Palvella QTORIN rapamycin receives FDA Priority Review
Palvella reported $236.7 million in cash, cash equivalents and short-term investments as of September 30, 2026.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Rhea-AI Filing Summary
Palvella Therapeutics (PVLA) said the FDA accepted its NDA for QTORIN™ rapamycin for microcystic lymphatic malformations and granted Priority Review, with a PDUFA target action date of February 28, 2027. The product remains investigational; preparations for a standalone U.S. launch are underway for the first half of 2027, if approved. Palvella estimates more than 30,000 diagnosed U.S. children and adults have the condition.
Cash, cash equivalents and short-term investments were $236.7 million as of September 30, 2026. Third-quarter R&D expenses were $10.8 million versus $6.5 million a year earlier; G&A expenses were $11.9 million versus $3.6 million, and net loss was $24.6 million versus $11.3 million. Palvella is enrolling patients in a Phase 2 angiokeratoma trial, with topline results expected in the second half of 2027; Phase 3 cutaneous venous malformation trial initiation remains on track for the fourth quarter of 2026, and QTORIN™ pitavastatin Phase 2 initiation is expected in the fourth quarter of 2026. Chief Medical Officer James R. Treat, M.D., is appointed effective December 21, 2026.
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Positive
- None.
Negative
- Moderate pointQ3 net loss rose to $24.6 million from $11.3 million.
Filing Explained
The release reports 15,844,715 shares outstanding as of
8-K Event Classification
Key Figures
Key Terms
Priority Review regulatory
PDUFA target action date regulatory
Fast Track regulatory
open-label extension study medical
pre-funded warrants financial
Earnings Snapshot
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What were PVLA's Q3 2026 financial results?
When is the FDA's target action date for QTORIN rapamycin?
AI-generated analysis. How Rhea-AI works. Not financial advice.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): |
(Exact name of registrant as specified in its charter)
(State or other jurisdiction |
(Commission File Number) |
(IRS Employer |
||
|
|
|
|
|
|
||||
|
||||
(Address of principal executive offices) |
|
(Zip Code) |
||
Registrant’s telephone number, including area code: ( |
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Securities registered pursuant to Section 12(b) of the Act:
Title of each class |
|
Trading Symbol(s) |
|
Name of each exchange on which registered |
|
|
The |
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 November 5, 2026, Palvella Therapeutics, Inc. (the “Company”) announced its financial results for the quarter ended September 30, 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 November 5, 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.
(d) Exhibits
Exhibit No. |
|
Description |
99.1 |
|
Press Release of Palvella Therapeutics, Inc., dated November 5, 2026* |
99.2 |
|
Earnings Call Presentation of Palvella Therapeutics, Inc., dated November 5, 2026* |
104 |
|
Cover Page Interactive Data File (embedded within the Inline XBRL document) |
* Furnished herewith
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.
|
|
|
Palvella Therapeutics, Inc. |
|
|
|
|
Date: |
November 5, 2026 |
By: |
/s/ Matthew Korenberg |
|
|
|
Matthew Korenberg |
|
|
|
Chief Financial Officer |
Palvella Therapeutics Reports Third Quarter 2026 Financial Results and Provides Corporate Update
FDA Accepts NDA for QTORIN™ Rapamycin and Grants Priority Review for Microcystic Lymphatic Malformations; PDUFA Target Action Date Set for February 28, 2027
QTORIN™ Rapamycin Could Become the First FDA-Approved Therapy for Microcystic Lymphatic Malformations, a Serious, Rare Lifelong Disease Affecting an Estimated More Than 30,000 Diagnosed Children and Adults in the U.S.
