First Patient Dosed in Phase 3 Trial of Rapamycin for Microcystic LMs

Palvella Therapeutics, Inc. announced the first patient has been dosed in SELVA, a multicenter, Phase 3 clinical trial designed to evaluate the safety and efficacy of its 3.9% rapamycin anhydrous gel, QTORIN™ rapamycin, for the treatment of microcystic lymphatic malformations (LMs).

"Microcystic LMs result from genetic changes that lead to hyperactivation of the causative PI3K/mTOR pathway resulting in malformed lymphatic networks that protrude through the skin. This results in persistent external lymph fluid drainage, as well as secondary infections and cellulitis that may require urgent medical attention and hospitalization,” said Joyce M. Teng, MD, PhD, Professor of Dermatology and Pediatrics at Stanford University School of Medicine and SELVA Principal Investigator. "Because this serious disease is present at birth and progresses over time without regression, it can result in significant morbidity beginning in childhood and have a lifelong impact."

SELVA is a Phase 3, single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin administered topically once daily for the treatment of microcystic LMs. The primary efficacy endpoint in SELVA is the change from baseline in the overall microcystic LM Investigator Global Assessment (mLM-IGA) at week 24. The Phase 3 study is expected to enroll approximately 40 participants, ages six and older, at leading vascular anomaly centers across the U.S. The FDA has granted Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation. Additionally, Palvella was awarded up to $2.6 million from the FDA’s Office of Orphan Products Development to support the SELVA study.

"We are pleased to have dosed the first patient in our Phase 3 SELVA trial, an important milestone towards our objective of advancing QTORIN™ rapamycin to potential regulatory approvals and U.S. commercialization,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the US for the estimated more than 30,000 diagnosed patients suffering from microcystic LMs in the US."

QTORIN rapamycin is a novel, patented 3.9% rapamycin anhydrous gel that aims to harness the potential therapeutic benefits of rapamycin, a mammalian target of rapamycin (mTOR) inhibitor, while minimizing systemic exposure of rapamycin and potential adverse reactions associated with systemic therapy. QTORIN rapamycin is currently under development for the treatment of microcystic LMs, cutaneous venous malformations, and other serious, functionally debilitating skin diseases driven by the overactivation of the mTOR pathway.