im Allgemeinen gut vertragen wurde.
Die Veröffentlichung ist im Journal of Vascular Anomalies, der offiziellen Zeitschrift der International Society for the Study of Vascular Anomalies (ISSVA), zugänglich. Palvella rekrutiert derzeit etwa 40 Probanden für die SELVA-Studie, die von einem Grant für Orphan Products des FDA-Büros für die Entwicklung von Orphan-Produkten unterstützt wird.
- 100% of participants were rated as 'Much Improved' or 'Very Much Improved' after 12 weeks of treatment.
- FDA has granted Breakthrough Therapy, Fast Track, and Orphan Drug Designations to QTORIN™ rapamycin.
- No drug-related serious adverse events were reported in the Phase 2 study.
- QTORIN™ rapamycin has potential to be the first approved therapy for microcystic LMs in the U.S.
- Topline data from the ongoing Phase 3 SELVA trial expected in Q1 2026.
- None.
Insights
The Phase 2 trial results for QTORIN™ rapamycin represent a significant clinical milestone in the treatment of microcystic lymphatic malformations. The 100% response rate in patient improvement is particularly noteworthy in the rare disease space, where such unanimous positive outcomes are uncommon. The drug's triple designation status from the FDA (Breakthrough Therapy, Fast Track and Orphan Drug) underscores its potential therapeutic value and expedited development pathway.
The ongoing Phase 3 SELVA trial with 40 subjects is relatively robust for a rare disease study. The single-arm, baseline-controlled design is appropriate given the orphan indication and lack of approved treatments. The 24-week duration will provide valuable long-term safety and efficacy data beyond the 12-week Phase 2 results. For a company with a
In simple terms: This gel-based treatment showed remarkable results in helping people with a rare skin condition that currently has no approved treatments. All patients in the early trial showed major improvements and the FDA is helping speed up its development. If the larger ongoing trial succeeds, this could become the first approved treatment for this condition.
From a market perspective, QTORIN™ rapamycin's position as a potential first-in-disease therapy creates a compelling commercial opportunity. The triple FDA designations not only facilitate faster development but also provide market exclusivity benefits - particularly the Orphan Drug Designation which grants 7 years of market exclusivity upon approval. For Palvella, with its focused rare disease strategy, this represents a significant value driver.
The publication in the Journal of Vascular Anomalies adds credibility and visibility within the specialist medical community, which is important for adoption in rare diseases where key opinion leaders significantly influence treatment patterns. The timing of topline data in Q1 2026 provides a clear catalyst timeline for investors. Given Palvella's current market cap of
Publication reports
FDA previously granted Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation to QTORIN™ rapamycin for microcystic lymphatic malformations (microcystic LMs)
Ongoing Phase 3 single-arm, baseline-controlled trial evaluating QTORIN™ rapamycin for the treatment of microcystic LMs with topline data expected in Q1 2026
QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for microcystic LMs
WAYNE, Pa., Jan. 10, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella), 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, today announced results from the Phase 2 study of QTORIN™
"The Phase 2 results highlight QTORIN™ rapamycin’s potential to be the first targeted therapy for children and adults living with microcystic lymphatic malformations, a serious, rare genetic disease," said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. "We look forward to further evaluating the potential of QTORIN™ rapamycin in the ongoing Phase 3 SELVA trial and to expediting this potential first-in-disease therapy to patients.”
As previously reported by Palvella, the publication presents results demonstrating nominal statistical significance across several of the efficacy endpoints assessing the change from pre-treatment baseline to end of treatment (Week 12) with once daily QTORIN™ rapamycin (n=12), including clinician and patient global impression assessments as well as assessments of individual clinical manifestations that are important disease burdens for individuals living with microcystic LMs. QTORIN™ rapamycin was generally well-tolerated with no participants experiencing drug related serious adverse events. The publication, titled “Phase 2 Study of the Safety and Efficacy of Topical QTORIN™ Rapamycin for the Treatment of Cutaneous Microcystic Lymphatic Malformations”, can be accessed here.
Palvella is currently enrolling approximately 40 subjects in SELVA, a 24-week, Phase 3, single-arm, baseline-controlled trial of QTORIN™ rapamycin for the treatment of microcystic LMs. The U.S. Food and Drug Administration (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 from FDA’s Office of Orphan Products Development.
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 drug disease drug development veterans, Palvella Therapeutics (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.
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, the sufficiency of Palvella’s capital resources; Palvella’s cash runway; statements regarding 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; the expected timing of initiating, as well as the design of Palvella’s Phase 2 clinical trial of QTORIN™ rapamycin in cutaneous venous malformations. 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 “Risk Factors” section of Palvella’s definitive proxy statement/information statement dated November 8, 2024 and other documents filed by Palvella from time to time with the Securities Exchange Commission. 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
Stephanie Jacobson
Managing Director, Argot Partners
palvella@argotpartners.com
FAQ
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