Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Investor Presentation (April 2026)
48 slides from Palvella Therapeutics's investor presentation, Apr 7, 2026
Slide 1: First-in-disease therapies for patients with rare diseases
First-in-disease therapies for patients with rare diseases Corporate Presentation April 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: Building the leading rare disease biopharma company
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 4: What's New at Palvella (April 2026)
What’s New at Palvella (April 2026): Building the Leading Rare Disease Company Microcystic LMs: Potential to be first and only FDA-approved therapy for >30,000 diagnosed U.S. patients Positive Phase 3 data announced Feb ’26: All pre-specified endpoints p<0.001; additional data to be presented at upc
Slide 5: A Clear Path Forward for Patients: QTORIN Rapamycin at Week 24
mLM-IGA = Microcystic Lymphatic Malformation Investigator Global Assessment. Note: Data analyzed per statistical analysis plan; non-completer data imputed per statistical analysis plan for primary endpoint; endpoints tested according to pre-specified hierarchical testing procedure; statistical signi
Slide 6: Multiple High-Impact Milestones Over Next 12+ Months
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
Slide 7: What Makes Palvella Stand Apart
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
Slide 8: Our Lead Product Candidate: QTORIN 3.9% Rapamycin Anhydrous Gel
OUR LEAD PRODUCT CANDIDATE QTORIN™ 3.9% RAPAMYCIN ANHYDROUS GEL
Slide 9: QTORIN: Reproducible Platform for Novel Topical Product Candidates
HIGH DRUG LOADING CAPACITY High solubility → high concentrations → potential for rapid onset and large magnitude treatment effect TOLERABILITY Retaining active drug in the skin while minimizing systemic absorption DERMAL ENGAGEMENT Delivery to deeper layers of skin, often the site of disease pathoph
Slide 10: Our Breakthrough Innovation: QTORIN 3.9% Rapamycin Anhydrous Gel
QTORIN™ 3.9% rapamycin anhydrous gel Our Breakthrough Innovation: QTORIN™ 3.9% Rapamycin Anhydrous Gel DERMAL ENGAGEMENT Rapamycin concentration in dermis exceeds IC90 for mTOR inhibition1 OPTIMIZED CONCENTRATION QTORIN™ synergistic solubility results in 3.9% concentration TOLERABILITY No traditiona
Slide 11: Pipeline-in-a-product strategy expands addressable patient pool
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 resea
Slide 12: Multi-Billion Dollar Market Opportunities in Two Lead Indications
QTORIN™ Rapamycin: Multi-Billion Dollar Market Opportunities in Two Lead Indications >75K diagnosed U.S. patients2 Cutaneous Venous Malformations Potential $1bn-$3bn+ U.S. peak sales potential >30K diagnosed U.S. patients1 Microcystic Lymphatic Malformations Annual ~$100k-$200k pricing range per pat
Slide 13: QTORIN 3.9% Rapamycin for Microcystic Lymphatic Malformations
QTORIN™ 3.9% RAPAMYCIN Microcystic Lymphatic Malformations FOR
Slide 14: Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong
Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong Genetics & Biology: Monogenic somatic PIK3CA mutations cause hyperactivated PI3K/mTOR Lymphorrhea: Persistent discharge of lymphatic fluid through skin layers Deep infections: Recurrent cellulitis and serious soft tissue infect
Slide 15: Phase 3 SELVA Trial Design
Phase 3 SELVA Trial Design Single-arm, baseline-controlled, QD dose Primary Endpoint: mLM-IGA (Investigator Global Assessment) Key Secondary Endpoint: Blinded mLM Multi-Component Static Scale (mLM-MCSS): independent, blinded review of randomized Baseline and Week 24 photos evaluating three key signs
Slide 16: QTORIN Rapamycin Exceeded Upside Case Profile
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 significan
Slide 17: SELVA: Primary and Key Secondary Endpoints Achieved
SELVA: Primary and Key Secondary Endpoints Achieved Mean Change at Week 24 (95% CI) p-value Primary Endpoint: Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA)* +2.13 (1.88, 2.38) p<0.001 Key Secondary Endpoint: Blinded Microcystic Lymphatic Malformation Multi-Component Sta
Slide 18: Phase 3 Results: Well-Tolerated and Favorable Safety Profile
