Palvella Therapeutics, Inc. (NASDAQ:PVLA) on Monday shared topline results from its Phase 2 TOIVA study of QTORIN 3.9% rapamycin anhydrous gel (QTORIN rapamycin) for cutaneous venous malformations (cutaneous VMs).
Cutaneous venous malformations are common, benign, slow-flow vascular anomalies appearing as blue/purple skin spots or soft, compressible lumps, causing pain, swelling, bleeding, or disfigurement.
The Overall Cutaneous Venous Malformations Investigator Global Assessment (Overall cVM-IGA) is a seven-point, clinician-assessed, single-item efficacy endpoint measuring change in severity from baseline, with the numeric rating scale ranging from “Very Much Worse” (-3) to “Very Much Improved” (+3).
On The Overall cVM-IGA at Week 12:
- 73% of trial participants (11/15 participants) improved.
- 67% of trial participants (10/15 participants) were either “Much Improved” (+2) or “Very Much Improved” (+3)
- No trial participants (0/15 participants) were “Minimally Worse” (-1), “Much Worse” (-2), or “Very Much Worse” (-3)
- In the Phase 2 TOIVA study, QTORIN rapamycin was generally well tolerated, with the most common treatment-emergent adverse events being application-site reactions (erythema, 25%).
- All treatment-related adverse events were moderate or mild, with no unexpected adverse events reported.
Rapamycin levels were below the lower limit of quantification (2 ng/mL) in systemic circulation on a standard lab assay for all participants at all timepoints in the study.
In November, Palvella Therapeutics announced a new product candidate, QTORIN pitavastatin, for the treatment of disseminated superficial actinic porokeratosis.
QTORIN pitavastatin was developed leveraging QTORIN, the company’s patented platform for reproducibly generating novel, topical product candidates for the targeted treatment of rare skin diseases.
Palvella plans to meet with the FDA in the first half of 2026 to discuss the proposed design of a Phase 2 trial, which is expected to start in the second half of 2026.
Price Action: Palvella stock is up 10.35% to $108.79 as of Monday’s last check.
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