1. 73% of trial participants showed improvement in cutaneous venous malformations. 2. QTORIN™ rapamycin achieved statistical significance in multiple efficacy endpoints. 3. Well-tolerated with no serious drug-related adverse events reported. 4. Palvella plans discussions with FDA for Breakthrough Therapy Designation. 5. Potential to be first FDA-approved treatment for 75,000 U.S. patients.
FAQ
Why Very Bullish?
Positive results bolster confidence in QTORIN™'s viability; FDA discussions enhance market outlook.
How important is it?
The article outlines significant clinical progress and regulatory intent, directly impacting PVLA's future.
Why Short Term?
Upcoming FDA discussions and potential Breakthrough Designation could quickly influence stock price.
Palvella Therapeutics Reports Positive Phase 2 Results for QTORIN™ Rapamycin in Treating Cutaneous Venous Malformations
WAYNE, Pa., Dec. 15, 2025 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. (Nasdaq: PVLA), a clinical-stage biopharmaceutical company dedicated to developing therapies for serious, rare skin diseases, has announced promising topline results from its Phase 2 TOIVA clinical trial. This study evaluated the effectiveness of QTORIN™ 3.9% rapamycin anhydrous gel for treating cutaneous venous malformations (cutaneous VMs), a condition that currently lacks FDA-approved therapies.
Key Findings from the TOIVA Study
The TOIVA trial demonstrated significant improvements among participants, with the following notable outcomes:
- 73% of trial participants (11 out of 15) exhibited improvement in the Overall Cutaneous Venous Malformations Investigator Global Assessment (Overall cVM-IGA) at Week 12.
- 67% rated their condition as “Much Improved” or “Very Much Improved” at Week 12.
- Statistical significance was achieved on multiple clinician-reported and patient-reported efficacy endpoints.
- QTORIN™ rapamycin was well-tolerated, with no reported serious adverse events related to the drug.
Objectives for Future Development
Following these results, Palvella plans to engage in discussions with the FDA regarding the possibility of obtaining Breakthrough Therapy Designation and initiating a Phase 3 pivotal study. The FDA has already granted Fast Track Designation for QTORIN™ rapamycin as a treatment for venous malformations.
Wes Kaupinen, Founder and Chief Executive Officer of Palvella, remarked that these findings highlight QTORIN™ rapamycin's potential to become the first FDA-approved therapy and the standard of care for the over 75,000 individuals in the U.S. suffering from cutaneous venous malformations.
Details of the Study Design
The TOIVA study is a Phase 2, single-arm, open-label trial involving a 12-week evaluation of QTORIN™ rapamycin. Sixteen participants, aged six and above, were enrolled at various leading vascular anomaly centers across the U.S. The study aimed to assess treatment efficacy through clinician and patient global impression assessments, as well as specific clinical manifestations contributing to the disease burden.
Key efficacy endpoints evaluated at Week 12 included:
- Overall cVM-IGA: Mean Change from Baseline: 1.5, p-value <0.001
- cVM-IGA Height/Engorgement: Mean Change from Baseline: 1.3, p-value <0.001
- cVM-IGA Appearance: Mean Change from Baseline: 1.5, p-value <0.001
These results indicate a clear improvement in both dynamic and static severity scales, reaffirming the clinical promise of QTORIN™ rapamycin.
Expert Insights and Future Directions
Dr. Megha Tollefson, pediatric dermatologist at Mayo Clinic and Principal Investigator of the study, emphasized the substantial impact of cutaneous venous malformations on patients’ quality of life. Current treatment options can often be painful and ineffective. She stated, “The Phase 2 data represent a milestone day for individuals living with cutaneous venous malformations. We look forward to working with Palvella to advance the development of this therapy for patients as quickly as possible.”
Data collection continues from participants for further analysis, with genotype-stratified results anticipated for future presentations. The company aims to establish a strong regulatory pathway based on the results of this pivotal study.
Conclusion
Palvella Therapeutics' strong Phase 2 results for QTORIN™ rapamycin bolster its potential as a leading treatment option for cutaneous venous malformations. The company is gearing up for potential conversations with the FDA, marking a significant step towards improving care for affected individuals.