Palvella (NASDAQ: PVLA) wins Phase 3 QTORIN data and $230M raise
Rhea-AI Filing Summary
Palvella Therapeutics reported a larger 2025 net loss while advancing its rare‑disease pipeline and bolstering its balance sheet. The company’s lead candidate QTORIN™ rapamycin showed highly positive Phase 3 SELVA results in microcystic lymphatic malformations, with a mean mLM-IGA improvement of +2.13 (p<0.001) and strong responder rates, supporting plans for a New Drug Application in the second half of 2026 and a potential first FDA approval in the first half of 2027.
Palvella is also expanding QTORIN™ rapamycin into cutaneous venous malformations and clinically significant angiokeratomas and advancing QTORIN™ pitavastatin for disseminated superficial actinic porokeratosis. Pro forma cash was about $274 million as of December 31, 2025, reflecting net proceeds of $215.8 million from a February 2026 equity financing, compared with cash and cash equivalents of $58.0 million at year-end. For 2025, research and development expenses rose to $22.8 million and general and administrative expenses to $15.8 million, driving a net loss attributable to common stockholders of $41.7 million, or $3.71 per share.
Positive
- Phase 3 success in lead indication: QTORIN™ rapamycin met the primary endpoint in the SELVA trial for microcystic lymphatic malformations with a mean mLM-IGA improvement of +2.13 (p<0.001), strong responder rates, and a favorable safety profile, supporting an NDA submission in 2H 2026.
- Strengthened balance sheet with major financing: Pro forma cash of about $274 million as of December 31, 2025 reflects approximately $215.8 million in net proceeds from an upsized, oversubscribed $230.0 million equity offering, providing resources for late-stage development and launch preparation.
Negative
- None.
Insights
Positive Phase 3 data and a $230M raise materially de-risk Palvella’s lead program.
Palvella delivered pivotal Phase 3 SELVA success for QTORIN™ rapamycin in microcystic lymphatic malformations, with a mean mLM-IGA improvement of +2.13 and p<0.001, plus strong patient-reported benefits and favorable safety. This validates the topical mTOR approach and underpins plans for a 2H 2026 NDA submission and potential 1H 2027 approval.
The company simultaneously strengthened its capital position, reporting pro forma cash of about $274 million as of December 31, 2025 after an upsized, oversubscribed $230.0 million equity financing that yielded approximately $215.8 million in net proceeds. Management indicates this could support the microcystic LM launch, a Phase 3 program in cutaneous venous malformations, and multiple Phase 2 trials across angiokeratomas and DSAP.
R&D and G&A expenses increased to $22.8 million and $15.8 million, respectively, with net loss widening to $41.7 million. For a clinical-stage rare-disease company, higher spending is consistent with scaling late-stage development and pre-commercial infrastructure. Future filings will clarify execution across the NDA process, launch buildout, and additional QTORIN™ indications.
8-K Event Classification
Key Figures
Key Terms
Breakthrough Therapy Designation regulatory
Fast Track Designation regulatory
microcystic lymphatic malformations medical
disseminated superficial actinic porokeratosis medical
505(b)(2) submission regulatory
orphan pricing financial
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 March 31, 2026, Palvella Therapeutics, Inc. (the “Company”) announced its financial results for the year ended December 31, 2025. 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 March 31, 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 March 31, 2026* |
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Earnings Call Presentation of Palvella Therapeutics, Inc., dated March 31, 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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March 31, 2026 |
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/s/ Matthew Korenberg |
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Matthew Korenberg |
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Chief Financial Officer |
Palvella Therapeutics Reports Full Year 2025 Financial Results and Provides Corporate Update
New Drug Application (NDA) for QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations (microcystic LMs) on track for planned submission in second half of 2026
Accelerating U.S. launch readiness for QTORIN™ rapamycin for microcystic LMs; potential to become the first FDA-approved therapy and first-line, standard-of-care treatment for serious, lifelong disease affecting an estimated more than 30,000 diagnosed patients in the U.S.