Phase 3 Results: Well-Tolerated and Favorable Safety Profile Rapamycin levels were below 2 ng/mL2 in systemic circulation on for all participants at all timepoints in the study Note: n=50 participants in efficacy evaluation evaluated for safety. 1. Denotes related or possibly related. 2. Validated a
Slide 19: Phase 3 Results: Age 10, Male, mLM-IGA +2
Week 24 Baseline Phase 3 Results: Age 10, Male, mLM-IGA: +2 “Much Improved” Note: Selective photos. Thigh Thigh
Slide 20: Phase 3 Results: Age 7, Female, mLM-IGA +3
Week 24 Baseline Phase 3 Results: Age 7, Female, mLM-IGA: +3 “Very Much Improved” Note: Selective photos. Elbow Elbow
Slide 21: Phase 2: Clinically Meaningful, Statistically Significant Improvements
* * * * * * Statistically significant across key clinician-assessed individual signs of microcystic LM at week 12 Height (p<0.0001) Leaking (p<0.005) Bleeding (p<0.05) Erythema (p<0.005) Hyperkeratosis (p<0.005) Phase 2: Clinically Meaningful, Statistically Significant Improvements Mean CGI-C: + 2.4
Slide 22: Regulatory: NDA Submission On Track for 2H 2026
Phase 3 SELVA data highly statistically significant and clinically meaningful, confirming positive Phase 2 results 1 Seeking traditional approval (not accelerated) based on clinical endpoints; intent to seek broad label age 3 and above 2 Previously granted Breakthrough, Fast Track, and Orphan Design
Slide 23: Strong Barriers Through Multi-Layered Exclusivity Strategy
Strong Barriers Through Multi-Layered Exclusivity Strategy Granted U.S. Patents Trade Secrets Regulatory Orphan drug designation and 7-year data exclusivity from anticipated FDA approval Multiple trade secrets related to proprietary formulation processes and manufacturing know-how 6 issued U.S. pate
Slide 24: QTORIN 3.9% Rapamycin U.S. Commercial Launch Planning
QTORIN™ 3.9% RAPAMYCIN U.S. Commercial Launch Planning
Slide 25: Multi-billion Dollar, Uncontested U.S. Market Opportunity
Microcystic LMs: Multi-billion Dollar, Uncontested U.S. Market Opportunity with Commercial Build-Out Underway Large orphan market: Claims analysis presented at 2025 Society for Investigative Dermatology (SID) Meeting verified > 30k estimated diagnosed U.S. patients, with >1,500 incident patients ann
Slide 26: Physician Market Research: Potential to be First Line Therapy
QTORIN™ Rapamycin Physician Market Research: Potential to be First Line, Standard of Care Therapy for Microcystic LMs Product X: Topical 3.9% rapamycin gel, including results from Phase 2 study of QTORIN™ rapamycin Core Intent to Prescribe Insights 98% of physicians Percent of my microcystic LM pati
Slide 27: Highly Concentrated Patient Population: ~400 High Volume Centers
Highly Concentrated Patient Population: ~400 Established High Volume Centers for Treating Vascular Malformations Comprise ~50% of Market Source: Palvella and ZS Associates claims analysis (2025). 1. Cohen-Cutler et al., The Journal of Pediatrics, (2023). CLAIMS ANALYSIS SUPPORTS THREE SEGMENTS ~50%
Slide 28: QTORIN Rapamycin Pricing: Prior First-in-Disease Launches
1. Medscape Medical News interview with Mandy Fahey, Insmed Director of Corporate Communications (2018). 2. Assumes one course of 8-week therapy for both eyes. 3. Horizon Therapeutics Management guidance and Wall Street research. Price dependent on weight-based dosing. 4. Insmed corporate presentati
Slide 29: QTORIN 3.9% Rapamycin for Cutaneous Venous Malformations
QTORIN™ 3.9% RAPAMYCIN Cutaneous Venous Malformations FOR
Slide 30: Cutaneous Venous Malformations: Serious, High Unmet Need
Cutaneous Venous Malformations: Serious, High Unmet Need Genetics & Pathophysiology: Monogenic TIE2 or PIK3CA over-activating mutations; part of the PI3K/mTOR pathway Dysregulated growth of malformed veins and hyperproliferation of immature venous endothelial cells Dysfunctional venous architecture
Slide 31: Progress Towards the Potential First Targeted Therapy for Cutaneous Disease
Venous Malformations: Progress Towards the Potential First Targeted Therapy for Unaddressed Cutaneous Disease Known Genetics / Clear Biology Real-world Evidence Real-world evidence supports rapamycin’s off-label use in primarily internal manifestations of VMs… …however, poor patient outcomes persist
