Palvella (NASDAQ: PVLA) boosts cash with $230M raise as QTORIN NDA nears
Rhea-AI Filing Summary
Palvella Therapeutics reported a wider first quarter 2026 loss while significantly strengthening its balance sheet and advancing its rare-disease pipeline. Net loss was $15.8 million, or $1.20 per share, compared with $8.2 million, or $0.74 per share, a year earlier, driven by higher research, manufacturing and public-company costs.
The company completed an upsized, oversubscribed equity financing in February 2026, generating $230.0 million in gross proceeds and $215.8 million in net proceeds. Cash, cash equivalents and short-term investments were $261.9 million as of March 31, 2026, compared with $59.6 million in total assets at year-end 2025, positioning Palvella to fund key clinical and commercial initiatives.
Palvella is preparing a New Drug Application for QTORIN™ rapamycin in microcystic lymphatic malformations, with a pre-NDA FDA meeting granted for the second quarter of 2026 and NDA submission on track for the second half of 2026, targeting a potential U.S. launch in the first half of 2027. It is also progressing QTORIN™ programs in cutaneous venous malformations, clinically significant angiokeratomas and disseminated superficial actinic porokeratosis, and has expanded its leadership team and board to support commercialization.
Positive
- Strengthened balance sheet with major equity financing: Palvella completed an upsized, oversubscribed public offering generating $230.0 million in gross proceeds and $215.8 million in net proceeds, lifting cash, cash equivalents and short-term investments to $261.9 million as of March 31, 2026.
- Late-stage progress for lead QTORIN™ program: Positive Phase 3 SELVA topline results in microcystic lymphatic malformations support a planned 505(b)(2) NDA submission for QTORIN™ rapamycin in the second half of 2026, with potential U.S. approval and commercial launch targeted for the first half of 2027.
- Broad rare-disease pipeline with multiple catalysts: Phase 3 initiation in cutaneous venous malformations, Phase 2 trials in clinically significant angiokeratomas and disseminated superficial actinic porokeratosis, plus additional QTORIN™ indications and a third platform program planned, create a dense set of value-driving milestones.
Negative
- Significantly higher operating expenses and net loss: Total operating expenses nearly doubled to $14.9 million for the quarter, and net loss increased to $15.8 million, or $1.20 per share, from $8.2 million, or $0.74 per share, reflecting heavier R&D and public-company costs.
- All programs remain pre-revenue and investigational: QTORIN™ rapamycin and QTORIN™ pitavastatin are still in clinical development with no FDA-approved indications, so Palvella remains dependent on external capital while it executes multiple concurrent trials and prepares for commercialization.
Insights
Large financing and late-stage data materially de-risk Palvella’s lead program despite rising losses.
Palvella combined positive Phase 3 SELVA results in microcystic lymphatic malformations with a substantial $230.0 million upsized equity raise, boosting cash, cash equivalents and short-term investments to $261.9 million as of March 31, 2026. This capital supports NDA preparation and early commercial build-out for QTORIN™ rapamycin.
Operating expenses rose sharply as the company scaled R&D and public-company infrastructure. Research and development reached $9.3 million and general and administrative $5.5 million for the quarter, nearly doubling total operating expenses year over year. Net loss of $15.8 million reflects this strategic investment phase.
The pipeline shows multiple shots on goal: an NDA in microcystic lymphatic malformations planned for the second half of 2026, a Phase 3 start in cutaneous venous malformations in the second half of 2026, ongoing Phase 2 trials in angiokeratomas and planned Phase 2 in disseminated superficial actinic porokeratosis. Future disclosures tied to these milestones will clarify how effectively the new capital converts into approvals and revenue.
8-K Event Classification
Key Figures
Key Terms
microcystic lymphatic malformations medical
Breakthrough Therapy Designation regulatory
Fast Track Designation regulatory
505(b)(2) submission regulatory
orphan drug designation regulatory
rare skin diseases medical
Earnings Snapshot
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 2.02 Results of Operations and Financial Condition.
On May 7, 2026, Palvella Therapeutics, Inc. (the “Company”) announced its financial results for the quarter ended March 31, 2026. A copy of the press release is being furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.
The information furnished pursuant to this Item 2.02, including Exhibit 99.1 attached hereto, is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended (the “Securities Act”), or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 7.01 Regulation FD Disclosure.
