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 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.
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™ 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 or cleared 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
[email protected]