Palvella Advancing Preparations for Standalone U.S. Commercial Launch of QTORIN™ Rapamycin in First Half of 2027, if Approved
Additional QTORIN™ Programs Accelerating in Cutaneous Venous Malformations, Clinically Significant Angiokeratomas, and Disseminated Superficial Actinic Porokeratosis; Two Additional Programs Anticipated to be Announced by Year-End
Cash, Cash Equivalents and Short-Term Investments of $236.7 Million as of September 30, 2026
Palvella to Host Third Quarter 2026 Financial Results and Corporate Update Conference Call at 8:30 a.m. ET Today
WAYNE, PA., November 5, 2026 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. (Palvella or “the Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies for serious, rare skin diseases and vascular anomalies for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today reported financial results for the third quarter ending September 30, 2026 and provided a corporate update.
“With the FDA’s acceptance of our NDA for QTORIN™ rapamycin in microcystic lymphatic malformations and granting of Priority Review, we believe we are closer than ever to bringing our first therapy to patients,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics. “Our commercial and medical leadership is now in place and we are actively recruiting additional top talent to shape an exceptional launch for the microcystic LM community, if approved by the FDA, with the goal of establishing QTORIN™ rapamycin as the first-line treatment and standard of care for this serious, lifelong disease. Our focus is clear: successfully obtain FDA approval and bring our first therapy to patients, advance multiple additional QTORIN™ programs to approval, and build the leading company addressing serious, rare skin diseases and vascular anomalies.”
Recent Program 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)
Pipeline expansion
Recent Corporate Highlights
Third Quarter 2026 Financial Results
Conference Call Details
Palvella will host a conference call and live audiovisual webcast to discuss the Company's third quarter 2026 financial results and provide a corporate update at 8:30 a.m. ET today. To access the live webcast, including presentation slides, please click here or visit the “Events & Presentations” section of Palvella’s website. To access the conference call by phone, register using this link, and you will be provided with dial-in details. A replay of the webcast will be available approximately two hours after the conclusion of the call and will remain 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 living with serious, rare skin diseases and vascular anomalies 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 anomalies, 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 and anticipated timing of regulatory submissions, Palvella’s expectations with respect to the potential approval of the NDA for QTORIN™ rapamycin, the potential impact of Priority Review on the FDA’s review timeline, any delays in the FDA’s review of Palvella’s accepted NDA, Palvella’s commercial preparedness and the timing and execution of a potential launch following regulatory approval, 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 make regulatory submissions on anticipated timelines; 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
Vice President of Investor Relations and Corporate Affairs
Palvella Therapeutics
marcy.nanus@palvellatx.com
PALVELLA THERAPEUTICS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
PALVELLA THERAPEUTICS, INC.
CONDENSED CONSOLIDATED BALANCE SHEET INFORMATION
(in thousands)
First-in-disease therapies for patients with rare diseases Q3 2026 Financial Results & Corporate Update November 5, 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 with respect to the potential approval of the NDA for QTORIN™ rapamycin, the potential impact of Priority Review on the FDA's review timeline, any delays in the FDA's review of Palvella's accepted NDA, 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 commercial preparedness and the timing and execution of a potential launch following regulatory approval, 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.
QTORIN™ Rapamycin: Microcystic LM Opens the Door to Many mTOR-driven Diseases October 2026 NDA Accepted with Priority Review 1H 2027 Standalone U.S. Launch if Approved >30,000 estimated diagnosed U.S. patients1 Potentially first FDA approved mLM therapy February 28, 2027 PDUFA Target Action Date 2029+ Additional Potential Approvals in mTOR-Driven Diseases >300,000 U.S. patients across diseases1 1. Clarity Pharma, Trinity Life Sciences, MedaCorp, and ZS Associates.
