Palvella Therapeutics (NASDAQ: PVLA) presented new clinical data from its Phase 3 SELVA and Phase 2 TOIVA studies at the International Society for the Study of Vascular Anomalies World Congress in Philadelphia. The data supports QTORIN rapamycin as a potential treatment for microcystic lymphatic malformations and cutaneous venous malformations.

In the SELVA Phase 3 study, all 13 participants aged 6-11 years showed improvement on the Microcystic Lymphatic Malformation Investigator Global Assessment scale at Week 24, with a mean improvement of +2.46 (p<0.001). Among participants with moderate or worse leaking/bleeding at baseline, 87% (20/23) showed improvement on the leaking/bleeding assessment, with a mean improvement of +2.48 (p<0.001).

All 43 participants who completed the efficacy evaluation period reported satisfaction with QTORIN rapamycin on the Treatment Satisfaction Questionnaire for Medication, with 84% reporting high levels of satisfaction. A blinded independent review demonstrated stability during the 8-week run-in period followed by improvement during treatment.

The TOIVA Phase 2 study for cutaneous venous malformations demonstrated statistically significant improvements in both height and appearance measures at all time points. At Week 24, the treatment showed a mean reduction of 1.50 points in height score and 1.43 points in appearance score (both p<0.001).

Palvella plans to submit a New Drug Application to the FDA for QTORIN rapamycin for microcystic lymphatic malformations in the second half of 2026. The company also plans to initiate a Phase 3 trial for cutaneous venous malformations in the second half of 2026. QTORIN rapamycin has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for microcystic lymphatic malformations.