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™ rapamycin for the treatment of clinically significant angiokeratomas planned for second quarter of 2026
Initiation of Phase 2 trial of QTORIN™ pitavastatin for the treatment of disseminated superficial actinic porokeratosis planned for second half of 2026
Pro forma cash of approximately $274 million as of December 31, 2025 reflects net proceeds from a February 2026 equity financing; cash and cash equivalents of $58.0 million as of December 31, 2025
Company to host conference call at 8:30 a.m. ET today
WAYNE, PA., March 31, 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 full year ending December 31, 2025 and provided a corporate update.
“2025 was a landmark year for Palvella, and we carried that momentum into 2026 with positive Phase 3 SELVA results in microcystic lymphatic malformations, marking a major milestone for the company and putting us on a path toward our first potential FDA approval in the first half of 2027,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “Following SELVA results, we significantly strengthened our balance sheet through an oversubscribed $230.0 million financing, enabling us to accelerate U.S. launch readiness and continue advancing novel topical product candidates from our QTORIN™ platform. Supported by Breakthrough Therapy, Fast Track, and Orphan designations, we are now focused on advancing QTORIN™ rapamycin toward an NDA submission while preparing for a planned standalone U.S. commercial launch. We believe this momentum positions us to advance our vision of building the leading rare disease biopharmaceutical company addressing serious, rare skin diseases and vascular malformations with no FDA-approved therapies.”
Recent Research and Development Highlights
QTORIN™ rapamycin for 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
Full Year 2025 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 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 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 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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Year Ended December 31, |
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2025 |
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2024 |
Operating expenses: |
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Research and development |
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$ 22,841 |
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$ 8,151 |
General and administrative |
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15,761 |
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5,944 |
Total operating expenses |
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38,602 |
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14,095 |
Loss from operations |
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(38,602) |
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(14,095) |
Total other income (expense), net |
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(3,113) |
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(3,339) |
Net loss |
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$ (41,715) |
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$ (17,434) |
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Net loss per share of Common Stock — basic and diluted |
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$ (3.71) |
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$ (7.83) |
Weighted-average shares used in computing net loss per share of Common Stock — basic and diluted |
11,251,250 |
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2,225,934 |
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PALVELLA THERAPEUTICS, INC.
CONDENSED CONSOLIDATED BALANCE SHEET INFORMATION
(in thousands)
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December 31, |
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December 31, |
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2025 |
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2024 |
Assets |
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Cash and cash equivalents |
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$ 57,982 |
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$ 83,602 |
Other current assets |
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1,005 |
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4,632 |
Total current assets |
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58,987 |
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88,234 |
Non-current assets |
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572 |
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— |
Total assets |
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$ 59,559 |
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$ 88,234 |
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Liabilities and Stockholders' Equity |
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Current liabilities |
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$ 11,344 |
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$ 12,038 |
Non-current liabilities |
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20,232 |
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13,589 |
Total liabilities |
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31,576 |
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25,627 |
Total stockholders' equity |
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27,983 |
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62,607 |
Total liabilities and stockholders’ equity |
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$ 59,559 |
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$ 88,234 |
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First-in-disease therapies for patients with rare diseases Full Year 2025 Financial Results & Corporate Update March 31, 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 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.
