Palvella Therapeutics secures new patent for rare skin disease gel

Published 22/04/2025, 12:38
Palvella Therapeutics secures new patent for rare skin disease gel

WAYNE, Pa. - Palvella Therapeutics Inc. (NASDAQ:PVLA), a clinical-stage biopharmaceutical company with a market capitalization of $280 million and an impressive 98% return over the past six months, announced today the issuance of a new U.S. patent for its leading product candidate QTORIN™ 3.9% rapamycin anhydrous gel, potentially the first therapy for microcystic lymphatic malformations (microcystic LMs) in the U.S. According to InvestingPro data, the company maintains strong financial health with a current ratio of 7.33, indicating robust short-term liquidity. The United States Patent and Trademark Office granted patent No. 12,268,673, extending the company’s intellectual property protection for QTORIN™ rapamycin into 2038.

Microcystic LMs are characterized by malformed lymphatic vessels that can lead to serious infections and lifelong morbidity, affecting over 30,000 patients in the U.S. with no current FDA-approved treatments. QTORIN™ rapamycin has received Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation by the U.S. FDA for the treatment of this condition.

The new patent, titled "Anhydrous Compositions of mTOR Inhibitors and Methods of Use," broadens the scope of protection for QTORIN™ rapamycin, which is based on Palvella’s patented QTORIN™ platform. This platform is focused on developing therapies for rare genetic skin diseases, many of which are lifelong conditions.

Palvella’s lead product candidate is currently under evaluation in the Phase 3 SELVA clinical trial for microcystic LMs and the Phase 2 TOIVA clinical trial for cutaneous venous malformations. If approved, QTORIN™ rapamycin is anticipated to be eligible for seven years of orphan drug market exclusivity in the U.S.

Wes Kaupinen, Founder and CEO of Palvella, stated that the fifth U.S. patent reflects significant innovation in the development of QTORIN™ rapamycin and strengthens their intellectual property portfolio.

The company, known for its focus on treating serious, rare genetic skin diseases, is advancing a broad pipeline of product candidates. Palvella’s approach is to provide targeted therapies for conditions with significant unmet medical needs. InvestingPro analysis reveals strong analyst confidence in the company’s potential, with price targets ranging from $38 to $70, significantly above current trading levels. Discover more insights and 10+ additional ProTips about PVLA with an InvestingPro subscription, including detailed financial health metrics and growth projections.

This news is based on a press release statement from Palvella Therapeutics and does not include any promotional content. QTORIN™ rapamycin remains investigational and has not yet been approved by the FDA or any other regulatory body.

In other recent news, Palvella Therapeutics has reported a net loss of $17.4 million for the year 2024, as outlined in their Q4 earnings call. Despite this, the company is advancing its lead product, QTORIN Rapamycin, through various clinical trials aimed at treating rare skin diseases. Chardan Capital Markets initiated coverage on Palvella Therapeutics with a Buy rating and a price target of $50.00, citing the potential of QTORIN topical rapamycin gel. This follows Stifel’s reaffirmation of a Buy rating with a $45.00 price target, highlighting the promising Phase 2 results for microcystic lymphatic malformations (mLMs). The company is currently in Phase 3 trials for mLMs and Phase 2 trials for cutaneous venous malformations (cVMs), with results expected in late 2025 and early 2026. Palvella ended 2024 with $83 million in cash, providing a financial runway into 2027. The company plans to spend $30 million in 2025, with a focus on research and development. These developments are part of Palvella’s broader strategy to address unmet needs in rare genetic skin diseases.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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