Palvella Therapeutics (NASDAQ:PVLA) saw its stock jump 15% on Tuesday after announcing topline results from the Phase 3 SELVA clinical study evaluating QTORIN™ 3.9% rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations.
The SELVA trial met its primary efficacy objective, demonstrating a statistically significant improvement on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA). The reported mean change was +2.13 points with p<0.0001. The study also achieved statistical significance on the pre-specified key secondary endpoint, the blinded mLM Multi-Component Static Scale, recording a mean improvement of 3.36 points with p<0.0001.
The trial enrolled 51 participants aged 3 years and older at vascular anomaly centers across the United States, with 50 individuals initiating treatment. Among the 49 participants aged 6 and older who completed the 24-week efficacy evaluation period, 95% showed improvement on the mLM-IGA and 86% were rated by investigators as "Much Improved" or "Very Much Improved."
QTORIN rapamycin was reported to be well-tolerated in the study population, with no drug-related serious adverse events observed. Systemic rapamycin concentrations remained below 2 ng/mL at all measured timepoints for every participant. Of those who completed the 24-week efficacy assessment, 98% chose to continue treatment in the ongoing extension phase of the study.
Following these results, Palvella plans to file a New Drug Application with the U.S. Food and Drug Administration in the second half of 2026. The company stated there is potential for U.S. approval in the first half of 2027. If approved, QTORIN rapamycin would be the first FDA-approved therapy for microcystic lymphatic malformations, a condition the company estimates affects about 30,000 people in the United States.
The development program for QTORIN rapamycin has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA.
Key points
- Phase 3 SELVA trial met both its primary endpoint (mLM-IGA) and a pre-specified key secondary endpoint (mLM Multi-Component Static Scale) with p<0.0001 for each result - relevant to the biotech and pharmaceutical sectors.
- Study enrolled 51 participants (50 treated); among 49 participants aged 6+ who completed 24-week efficacy assessment, 95% improved on mLM-IGA and 86% were rated as "Much Improved" or "Very Much Improved" - pertinent to clinical development and medical devices/therapeutics markets.
- QTORIN rapamycin showed favorable tolerability with no drug-related serious adverse events and systemic rapamycin levels below 2 ng/mL at all timepoints; 98% of completers entered the extension period - important information for investors and healthcare stakeholders assessing safety and retention.
Risks and uncertainties
- The therapy still requires formal regulatory review - Palvella plans an NDA submission in the second half of 2026 with potential approval in the first half of 2027, so a regulatory decision is not yet finalized - impacting biotech investors and the regulatory landscape.
- The reported findings come from a 51-participant trial with 50 initiating treatment and 49 in the primary 24-week efficacy cohort; the trial size and duration reported may limit the scope of conclusions until additional data from the ongoing extension become available - relevant to clinical development assessments and market expectations.
- Longer-term safety and efficacy beyond the 24-week efficacy period are not detailed in the topline announcement; continued observation in the extension period will be necessary to further characterize durability and safety - important for clinicians, payers, and investors.