Palvella Therapeutics Reports Second Quarter 2025 Financial Results and Provides Corporate Update
Palvella Therapeutics (NASDAQ:PVLA) reported Q2 2025 financial results and provided updates on its clinical programs. The company's Phase 3 SELVA trial for QTORIN™ rapamycin in microcystic lymphatic malformations exceeded enrollment targets by 25%, with top-line results expected in Q1 2026. The Phase 2 TOIVA trial for cutaneous venous malformations remains on track for Q4 2025 results.
Financial highlights include $70.4 million in cash as of June 30, 2025, expected to fund operations into H2 2027. Q2 2025 saw a net loss of $9.5 million ($0.86 per share). The company plans to announce both a third clinical indication for QTORIN™ rapamycin and a second QTORIN™ platform candidate by year-end 2025.
Palvella Therapeutics (NASDAQ:PVLA) ha comunicato i risultati finanziari del secondo trimestre 2025 e ha fornito aggiornamenti sui suoi programmi clinici. Lo studio di Fase 3 SELVA su QTORIN™ rapamycin nelle malformazioni linfatiche microcistiche ha superato gli obiettivi di arruolamento del 25%, con i risultati principali attesi nel primo trimestre 2026. Lo studio di Fase 2 TOIVA per le malformazioni venose cutanee rimane in linea per i risultati nel quarto trimestre 2025.
I punti finanziari evidenziano una liquidità di 70,4 milioni di dollari al 30 giugno 2025, ritenuta sufficiente a finanziare le operazioni fino al secondo semestre 2027. Nel Q2 2025 la società ha registrato una perdita netta di 9,5 milioni di dollari (0,86 $ per azione). L'azienda prevede di annunciare entro la fine del 2025 sia una terza indicazione clinica per QTORIN™ rapamycin sia un secondo candidato della piattaforma QTORIN™.
Palvella Therapeutics (NASDAQ:PVLA) informó los resultados financieros del segundo trimestre de 2025 y ofreció actualizaciones sobre sus programas clínicos. El ensayo de Fase 3 SELVA para QTORIN™ rapamycin en malformaciones linfáticas microquísticas superó las metas de reclutamiento en un 25%, y se esperan los resultados principales en el primer trimestre de 2026. El ensayo de Fase 2 TOIVA para malformaciones venosas cutáneas continúa según lo previsto y mantiene la expectativa de resultados en el cuarto trimestre de 2025.
Entre los aspectos financieros destaca una liquidez de 70,4 millones de dólares al 30 de junio de 2025, que se espera financie las operaciones hasta la segunda mitad de 2027. En el segundo trimestre de 2025 registró una pérdida neta de 9,5 millones de dólares (0,86 $ por acción). La compañía planea anunciar antes de finales de 2025 tanto una tercera indicación clínica para QTORIN™ rapamycin como un segundo candidato de la plataforma QTORIN™.
Palvella Therapeutics (NASDAQ:PVLA)는 2025년 2분기 실적을 발표하고 임상 프로그램 현황을 업데이트했습니다. 미세낭성 림프관 기형에 대한 QTORIN™ rapamycin의 임상 3상 SELVA 연구는 모집 목표를 25% 초과 달성했으며, 주요 결과는 2026년 1분기에 발표될 예정입니다. 피부 정맥 기형을 대상으로 한 2상 TOIVA 연구는 2025년 4분기 결과 발표 일정에 맞춰 순조롭게 진행 중입니다.
재무 하이라이트로는 2025년 6월 30일 기준 현금 7,040만 달러를 보유하고 있어 2027년 하반기까지 운영 자금을 충당할 것으로 예상됩니다. 2025년 2분기 순손실은 950만 달러(주당 0.86달러)를 기록했습니다. 회사는 2025년 말까지 QTORIN™ rapamycin의 세 번째 임상 적응증과 QTORIN™ 플랫폼의 두 번째 후보 물질을 발표할 계획입니다.
Palvella Therapeutics (NASDAQ:PVLA) a publié ses résultats financiers du deuxième trimestre 2025 et a fourni des mises à jour sur ses programmes cliniques. L'essai de phase 3 SELVA portant sur QTORIN™ rapamycin dans les malformations lymphatiques microkystiques a dépassé les objectifs de recrutement de 25 %, les résultats principaux étant attendus au premier trimestre 2026. L'essai de phase 2 TOIVA pour les malformations veineuses cutanées reste en bonne voie pour des résultats au quatrième trimestre 2025.
