WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) has announced positive outcomes from a Phase 2 study evaluating QTORIN™ 3.9% rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations (microcystic LMs). The findings were published in the Journal of Vascular Anomalies (JoVA), a peer-reviewed publication dedicated to vascular anomaly research.
The study results demonstrated substantial improvements, with 100% of the participants receiving ratings of “Much Improved” or “Very Much Improved” on the Clinician Global Impression of Change after 12 weeks of treatment. QTORIN™ rapamycin, which has previously received Breakthrough Therapy, Fast Track, and Orphan Drug Designations from the FDA, has the potential to become the first approved therapy for this rare and debilitating disease in the United States.
Microcystic LMs are a genetic condition caused by disruptions in the PI3K/mTOR pathway, leading to malformed lymphatic vessels that can cause persistent leakage, infections, and chronic complications. The disease affects over 30,000 diagnosed individuals in the U.S., with no current FDA-approved treatments.
The Phase 2 study results showed statistically significant improvements in key efficacy measures, such as clinician and patient impressions of change, as well as reductions in clinical manifestations of the disease. The treatment was well-tolerated, with no serious adverse events linked to the drug.
Building on this success, Palvella is currently conducting a Phase 3 clinical trial, dubbed SELVA, which will evaluate the safety and efficacy of the therapy in approximately 40 participants over 24 weeks. Topline data from the study is expected in the first quarter of 2026. This trial is supported by an Orphan Products Grant from the FDA.
“This therapy represents an important step forward for individuals living with microcystic lymphatic malformations,” said Wes Kaupinen, Founder and CEO of Palvella Therapeutics. “We are committed to advancing the ongoing Phase 3 SELVA trial and delivering this breakthrough treatment to patients as soon as possible.”
If successful, QTORIN™ rapamycin could set a new standard of care for microcystic LMs, addressing a critical unmet need and markedly improving the lives of those affected by this chronic disease.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and MSN.