On May 7, 2026, the Company will hold its earnings call and use a slide presentation in conjunction with the earnings call. A copy of the presentation is furnished herewith as Exhibit 99.2, and incorporated herein by reference.
The information furnished pursuant to Item 7.01, including Exhibit 99.2, shall not be deemed “filed” for purposes of Section 18 of the Exchange Act or otherwise subject to the liabilities of that section, and shall not be deemed to be incorporated by reference in any filing under the Securities Act or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
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Press Release of Palvella Therapeutics, Inc., dated May 7, 2026* |
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Earnings Call Presentation of Palvella Therapeutics, Inc., dated May 7, 2026* |
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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Palvella Therapeutics, Inc. |
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May 7, 2026 |
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/s/ Matthew Korenberg |
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Matthew Korenberg |
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Chief Financial Officer |
Palvella Therapeutics Reports First Quarter 2026 Financial Results and Provides Corporate Update
FDA Pre-New Drug Application (NDA) meeting granted for QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations, with meeting expected in second quarter of 2026; NDA submission on track for second half of 2026
Accelerating U.S. launch readiness for QTORIN™ rapamycin for microcystic lymphatic malformations; BEYONDmLM.com disease awareness campaign launched to educate, engage, and empower patients, caregivers, and healthcare professionals
Initiation of Phase 3 trial of QTORIN™ rapamycin for the treatment of cutaneous venous malformations planned for second half of 2026
Initiation of Phase 2 trial of QTORIN™ pitavastatin for the treatment of disseminated superficial actinic porokeratosis planned for second half of 2026
Phase 2 LOTU trial of QTORIN™ rapamycin for clinically significant angiokeratomas initiated with topline results expected in second half of 2027
Completed upsized, oversubscribed equity financing of $230.0 million in February 2026; cash, cash equivalents and short-term investments of $261.9 million as of March 31, 2026
Company to host conference call at 8:30 a.m. ET today
WAYNE, PA., May 7, 2026 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. (Palvella or “the Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today reported financial results for the first quarter ending March 31, 2026 and provided a corporate update.
“Following the positive Phase 3 SELVA topline results, we believe Palvella is on a clear path toward near-term NDA submission for QTORIN™ rapamycin in microcystic lymphatic malformations, with the potential for approval and U.S. commercial launch in the first half of 2027,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “Our $230.0 million financing with participation from high-quality new and existing investors meaningfully strengthens our balance sheet, which supports our plan for U.S. commercialization, and enables us to attract key talent as we prepare for a potential first-in-disease launch. At the same time, we continue to rapidly advance our pipeline beyond microcystic LMs, with the goal of expanding our QTORIN™-derived programs across six serious, rare skin diseases and vascular malformations by year-end.”
Recent Research and Development Highlights
QTORIN™ rapamycin for microcystic lymphatic malformations (microcystic LMs)
QTORIN™ rapamycin for cutaneous venous malformations (cutaneous VMs)
QTORIN™ rapamycin for clinically significant angiokeratomas
QTORIN™ pitavastatin for disseminated superficial actinic porokeratosis (DSAP)
QTORIN™ rapamycin and QTORIN™ platform expansion
Recent Corporate Highlights
First Quarter 2026 Financial Results
Conference Call Details
Palvella will host a conference call and live audiovisual webcast to discuss the Company's full year 2025 financial results and provide a corporate update at 8:30 a.m. ET today. To access the live webcast, including slides, please click here or visit the “Events & Presentations” section of Palvella’s website. To join the conference call by phone, dial 800-715-9871 (domestic) or +1 646-307-1963 (international) and provide Conference ID 9970701. Participants are encouraged to dial in approximately 15 minutes prior to the start of the call.
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 biotech 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 skin diseases and vascular malformations 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 skin diseases and vascular malformations, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being developed for the treatment of microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas. Palvella’s second product candidate, QTORIN™ pitavastatin, is currently being developed for the treatment of disseminated superficial actinic porokeratosis. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter).
QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and neither has been approved 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 clinical trials, Palvella’s clinical development plans and related anticipated development milestones, Palvella’s plans to pursue Breakthrough Therapy Designation, Palvella’s plans to meet with regulatory authorities, Palvella’s expectations regarding the benefits of orphan drug designation and potential benefit of orphan drug exclusivity for QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations, Palvella’s cash, financial resources and expected runway, Palvella’s expectations regarding its programs, including QTORIN™ rapamycin and QTORIN™ pitavastatin, and its research-stage opportunities, 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 and QTORIN™ pitavastatin; 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; Palvella’s limited experience in commercial manufacturing; 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)
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Three Months Ended |
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2026 |
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2025 |
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Operating expenses: |
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Research and development |
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$ |
9,334 |
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$ |
4,074 |
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General and administrative |
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5,521 |
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|
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3,797 |
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Total operating expenses |
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14,855 |
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7,871 |
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Loss from operations |
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(14,855 |
) |
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(7,871 |
) |
Total other income (expense), net |
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(912 |
) |
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(314 |
) |
Net loss |
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$ |
(15,767 |
) |
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$ |
(8,185 |
) |
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Net loss per share of Common Stock — basic and diluted |
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$ |
(1.20 |
) |
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$ |
(0.74 |
) |
Weighted-average shares used in computing net loss per share of Common Stock — basic and diluted |
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13,085,271 |
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11,013,697 |
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PALVELLA THERAPEUTICS, INC.
CONDENSED CONSOLIDATED BALANCE SHEET INFORMATION
(in thousands)
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March 31, |
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December 31, |
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2026 |
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2025 |
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Assets |
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Cash and cash equivalents |
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$ |
206,394 |
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$ |
57,982 |
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Short-term investments |
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55,459 |
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— |
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Other current assets |
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1,378 |
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1,005 |
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Total current assets |
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263,231 |
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58,987 |
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Non-current assets |
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528 |
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572 |
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Total assets |
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$ |
263,759 |
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$ |
59,559 |
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Liabilities and Stockholders' Equity |
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Current liabilities |
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$ |
9,115 |
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$ |
11,344 |
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Non-current liabilities |
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22,331 |
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20,232 |
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Total liabilities |
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31,446 |
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31,576 |
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Total stockholders' equity |
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232,313 |
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27,983 |
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Total liabilities and stockholders’ equity |
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$ |
263,759 |
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$ |
59,559 |
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First-in-disease therapies for patients with rare diseases Q1 2026 Financial Results & Corporate Update May 7, 2026
Forward Looking Statements This presentation contains forward-looking statements of Palvella Therapeutics, Inc. (“the Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that are not historical facts, and in some cases, can be identified by terms such as “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “objective,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue,” “ongoing,” or the negative of these terms, or other comparable terminology intended to identify statements about the future. Forward-looking statements contained in this presentation include, but are not limited to, statements regarding the Company’s future financial or business performance, conditions, plans, prospects, trends or strategies and other financial and business matters, the Company’s current and prospective product candidates and any additional indications or platform candidates, the Company's planned research and development activities, the Company's planned clinical trials, including timing of receipt of data from the same, the planned regulatory framework for the Company's product candidates, the Company's expectations regarding the benefits of orphan drug designation and potential benefit of orphan drug exclusivity for QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations, the Company's ability and the ability of third-party manufacturers the Company engages to optimize and scale manufacturing, the strength of the Company's intellectual property portfolio, and projections of the Company’s future financial results and other metrics. Such forward-looking statements are subject to risks, uncertainties, and other factors which could cause actual results to differ materially from those expressed or implied by such forward looking statements. These forward-looking statements are based upon current estimates and assumptions of the Company and its management and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this presentation. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: competition, the ability of the Company to grow and manage growth, maintain relationships with suppliers and retain its management and key employees; the success, cost and timing of the Company’s product development activities, studies and clinical trials; changes in applicable laws or regulations; the possibility that the Company may be adversely affected by other economic, business or competitive factors; the Company’s estimates of expenses and profitability; the evolution of the markets in which the Company competes; the ability of the Company to implement its strategic initiatives and continue to innovate its existing products; and the ability of the Company to defend its intellectual property. Nothing in this Presentation should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements, which speak only as of the date they are made. The Company undertakes no duty to update these forward-looking statements. Industry and Market Data The Company may from time to time provide estimates, projections and other information concerning its industry, the general business environment, and the markets for certain conditions, including estimates regarding the potential size of those markets and the estimated incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties, and actual events, circumstances or numbers, including actual disease prevalence rates and market size, may differ materially from the information reflected in this presentation. Unless otherwise expressly stated, we obtained this industry, business information, market data, prevalence information and other data from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data, and similar sources, in some cases applying our own assumptions and analysis that may, in the future, prove not to have been accurate. Trademarks This Presentation may contain trademarks, service marks, trade names and copyrights of other companies, which are the property of their respective owners. Solely for convenience, some of the trademarks, service marks, trade names and copyrights referred to in this Presentation may be listed without the TM, SM © or ® symbols, but the Company will assert, to the fullest extent under applicable law, the rights of the applicable owners, if any, to these trademarks, service marks, trade names and copyrights.
