WAYNE, PA — Palvella Therapeutics (Nasdaq: PVLA) reported new late-stage clinical data showing strong patient response rates for its experimental QTORIN rapamycin treatment in rare vascular malformations, positioning the company to pursue what could become the first FDA-approved therapy for microcystic lymphatic malformations.
The data, presented at the International Society for the Study of Vascular Anomalies World Congress in Philadelphia, included results from the Phase 3 SELVA study and Phase 2 TOIVA study evaluating topical QTORIN rapamycin in patients with microcystic lymphatic malformations and cutaneous venous malformations.
In the Phase 3 SELVA trial, all 13 participants between ages 6 and 11 were rated “Much Improved” or “Very Much Improved” after 24 weeks of treatment under the Microcystic Lymphatic Malformation Investigator Global Assessment scale.
Among participants entering the study with moderate or worse leaking or bleeding symptoms, 87% demonstrated similar improvement ratings at Week 24.
Palvella also reported that all 43 patients completing the efficacy evaluation period expressed at least some satisfaction with treatment on the TSQM-9 satisfaction assessment, while 84% reported being satisfied, very satisfied, or extremely satisfied.
The company indicated blinded independent review showed little measurable disease change during the study’s pre-treatment run-in period, followed by marked improvement after treatment began, supporting the trial’s single-arm design.
QTORIN rapamycin targets the mTOR signaling pathway, which researchers increasingly associate with lymphatic and venous malformation disorders. No FDA-approved therapies currently exist for either indication.
Chief Executive Officer Wes Kaupinen stated the company remains on track to submit a New Drug Application to the FDA during the second half of 2026 for treatment of microcystic lymphatic malformations, with potential approval targeted for the first half of 2027.
Additional data from the Phase 2 TOIVA study showed statistically significant improvement in lesion height and appearance scores among patients with cutaneous venous malformations.
At Week 24, patients in the TOIVA study demonstrated mean reductions of 1.50 points in lesion height scores and 1.43 points in appearance scores, according to the company.
Palvella plans to initiate a Phase 3 trial for cutaneous venous malformations during the second half of 2026.
QTORIN rapamycin has received FDA Breakthrough Therapy, Fast Track, and Orphan Drug designations for microcystic lymphatic malformations. The therapy has also received Fast Track designation for cutaneous venous malformations.
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