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WAYNE, Pa. - Palvella Therapeutics, Inc. (NASDAQ:PVLA), a biopharmaceutical company focused on rare genetic skin diseases with a market capitalization of $281 million, has received initial funding from a grant awarded by the FDA’s Office of Orphan Products Development. According to InvestingPro data, the company maintains a strong financial position with more cash than debt on its balance sheet. The grant is allocated for the company’s ongoing Phase 3 SELVA trial, testing QTORIN™ 3.9% rapamycin anhydrous gel for treating microcystic lymphatic malformations (microcystic LMs).
The SELVA trial, which is currently enrolling participants at various vascular anomaly centers in the U.S., is a 24-week study that remains on schedule to report its top-line results in the first quarter of 2026. Approximately 40 subjects aged three and older are expected to participate in this single-arm, baseline-controlled clinical trial. The company’s stock has shown remarkable momentum, with a year-to-date return of 112%, while analyst price targets range from $38 to $70 per share.
Microcystic LMs are characterized by malformed lymphatic vessels that can lead to complications such as persistent leakage, bleeding, serious infections, and cellulitis. With over 30,000 diagnosed patients in the United States, there are presently no FDA-approved treatments for this condition.
Palvella’s QTORIN™ rapamycin has been identified as a potential first approved therapy for microcystic LMs, aiming to establish a new standard of care for those affected by the disease. With a healthy current ratio of 8.84, the company appears well-positioned to fund its development programs. Discover more detailed financial metrics and analysis with a InvestingPro subscription, which includes comprehensive research reports for over 1,400 US stocks. The FDA Orphan Products Grants Program, which has been instrumental in the approval of over 85 products since its inception, rigorously reviews grant applications to ensure scientific and technical merit.
Wes Kaupinen, CEO of Palvella, expressed honor in receiving the prestigious grant, emphasizing the lack of approved therapies for microcystic LMs and the potential impact of QTORIN™ rapamycin for patients living with this rare disease.
Palvella Therapeutics, which prides itself on addressing unmet medical needs in the rare disease space, has a pipeline of product candidates based on its patented QTORIN™ platform. The company’s approach to drug development is underscored by its commitment to providing novel treatments for serious genetic skin diseases.
The information in this article is based on a press release statement from Palvella Therapeutics and financial data from InvestingPro, which indicates the stock is currently trading slightly above its Fair Value.
In other recent news, Palvella Therapeutics reported a net loss of $8.2 million for the first quarter of 2025, with increased research and development expenses contributing to the loss. The company has $75.6 million in cash reserves, providing a cash runway for the next two years. Palvella is advancing its lead product, QTORIN™ rapamycin, with ongoing Phase 3 trials for microcystic lymphatic malformations and Phase 2 trials for cutaneous venous malformations. Additionally, Palvella anticipates announcing new programs in the latter half of 2025.
In a strategic move, Palvella Therapeutics appointed Ashley Kline as Chief Commercial Officer to lead the commercial strategy for QTORIN™, pending regulatory approval. Kline brings experience from Dompé Pharmaceuticals, where she successfully launched Oxervate®. The company remains focused on developing treatments for serious, rare genetic skin diseases. CEO Wes Coffinen highlighted the company’s commitment to being a leader in this niche market. Palvella continues to prioritize innovation, with plans to release top-line data from its Phase 3 study in early 2026.
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