Palvella Therapeutics Announces New QTORIN™ Product Candidate, QTORIN™ Pitavastatin, for the Treatment of Disseminated Superficial Actinic Porokeratosis (DSAP), a Rare, Chronic, and Pre-Cancerous Genetic Skin Disease with No FDA-Approved Therapies
Palvella Therapeutics (NASDAQ: PVLA) announced a new topical product candidate, QTORIN™ pitavastatin, for disseminated superficial actinic porokeratosis (DSAP), a rare premalignant genetic skin disease with no FDA‑approved therapies and an estimated >50,000 diagnosed U.S. patients.
The company plans an FDA meeting in the first half of 2026 to discuss a proposed Phase 2 design and anticipates initiating the Phase 2 trial in the second half of 2026. Palvella cited genetic evidence and published case studies supporting topical statins and highlighted pitavastatin's potency as rationale for development. A webcast conference call was held on November 5, 2025 to discuss the candidate.
Palvella Therapeutics (NASDAQ: PVLA) ha annunciato un nuovo candidato di prodotto topico, QTORIN™ pitavastatina, per porokeratosi attinica disseminata superficiale (DSAP), una rara malattia genetica cutanea premalignante senza terapie approvate dalla FDA e con oltre 50.000 pazienti diagnosticati negli Stati Uniti.
L'azienda prevede un incontro con la FDA nella prima metà del 2026 per discutere un design proposto di Fase 2 e anticipa di avviare il trial di Fase 2 nella seconda metà del 2026. Palvella ha citato evidenze genetiche e casi clinici pubblicati a sostegno dell'uso topico di statine e ha evidenziato la potenza della pitavastatina come base per lo sviluppo. Una conference call in webcast è stata tenuta il 5 novembre 2025 per discutere il candidato.
Palvella Therapeutics (NASDAQ: PVLA) anunció un nuevo candidato de producto tópico, QTORIN™ pitavastatina, para la poroqueratosis actínica difusa superficial (DSAP), una rara enfermedad genética de la piel premaligna sin terapias aprobadas por la FDA y con más de 50,000 pacientes diagnosticados en EE. UU.
La empresa planifica una reunión con la FDA en la primera mitad de 2026 para discutir un diseño propuesto de Fase 2 y anticipa iniciar el ensayo de Fase 2 en la segunda mitad de 2026. Palvella citó evidencias genéticas y estudios de casos publicados que respaldan las estatinas tópicas y destacó la potencia de la pitavastatina como base para el desarrollo. Una conferencia web se realizó el 5 de noviembre de 2025 para discutir el candidato.
Palvella Therapeutics (NASDAQ: PVLA)는 표피성 확산성 태양노화 포로커라토시스(DSAP)용 새로운 국소 후보물질 QTORIN™ 피타바스타틴을 발표했다. 미국에서 진단된 환자 수가 >50,000명으로 FDA 승인을 받은 치료제가 없는 희귀한 전유전적 피부질환이다.
회사 측은 2026년 상반기에 FDA 회의를 계획하고 제2상 설계 초안을 논의하고 2026년 하반기에 제2상 시험을 시작할 것으로 예상한다. Palvella는 유전적 증거와 발표된 사례 연구가 국소 스타틴의 근거를 뒷받침한다고 언급했고 피타바스타틴의 효능을 개발의 타당성으로 강조했다. 후보 물질을 논의하기 위한 웹캐스트 컨퍼런스 콜은 2025년 11월 5일에 개최됐다.
Palvella Therapeutics (NASDAQ: PVLA) a annoncé un nouveau candidat de produit topique, QTORIN™ pitavastatine, pour la porokératose actinique superficielle disséminée (DSAP), une maladie cutanée génétique rare et prémalignante sans thérapies approuvées par la FDA et estimée à plus de 50 000 patients diagnostiqués aux États-Unis.
L'entreprise prévoit une réunion avec la FDA dans la première moitié de 2026 pour discuter d'un design de phase 2 proposé et prévoit d'initier l'essai de phase 2 dans la deuxième moitié de 2026. Palvella a cité des preuves génétiques et des études de cas publiées soutenant les statines topiques et a mis en avant la puissance de la pitavastatine comme argument en faveur du développement. Une web conférence a eu lieu le 5 novembre 2025 pour discuter du candidat.
Palvella Therapeutics (NASDAQ: PVLA) kündigte einen neuen topischen Produktkandidaten QTORIN™ Pitavastatin für disseminierte superficielle aktinische Porokeratose (DSAP) an, eine seltene prämalignante genetische Hautkrankheit ohne FDA-zugelassene Therapien und schätzungsweise >50.000 diagnostizierte US‑Patienten.
Das Unternehmen plant eine FDA-Sitzung in der ersten Hälfte von 2026, um ein vorgeschlagenes Phase-2-Design zu besprechen, und rechnet damit, die Phase-2-Studie in der zweiten Hälfte von 2026 zu beginnen. Palvella verwies auf genetische Belege und veröffentlichte Fallstudien, die topische Statine unterstützen, und hob die Potenz von Pitavastatin als Begründung für die Entwicklung hervor. Eine Webcast-Konferenzschaltung wurde am 5. November 2025 abgehalten, um den Kandidaten zu erläutern.
Palvella Therapeutics (NASDAQ: PVLA) أعلنت عن مرشح منتج موضعي جديد، QTORIN™ بيتافاستاتين، لمرض البوروكيراتوس السطحي الانتشاري (DSAP)، وهو اضطراب جلدي وراثي نادر قبل الخبري مع عدم وجود علاجات معتمدة من FDA ويُقدَّر عدد المرضى الذين تم تشخيصهم في الولايات المتحدة بأكثر من 50,000.
