Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Q1 2026 Earnings Presentation
30 slides from Palvella Therapeutics's earnings presentation, May 7, 2026
Slide 1: Q1 2026 Financial Results & Corporate Update
First-in-disease therapies for patients with rare diseases Q1 2026 Financial Results & Corporate Update May 7, 2026
Slide 2: Forward Looking Statements
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 cas
Slide 3: Q1 2026 Achievements Position Company for First Potential Approval and Launch
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
Slide 4: Rare Skin Diseases
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
Slide 5: Driving the Next Wave of Innovation in Under-appreciated 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,
Slide 6: Attracting proven commercial talents
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 ce
Slide 7: QTORIN Rapamycin: >$1bn U.S. Peak Sales Potential in Microcystic LMs
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 ch
Slide 8: Our Platform
OUR PLATFORM QTORIN™ palvella THERAPEUTICS
Slide 9: QTORIN: Repeatable New Product Development Engine
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-effici
Slide 10: Rare Disease Pipeline Overview
Rare Disease Pipeline Overview
Slide 11: SELVA: Additional Analyses Support Clinical Meaningfulness
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 a
Slide 12: Two prospective studies with statistical significance; planned 505(b)(2) NDA
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 strea
Slide 13: Strong Medical Affairs Presence at Key Medical Congresses
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 (Ph
Slide 14: Cutaneous Venous Malformations: 73% of Patients Improved in Phase 2
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 TH
Slide 15: Clinically Significant Angiokeratomas: Superficial Lymphatic Malformations
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
Slide 16: Angiokeratomas: First Patients Dosed in Phase 2 Trial
> 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
Slide 17: Disseminated Superficial Actinic Porokeratosis (DSAP)
> 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 T
Slide 18: QTORIN Rapamycin: Aligned With Palvella's Pipeline Strategy
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
Slide 19: Finance & Upcoming Catalysts
Finance & Upcoming Catalysts
Slide 20: Well-Capitalized with $230mm Oversubscribed Feb. 2026 Financing
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 QTO
Slide 21: Microcystic LMs: NDA Submission on Track for 2H 2026
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 h
Slide 22: Multiple High-Impact Pipeline Milestones in 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 ha
Slide 23: Multiple High-Impact Pipeline Milestones in 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 ha
Slide 24: Multiple High-Impact Pipeline Milestones in 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 ha
Slide 25: Multiple High-Impact Pipeline Milestones in 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 ha
Slide 26: Multiple High-Impact Pipeline Milestones in 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 ha
Slide 27: Closing Remarks
Closing Remarks palvella THERAPEUTICS
Slide 28: What Makes Palvella Stand Apart
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 Ge
Slide 29: Palvella: To Serve
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
Slide 30: Thank You
Striving to be first for rare disease patients Thank You
Palvella Therapeutics — Q1 2026 Earnings Presentation on 6ix