QTORIN Rapamycin Gel Demonstrates Clinical Efficacy in Phase 2 Venous Malformation Study

Palvella Therapeutics, Inc. (PVLA) unveiled encouraging findings from its Phase 2 TOIVA study evaluating QTORIN 3.9% rapamycin anhydrous gel as a treatment option for cutaneous venous malformations—congenital skin lesions that worsen progressively throughout a patient’s lifetime. The topical formulation was applied once daily over a 12-week treatment phase, with an additional 12-week monitoring extension.

Clinical Efficacy and Safety Profile

The trial demonstrated substantial therapeutic benefit across key measurement criteria. Among study participants, 73% achieved measurable improvement based on the Overall Cutaneous Venous Malformations Investigator Global Assessment (cVM-IGA) scale at the Week 12 checkpoint. Furthermore, two-thirds of patients (67%) received ratings of Much Improved or Very Much Improved, indicating clinically meaningful responses. Palvella reported that efficacy endpoints achieved statistical significance across multiple parameters, encompassing both measures of dynamic improvement and static lesion severity assessment.

Importantly, QTORIN rapamycin demonstrated a favorable safety profile, with no drug-related serious adverse events documented during the study period. This safety record strengthens the candidate’s profile for continued development.

Regulatory Path Forward and Market Response

Following these Phase 2 successes, Palvella intends to engage with the U.S. Food and Drug Administration regarding a potential Breakthrough Therapy Designation and the design of a Phase 3 pivotal trial. The FDA previously granted Fast Track Designation status to QTORIN rapamycin, recognizing the unmet medical need in venous malformation treatment.

Market participants responded positively to the clinical milestone. PVLA shares gained 3% in pre-market trading on the announcement, building on Friday’s 1.86% advance that brought the stock to $98.58 at close. The dual regulatory designation status positions QTORIN rapamycin for expedited development toward potential commercial approval.