Parmi les points financiers, on relève une trésorerie de 70,4 millions de dollars au 30 juin 2025, qui devrait permettre de financer les opérations jusqu'à la seconde moitié de 2027. Le deuxième trimestre 2025 a affiché une perte nette de 9,5 millions de dollars (0,86 $ par action). La société prévoit d'annoncer d'ici la fin 2025 une troisième indication clinique pour QTORIN™ rapamycin ainsi qu'un deuxième candidat de la plateforme QTORIN™.
Palvella Therapeutics (NASDAQ:PVLA) veröffentlichte die Finanzergebnisse für das zweite Quartal 2025 und gab Updates zu seinen klinischen Programmen. Die Phase‑3‑Studie SELVA mit QTORIN™ rapamycin bei mikrozystischen lymphatischen Fehlbildungen übertraf die Einschreibeziele um 25 %; die Topline‑Ergebnisse werden für Q1 2026 erwartet. Die Phase‑2‑Studie TOIVA zu kutanen venösen Malformationen liegt weiterhin im Zeitplan für Ergebnisse im Q4 2025.
Zu den finanziellen Eckdaten gehören 70,4 Mio. US‑Dollar liquide Mittel zum 30. Juni 2025, die voraussichtlich zur Finanzierung der Geschäftstätigkeit bis in die zweite Hälfte 2027 ausreichen. Im Q2 2025 wurde ein Nettoverlust von 9,5 Mio. US‑Dollar (0,86 USD je Aktie) ausgewiesen. Das Unternehmen plant, noch bis Ende 2025 sowohl eine dritte klinische Indikation für QTORIN™ rapamycin als auch einen zweiten Kandidaten der QTORIN™‑Plattform bekannt zu geben.
- None.
- Increased net loss to $9.5M in Q2 2025 vs $4.4M in Q2 2024
- R&D expenses increased significantly to $5.1M from $1.4M year-over-year
- G&A expenses nearly tripled to $4.1M from $1.5M year-over-year
Insights
Palvella shows strong clinical progress with QTORIN rapamycin, exceeding enrollment targets and maintaining cash runway into 2027.
Palvella Therapeutics is advancing well in its clinical development programs for QTORIN rapamycin, demonstrating significant enrollment momentum in its Phase 3 SELVA trial for microcystic lymphatic malformations (LMs). The company exceeded its enrollment target by over 25%, recruiting 51 subjects versus the planned 40, which suggests strong investigator interest and potentially accelerated timelines.
The company's dual-indication strategy for QTORIN rapamycin puts it on track for two important data readouts: Phase 3 SELVA results in Q1 2026 and Phase 2 TOIVA results for cutaneous venous malformations in Q4 2025. Both indications represent first-in-disease opportunities where no FDA-approved therapies currently exist, positioning Palvella to potentially capture entire treatment markets if successful.
From a financial perspective, Palvella reported $70.4 million in cash as of June 30, 2025, providing runway into the second half of 2027. This is a critical strength, as it fully funds the company through both key clinical readouts and potential NDA submission. The non-dilutive FDA Orphan Products Grant (up to $2.6 million) further extends their runway.
R&D expenses increased to $5.1 million for Q2 2025 (versus $1.4 million in Q2 2024), reflecting appropriate investment in clinical trials. Similarly, G&A expenses rose to $4.1 million (from $1.5 million), partly due to public company costs. The net loss was $9.5 million ($0.86 per share).
The company's patent portfolio strengthening is noteworthy, with two new patents extending protection for QTORIN rapamycin until 2038, providing substantial commercial runway if approved. Additionally, plans to announce both a third indication for QTORIN rapamycin and a second platform candidate by year-end 2025 suggest pipeline expansion that could create additional value inflection points.
The addition of Ashley Kline as CCO, with experience launching Oxervate (another first-in-disease topical therapy), indicates preparation for potential commercialization following positive clinical results and regulatory approval.
Phase 3 SELVA trial evaluating QTORIN™
Top-line results for Phase 2 TOIVA trial evaluating QTORIN™ rapamycin for cutaneous venous malformations remain on track for the fourth quarter of 2025
QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for microcystic lymphatic malformations and cutaneous venous malformations
Company plans to announce both a third clinical indication for QTORIN™ rapamycin and a second QTORIN™ platform candidate before year-end 2025
Cash and cash equivalents of
Company to host conference call at 8:30 a.m. ET today
WAYNE, Pa., Aug. 14, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) 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 genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, reported financial results for the second quarter ending June 30, 2025 and provided a corporate update.
