Palvella Therapeutics, Inc. has announced the dosing of the first patient in its Phase 3 SELVA clinical trial, evaluating QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs). This milestone marks a significant step toward potentially providing the first FDA-approved therapy for this rare and debilitating genetic disease.
The SELVA trial is a multicenter, single-arm, baseline-controlled study designed to assess the safety and efficacy of QTORIN™ rapamycin when administered topically once daily. The trial aims to enroll approximately 40 participants, ages six and older, across leading vascular anomaly centers in the U.S. The primary efficacy endpoint is the change from baseline in the overall microcystic LM Investigator Global Assessment (mLM-IGA) at week 24.
Clinical Significance of QTORIN™ Rapamycin
Microcystic LMs are characterized by malformed lymphatic vessels that protrude through the skin, leading to persistent lymph fluid drainage, recurrent infections, and cellulitis. These complications can necessitate urgent medical attention and hospitalization. According to Joyce M. Teng, M.D., Ph.D., Professor of Dermatology and Pediatrics at Stanford University School of Medicine and SELVA Principal Investigator, the disease's progression without spontaneous resolution results in significant morbidity, beginning in childhood and having lifelong impacts.
QTORIN™ rapamycin, a novel, patented 3.9% rapamycin anhydrous gel, aims to leverage the therapeutic benefits of rapamycin, a mammalian target of rapamycin (mTOR) inhibitor, while minimizing systemic exposure and potential adverse reactions associated with systemic therapy. The FDA has granted QTORIN™ rapamycin Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation for the treatment of microcystic LMs.
Addressing an Unmet Need
Currently, there are no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States. The disease is caused by dysregulation of the phosphatidylinositol 3-kinase (PI3K)/mTOR pathway, leading to the development of malformed lymphatic vessels. Palvella's QTORIN™ rapamycin represents a potential standard of care for these patients.
Palvella's Broader Pipeline
QTORIN™ rapamycin is the lead product candidate from Palvella’s QTORIN™ platform. It is also under development for the treatment of cutaneous venous malformations and other serious skin diseases driven by mTOR pathway overactivation. Palvella Therapeutics is focused on developing and commercializing novel therapies for rare genetic skin diseases with no FDA-approved treatments.
Wes Kaupinen, Founder and Chief Executive Officer of Palvella, stated, "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."
