Palvella’s QTORIN Rapamycin Delivers +2.5-Point Bleeding Score Gain in Phase 2 Trial
Palvella Therapeutics reported that in its Phase 2 TOIVA trial, 100% of patients with bleeding at baseline (n=4) demonstrated a +2.5-point improvement on the cVM-IGA Bleeding scale at Week 12 (p=0.003). All such patients reported being “satisfied” or “very satisfied” with QTORIN rapamycin, highlighting strong patient-reported outcomes.
1. Phase 2 TOIVA Trial Results
QTORIN rapamycin was administered topically once daily for 12 weeks in a single-arm, open-label Phase 2 trial of cutaneous venous malformations. All four patients with baseline bleeding achieved statistically significant improvements on the cVM-IGA Bleeding scale at Week 12, with a mean +2.5-point gain (p=0.003).
2. Patient-Reported Outcomes
By Week 12, 100% of bleeding patients reported being “satisfied” or “very satisfied” with treatment. Baseline qualitative interviews underscored the substantial physical, functional, and psychosocial burden of cutaneous venous malformations, including pain, discoloration, swelling and social limitations.
3. Next Development Steps
These results reinforce QTORIN’s potential to become the first FDA-approved therapy for cutaneous venous malformations affecting over 75,000 U.S. patients. Palvella plans to advance to pivotal studies and engage with regulators on approval pathways based on these Phase 2 findings.