“In the second quarter of 2025, we continued to advance Palvella’s mission of pioneering first-in-disease therapies for individuals with serious, rare genetic skin diseases,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “We remain on track for two near-term data readouts for QTORIN™ rapamycin, including our Phase 3 SELVA study in microcystic LMs which we believe will support a New Drug Application submission in 2026. In parallel, we are preparing to announce two new development programs in 2025, further expanding our rare disease pipeline. With strong momentum and a solid financial foundation, Palvella is well-positioned for continued growth and execution on our vision.”
Recent Research and Development Highlights
QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations (microcystic LMs)
- Completed enrollment in the Phase 3 SELVA trial, a single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin for the treatment of microcystic LMs; trial enrolled 51 subjects, exceeding its original target of 40 subjects by over
25% .
- The Company received initial proceeds from an FDA Orphan Products Grant which could total up to
$2.6 million to support the Phase 3 SELVA trial. The FDA Orphan Products Grants Program provides non-dilutive funding to advance the development of promising therapies for rare diseases.
- Key presentations and posters featured at major scientific meetings included:
- Oral presentation highlighting QTORIN™ rapamycin for the treatment of microcystic LMs was presented by Amy Paller, M.S., M.D., Chair of Dermatology at Northwestern University’s Feinberg School of Medicine, at the 15th World Congress of Pediatric Dermatology; the presentation highlighted the Phase 3 SELVA trial design and reviewed Phase 2 results.
- Poster highlighting the estimated diagnosed U.S. prevalence and U.S. annual incidence of lymphatic malformations was presented at the 82nd Annual Meeting of the Society for Investigative Dermatology.
- The United States Patent and Trademark Office (USPTO) issued patent No. 12,268,673 with claims to QTORIN™ rapamycin for the treatment of microcystic LMs with anticipated patent life extending into 2038.
- Top-line results from SELVA are on track for the first quarter of 2026.
QTORIN™ rapamycin for the treatment of cutaneous venous malformations (cutaneous VMs)
- Continued execution of ongoing Phase 2 TOIVA trial, a single-arm, open-label, baseline-controlled clinical trial evaluating QTORIN™ rapamycin for the treatment of cutaneous VMs. Enrollment is ongoing, targeting approximately 15 subjects.
- The Company, in collaboration with key opinion leaders in treating venous malformations, completed a nationally representative, blinded, real-world observational study estimating the U.S. annual prevalence of cutaneous VMs, with findings submitted to a peer reviewed journal.
- Top-line results from TOIVA are on track for the fourth quarter of 2025.
QTORIN™ pipeline development
- The expansion of QTORIN™ rapamycin into a third serious, rare skin disease with no FDA-approved therapies is progressing, with clinical and regulatory planning ongoing, and announcement of the program is planned for the second half of 2025.
- The second product candidate from the QTORIN™ platform for a serious, rare skin disease with no FDA-approved therapies, has achieved key pre-clinical development milestones, and announcement of the program is planned for the second half of 2025.
Recent Corporate Highlights
- Wes Kaupinen, Palvella’s Founder and CEO, participated in the FDA’s CEO Forum with Commissioner Marty A. Makary, M.D., M.P.H, and other senior FDA officials on June 5th.
- Strengthened executive leadership team with the appointment of Ashley Kline, a rare disease commercial veteran, as Chief Commercial Officer. Ashley previously led the successful launch of Oxervate®, a first-in-disease topical therapy for neurotrophic keratitis, a rare, serious, and progressive eye disease that previously had no FDA-approved therapies.
- The USPTO issued patent No. 12,329,748 encompassing a wide range of composition and method-of-use claims for QTORIN™ rapamycin and other mTOR inhibitors formulated in 0.1–
20% anhydrous compositions for a broad spectrum of rare and common skin diseases, with patent term expected through at least 2038.
- The Company was added to the Russell 3000® and Russell 2000® indexes on June 27, 2025.
Second Quarter 2025 Financial Results
- Cash and cash equivalents as of June 30, 2025 were
$70.4 million . Palvella expects such resources will be sufficient to fund its operations into the second half of 2027, and sufficient to accomplish its current strategic agenda.