Q1 2026 Achievements Position Company for First Potential Approval and Launch of QTORIN™ Rapamycin $230mm raised in gross proceeds in upsized equity financing Microcystic Lymphatic Malformations: Advancing Towards Potential 1H 2027 Approval Phase 3 results exceeded upside case profile NDA remains on track for 2H 2026; pre-NDA meeting granted for Q2 2026 Exceptional veteran commercial leadership recruited; launch planning accelerating Phase 3 initiation on track Cutaneous VMs Angiokeratomas Accelerated towards first patients dosed ahead of schedule DSAP Review published in Clinical and Experimental Dermatology >40 patient inbounds for clinical trial
597 Rare Skin Diseases Often serious and chronically debilitating Over 98% Have No Approved Therapies1 Lower industry innovation and investment compared to other therapeutic areas (e.g., oncology, neurology, pulmonary) Note: John Doux, M.D. is a member of the Palvella Board of Directors. 1. Licholai G., Rare Dermatology Orphan Disease Drug Development Opportunities & Challenges, Presented at Dermatology Summit, Jan 2019. “We have begun to see interest from investors and companies in developing treatments for a rare disease such as epidermolysis bullosa, but there are many other diseases within dermatology that remain unaddressed” John Doux, M.D., Barriers and Opportunities Across the Development Divide, The Society of Investigative Dermatology, 2015 Palvella: Vision and Leadership in Treating Serious, Rare Skin Diseases
Palvella: Driving the Next Wave of Innovation in Under-appreciated Rare Skin Diseases Diagnosed U.S. Prevalence1 30k+ Microcystic LMs 75k+ Cutaneous VMs 50k+ Clinically Significant Angiokeratomas 50k+ Disseminated Superficial Actinic Porokeratosis 1. Clarity Pharma, Trinity Life Sciences, MedaCorp, and ZS Associates. 2. MedaCorp market research. Market Research: % Physicians Who Would Consider QTORIN™ as First-line Therapy2 >80% across four indications None # of FDA-approved Therapies
Attracting proven commercial talents Positive Phase 3 data in microcystic LMs and positive Phase 2 data in cutaneous VMs Positive Clinical Data and Upsized Equity Financing Enable Accelerated U.S. Launch Planning $230 million Gross proceeds in upsized equity financing Engaging top 400 high-volume centers, including VACs* Jen McDonough SVP Market Access Prev. Krystal Biotech Kent Taylor SVP Sales Prev. Arcutis Targeting higher end of range for field sales force (30-40 reps) with plans to hire prior to PDUFA 1 2 Two MSLs hired, with plans for additional hires, to drive disease state awareness *VACs = vascular anomaly centers.
QTORIN™ Rapamycin: >$1bn U.S. Peak Sales Potential in Microcystic LMs QTORIN™ Rapamycin Potential to Achieve >$1bn Peak Sales >30k diagnosed U.S. patients, with concentration in vascular anomaly centers Microcystic Lymphatic Malformations Annual ~$100k-$200k pricing range per patient Recent field checks, annual incidence estimates, published claims analysis Supported by Phase 3 SELVA data, payor testing, analogues QTORIN™ 3.9% rapamycin anhydrous gel QTORIN™ 3.9% rapamycin anhydrous gel is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency. The safety or efficacy has not been established for any use.
