WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA), a biopharmaceutical company focused on developing treatments for rare genetic skin diseases, has announced its financial results for 2024, alongside key corporate and clinical updates. Following its recent merger with Pieris Pharmaceuticals and a $78.9 million private placement, Palvella is poised for significant growth with its late-stage pipeline of innovative therapies.
Clinical Pipeline Milestones
Palvella spotlighted advancements in its lead candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), aimed at addressing chronically debilitating genetic skin conditions for which there are currently no FDA-approved treatments.
Microcystic Lymphatic Malformations (microcystic LMs)
QTORIN™ rapamycin is currently in a Phase 3 clinical trial (SELVA) for the treatment of microcystic lymphatic malformations, a rare disorder affecting over 30,000 individuals in the U.S. Microcystic LMs, caused by dysregulation of the PI3K/mTOR pathway, lead to malformed, leaking lymphatic vessels that can cause infections and potentially life-threatening complications.
In October 2024, Palvella received a $2.6 million grant from the FDA Office of Orphan Products Development to support its Phase 3 SELVA study. “Microcystic LMs and cutaneous venous malformations represent serious, unmet medical needs, and QTORIN™ rapamycin has the potential to become the first FDA-approved standard of care in the U.S. for these conditions,” said Wes Kaupinen, Founder and CEO of Palvella.
Enrollment for the SELVA trial began in November 2024, with top-line results expected in Q1 2026. The FDA has granted QTORIN™ rapamycin multiple designations, including Breakthrough Therapy, Orphan Drug, and Fast Track.
Cutaneous Venous Malformations (cutaneous VMs)
Meanwhile, the Phase 2 study (TOIVA) for cutaneous venous malformations, which affect approximately 75,000 people in the U.S., is on track to deliver top-line results by the end of 2025. Cutaneous VMs, driven by PI3K/mTOR pathway mutations, result in vascular abnormalities that can severely impact quality of life. Patients enrolled in the TOIVA trial began receiving treatment in January 2025, with the FDA granting Fast Track Designation for this indication.
Palvella’s QTORIN™ platform is also set to expand in 2025, with new programs targeting additional rare genetic disorders driven by mTOR pathway dysregulation. A second product from the QTORIN™ platform is also anticipated, further solidifying the company’s presence in the rare disease space.
Strategic Corporate Updates
Palvella’s transformation into a publicly traded company followed a December 2024 merger with Pieris Pharmaceuticals, which coincided with a $78.9 million private placement co-led by BVF Partners L.P. and Frazier Life Sciences. The financing also saw support from top-tier healthcare investors including Blue Owl Healthcare Opportunities, Samsara BioCapital, and Gore Range Capital.
Kaupinen said, “2024 was marked by significant progress toward achieving our vision of becoming the leading rare disease biopharmaceutical company focused on serious, rare genetic skin diseases.” He added that Palvella is well-positioned to deliver impactful therapies to underserved patient populations in the years ahead.
Additionally, the company welcomed industry veteran Matthew Korenberg as Chief Financial Officer in October 2024. With extensive leadership experience at Ligand Pharmaceuticals and in healthcare investment banking, Korenberg is set to strengthen Palvella’s operational and financial strategy.
Financial Performance
Palvella ended 2024 with $83.6 million in cash and equivalents, ensuring sufficient funding for operations into the second half of 2027. Research and development expenses totaled $8.2 million for the year, consistent with its focus on advancing its clinical pipeline. General and administrative expenses rose to $5.9 million, reflecting costs associated with becoming a public company.
While the company reported a net loss of $17.4 million for the year, this reflects significant investments in its pipeline and strategic initiatives. Kaupinen emphasized that these resources are integral to advancing Palvella’s mission to deliver first-of-its-kind therapies for rare genetic skin diseases.
Looking Ahead
With a late-stage clinical pipeline, expanding resources, and a robust financial foundation, Palvella Therapeutics is positioned for a pivotal year in 2025. The company aims to achieve critical milestones, including advancing QTORIN™ rapamycin through regulatory milestones and clinical readouts, while expanding its platform to address broader rare disease challenges.
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