Palvella expands QTORIN rapamycin development to angiokeratomas

Palvella Therapeutics Inc. (NASDAQ: PVLA) announced it will expand development of its QTORIN 3.9% rapamycin anhydrous gel into clinically significant angiokeratomas, a rare lymphatic disease affecting an estimated 50,000 diagnosed patients in the United States.

The company plans to initiate a Phase 2 trial in the second half of 2026 to evaluate the treatment for clinically significant angiokeratomas, which are characterized by lymphatic-derived skin lesions that can cause persistent bleeding and impact quality of life. Currently, no FDA-approved therapies exist for this condition.

Palvella will meet with the FDA in the first half of 2026 to discuss the proposed Phase 2 study design, which is expected to include approximately 10-20 patients. The International Society for the Study of Vascular Anomalies classified angiokeratomas as an isolated lymphatic malformation in 2025.

This represents the third target indication for QTORIN rapamycin. The treatment is currently being evaluated in a Phase 3 trial called SELVA for microcystic lymphatic malformations, with top-line results expected in the first quarter of 2026. It is also being studied in a Phase 2 trial called TOIVA for cutaneous venous malformations, with results anticipated in mid-December 2025.

"Clinically significant angiokeratomas represent our third target clinical indication for QTORIN rapamycin and align with Palvella's strategy of selecting serious, rare skin diseases in which Palvella has the opportunity to introduce the first FDA-approved therapy," said Wes Kaupinen, founder and chief executive officer of Palvella.

The company hosted a webcast conference call to discuss the development expansion.

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