OUR PLATFORM QTORIN™
QTORIN™: Repeatable New Product Development Engine PARADIGM SHIFT towards targeted and well-tolerated delivery to the dermis BROAD PLATFORM CAPABILITY across diverse range of molecules NEW LONG-DURATION IP opportunity with each product candidate Rapid pipeline expansion with capital- and time-efficient QTORIN™ programs
Rare Disease Pipeline Overview
SELVA: Additional Analyses Support Clinical Meaningfulness IGA: +3 “ It doesn’t bleed anymore and it’s really clear... It just looks like my normal skin. …it doesn't leak constantly like it used to. KEY INSIGHTS “ Week 24 [The lesion] became closer to the color of my skin tone before it fully went away A pre-specified embedded patient interview sub-study provided qualitative evidence supporting the clinical meaningfulness of the improvements observed in SELVA Patient findings strengthen overall risk/benefit narrative for planned NDA submission Interviews reinforced that SELVA endpoints captured meaningful changes to patients’ lives Improvements in disease burden were consistently reported by patients
Two prospective studies with statistical significance, with 95% of patients improving on mLM-IGA at Week 24 in SELVA Seeking traditional approval based on clinical endpoints (not biomarkers for accelerated approval) Planned 505(b)(2) submission: leveraging FDA’s prior findings for rapamycin to streamline review process Planned 505(b)(2)-Enabled NDA Submission Pre-NDA meeting granted by FDA, with meeting expected Q2 2026 Real-world studies from off-label rapamycin use as supportive evidence Supported by FDA Orphan Products Grant: Two tranches of non-dilutive funding received
Strong Medical Affairs Presence with Sponsorship and Presentations at Key Medical Congresses Platinum Sponsor at ISSVA World Congress (May 19-22, 2026) Title: QTORIN™ 3.9% Rapamycin Anhydrous Gel: Statistically Significant, Clinically Meaningful Improvement in Microcystic Lymphatic Malformations (Phase 3 SELVA Study) and Cutaneous Venous Malformations (Phase 2 TOIVA Study) Date/Time: Wednesday, May 20, 2026 at 4:30pm ET LATE-BREAKER PRESENTATION SCIENTIFIC SYMPOSIUM Title: Clinical Development in Rare Cutaneous Vascular Disorders: Lessons Learned from SELVA and TOIVA Trials Date/Time: Thursday, May 21, 2026 at 12:30pm ET BEYOND mLM LOUNGE Attendees can participate in discussions on mLM and sign up to receive educational resources and communications for both themselves and their patients Planned Presentations at Additional Medical Congresses: Epidermal Differentiation Disorders Symposium (Platinum Sponsor) May 12, 2026, Chicago, IL Jul 22-25, 2026 Minneapolis, MN Oct 15-17, 2026 Alexandria, VA JAMES TREAT, MD Professor of Clinical Pediatrics and Dermatology, CHOP
Cutaneous Venous Malformations: 73% of Patients Improved in Phase 2 No FDA-approved therapies Fast Track Designation Granted Breakthrough Therapy Designation application submitted Pipeline-in-a-product: sNDA planned > 75k patients ESTIMATED DIAGNOSED IN THE U.S. POTENTIAL TO BE FIRST FDA-APPROVED THERAPY Why this fits Palvella’s strategy: Serious, rare, mTOR-driven Meaningful overlap with microcystic LM physicians at centers of excellence On track for Phase 3 trial initiation in 2H 2026 Additional findings from Phase 2 TOIVA study to be presented at ISSVA World Congress later this month Market research (n=50 physicians): 86% would consider QTORIN™ rapamycin as first-line therapy for cutaneous VMs
Clinically Significant Angiokeratomas: Superficial Lymphatic Malformations Lymphatic origin1,5 Superficial dermal location3,5 Clinically impactful, causing bleeding, functional impairment, and risk of infection4,5 Microcystic LMs Angiokeratomas Scientific rationale and real-world evidence supporting targeted therapy with rapamycin2,5 P P P P P P P P Shared Clinical Features between Clinically Significant Angiokeratomas and Microcystic LMs 1. Trindade F, et al. Am J Dermatopathol, Sep 30, 2014. 2. Bell KA, et al. JAAD Case Reports, Nov 30, 2020; Camacho I, et al. Dermatologic Therapy, Jun 27, 2020; Moeineddin F, et al. Clinical Case Reports, May 31, 2024; Farajzadeh S, et al. Indian J Dermatol Venereol Leprol, Jun 20, 2023; Fernández Ginés Fd, et al. Eur J Hosp Pharm, Feb 28, 2018; Kang Y, et al. J Korean Association of Oral Maxillofacial Surgery July 12, 2014. 3. Trindade F, et al. Am J Dermatopathol, Sep 30, 2014. 4. Philip C, et al. Dermatological Therapeutics March 31, 2020; Hobbs et al, Journal of Dermatology Surg Onco, 1987. 5. Teng et al, Lymphatic Research and Biology, 2022.
