Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Investor Presentation (January 2026)
56 slides from Palvella Therapeutics's investor presentation, Jan 29, 2026
Slide 1: First-in-disease therapies for patients with rare diseases
First-in-disease therapies for patients with rare diseases Corporate Presentation January 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: Leadership in Treating Serious, Rare Skin Diseases and Vascular Malformations
Palvella: Leadership in Treating Serious, Rare Skin Diseases and Vascular Malformations Repeatably unlocking multi-billion dollar market opportunities in previously untreated orphan diseases …through: First-in-Disease Focus Molecules Aimed at Addressing Causal Disease Biology Innovative QTORIN™ Plat
Slide 5: What's New at Palvella (January 2026)
What’s New at Palvella (January 2026): Advancing Our Late-Stage Pipeline and QTORIN™ Platform Expanded pipeline: Four programs with first-in-disease potential in high unmet need, commercially attractive rare diseases 1 QTORIN rapamycin: $1-$3bn+ peak U.S. sales potential; pipeline-in-a-product oppor
Slide 6: QTORIN Rapamycin: Potential First FDA-Approved Therapy in Two Vascular Malformations
QTORIN™ Rapamycin: Potential First FDA-Approved Therapy and First-Line, Standard-of-Care in Two Vascular Malformations Microcystic Lymphatic Malformations > 30k U.S. patients Senior Commercial and Medical Leadership Accelerating U.S. Launch Readiness Market research indicates strong physician enthus
Slide 7: QTORIN Rapamycin: 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 8: 2025 Established the Foundation for a Catalyst-Rich 2026 and Beyond
2025 Established the Foundation for a Catalyst-Rich 2026 and Beyond 2025 Accomplishments Anticipated By YE 2026 Vision: An enduring rare disease biopharma company with 10+ first-in-disease therapies launched or in late-stage development Positive Phase 2 cVM data (December 2025) Phase 3 mLM SELVA tri
Slide 9: Our Lead Product Candidate: QTORIN 3.9% Rapamycin Anhydrous Gel
OUR LEAD PRODUCT CANDIDATE QTORIN™ 3.9% RAPAMYCIN ANHYDROUS GEL
Slide 10: 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 11: 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 12: QTORIN Rapamycin: Pipeline-in-a-Product for mTOR-Driven Skin Diseases
QTORIN™ Rapamycin: “Pipeline-in-a-Product” to Address Broad Range of mTOR-Driven Skin Diseases Larger, controlled studies are needed to define optimal concentrations, formulations, and dosing schedules. “ “ > 25 mTOR-driven skin diseases Vascular anomalies, genodermatoses, non-vascular neoplasms, an
Slide 13: Three Announced Indications for QTORIN Rapamycin in Multi-Billion Dollar Markets
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 Three Announced Indications for QTORIN™ Rapamycin in Multi-Billion Dollar Markets 1.
Slide 14: QTORIN 3.9% Rapamycin for Microcystic Lymphatic Malformations
QTORIN™ 3.9% RAPAMYCIN Microcystic Lymphatic Malformations FOR
Slide 15: Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong
Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong Genetics & Pathophysiology: Monogenic somatic PIK3CA mutations cause hyperactivated PI3K/mTOR Lymphorrhea: Persistent discharge of lymphatic fluid through skin layers Deep infections: Cellulitis and other serious infections Nat
Slide 16: QTORIN Rapamycin for Micro LMs: On Target, In Tissue
QTORIN™ Rapamycin for Micro LMs: On Target, In Tissue rapamycin Target: mTOR Tissue: Dermis Monogenic somatic mutations leads to overactivated PI3K/mTOR signaling QTORIN™ delivers pharmacologically active levels of rapamycin to diseased tissue QTORIN™ 3.9% rapamycin anhydrous gel is for investigatio
Slide 17: QTORIN Rapamycin: Phase 2 Study in Microcystic LMs
QTORIN™ Rapamycin: Phase 2 Study in Microcystic LMs n=12; QD dose James Treat, MD Joyce Teng, MD, PhD Alison Small, MD Milton Waner, MD Steve Kempers, MD Single arm, QTORIN™ rapamycin treatment (QD) (12 weeks) Baseline (4 weeks) Study Objectives: Safety and efficacy Results Clinically & statisticall