What Makes Palvella Stand Apart Repeatably unlocking multi-billion dollar market opportunities in previously untreated orphan diseases Zero to One: 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
Shaping an Exceptional Launch for mLM Patients
Ashley Kline Chief Commercial Officer Led U.S. launch of Oxervate® a topical medicine for neurotrophic keratitis, a rare eye disease Oxervate® reached >$1 billion in U.S. sales seven years after launch Joined Palvella ~18 months ago; prior roles at Dompé, Genentech, and Bain Consulting Proven Orphan Launch Leadership, Applied With Urgency Significant progress across crucial keys to launch success Strong Prescribing Intent >70% plan to prescribe within 3 months of approval Clear Path to Coverage Payer research has confirmed pricing and access assumptions Precision Patient-Finding Multiple patient identification approaches deployed Keys to a Successful Orphan Launch
>70% of surveyed physicians expect to begin prescribing within 3 months of approval Physician Survey (~150 treating U.S. physicians) Product profile supported by compelling clinical evidence package Responses weighted by patient volume and adjusted for overstatement. Physicians evaluated a blinded product profile. Source: Palvella-sponsored market research (survey of 150 U.S. physicians; interviews with 14 physicians and 10 payers), April–June 2026. >75% would start several patients at once, then expand Payer Survey (10 U.S. payers) mLM is recognized as a serious, genetic vascular anomaly Clinical diagnosis with no step-through anticipated Favorable coverage anticipated at orphan pricing corridor New Market Research Reinforces Potential for Strong Orphan Launch Post-SELVA market research further substantiates previous launch positioning
Proven Launch Leaders Recruited to Serve the mLM Community Anticipated launch in 1H 2027 >70 field-based team members at launch Sales Focused on winners from first-in-indication launches Field Marketing HCP education beyond sales call points In-House Patient Team Personalized support for patients Medical Affairs Deep discussions with providers on our science Precision Digital Effort Physician National Prescriber Identification (NPI)-targeted strategy informed by claims data Leadership team brings deep expertise in both rare disease and dermatology
Broad applicability potential: pediatric and adult, all severities, treatment-naïve and experienced Long-term chronic administration anticipated Position as first-line and establish as the new standard of care QTORIN™ Rapamycin: Opportunity to be the First FDA-approved Therapy for Estimated >30K Microcystic LM Patients in the U.S. 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. QTORIN™ 3.9% Rapamycin Microcystic Lymphatic Malformations
Rare Disease Pipeline Updates
FDA Rare Disease Framework Public Meeting (October 2026) FDA PUBLIC MEETING Rare Disease Framework Initiative Public Meeting October 28, 2026 FDA White Oak Campus, Silver Spring, MD Hosted by the Reagan-Udall Foundation for the FDA “ “Dermatology has more rare diseases than any other specialty.” Dr. Matthew Zirwas, Board-certified dermatologist Public comment, October 28, 2026
Deep Pipeline in Serious, Rare Skin Diseases and Vascular Anomalies Phase 3 PRODUCT CANDIDATE DISEASE ESTIMATED U.S. PREVALENCE1 CLINICAL STATUS Preclinical Phase 1 Phase 2 UPCOMING MILESTONES QTORIN™ Rapamycin 3.9% anhydrous gel Microcystic Lymphatic Malformations PDUFA: Feb 28, 2027 Clinically Significant Angiokeratomas Phase 2 data expected 2H 2027 Cutaneous Venous Malformations >75,000 Phase 3 trial initiation expected Q4 2026 QTORIN™ Pitavastatin Disseminated Superficial Actinic Porokeratosis (DSAP) PDUFA: Feb 28, 2027 Phase 2 Completed Phase 2 Ongoing Phase 2 Ready >30,000 FDA DESIGNATIONS Breakthrough Therapy Fast Track Orphan Fast Track Phase 2 trial initiation expected Q4 2026 >50,000 >50,000 1. Clarity Pharma, Trinity Life Sciences, MedaCorp, and ZS Associates. Announcement expected Q4 2026 Third QTORIN™ Program Fourth QTORIN™ rapamycin indication Phase 2 Ready TBD Announcement expected Q4 2026 Serious, rare disease TBD Fast Track Planned Planned Planned Announcement expected 2027+ Additional New QTORIN™ Programs Serious, rare diseases TBD Planned
Why the Microcystic LM Field is Moving Toward Early Intervention THE BIOLOGY Somatic PIK3CA mutations keep the growth pathway always “on”1 LM is proliferative and progressive, not a static lesion Luks et al., J Pediatr 2015 (Boston Children’s)1 85% Boston Children’s2 of childhood LMs progressed by adulthood 2.8x Stanford3 higher odds of progression without treatment “Early intervention should be considered…because of their tendency to progress.”2 1. Luks VL et al. J Pediatr. 2015;166:1048–54. 2. Hassanein AH et al. J Craniofac Surg. 2012;23(1):149–52. 3. Patel V et al. J Clin Exp Dermatol Res. 2017;8(6):424.
