Poster #76954 highlights QTORIN™ rapamycin’s single phase anhydrous gel formulation designed to optimize dermal bioavailability of rapamycin for mTOR-driven skin diseases while overcoming the crystallization, stability, and dermal penetration challenges posed by rapamycin

Poster #76929 highlights a qualitative patient and caregiver interview study evaluating the burden of living with porokeratosis, a serious, rare genetic skin disease characterized by numerous pre-cancerous, pruritic lesions and substantial functional and psychosocial burden

WAYNE, Pa., March 27, 2026 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. , (“Palvella” or “the Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced two poster presentations at the 2026 American Academy of Dermatology Annual Meeting to be held March 27-31 in Denver, Colorado.

“The data presented in these posters highlight the innovative capabilities of the QTORIN™ platform as well as Palvella’s unwavering commitment to incorporating the voice of the patient in our development programs,” said Dr. Jeff Martini, Chief Scientific Officer of Palvella Therapeutics. “With patients in mind, we developed QTORIN™ rapamycin as a single-phase anhydrous gel designed to prevent crystallization and degradation of rapamycin with the goal of ensuring rapamycin bioavailability in the dermis, the site of disease pathology for many mTOR-driven skin diseases. Furthermore, our porokeratosis qualitative burden-of-living study, incorporating both patient and caregiver perspectives, confirms that porokeratosis is a life-altering condition with no FDA-approved therapies, imposing substantial physical, psychosocial, and cancer-related burdens that affect daily life in profound ways.”

The details of the poster presentations are as follows:

Poster #76954: QTORIN™ Rapamycin: Advancing Topical Formulation Science for Rare Dermatologic Diseases

QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) was developed over many years by Palvella and an international team of leading topical formulation scientists. This work addressed rapamycin’s crystallization, stability, and skin penetration challenges, which have historically limited its use in skin diseases despite the identification of multiple mTOR pathway-driven conditions.

  • QTORIN™ rapamycin is being developed under an active FDA-regulated Investigational New Drug application, with rigorous clinical, nonclinical, and chemistry, manufacturing, and controls evaluation, as well as manufacturing in accordance with current Good Manufacturing Practice (GMP) standards.
  • Compounded rapamycin formulations do not undergo the FDA drug approval process and typically are not manufactured under GMP standards, which can contribute to poor solubility, instability, inconsistent drug delivery, and potential patient safety concerns.
  • Microscopic evaluation of compounded rapamycin formulations revealed drug crystallization, indicating rapamycin was bound in crystals and therefore less available for skin penetration; notably, all compounded formulations tested demonstrated rapid chemical degradation when assayed for drug product stability.

Supporting QTORIN™ rapamycin’s potential in serious, rare skin diseases and vascular malformations, the Phase 3 SELVA study of QTORIN™ rapamycin in microcystic lymphatic malformations met its primary endpoint with statistically significant improvement on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA) (p<0.001), as well as its key secondary endpoint and all four additional secondary endpoints (all p<0.001). QTORIN™ rapamycin was well tolerated with systemic levels of rapamycin below 2ng/mL at all timepoints measured.

Palvella is currently advancing QTORIN™ rapamycin across multiple serious, rare skin diseases and vascular malformations for which there are no FDA-approved therapies, including microcystic lymphatic malformations, cutaneous venous malformations, clinically significant angiokeratomas, and a fourth target clinical indication which the Company anticipates announcing in the second half of 2026.

Poster #76929: The Burden of Living with Porokeratosis: Patient and Caregiver Perspectives on Serious Rare Diseases

  • Porokeratosis is a serious, rare genetic skin disease with no FDA-approved therapies. It is driven by mutations in the mevalonate pathway and is characterized by persistent, pre-cancerous, pruritic lesions that expand over time. According to a 2021 study published in Cureus by Novice et al., these lesions have been reported to carry malignant transformation rates of up to 16%.
  • To better understand the burden of this rare, pre-cancerous disease, ten in-depth qualitative interviews focused on physical symptoms, daily function, psychosocial impact, cancer impact, and treatment challenges were conducted with individuals living with porokeratosis (n=9) and a caregiver (n=1).
  • Structured interviews highlighted a wide range of functional and psychosocial experiences caused by porokeratosis, including:
    • The persistent physical burden on daily life, characterized by widespread lesions with common signs and symptoms such as redness, scaling, and itch.
    • Diagnoses of skin cancer resulting from disease progression and transformation of the lesions from pre-cancerous to malignant.
    • Limitations to daily life, including avoiding pools, gyms, and other social environments, contributing to feelings of social isolation, frustration, and challenges in intimate and personal relationships.
    • Reduced exercise and mobility, with some reporting episodes of severe pain that limited routine activities.
    • Significant psychosocial burden, including awareness of the associated skin cancer risk causing anxiety, self-consciousness, and embarrassment about visible lesions.
  • The results support the urgency of developing a pathogenesis-directed therapy for the treatment of porokeratosis.

Palvella is developing QTORIN™ pitavastatin for disseminated superficial actinic porokeratosis (DSAP), with a Phase 2 trial anticipated to begin in the second half of 2026. No FDA-approved therapies currently exist for the estimated more than 50,000 diagnosed U.S. patients. Additional information on Palvella's DSAP program can be found here .

About Palvella Therapeutics

Founded and led by rare disease biotech 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 and vascular malformations 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 and vascular malformations, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being developed for the treatment of microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas. Palvella’s second product candidate, QTORIN™ pitavastatin, is currently being developed for the treatment of disseminated superficial actinic porokeratosis. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter).

QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and neither has been approved 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 clinical trials, Palvella’s clinical development plans and related anticipated development milestones, Palvella’s plans to pursue Breakthrough Therapy Designation, Palvella’s plans to meet with regulatory authorities, Palvella’s cash, financial resources and expected runway, 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
[email protected]

Media
Marcy Nanus
Managing Partner, Trilon Advisors LLC
[email protected]