WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) reported positive topline results from a mid-stage clinical trial that positions its lead drug candidate as a potential first approved treatment for a rare and debilitating genetic skin disorder.
The clinical-stage biopharmaceutical company said Monday that its Phase 2 TOIVA trial of QTORIN™ 3.9% rapamycin anhydrous gel met multiple pre-specified efficacy endpoints in patients with cutaneous venous malformations, a lifelong condition for which no FDA-approved therapies currently exist. The data showed measurable improvements across both clinician- and patient-reported outcomes after 12 weeks of treatment.
According to the company, 73 percent of trial participants demonstrated improvement on the Overall Cutaneous Venous Malformations Investigator Global Assessment, with 67 percent rated as “Much Improved” or “Very Much Improved.” No participants experienced worsening of disease severity during the study period. The trial enrolled 16 patients aged six and older, with data from 15 participants included in the primary analysis.
Palvella said QTORIN™ rapamycin was generally well tolerated, with no drug-related serious adverse events reported. The most common treatment-related side effect was mild to moderate application site irritation. Systemic exposure to rapamycin remained below detectable levels throughout the trial, an outcome the company views as supportive of the therapy’s localized, topical design.
Cutaneous venous malformations are caused by genetic mutations that lead to abnormal vein development in the skin, often resulting in pain, bleeding, functional impairment, and long-term quality-of-life challenges. Palvella estimates more than 75,000 individuals in the United States are affected by the condition.
Based on the Phase 2 results, the company said it plans to engage with federal regulators to discuss next steps, including a potential late-stage pivotal study. The FDA has previously granted Fast Track Designation to QTORIN™ rapamycin for venous malformations.
Palvella, founded by veterans of rare disease drug development, is building a pipeline around its proprietary QTORIN™ platform, with an emphasis on serious dermatologic conditions lacking approved treatment options. The company’s lead program is also being evaluated in other vascular and skin disorders.
QTORIN™ rapamycin remains an investigational therapy and has not been approved by the FDA for any indication.
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