Palvella Therapeutics (NASDAQ:PVLA) reported encouraging topline results from its Phase 2 TOIVA trial evaluating QTORIN™ 3.9% rapamycin gel for the treatment of cutaneous venous malformations (cVM), a chronic and disfiguring vascular condition with limited therapeutic options.
At Week 12, 73% of patients (11 of 15) achieved improvement on the Overall cVM–Investigator Global Assessment (cVM-IGA), while 67% (10 of 15) were rated by investigators as “Much Improved” or “Very Much Improved.”
Multiple secondary endpoints captured through both clinician and patient assessments reached nominal statistical significance, underscoring the consistency of benefit observed in the trial.
QTORIN was reported to be generally well tolerated, with no drug-related serious adverse events, an important consideration for a chronic-use topical therapy.
Systemic rapamycin exposure remained below quantifiable limits, reinforcing QTORIN’s targeted dermal delivery profile.
Palvella said it intends to engage the FDA in the near term regarding Breakthrough Therapy designation and to discuss plans for a pivotal Phase 3 study.
The FDA has already granted Fast Track designation to QTORIN for cVM, reflecting the significant unmet medical need and the potential for a first-of-its-kind approved therapy.
If successful in Phase 3, QTORIN could become the first FDA-approved pharmacologic treatment for cutaneous venous malformations, positioning Palvella to move into late-stage development with strengthened clinical rationale.