WAYNE, Pa. — (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 and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced the publication of a systematic review in Clinical and Experimental Dermatology which synthesizes available published clinical evidence on off-label statin use for cutaneous application in porokeratosis. The systematic review supports the scientific rationale and clinical potential for developing Palvella’s QTORIN™ pitavastatin for the treatment of disseminated superficial actinic porokeratosis (DSAP), currently anticipated to enter Phase 2 development in the second half of 2026.
“Porokeratosis is a serious, progressive genetic skin disease that carries a meaningful risk of malignant transformation, particularly in patients with DSAP,” said Maria Gnarra Buethe, MD, PhD, FAAD, Pediatric Dermatology Director at the University of California, Irvine, and Division Chief of Dermatology at Rady Children’s Hospital of Orange County. “Advances in our understanding of porokeratosis have identified mutations in the mevalonate pathway as central drivers of disease pathogenesis. While systemic statins have not demonstrated clinical benefit—likely due to limited skin bioavailability—this systematic review underscores the mechanistic rationale and emerging clinical evidence supporting topical application of statins in porokeratosis as a potential targeted therapeutic approach for a condition with no FDA-approved treatments.”
Clinical and Experimental Dermatology is a peer-reviewed journal published by Oxford University Press on behalf of the British Association of Dermatologists that provides clinically relevant research, reviews, case reports, and continuing professional development content to advance the understanding and management of skin disease worldwide. The publication, titled “Topical Statins in the Treatment of Porokeratosis: A Systematic Review,” reviews 24 studies comprising 95 patients with porokeratosis treated via topical application of statins originally developed for systemic use, including atorvastatin, fluvastatin, lovastatin, rosuvastatin, and simvastatin. Dr. Maria Gnarra Buethe, Lihi Atzmony Maoz, MD, an Assistant Professor at the Yale School of Medicine’s Department of Dermatology, and Jeff Martini, PhD, Chief Scientific Officer of Palvella, served as authors on the publication. Key findings from the review article include:
- Recent genetic studies have identified loss-of-function mutations in the mevalonate metabolic pathway as a major driver of porokeratosis pathogenesis, prompting real-world, off-label use of statins designed to inhibit HMG-CoA reductase, a key enzyme in the mevalonate pathway, in porokeratosis.
- The majority of patients in the systematic review experienced at least partial clinical benefit, with symptom relief and reductions in lesion size observed across a broad age range (2 to 85 years).
- The need for controlled clinical trials using standardized, optimized statin formulations and standardized endpoints to robustly evaluate safety and efficacy.
“There is a significant unmet need in porokeratosis, with no FDA-approved therapies and a high burden of disease characterized by numerous expanding lesions that do not spontaneously regress, which significantly impact quality-of-life,” said Jeff Martini, Ph.D., Chief Scientific Officer of Palvella. “This systematic review highlights a potentially important role for QTORIN™ pitavastatin in porokeratosis. It also further validates Palvella’s approach to addressing DSAP, the most common subtype of the disease, which affects an estimated more than 50,000 diagnosed patients in the U.S., with QTORIN™ pitavastatin, if approved.”
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 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, 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 topical 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.
Contact Information
Investors
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
[email protected]
Media
Marcy Nanus
Managing Partner, Trilon Advisors LLC
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