- Research and development expenses were
$5.1 million for the three months ended June 30, 2025, as compared to$1.4 million for the three months ended June 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 2H 2024.
- General and administrative expenses were
$4.1 million for the three months ended June 30, 2025, as compared to$1.5 million for the three months ended June 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 expenses related to operating as a publicly-traded company.
- Net loss attributable to common stockholders was
$9.5 million , or$0.86 per basic and diluted share, for the three months ended June 30, 2025, as compared to net loss attributable to common stockholders of$4.4 million , or$2.47 per basic and diluted share, for the three months ended June 30, 2024.
- Shares outstanding were 13,735,390 as of August 8, 2025, including 11,059,665 shares of common stock and 2,675,725 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 second quarter 2025 financial results and provide a corporate update at 8:30 a.m. ET today. 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.
About Palvella Therapeutics
Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™
QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication.
Forward-Looking Statements
This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)). These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella, as well as assumptions made by, and information currently available to, the management of Palvella. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from ongoing clinical trials, Palvella’s clinical development plans and related anticipated development milestones, Palvella’s cash and financial resources and expected cash runway, and the potential of, and expectations regarding, Palvella’s programs, including QTORIN™ rapamycin, and its research-stage opportunities and any additional indications or platform candidates, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin; the outcome of early clinical trials for Palvella’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella’s current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that Palvella may face. Except as required by applicable law, Palvella does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release.
Contact Information
Investors
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
wes.kaupinen@palvellatx.com
Media
Marcy Nanus
Managing Partner, Trilon Advisors LLC
mnanus@trilonadvisors.com
PALVELLA THERAPEUTICS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 5,118 | $ | 1,442 | $ | 9,192 | $ | 2,426 | ||||||||
| General and administrative | 4,132 | 1,466 | 7,929 | 2,241 | ||||||||||||
| Total operating expenses | 9,250 | 2,908 | 17,121 | 4,667 | ||||||||||||
| Loss from operations | (9,250 | ) | (2,908 | ) | (17,121 | ) | (4,667 | ) | ||||||||
| Total other income (expense), net | (221 | ) | (1,264 | ) | (535 | ) | (2,041 | ) | ||||||||
| Net loss | $ | (9,471 | ) | $ | (4,172 | ) | $ | (17,656 | ) | $ | (6,708 | ) | ||||
| Less: Cumulative Series D preferred dividends | — | (194 | ) | — | (388 | ) | ||||||||||
| Net loss attributable to common stockholders | $ | (9,471 | ) | $ | (4,366 | ) | $ | (17,656 | ) | $ | (7,096 | ) | ||||
| Net loss per share — basic and diluted | $ | (0.86 | ) | $ | (2.47 | ) | $ | (1.60 | ) | $ | (4.01 | ) | ||||
| Weighted-average number of common shares used in computing net loss per share — basic and diluted | 11,052,741 | 1,770,167 | 11,033,327 | 1,770,167 | ||||||||||||
PALVELLA THERAPEUTICS, INC.
CONDENSED CONSOLIDATED BALANCE SHEET INFORMATION
(in thousands)
| June 30, 2025 | December 31, 2024 | |||||
| Assets | ||||||
| Cash and cash equivalents | $ | 70,433 | $ | 83,602 | ||
| Other current assets | 3,314 | 4,632 | ||||
| Total current assets | 73,747 | 88,234 | ||||
| Total assets | $ | 73,747 | $ | 88,234 | ||
| Liabilities and Stockholders' Equity | ||||||
| Current liabilities | $ | 9,616 | $ | 12,038 | ||
| Non-current liabilities | 16,351 | 13,589 | ||||
| Total liabilities | 25,967 | 25,627 | ||||
| Total stockholders' equity | 47,780 | 62,607 | ||||
| Total liabilities and stockholders’ equity | $ | 73,747 | $ | 88,234 | ||
FAQ
What were Palvella Therapeutics' (PVLA) key financial results for Q2 2025?
When will Palvella's Phase 3 SELVA trial results for QTORIN rapamycin be available?
How many patients were enrolled in PVLA's Phase 3 SELVA trial?
What is PVLA's cash runway following Q2 2025 results?
What new developments does Palvella plan to announce by the end of 2025?
