On November 11, 2025 Palvella Therapeutics, Inc. (Palvella or "the Company"), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, reported financial results for the third quarter ending September 30, 2025 and provided a corporate update.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"As we enter year-end 2025, Palvella is now advancing innovative QTORIN-derived therapies for four serious, rare skin diseases, each lacking a single FDA-approved therapy, giving us the opportunity to potentially be first for each of these deserving rare disease communities," said Wes Kaupinen, Founder and CEO of Palvella Therapeutics. "Our lead product candidate, QTORIN rapamycin, continues to demonstrate its potential as a pipeline-in-a-product for mTOR-driven skin diseases, with a planned Phase 2 top-line readout in cutaneous venous malformations expected in mid-December. This will be followed by a Phase 3 topline readout in microcystic lymphatic malformations which we anticipate in the first quarter of 2026. Overall, we anticipate the next 18 months will be a catalyst-rich period highlighted by our objective to advance QTORIN
rapamycin toward its first potential regulatory approval, with a steady flow of clinical, pre-commercialization, regulatory, and indication expansion milestones for QTORIN rapamycin and our additional QTORIN pipeline programs expected. Achievement of these milestones will advance our vision of becoming the leading biopharmaceutical company addressing serious, rare skin diseases with no FDA-approved therapies."
Recent Corporate Highlights
•
Appointed David W. Osborne, Ph.D., as Chief Innovation Officer to provide leadership across Palvella’s early-stage R&D pipeline, including maximizing the potential of the Company’s proprietary QTORIN platform. Dr. Osborne brings extensive topical product development experience and a track record of translating science into commercially available therapies, including contributing to the development of ZORYVE (roflumilast) cream and foam while serving as Co-Founder and Chief Technical Officer of Arcutis Biotherapeutics.
•
Jeffrey Martini, Ph.D., Chief Scientific Officer, presented at the Center for Dermal Research’s Innovations in Dermatological Sciences Conference at Rutgers University, showcasing the QTORIN platform and its lead product candidate, QTORIN rapamycin, in his presentation entitled "Unlocking the Potential of Topical Therapy in Rare Skin Diseases."
Recent Research and Development Highlights
QTORIN rapamycin for the treatment of microcystic lymphatic malformations (microcystic LMs)
•
Following FDA’s review of an annual performance progress report submitted by Palvella, FDA awarded the year two proceeds from the FDA Orphan Products Grant program to support the ongoing SELVA trial, a single arm, baseline-controlled Phase 3 study of QTORIN rapamycin for microcystic lymphatic malformations.
•
Top-line results from SELVA remain on track for the first quarter of 2026.
QTORIN rapamycin for the treatment of cutaneous venous malformations (cutaneous VMs)
•
The Company completed enrollment in the Phase 2 TOIVA trial of QTORIN rapamycin for cutaneous VMs, meeting its recruitment target of 16 subjects enrolled at leading vascular anomaly centers.
•
A peer-reviewed publication in Lymphatic Research and Biology highlighted 26 published studies evaluating the real-world use of off-label rapamycin in treating venous malformations, supporting rapamycin’s potential as a targeted therapy for the disease. The authors also noted the continued unmet need for a topical treatment option, reinforcing the scientific rationale for the development of Palvella’s QTORIN rapamycin for cutaneous VMs.
•
A nationally representative, retrospective, real-world, subject-blinded, physician-observational probability study published in Orphanet Journal of Rare Diseases estimated an annual U.S. treatment prevalence of more than 190,000 diagnosed patients with cutaneous VMs. The findings further underscored the need for the development of targeted therapies for this understudied, debilitating condition.
•
Top-line results from TOIVA are on track for mid-December 2025.
QTORIN rapamycin for the treatment of clinically significant angiokeratomas
•
Palvella expanded the development of QTORIN rapamycin into clinically significant angiokeratomas, a disease characterized by superficial vascular malformations of lymphatic origin which can cause bleeding, pain, functional impairment, and risk of infection.
•
Angiokeratomas were recently classified as an isolated lymphatic malformation in 2025 by the International Society for the Study of Vascular Anomalies (ISSVA).
•
There are currently no FDA-approved therapies available for the estimated more than 50,000 patients in the U.S. with clinically significant angiokeratomas.
•
Palvella plans to meet with the FDA in the first half of 2026 to discuss the proposed design of a Phase 2 study of approximately 10-20 patients; study initiation is anticipated in the second half of 2026.
QTORIN pitavastatin for the treatment of disseminated superficial actinic porokeratosis (DSAP)
•
The Company announced a new QTORIN product candidate, QTORIN pitavastatin, for the topical treatment of disseminated superficial actinic porokeratosis, a rare, chronic, and pre-cancerous genetic skin disease which presents as persistent, often extensive lesions that enlarge and increase in size, number, and extent over time.
•
Recent breakthrough discoveries on the genetics and biology of porokeratosis by Keith Choate, M.D, Ph.D., Chair of Dermatology at Yale School of Medicine, enable the development of QTORIN pitavastatin as the first potential pathogenesis-directed therapy for DSAP, a subtype of porokeratosis, by locally targeting the causal mevalonate pathway in the pathogenic epidermal and dermal tissue.
•
There are currently no FDA-approved therapies available for the estimated more than 50,000 patients in the U.S. with DSAP.
•
Palvella plans to meet with the FDA in the first half of 2026 to discuss the proposed design of a Phase 2 study evaluating QTORIN pitavastatin in subjects with DSAP; study initiation is anticipated in the second half of 2026.
Third Quarter 2025 Financial Results
•
Cash and cash equivalents as of September 30, 2025 were $63.6 million. Palvella expects these resources will be sufficient to fund its operations into the second half of 2027.
•
Research and development expenses were $6.5 million for the three months ended September 30, 2025, as compared to $3.2 million for the three months ended September 30, 2024. The increase in research and development expenses was primarily due to increased spending on the clinical development of QTORIN rapamycin for the treatment of microcystic LMs and cutaneous VMs, including conducting the Phase 3 SELVA and Phase 2 TOIVA trials, which were initiated in the second half of 2024.
•
General and administrative expenses were $3.6 million for the three months ended September 30, 2025, as compared to $1.9 million for the three months ended September 30, 2024. The increase in general and administrative expenses was primarily driven by increased employee compensation expense due to headcount additions, as well as increases in costs related to being a publicly-traded company.
•
Net loss attributable to common stockholders was $11.3 million, or $1.03 per basic and diluted share, for the three months ended September 30, 2025, as compared to net loss attributable to common stockholders of $7.0 million, or $3.94 per basic and diluted share, for the three months ended September 30, 2024.
•
Shares outstanding were 13,768,036 as of November 7, 2025, including 11,836,490 shares of common stock and 1,931,546 common share equivalents assuming conversion of outstanding preferred shares and prefunded warrants.
Conference Call Details
Palvella will host a conference call and live audiovisual webcast to discuss the Company’s third quarter 2025 financial results and provide a corporate update at 8:30 a.m. ET on November 11, 2025. To access the live webcast of the call with slides, please click here or visit the "Events & Presentations" section of Palvella’s website. To access the call by phone, please use this registration link, and you will be provided with dial in details. A replay of the webcast will be available approximately 2 hours after the conclusion of the call and archived for 90 days under the "Events & Presentations" section of the Company’s website at www.palvellatx.com.
(Press release, Palvella Therapeutics, NOV 11, 2025, View Source [SID1234659724])