Slide 18: 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 19: Phase 2 Results: Visible Improvement
Week 12 Phase 2 Results: Visible Improvement Baseline Patient CGI-C: Very Much Improved (+3). 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 an
Slide 20: Phase 2 Results: Visible Improvement
Week 12 Baseline Phase 2 Results: Visible Improvement Wrist Wrist Patient CGI-C: Very Much Improved (+3). 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 establ
Slide 21: Phase 2 Results: Safety and Tolerability
Phase 2 Results: Safety and Tolerability TREATMENT-AFFECTED DRUG-RELATED AES RELATED ANY GRADE EVENTS (%, N=12) Application site pain 3 (25) Application site pruritus 3 (25) Application site discharge, Application site erythema, Application site paraesthesia, Nodule, Eczema, Skin exfoliation, Diarrh
Slide 22: Phase 3 SELVA Trial Design
Phase 3 SELVA Trial Design Single-arm, baseline-controlled, 24-week efficacy evaluation, QD dose, age 3+(1) 1. Patients ages 3-5 will be excluded from primary endpoint. Primary Endpoint: mLM-IGA (Investigator Global Assessment), a 7-point clinician change scale with similarities to CGI-C (used in Ph
Slide 23: Regulatory: NDA Submission Planned for 2H 2026
Regulatory: NDA Submission Planned for 2H 2026 Breakthrough, Fast Track, and Orphan Designations granted Anticipating expedited regulatory pathway: Breakthrough Therapy, Fast Track, and Orphan Drug Designations Planned 505(b)(2) pathway Potential for six-month priority review Potential for rolling N
Slide 24: 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 25: QTORIN 3.9% Rapamycin U.S. Commercial Launch Planning
QTORIN™ 3.9% RAPAMYCIN U.S. Commercial Launch Planning
Slide 26: Microcystic LMs: Multi-billion Dollar, Uncontested U.S. Market Opportunity
Microcystic LMs: Multi-billion Dollar, Uncontested U.S. Market Opportunity with Commercial Build-Out Underway ASHLEY KLINE Chief Commercial Officer Joined May 2025 Recently elucidated genetics enable development of first targeted therapy to address causal mTOR pathway with minimal side effects Large
Slide 27: Physician Market Research: Potential to be First Line, Standard of Care 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 28: Highly Concentrated Patient Population: ~400 Established 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 29: Payor Research (Q2 2025): High Unmet Need, Low Budget Impact
Payor Research (Q2 2025): High Unmet Need, Low Budget Impact Rare Disease with Positive Perception of Potential Efficacy and Safety Safety & Tolerability of Product X Clinical Efficacy of Product X L H L H Budget Impact of Disease (mLM) L H “ We are very hands off here, we leave it to the specialist
Slide 30: QTORIN Rapamycin Pricing: Prior First-in-Disease and Topical Orphan 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 31: Strong Medical Affairs Presence in 2026 and Beyond
Strong Medical Affairs Presence in 2026 and Beyond Educating on underlying genetic pathology of disease and relevance of a targeted therapeutic approach Informing on importance of early therapeutic intervention to prevent progression, especially during early childhood and puberty High touch physicia
Slide 32: QTORIN 3.9% Rapamycin for Cutaneous Venous Malformations
QTORIN™ 3.9% RAPAMYCIN Cutaneous Venous Malformations FOR
Slide 33: 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 34: Cutaneous Venous Malformations: Relentless Cycle of Microthromboses and Functional Loss
Cutaneous Venous Malformations: Relentless Cycle of Microthromboses and Functional Loss Abnormal Vessels Bleeding Chronic Micro-thromboses Height (elevation/ engorgement) Phleboliths (painful calcium deposits) TEK/PIK3CA/Other VM Mutations mTOR Hyperactivation Endothelial Dysfunction Therapeutic goa
Slide 35: Venous Malformations: Progress Towards the Potential First Targeted Therapy
