WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) announced the presentation of its investigational therapy QTORIN™ rapamycin 3.9% anhydrous gel for microcystic lymphatic malformations (microcystic LMs) at the 15th World Congress of Pediatric Dermatology in Buenos Aires, Argentina. Dr. Amy Paller, Chair of Dermatology at Northwestern University’s Feinberg School of Medicine, delivered the presentation highlighting the promising potential of the therapy.
“There is an urgent need for a safe and effective targeted topical therapy for mosaic genetic skin disorders, including microcystic lymphatic malformation,” said Dr. Paller. “Many patients have considerable complications associated with this disease, and I am looking forward to the Phase 3 results early next year.”
The rare, pediatric-onset condition is characterized by congenital mosaic lesions that grow progressively, leading to complications and requiring early intervention. Designed to inhibit the mammalian target of rapamycin (mTOR) signaling pathway, QTORIN™ rapamycin aims to address the hyper-proliferative endothelial cells underpinning microcystic LMs.
Palvella’s ongoing Phase 3 SELVA study builds on earlier results from a 12-week Phase 2 trial, where all participants were rated as either “Very Much Improved” (41.7%) or “Much Improved” (58.3%) based on clinician assessment. The Phase 3 single-arm trial has been expanded to include patients aged three years and older, extending the treatment period to 24 weeks, with a target enrollment of 40 participants across 13 U.S. centers.
The U.S. Food and Drug Administration has granted QTORIN™ rapamycin Breakthrough Therapy, Fast Track, and Orphan Drug designations, along with a $2.6 million Orphan Products Grant to support the SELVA study. Top-line data is expected in early 2026.
“Microcystic LMs are a serious, rare, and chronically debilitating genetic disease with a pediatric onset and lifelong course,” said Wes Kaupinen, Founder and CEO of Palvella Therapeutics. “Early intervention is essential, and we are pleased to include younger patients in the SELVA study.”
The presentation has added momentum to Palvella’s aim of delivering a much-needed therapy for this underserved patient population.
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