No FDA-approved therapies Fast Track Designation Granted Pipeline-in-a-product: sNDA planned > 75k patients ESTIMATED DIAGNOSED IN THE U.S. Phase 3 initiation on track for Q4 2026 Significant treater overlap with microcystic lymphatic malformations Positive Phase 2 TOIVA data with 73% of patients improved on cVM-IGA at Week 12 24-week data presented at PeDRA October 2026 Additional improvement between Week 12 and Week 24 on multiple endpoints Data demonstrated reduction in disease severity and bleeding in pediatric patients after treatment Cutaneous Venous Malformations Near-term Phase 3 initiation on track QTORIN™ RAPAMYCIN HAS POTENTIAL TO BE FIRST APPROVED THERAPY
Topline results (n=~15) expected in 2H 2027 Phase 2 LOTU study is a 12-week, single-arm, baseline-controlled clinical trial evaluating QTORIN™ rapamycin topically once daily Strong enrollment momentum and site engagement Highly symptomatic with bleeding and painful clots; sensitive location limits destructive interventional treatments Clinically Significant Angiokeratomas Superficial lymphatic malformations UNDER-RECOGNIZED MARKET OPPORTUNITY Market research (n=50 physicians): 96% would incorporate QTORIN™ rapamycin into their practice > 50k patients ESTIMATED DIAGNOSED IN THE U.S. No FDA-approved therapies Fast Track Designation Granted Pipeline-in-a-product: sNDA planned
QTORIN™ pitavastatin: First pathogenesis-directed therapy Milestones: No FDA-approved therapies Current options: Laser, surgery, and off-label topical chemo agents & mevalonate pathway inhibitors POTENTIAL TO BE FIRST FDA-APPROVED THERAPY AND STANDARD OF CARE Disseminated Superficial Actinic Porokeratosis Chronic, pre-cancerous, and progressive FALL CLINICAL DERMATOLOGY CONFERENCE® New claims analysis
October 2026 ~155K diagnosed U.S. DSAP patients First real-world claims analysis-based characterization of the diagnosed U.S. DSAP population Formulation development completed Q4 2026: Phase 2 initiation anticipated Licensed Yale U.S. patent and filed IP Multiple IND-enabling tox studies completed Yale collaboration: pharmacodynamic proof of principle with pitavastatin reversing hyperproliferation in a human porokeratosis cell model
Finance & Cash Update
Key strategic priorities accelerating: NDA accepted with Priority Review; PDUFA date of February 28, 2027 Commercial and medical affairs expansion Major QTORIN™ pipeline and platform value drivers $236.7 million 9/30/26 cash Cash, cash equivalents and short-term investments U.S. Launch and Rare Disease Pipeline are Well-Funded with $236.7 Million on Balance Sheet as of 9/30/26
Closing Remarks
Palvella: Driving the Next Wave of Innovation in Under-Appreciated Rare Skin Diseases and Vascular Malformations 1. Clarity Pharma, Trinity Life Sciences, MedaCorp, and ZS Associates. 2. MedaCorp market research. Diagnosed US Prevalence (Estimated)1 Number of FDA Approved Therapies Physicians Who Would Consider QTORIN™ Rapamycin / Pitavastatin as First-line Therapy2 30k+ Microcystic LMs 75k+ Cutaneous VMs 50k+ Clinically Significant Angiokeratomas 50k+ Disseminated Superficial Actinic Porokeratosis None > 80%
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