WAYNE, Pa. - Palvella Therapeutics Inc. (NASDAQ:PVLA), whose stock has gained nearly 19% year-to-date and currently trades at $14.25, announced today that results from its Phase 2 study of QTORIN™ 3.9% rapamycin anhydrous gel for treating microcystic lymphatic malformations (microcystic LMs) have been published in the Journal of Vascular Anomalies. The study showed that 100% of participants experienced significant improvement after a 12-week treatment period. According to InvestingPro data, analysts maintain a strong buy consensus with price targets ranging from $30 to $38.
The trial's findings, which evaluated the safety and efficacy of QTORIN™ rapamycin, indicated nominal statistical significance across several efficacy endpoints. Notably, clinician and patient global impression assessments, along with evaluations of individual clinical manifestations, demonstrated positive outcomes. The treatment was generally well-tolerated, with no reported serious adverse events related to the drug. InvestingPro analysis shows the company maintains a "GOOD" financial health score of 2.76, with a healthy current ratio of 2.14.
Microcystic LMs, a rare genetic disease characterized by malformed lymphatic vessels, lead to recurrent infections and chronic debilitation. Currently, over 30,000 patients in the United States are diagnosed with this condition, with no FDA-approved treatments available.
Palvella Therapeutics, a company specializing in rare genetic skin diseases, is now conducting a Phase 3 single-arm, baseline-controlled trial named SELVA, enrolling approximately 40 subjects. The U.S. Food and Drug Administration has granted QTORIN™ rapamycin Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation.
The ongoing SELVA trial aims to further assess the potential of QTORIN™ rapamycin, with topline data expected in Q1 2026. If successful, QTORIN™ rapamycin could become the first approved therapy for microcystic LMs in the U.S., potentially establishing a new standard of care.
This development is based on a press release statement from Palvella Therapeutics Inc. and does not constitute an endorsement of the company or its products. For deeper insights into PVLA's financial metrics and growth potential, including exclusive ProTips and detailed valuation analysis, visit InvestingPro.
In other recent news, Palvella Therapeutics and Pieris Pharmaceuticals (NASDAQ:PVLA) are making significant progress in their respective fields. Palvella is anticipating the results from the Phase 2 TOIVA trial, which is evaluating QTORIN rapamycin for cutaneous venous malformations (VMs), in late 2025. The company is also targeting microcystic lymphatic malformations (microcystic LMs) with Phase 3 data expected in early 2026. H.C. Wainwright has shown confidence in Palvella's potential, reaffirming a Buy rating on the company's stock.
Meanwhile, Pieris Pharmaceuticals has announced board changes and updates on its ongoing merger with Palvella Therapeutics. The merger is progressing with Palvella slated to become a wholly owned subsidiary of Pieris upon completion. Responding to demands for additional information regarding the merger, Pieris provided supplemental disclosures, including updated financial projections for Palvella through 2038.
These developments highlight the companies' commitment to advancing their respective medical innovations and strategic plans. While these are recent developments, investors are advised to keep a close eye on future updates from both Palvella Therapeutics and Pieris Pharmaceuticals.
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