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
What Makes Palvella Stand Apart Repeatably unlocking multi-billion dollar market opportunities in previously untreated orphan diseases …through: First-in-Disease Focus Rare Diseases with Clear Disease Biology Leveraging Existing Human Proof-of-Concept Data Innovative QTORIN™ Platform: On Target, In Tissue …resulting in first-in-disease therapies with potential in: Microcystic Lymphatic Malformations Cutaneous Venous Malformations Clinically Significant Angiokeratomas Disseminated Superficial Actinic Porokeratosis …with two more to come in 2026 Focused rare disease development model designed to reduce time and capital to FDA approval
Multiple High-Impact Milestones Over Next 12+ Months: Anticipated U.S. Commercialization + Pipeline Expansion to Six Rare Diseases 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. Microcystic Lymphatic Malformations 1 Positive Phase 3 data NDA submission in 2H 2026 Cutaneous Venous Malformations 2 Positive Phase 2 data BTD application expected to be submitted Q2 2026 P3 initiation 2H 2026 Clinically Significant Angiokeratomas 3 Fast Track Designation granted Phase 2 initiation ahead of schedule, expected Q2 2026 Disseminated Superficial Actinic Porokeratosis 4 QTORIN™ pitavastatin formulation developed, IP filed Phase 2 initiation expected 2H 2026 QTORIN™ rapamycin fourth indication expected 2H 2026 QTORIN™ third program expected 2H 2026 QTORIN™ Pipeline and Platform 5 Potential FDA approval 1H 2027
QTORIN™ 3.9% RAPAMYCIN Microcystic Lymphatic Malformations FOR
QTORIN™ Rapamycin in Microcystic Lymphatic Malformations: NDA On Track for 2H 2026 with Potential Approval in 1H 2027 Potential to be first-line and standard of care for >30,000 U.S. diagnosed patients 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. Targets underlying pathobiology Large magnitude treatment effect in two prospective trials; favorable safety profile QTORIN™ 3.9% Rapamycin
Upside Target Clinical Profile SELVA Outcome Statistically significant primary endpoint with mean mLM-IGA of ≥+1.5 at Week 24 Statistical significance on independent, blinded key secondary endpoint Safety profile: well-tolerated and similar to previous clinical trials Highly statistically significant with mean mLM-IGA of +2.13 at Week 24 (p<0.001) Highly statistically significant (p<0.001), including on all three clinical signs: vesicle appearance, height, leaking/bleeding Well-tolerated across both adult and pediatric patients, supporting chronic administration : QTORIN™ Rapamycin Exceeded Upside Case Profile Rollover into extension period in line with best-in-class drugs for rare diseases 98% of Week 24 completers (43/44) rolled over to Extension period Note: Data analyzed per statistical analysis plan; non-completer data handled via multiple imputation per statistical analysis plan; endpoints tested sequentially according to pre-specified hierarchical testing; statistical significance (p<0.05). Week 24 completers included 43 participants ≥6 years old and 1 participant 3-5 years old.
Phase 3 SELVA data highly statistically significant and clinically meaningful, confirming positive Phase 2 results 1 Intent to seek broad label: age 3 and above based on clinical rationale for earlier intervention 2 Previously granted Breakthrough, Fast Track, and Orphan Designations; 505(b)(2) submission 3 Collaborating with FDA Office of Orphan Products Development, which is providing non-dilutive funding, to participate in Pre-NDA meeting 4 Regulatory: NDA Submission On Track for 2H 2026 Breakthrough, Fast Track, and Orphan Designations granted Submitted Pre-NDA meeting request to FDA, with meeting anticipated Q2 2026
QTORIN™ Rapamycin in Microcystic LMs: Characteristics of an Attractive Potential Orphan Launch Serious, rare disease with high unmet medical need and no FDA-approved therapies Enthusiasm from key opinion leaders to drive adoption Anticipated orphan pricing Positive Phase 3 data that support a favorable risk/benefit profile (February 2026) Large population of existing diagnosed patients (>30,000) with high patient concentration in vascular anomaly centers
Microcystic LMs: Significant Pre-Launch Investment Underway for Potential 1H 2027 Launch Leadership Medical Affairs Patient Identification MSL hiring underway Recruited Ashley Kline as Chief Commercial Officer (May 2025) Recruited Jen McDonough as SVP Market Access; previously helped lead launch of Vyjuvek at Krystal Biotech Next planned key hire: Head of Sales Estimated >30K diagnosed U.S. patients with ~1,500 annual incidence Concentrating resources on the highest value centers and treating physicians Focus on top 400 high volume centers (including vascular anomaly centers) comprising 50% of market