> 50k patients ESTIMATED DIAGNOSED IN THE U.S. Clinically Significant Angiokeratomas: Superficial Lymphatic Malformations FIRST PATIENTS DOSED IN PHASE 2 TRIAL MAY 2026 A type of isolated lymphatic malformation: direct scientific adjacency to microcystic LMs FDA granted Fast Track Designation in Dec 2025 Phase 2 LOTU study is a single-arm, baseline-controlled clinical trial evaluating QTORIN™ rapamycin applied topically once daily Enrolling up to 15 patients Topline results are expected in 2H 2027 Market research (n=50 physicians): 96% would incorporate QTORIN™ rapamycin into their practice No FDA-approved therapies Fast Track Designation Granted Pipeline-in-a-product: sNDA planned
> 50k patients ESTIMATED DIAGNOSED IN THE U.S. Disseminated Superficial Actinic Porokeratosis (DSAP): Chronic, Pre-Cancerous, and Progressive No FDA-approved therapies Current options: Laser, surgery, and off-label topical chemo agents & mevalonate pathway inhibitors QTORIN™ PITAVASTATIN POTENTIAL TO BE FIRST FDA-APPROVED THERAPY AND STANDARD OF CARE First pathogenesis-directed therapy targeting the casual mevalonate pathway Phase 2 initiation on track for 2H 2026 Strong patient interest for planned Phase 2 study; over 40 inbound patient inquiries, including: “I tried several unsuccessful treatments. This is a disfiguring… potentially cancerous condition. After years of waiting, the prospect of a successful treatment is exciting. If I can be part of the solution by participating in a clinical trial, count me in.” “Having tried just about everything to treat this rare disorder, I am EXCEPTIONALLY interested in your clinical trial…I am desperate for any treatment”
QTORIN™ Rapamycin Rare Serious Strong Scientific & Biologic Rationale Commercially Attractive No FDA-approved Therapies Chronic, extensive lesions, malignant transformation >50k U.S. patients Mutations in mevalonate pathway lead to accumulation of toxic intermediates None Microcystic LMs, Cutaneous VMs, Clinically Significant Angiokeratomas DSAP Significant unmet medical need Multi-billion dollar U.S. TAM1 Optimizing likelihood of clinical success Opportunity to be first-in-disease and SOC Published Case Studies & Off-label Use of API Multiple published case studies + use in academic centers P P P P P P Based on internal and third-party estimates. QTORIN™ Pitavastatin Disseminated Superficial Actinic Porokeratosis (DSAP): Aligned With Palvella’s Pipeline Strategy
Finance & Upcoming Catalysts
Well-Capitalized with High-Quality Investor Participation in $230mm Oversubscribed Feb. 2026 Financing Following Positive SELVA Data Potential to Fund Through: QTORIN™ + 505(b)(2) + rare disease focus offers potential for attractive ROI NDA filing, FDA approval, and, if approved, U.S. launch for QTORIN™ rapamycin in microcystic LMs NDA filing for QTORIN™ rapamycin in cutaneous VMs Multiple Phase 2 data readouts from pipeline programs $262 million 3/31/26 cash
Microcystic Lymphatic Malformations: NDA Submission on Track for 2H 2026 with Anticipated U.S. Commercialization in 1H 2027 QTORIN™ 3.9% rapamycin anhydrous gel is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency. The safety or efficacy has not been established for any use. Microcystic Lymphatic Malformations 1 Positive Phase 3 SELVA data In-person pre-NDA meeting granted by FDA, to occur in Q2 2026 Findings to be presented at ISSVA World Congress, May 2026 NDA submission on track for 2H 2026 Potential FDA approval and U.S. launch 1H 2027
Multiple High-Impact Pipeline Milestones in 2026: Density of Catalyst Creation Driven by QTORIN™ QTORIN™ 3.9% rapamycin anhydrous gel and QTORIN™ pitavastatin are for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency. The safety or efficacy has not been established for any use. Cutaneous Venous Malformations 2 Positive Phase 2 data BTD application submitted Q2 2026 P3 initiation expected 2H 2026
