EX-99.2 3 pvla-ex99_2.htm EX-99.2

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First-in-disease therapies for patients with rare diseases Full Year 2025 Financial Results & Corporate Update March 31, 2026


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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.


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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


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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


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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


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QTORIN™ 3.9% RAPAMYCIN Microcystic Lymphatic Malformations FOR


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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


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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.


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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


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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


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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


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QTORIN™ 3.9% RAPAMYCIN Cutaneous Venous Malformations FOR


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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


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QTORIN™ 3.9% RAPAMYCIN Clinically Significant Angiokeratomas FOR


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> 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


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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


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QTORIN™ PITAVASTATIN Disseminated Superficial Actinic Porokeratosis FOR


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> 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


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OUR PLATFORM QTORIN™


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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


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Finance


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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.


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Closing Remarks Wes Kaupinen Founder and CEO


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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


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Striving to be first for rare disease patients Thank You