QTORIN™ 3.9% RAPAMYCIN Cutaneous Venous Malformations FOR
Cutaneous Venous Malformations: 73% of Patients Improved in Phase 2 No FDA-approved therapies Pipeline-in-a-product: sNDA planned > 75k patients ESTIMATED DIAGNOSED IN THE U.S.1 POSITIVE PHASE 2 DATA IN DEC 2025 DENISE ADAMS, MD Children’s Hospital of Philadelphia Presented at BTD Preliminary Advice meeting, guiding Phase 3 design Phase 3 planning underway: align on study design mid-2026 and initiate trial in 2H 2026 Submitting for Breakthrough Therapy Designation in Q2 2026 Breakthrough Therapy Preliminary Advice meeting completed Evidence package to include patient qualitative interviews and Phase 2 investigator letter of support to augment data Treatment extension ongoing; planned presentation of final data set at medical meeting later this year
QTORIN™ 3.9% RAPAMYCIN Clinically Significant Angiokeratomas FOR
> 50k patients ESTIMATED DIAGNOSED IN THE U.S.1 Clinically Significant Angiokeratomas: Superficial Lymphatic Malformations PHASE 2 TRIAL INITIATION EXPECTED IN Q2, EARLIER THAN PRIOR GUIDANCE OF 2H 2026 JAMES TREAT, MD Children’s Hospital of Philadelphia Member of Palvella MSAB Angiokeratomas share overlapping clinical and pathological features with microcystic LMs Aberrant lymphatic biology mTOR-driven No tendency for spontaneous regression Leveraging existing human proof-of-concept data Five case reports demonstrate preliminary clinical benefit of off-label rapamycin (Camacho, Farajzadeh, Bell, Gines, and Moeineddin) No FDA-approved therapies Pipeline-in-a-product: sNDA planned
Microcystic LMs Cutaneous VMs Clinically Significant Angiokeratomas Additional Potential Future Indications2 TODAY FUTURE Announced September 2025 Targeting announcement in 2026 and beyond = 5k 30k+ 75k+ 200k+ 50k+1 QTORIN™ Rapamycin: Fourth Indication Announcement in 2H 2026 1. Clarity Pharma research (July 2025), n=643 physicians surveyed. 2. Lapa et al., Journal of Cutaneous Medicine and Surgery, (2025). Pipeline-in-a-product strategy expands addressable U.S. patient pool by 10x beyond initial indication Estimated timeline for potential regulatory approval 2031+ 2032+ 2027 2029
QTORIN™ PITAVASTATIN Disseminated Superficial Actinic Porokeratosis FOR
> 50k patients ESTIMATED DIAGNOSED IN THE U.S.1 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 PHASE 2 INITIATION ON TRACK FOR 2H 2026 KEITH CHOATE, MD, PhD Yale School of Medicine Member of Palvella MSAB First pathogenesis-directed therapy with potential to be first-line and standard of care Recent scientific publications: Experimental Dermatology article highlighting real-world statin evidence and treatment gaps in porokeratosis AAD presentation on burden of disease Strong inbound patient interest for planned Phase 2 study
OUR PLATFORM QTORIN™
QTORIN™: New Product Development Engine VALIDATION OF QTORIN™ PLATFORM Demonstrated by two positive clinical study readouts (Phase 3 SELVA trial and Phase 2 TOIVA trial results) PLANNING TO PURSUE PLATFORM DESIGNATION We expect to apply for FDA’s Platform Technology Designation Program following QTORIN™ rapamycin’s targeted approval in 2027 SCALING OF PIPELINE WITH NEW QTORIN™ PROGRAMS Rapidly advancing and testing multiple molecules through QTORIN™ platform in capital- and time-efficient manner We plan to announce one new QTORIN™ program and one new QTORIN™ rapamycin indication later this year DAVID OSBORNE, PhD Chief Innovation Officer
Finance
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 ~$274 million pro forma cash1 1. Pro forma cash reflects $58.0 million of cash and cash equivalents as of December 31, 2025, together with the $215.8 million of net proceeds from February 2026 public offering.
Closing Remarks Wes Kaupinen Founder and CEO
What Sets Palvella Apart: Building The Leader in Rare Skin Diseases and Vascular Malformations Positive Phase 3 data in microcystic lymphatic malformations, focused on NDA submission and potential U.S. commercial launch QTORIN™ rapamycin: potential to be first approved therapy and SOC in U.S. for microcystic LMs, cutaneous VMs, and angiokeratomas U.S. commercial opportunity: multi-billion dollar TAM in mLMs, with potential to expand addressable pool of patients by 10x Striving to be first for rare disease patients Deep pipeline of rare disease therapies: six diseases anticipated by year end 2026
Striving to be first for rare disease patients Thank You