Slide 32: Phase 2 TOIVA Study in cVMs: 24-Week Study
Phase 2 TOIVA Study in cVMs: 24-Week Study Single-arm, baseline-controlled, QD dose, age 6+ Safety and Tolerability Efficacy (no pre-specified primary endpoint): cVM-IGA (7-point clinician change scale), cutaneous VM multi-component static scale (cVM-MCSS), other clinician- and patient-reported outc
Slide 33: Overall cVM-IGA: 73% of Participants Demonstrated Improvement at Week 12
Overall cVM-IGA: 73% of Participants Demonstrated Improvement at Week 12 Single-arm, baseline-controlled, QD dose, age 6+ Overall cVM-IGA: 7-point clinician-assessed dynamic change scale ranging from “Very Much Worse” (-3) to “Very Much Improved” (+3) Mean effect size at week 12: +1.5 (p<0.001) Medi
Slide 34: Phase 2 Results: Visible Improvement
Site: CHOP Investigator: Dr. Denise Adams Participant Age: 17 Mutation: TEK cVM-IGA at Week 12: Very Much Improved (+3) Week 12 Baseline Phase 2 Results: Visible Improvement Participant Qualitative Interview: “I’ve definitely noticed some improvements…it’s definitely had a positive effect…It’s more
Slide 35: Phase 2 Results: Safety and Tolerability
Phase 2 Results: Safety and Tolerability QTORIN™ rapamycin was generally well-tolerated, similar to previous clinical trials Most common Treatment-Emergent Adverse Events were application site reactions (erythema, 25%) All Treatment-Related Adverse Events were moderate or mild (no severe events) Maj
Slide 36: QTORIN 3.9% Rapamycin for Clinically Significant Angiokeratomas
QTORIN™ 3.9% RAPAMYCIN Clinically Significant Angiokeratomas FOR
Slide 37: Clinically Significant Angiokeratomas: Superficial Lymphatic Malformations
> 50k patients ESTIMATED DIAGNOSED IN THE U.S.1 Clinically Significant Angiokeratomas: Superficial Lymphatic Malformations Persistent and extensive: Lesions can be large and increase in size, number, and extent over time Chronically debilitating lymphatic-derived skin lesions associated with bleedin
Slide 38: QTORIN Pitavastatin for Disseminated Superficial Actinic Porokeratosis
QTORIN™ PITAVASTATIN Disseminated Superficial Actinic Porokeratosis FOR
Slide 39: DSAP: Chronic, Pre-Cancerous, and Progressive
> 50k patients ESTIMATED DIAGNOSED IN THE U.S.1 Disseminated Superficial Actinic Porokeratosis (DSAP): Chronic, Pre-Cancerous, and Progressive Persistent and extensive: Clonal proliferation of abnormal keratinocytes leads to increased number and size of lesions Risk of malignant transformation: Prem
Slide 40: Unmet Need for First FDA-approved Topical Mevalonate Pathway Inhibitor for DSAP
Unmet Need for First FDA-approved Topical Mevalonate Pathway Inhibitor for DSAP Proof-of-concept study, demonstrating a plausible mechanistic approach Significant need for an FDA-approved topical mevalonate pathway inhibitor Recently published systematic review in Clinical and Experimental Dermatolo
Slide 41: Clear Biology: Targeting the Causal Mevalonate Pathway
Clear Biology: Targeting the Causal Mevalonate Pathway Target: Mevalonate Pathway Tissue: Epidermis & Dermis Site of pathogenesis-directed therapy An on-target, in-tissue approach could result in significant clinical improvement *HMGCR = 3-hydroxy-3-methylglutaryl-coenzyme A reductase. Image sources
Slide 42: QTORIN Pitavastatin: On Target, In Tissue
Using QTORIN™, we considered and tested a wide range of mevalonate pathway inhibitors QTORIN™ Pitavastatin: On Target, In Tissue QTORIN™ PITAVASTATIN Molecule Potency Optimal Skin PK Stability Pitavastatin Mev. Inhibitor 2 Mev. Inhibitor 3 Mev. Inhibitor 4 Mev. Inhibitor 5 Mev. Inhibitor 6 Mev. Inhi
Slide 43: QTORIN Platform
QTORIN™ Platform palvella THERAPEUTICS
Slide 44: QTORIN: New Product Development Engine
QTORIN™: New Product Development Engine VALIDATION OF QTORIN™ PLATFORM 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
Slide 45: Finance
Finance palvella THERAPEUTICS
Slide 46: Well-Capitalized Following $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 47: What Sets Palvella Apart
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 mi
Slide 48: Thank You
Striving to be first for rare disease patients Thank You
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