WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) has announced that the first group of patients has been dosed in a pivotal Phase 2 clinical trial evaluating its QTORIN™ 3.9% rapamycin gel. This trial aims to assess the safety and effectiveness of the topical treatment for cutaneous venous malformations (cutaneous VMs), an uncommon and serious genetic condition affecting the skin.
This single-arm, baseline-controlled study, known as TOIVA, will enroll 15 patients at prominent vascular anomaly centers across the United States. The trial will evaluate the treatment’s safety alongside its ability to reduce the burden associated with the disease, which impacts over 75,000 diagnosed patients in the United States.
Cutaneous VMs are caused by genetic mutations that overstimulate the PI3K/mTOR signaling pathway, leading to malformed and dysfunctional veins in the skin. Patients often endure complications such as severe pain, bleeding, ulceration, and clotting, significantly affecting their daily lives. There are currently no FDA-approved therapies to treat this condition.
QTORIN™ rapamycin is a topical gel designed to target the underlying drivers of cutaneous VMs by inhibiting the mTOR pathway. Unlike the off-label systemic mTOR inhibitors used in some cases, the QTORIN formulation minimizes systemic exposure and the potential side effects associated with broader treatments.
“Cutaneous VMs are a serious, lifelong disease which leads to significant disease burden for children and adults,” said Dr. Megha M. Tollefson, Medical Director of the Mayo Clinic Vascular Malformation Clinic. She added that the TOIVA study represents a significant step forward, bringing hope for a focused, FDA-approved therapy to patients who currently rely on limited options.
The trial will monitor safety through reports of adverse events and effectiveness through a variety of measures, including clinical assessments and patient reports. Participants six years and older will apply the gel daily for 12 weeks to determine its impact on the disease’s challenging physical manifestations.
QTORIN™ rapamycin received FDA Fast Track Designation in 2024, signaling its potential to address a high-priority unmet medical need. If successful, the gel could become the first FDA-approved treatment for cutaneous VMs, offering a targeted solution for patients where none currently exists.
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