تخطط الشركة لاجتماع مع FDA في النصف الأول من 2026 لمناقشة تصميم مقترح للمرحلة الثانية وتتوقع بدء تجربة المرحلة الثانية في النصف الثاني من 2026. وأشارت Palvella إلى الأدلة الوراثية ودراسات الحالة المنشورة التي تدعم الستاتينات الموضعية وأبرزت قوة بيتافاستاتين كحجة للتطوير. جرى بث عبر الويب لمكالمة مؤتمر في 5 نوفمبر 2025 لمناقشة المرشح.
- FDA meeting planned in first half of 2026
- Phase 2 trial anticipated to start in second half of 2026
- Targets an estimated >50,000 diagnosed U.S. DSAP patients
- Pathogenesis-directed approach targeting mevalonate pathway
- No completed clinical trials or reported efficacy data for QTORIN™ pitavastatin
- Regulatory progress depends on FDA meeting outcome before Phase 2 initiation
Insights
Palvella advances a novel topical pitavastatin into Phase 2 plans for DSAP; FDA meeting set for H1 2026 and trial start expected H2 2026.
The announcement describes a new topical candidate, QTORIN™ pitavastatin, aimed at treating disseminated superficial actinic porokeratosis (DSAP). It states DSAP arises from mutations in the mevalonate pathway and that no FDA-approved therapies exist for the estimated more than 50,000 diagnosed U.S. patients. The company plans an FDA meeting in
Key dependencies and risks are clearly procedural: regulatory feedback from the FDA, the Phase 2 design and execution, and the ability of a topical formulation to meaningfully engage the mevalonate pathway in affected skin. The release cites published off-label case studies and mechanistic rationale but provides no clinical data for this candidate, so clinical and regulatory outcomes remain unreported.
Watch for the scheduled FDA meeting in
DSAP is a premalignant, progressive disease characterized by numerous expanding lesions which significantly impact quality-of-life; no FDA-approved therapies exist for the estimated more than 50,000 diagnosed U.S. patients
QTORIN™ pitavastatin has the potential to be the first pathogenesis-directed therapy designed to directly inhibit the mevalonate pathway, the causal driver of DSAP, in the pathogenic skin tissue
Company plans to initiate a Phase 2 trial evaluating QTORIN™ pitavastatin for DSAP in the second half of 2026
Company to host webcast conference call today, November 5, 2025 at 8:30am ET
WAYNE, Pa., Nov. 05, 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 skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced a new product candidate, QTORIN™ pitavastatin, for the treatment of disseminated superficial actinic porokeratosis (DSAP). QTORIN™ pitavastatin was developed leveraging QTORIN™, the company's patented platform for reproducibly generating novel, topical product candidates for the targeted treatment of serious, rare skin diseases.
DSAP is a genetic skin disease that results from mutations in the mevalonate pathway, leading to the accumulation of toxic intermediates. Clinically, DSAP presents as persistent, often extensive lesions that enlarge and increase in size, number, and extent over time. These lesions cause chronic loss of skin integrity, which can severely impact quality-of-life. DSAP is premalignant, with potential transformation to squamous cell carcinoma, particularly in long-standing or widespread cases. Spontaneous regression is extremely rare, and no FDA-approved therapies currently exist for the estimated more than 50,000 diagnosed patients in the United States.
"QTORIN™ pitavastatin has the potential to be the first pathogenesis-directed therapy for the treatment of DSAP, a serious, rare skin disease which currently has no FDA-approved therapies,” said Wes Kaupinen, Founder and Chief Executive Officer. “Recent breakthrough scientific discoveries further characterizing the genetics and biology of DSAP, as well as published case studies on the use of off-label topical statins, provide strong scientific rationale for advancing the development of QTORIN™ pitavastatin. With its superior potency relative to other mevalonate pathway inhibitors, pitavastatin represents a next-generation statin ideally suited for QTORIN™ development in DSAP.”
Palvella plans to meet with the FDA in the first half of 2026 to discuss the proposed design of a Phase 2 clinical trial evaluating QTORIN™ pitavastatin in subjects with disseminated superficial actinic porokeratosis. Trial initiation is anticipated in the second half of 2026.
Webcast Conference Call Details
Palvella will host a conference call and live audiovisual webcast to discuss its new clinical candidate QTORIN™ pitavastatin at 8:30 a.m. ET today. The conference call format will be presentation only. To access the live webcast of the call with slides, please click here or visit the “Events & Presentations” section of Palvella’s website. A replay of the webcast will be available approximately 2 hours after the conclusion of the call and archived for 90 days under the “Events & Presentations” section of the Company's website at www.palvellatx.com.
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 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 skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™
QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and neither has 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 expectations regarding its programs, including QTORIN™ rapamycin and QTORIN™ pitavastatin, 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 and QTORIN™ pitavastatin; 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. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release.
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 is Palvella Therapeutics announcing about QTORIN™ pitavastatin (PVLA) on November 5, 2025?
When does PVLA plan to meet the FDA and start the Phase 2 trial for QTORIN™ pitavastatin?
How many U.S. patients could QTORIN™ pitavastatin potentially address (PVLA)?
What is the proposed mechanism of QTORIN™ pitavastatin for DSAP (PVLA)?
Are there any FDA‑approved therapies for DSAP and has QTORIN™ pitavastatin shown clinical efficacy (PVLA)?
Where can investors view Palvella's November 5, 2025 webcast about QTORIN™ pitavastatin (PVLA)?
