Palvella Therapeutics, Inc. (PVLA) is up more than 25% in pre-market trading after the company reported positive topline results from its pivotal Phase 3 SELVA clinical study evaluating QTORIN 3.9% rapamycin anhydrous gel for microcystic lymphatic malformations.
Microcystic lymphatic malformations are rare, chronically debilitating genetic conditions driven by dysregulation of the PI3K/mTOR pathway. Patients often experience persistent lymph leakage, bleeding, recurrent infections, and functional impairment. With no FDA-approved therapies available, treatment options remain limited for the estimated 30,000 affected individuals in the U.S.
The SELVA study, a single-arm, baseline-controlled Phase 3 trial, evaluated once-daily QTORIN rapamycin in individuals aged three years and older. Of the 51 participants enrolled, 49 aged six and above formed the intent-to-treat population. The trial met its primary endpoint, demonstrating a statistically significant improvement on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA), with a mean change of +2.13.
The therapy also achieved statistical significance across its key secondary endpoint- the blinded mLM Multi-Component Static Scale- which improved by 3.36 points. All four additional secondary endpoints, including patient-reported global impression measures and clinician-rated severity scales, also reached statistical significance.
At Week 24, 95% of participants aged six and older who completed the efficacy evaluation period achieved at least a one-point improvement on the mLM-IGA, and 86% were rated as "Much Improved" or "Very Much Improved."
QTORIN rapamycin was well-tolerated, with no drug-related serious adverse events reported. Systemic rapamycin levels remained below 2 ng/mL at all timepoints. Of the 50 participants who initiated treatment, 88% completed the 24-week efficacy period, and 98% of eligible participants chose to continue into the ongoing extension study.
Based on these results, Palvella plans to submit an NDA to the U.S. FDA in the second half of 2026, positioning QTORIN rapamycin to potentially become the first FDA-approved therapy for microcystic LMs as early as 2027.
PVLA has traded between $18.23 and $114.69 over the past year. The stock closed Monday's session at $87.84, up 2.54%. In pre-market trading Tuesday, the stock is up 25% at $110.62.
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