Multiple High-Impact Pipeline Milestones in 2026: Density of Catalyst Creation Driven by QTORIN™ QTORIN™ 3.9% rapamycin anhydrous gel and QTORIN™ pitavastatin are for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency. The safety or efficacy has not been established for any use. Clinically Significant Angiokeratomas 3 Fast Track Designated Phase 2 initiated May 2026, ahead of schedule Phase 2 data expected 2H 2027 Cutaneous Venous Malformations 2 Positive Phase 2 data BTD application submitted Q2 2026 P3 initiation expected 2H 2026
Multiple High-Impact Pipeline Milestones in 2026: Density of Catalyst Creation Driven by QTORIN™ QTORIN™ 3.9% rapamycin anhydrous gel and QTORIN™ pitavastatin are for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency. The safety or efficacy has not been established for any use. Clinically Significant Angiokeratomas 3 Fast Track Designated Phase 2 initiated May 2026, ahead of schedule Phase 2 data expected 2H 2027 Disseminated Superficial Actinic Porokeratosis 4 QTORIN™ pitavastatin formulation developed, IP filed Phase 2 initiation expected 2H 2026 Cutaneous Venous Malformations 2 Positive Phase 2 data BTD application submitted Q2 2026 P3 initiation expected 2H 2026
Multiple High-Impact Pipeline Milestones in 2026: Density of Catalyst Creation Driven by QTORIN™ QTORIN™ 3.9% rapamycin anhydrous gel and QTORIN™ pitavastatin are for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency. The safety or efficacy has not been established for any use. Clinically Significant Angiokeratomas 3 Fast Track Designated Phase 2 initiated May 2026, ahead of schedule Phase 2 data expected 2H 2027 Disseminated Superficial Actinic Porokeratosis 4 QTORIN™ pitavastatin formulation developed, IP filed Phase 2 initiation expected 2H 2026 Announcement expected 2H 2026 Potential pipeline-in-a-product QTORIN™ Third Program 5 DAVID OSBORNE, PhD Chief Innovation Officer Cutaneous Venous Malformations 2 Positive Phase 2 data BTD application submitted Q2 2026 P3 initiation expected 2H 2026
Multiple High-Impact Pipeline Milestones in 2026: Density of Catalyst Creation Driven by QTORIN™ QTORIN™ 3.9% rapamycin anhydrous gel and QTORIN™ pitavastatin are for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency. The safety or efficacy has not been established for any use. Clinically Significant Angiokeratomas 3 Fast Track Designated Phase 2 initiated May 2026, ahead of schedule Phase 2 data expected 2H 2027 Disseminated Superficial Actinic Porokeratosis 4 QTORIN™ pitavastatin formulation developed, IP filed Phase 2 initiation expected 2H 2026 Announcement expected 2H 2026 Potential pipeline-in-a-product QTORIN™ Third Program 5 DAVID OSBORNE, PhD Chief Innovation Officer QTORIN™ rapamycin fourth indication announcement expected 2H 2026 QTORIN™ Rapamycin Fourth Indication 6 Cutaneous Venous Malformations 2 Positive Phase 2 data BTD application submitted Q2 2026 P3 initiation expected 2H 2026
Closing Remarks
What Makes Palvella Stand Apart Repeatably unlocking multi-billion dollar market opportunities in previously untreated orphan diseases First-in-Disease Focus Rare Diseases with Clear Disease Biology Leveraging Existing Human Proof-of-Concept and Safety Data Innovative QTORIN™ Platform: Durable IP Generation Veteran team executing rare disease model designed to reduce time and capital to FDA approval 1 2 3 4
PALVELLA (pɑlʋelːɑ, Finnish): TO SERVE Building the leading rare disease biopharma company focused on developing and commercializing first-in-disease therapies for serious, rare skin diseases and vascular malformations
Striving to be first for rare disease patients Thank You