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 36: 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 37: 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 38: Overall cVM-IGA: Rapid, Time Dependent Response Observed
Overall cVM-IGA: Rapid, Time Dependent Response Observed % of Participants with At Least a 2-point Improvement on Overall cVM-IGA Single-arm, baseline-controlled, QD dose, age 6+ Note: Statistical significance (p<0.05) is nominal as there was no adjustment for multiplicity amongst efficacy endpoints
Slide 39: 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 40: Phase 2 Results: Visible Improvement
Site: Cleveland Clinic Investigator: Dr. Michael Kelly Participant Age: 13 Mutation: PIK3CA cVM-IGA at Week 12: Much Improved (+2) Week 12 Phase 2 Results: Visible Improvement Note: Selective photos. Week 12 Baseline Participant Qualitative Interview: “It’s slowed down bleeding and the color has cha
Slide 41: 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 42: Market Research in Cutaneous VMs: Potential as First Line Therapy
Would you consider Product X over oral mTOR and PI3K inhibitors? Would you consider this as a first-line therapy for cutaneous VM patients? 90% 86% Survey of 50 high-volume dermatologists and hematologists with an average of 10.6 cutaneous VM patients seen per month % Yes Current options: laser trea
Slide 43: QTORIN 3.9% Rapamycin for Clinically Significant Angiokeratomas
QTORIN™ 3.9% RAPAMYCIN Clinically Significant Angiokeratomas FOR
Slide 44: 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 45: Pipeline-in-a-Product: Advancing Program to Angiokeratoma Patients
QTORIN™ Rapamycin as a “Pipeline-in-a-Product”: Advancing Program to Angiokeratoma Patients FDA meeting planned 1Q 2026 Discuss proposed Phase 2 study design Longer-term, supplemental NDA (sNDA) submission planned (if approval achieved) in microcystic LMs and/or cutaneous VMs Fast Track Designation
Slide 46: QTORIN Pitavastatin for Disseminated Superficial Actinic Porokeratosis
QTORIN™ PITAVASTATIN Disseminated Superficial Actinic Porokeratosis FOR
Slide 47: 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 48: 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 >20 subsequent supportive studies of off-label use of topical statin therap
Slide 49: 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 50: 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 51: QTORIN Pitavastatin Clinical Pathway: Planned Phase 2 in 2H 2026
QTORIN™ Pitavastatin Clinical Pathway: Planned Initiation of Phase 2 in 2H 2026 FDA meeting planned 1H 2026 Discuss proposed Phase 2 study design Discuss eligibility for expedited programs (Fast Track Designation) QTORIN™ Pitavastatin: From Concept to Clinic QTORIN™ pitavastatin optimized for stabil
Slide 52: QTORIN Pitavastatin: Future Expansion Opportunities
QTORIN™ Pitavastatin: Future Expansion Opportunities First Clinical Trial: DSAP Patients Subsequent Studies: Expand into Other Porokeratosis Subtypes DSAP: 50k+ U.S. pts Clarity Pharma (September 2025), n=277 physicians surveyed, MedaCorp Survey (August 2025), n=55 physicians surveyed. Porokeratosis
Slide 53: Finance
Finance THERAPEUTICS
Slide 54: Well-Capitalized Through Multiple Inflection Points
Well-Capitalized Through Multiple Inflection Points with Funding from Leading Healthcare-Dedicated Investors Dec. 2024 Financing Strong Cash Position Innovative operating model prioritizing capital efficiency Oversubscribed $78.9mm Financing co-led by $63.6 million Cash at 9/30/20251 $10.2 million R
Slide 55: What Sets Palvella Apart
What Sets Palvella Apart: Building The Leader in Rare Skin Diseases and Vascular Malformations Late-stage rare disease pipeline and QTORIN™ platform QTORIN™ rapamycin in two late-stage indications: Microcystic LMs (Phase 3) and cutaneous VMs (positive Phase 2 data), with clinically significant angio
Slide 56: Thank You
Striving to be first for rare disease patients Thank You
Palvella Therapeutics — Investor Presentation (January 2026) on 6ix