Palvella Completes Phase 2 Trial for QTORIN Rapamycin in Cutaneous Venous Malformations

Palvella Therapeutics has completed enrollment in its Phase 2 TOIVA trial of QTORIN™ rapamycin for cutaneous venous malformations. The trial has successfully met its recruitment target of 16 subjects at leading vascular anomaly centers. Top-line data is expected in mid-December 2025. Cutaneous VMs are a rare genetic disease with no FDA-approved therapies, impacting an estimated 50-80% of patients with bleeding, thrombosis, ulceration, disfigurement, and proliferation. QTORIN™ rapamycin has the potential to be the first approved therapy in the U.S. for more than 75,000 U.S. patients with cutaneous VMs.

Palvella Therapeutics (NASDAQ: PVLA) has successfully completed enrollment in its Phase 2 TOIVA trial for QTORIN™ rapamycin, a potential first-in-class treatment for cutaneous venous malformations (VMs). The trial enrolled 16 subjects at leading vascular anomaly centers, meeting its target of approximately 15 participants Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial of QTORIN™ Rapamycin for Cutaneous Venous Malformations^[1].

QTORIN™ rapamycin is a novel 3.9% rapamycin anhydrous gel designed to treat VMs, which affect an estimated 75,000+ U.S. patients. The drug aims to inhibit mTOR signaling while minimizing systemic exposure. The single-arm, open-label study will evaluate safety, tolerability, and efficacy through multiple measures, with top-line results expected in mid-December 2025 Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial of QTORIN™ Rapamycin for Cutaneous Venous Malformations^[1].

Cutaneous VMs are a serious, rare genetic disease caused by mutations in genes that cause overactivation of the PI3K/mTOR signaling pathway, leading to dysfunctional veins within the skin. The Phase 2 TOIVA study is a single-arm, open-label, baseline-controlled clinical trial of QTORIN™ rapamycin, which aims to harness the potential therapeutic benefits of rapamycin, an mTOR inhibitor, while minimizing systemic exposure and potential adverse reactions associated with systemic therapy Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial of QTORIN™ Rapamycin for Cutaneous Venous Malformations^[1].

Palvella's successful enrollment of 16 subjects in their Phase 2 TOIVA trial represents a significant operational milestone for the company's lead candidate, QTORIN™ rapamycin. This achievement keeps the company on track with their clinical timeline, with top-line data expected by mid-December 2025 Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial of QTORIN™ Rapamycin for Cutaneous Venous Malformations^[1].

While enrollment completion is positive, investors should recognize that Phase 2 represents mid-stage development, with significant regulatory hurdles still ahead. The upcoming data readout in December 2025 will be the true value inflection point, determining whether QTORIN™ rapamycin demonstrates sufficient safety and efficacy to advance to Phase 3 Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial of QTORIN™ Rapamycin for Cutaneous Venous Malformations^[1].

Venous malformations are the most common type of vascular malformation, with skin involvement impacting an estimated approximately 50-80% of patients, which can result in bleeding, thrombosis, ulceration, disfigurement, and proliferation Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial of QTORIN™ Rapamycin for Cutaneous Venous Malformations^[1]. QTORIN™ rapamycin has the potential to be the first approved therapy in the U.S. for more than an estimated 75,000 U.S. patients with cutaneous VMs Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial of QTORIN™ Rapamycin for Cutaneous Venous Malformations^[1].