Palvella Therapeutics completes TOIVA Phase 2 trial recruitment target

Palvella Therapeutics announced the successful completion of the TOIVA Phase 2 trial, enrolling 16 subjects with venous malformations. The trial met its recruitment target, and top-line data is expected in mid-December 2025. QTORIN™ rapamycin has the potential to be the first approved therapy in the US for approximately 75,000 patients with cutaneous VMs, a common type of vascular malformation.

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, which aimed to enroll approximately 15 participants, successfully recruited 16 subjects at leading vascular anomaly centers, meeting its target 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].

Palvella's successful enrollment of 16 subjects in its 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. The trial addresses cutaneous venous malformations (VMs), the most common type of vascular malformation, for which there are currently no FDA-approved therapies. This positions Palvella to potentially capture an untapped market of approximately 75,000 patients in the U.S. alone Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial of QTORIN™ Rapamycin for Cutaneous Venous Malformations^[1].

From a mechanistic perspective, QTORIN™ rapamycin targets the underlying disease pathophysiology - mutations causing overactivation of the PI3K/mTOR pathway. The novel 3.9% rapamycin anhydrous gel formulation aims to deliver the therapeutic benefits of rapamycin (an established mTOR inhibitor) while minimizing systemic exposure and related adverse effects. The trial design is appropriately structured for a Phase 2 proof-of-concept study, with multiple efficacy endpoints including clinician and patient global impression assessments alongside evaluation of specific disease manifestations. These endpoints will provide comprehensive data to inform potential pivotal trials 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].