Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Investor Presentation (September 2025)
48 slides from Palvella Therapeutics's investor presentation, Sep 3, 2025
Slide 1: First-in-disease therapies for patients with rare genetic skin diseases
1 First - in - disease therapies for patients with rare genetic skin diseases Corporate Presentation September 2025
Slide 2: Forward Looking Statements
2 Forward Looking Statements This presentation contains forward - looking statements of Palvella Therapeutics, Inc. (the Company”) within the meaning of the Pr ivate Securities Litigation Reform Act of 1995. Forward - looking statements include all statements that are not historical facts, and in so
Slide 3: Building an enduring rare disease biopharma company
3 PALVELLA ( p ɑ lʋel ːɑ , Finnish) : TO SERVE Building an enduring rare disease biopharma company to address serious, rare genetic skin diseases
Slide 4: Leadership in Treating Serious, Rare Skin Diseases
4 Palvella: Leadership in Treating Serious, Rare Skin Diseases 597 Rare Skin Diseases Often chronic and debilitating; many have known genetic drivers Over 98% Have No Approved Therapies 1 Lack of industry innovation and investment compared to other therapeutic areas (e.g., oncology, neurology, pulmo
Slide 5: What's New at Palvella – September 2025
5 What’s New at Palvella – September 2025 U.S. launch planning is accelerating for microcystic LMs (first indication) • Hired Ashley Kline as Chief Commercial Officer (May 2025); previously led launch of Oxervate , first - in - disease topical therapy which surpassed $500 million in annual U.S. sale
Slide 6: Four High-Impact Milestones Between Now and End of Q1 26
6 Four High - Impact Milestones Between Now and End of Q1 2026 SEPTEMBER 2025 Q4 2025 x Serious, rare, no FDA - approved therapies x Commercially attractive QTORIN 3.9% rapamycin anhydrous gel Third Indication for QTORIN Rapamycin 1 Phase 2 Topline Data in Cutaneous VMs 3 x Targeting ~15 subjects DE
Slide 7: Our Lead Product Candidate QTORIN 3.9% Rapamycin Anhydrous Gel
7 OUR LEAD PRODUCT CANDIDATE QTORIN 3.9% RAPAMYCIN ANHYDROUS GEL
Slide 8: QTORIN: Reproducible Platform for Novel Topical Product Candidates
8 HIGH DRUG LOADING CAPACITY High solubility → high concentrations → potential for rapid onset and large magnitude treatment effect SAFETY AND TOLERABILITY Retaining active drug in the skin while minimizing systemic absorption DERMAL ENGAGEMENT Delivery to deeper layers of skin, often the site of di
Slide 9: Oral Rapamycin: Limitations Restrict Use in Skin Diseases
9 Rapamycin Oral Rapamycin: Limitations Restrict Use in Skin Diseases Significant Barriers to Commercially Viable Topical Rapamycin Formulation Palvella’s QTORIN is designed to overcome these major obstacles Current Rapamycin Options Inadequate for Skin Diseases Strong immunosuppressive activity 1 P
Slide 10: Our Breakthrough Innovation: QTORIN 3.9% Rapamycin Anhydrous Gel
10 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 inhibition 1 OPTIMIZED CONCENTRATION QTORIN synergistic solubility results in 3.9% concentration TOLERABILITY No tradition
Slide 11: Growing Commercial Opportunity: Pool of Addressable Patients
11 Micro LM Cutaneous VM Next Indication Additional Future Indications 1 TODAY FUTURE INDICATIONS Targeting announcement in September 2025 Targeting announcement in 2026 and beyond = 5k 30k+ 75k+ 200k+ 50 k+ Growing Commercial Opportunity for QTORIN Rapamycin: Pool of Addressable Patients Projected
Slide 12: QTORIN 3.9% Rapamycin for Microcystic Lymphatic Malformations
12 QTORIN 3.9% RAPAMYCIN Microcystic Lymphatic Malformations FOR
Slide 13: Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong
13 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 infection
Slide 14: QTORIN Rapamycin for Micro LMs: On Target, In Tissue
14 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 investig
Slide 15: QTORIN Rapamycin: Phase 2 Study in Microcystic LMs
15 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 & statis
Slide 16: Phase 2: Clinically Meaningful, Statistically Significant Improvements
16 -3 -2 -1 0 1 2 3 * * * * * * 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 Significan
Slide 17: Phase 2 Results: Visible Improvement
17 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 fo
Slide 18: Phase 2 Results: Visible Improvement
18 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 es
Slide 19: Phase 2 Results: Safety and Tolerability
19 Phase 2 Results: Safety and Tolerability RELATED ANY GRADE EVENTS (%, N=12) TREATMENT - AFFECTED DRUG - RELATED AES 3 (25) Application site pain 3 (25) Application site pruritus 1 (8.3) each Application site discharge, Application site erythema, Application site paraesthesia, Nodule, Eczema, Skin
Slide 20: Phase 3 SELVA Enrollment: Exceeded Target
20 Phase 3 SELVA Enrollment: Exceeded Target of 40 Patients Single - arm, baseline - controlled, 24 - week efficacy evaluation, QD dose, age 3+ (1) *Denotes sites that also participated in Phase 2 trial . 1. Patients ages 3 - 5 will be excluded from primary endpoint. Enrollment closed in June 2025 w
Slide 21: Phase 3 Study Design Mimics Phase 2 Study
21 Phase 3 Study Design Mimics Phase 2 Study Phase 3 trial design based on statistical significance with n=12 in Phase 2 Phase 2 n=12 Size CGI - C, a 7 - point clinician change scale 1 (p<0.0001) Key Efficacy Endpoints 12 weeks Study duration x Baseline Controlled x QD Dosing x Moderate to severe st
Slide 22: Regulatory: NDA Submission Planned for H2 2026
22 Regulatory: NDA Submission Planned for H2 2026 Breakthrough, Fast Track, and Orphan Designations granted Anticipating expedited regulatory pathway: i. Breakthrough Therapy, Fast Track, and Orphan Drug Designations ii. Planned 505(b)(2) pathway iii. Potential for six - month priority review iv. Po
Slide 23: QTORIN 3.9% Rapamycin U.S. Commercial Launch Planning
23 QTORIN 3.9% RAPAMYCIN U.S. Commercial Launch Planning
Slide 24: Microcystic LMs: Pre-Commercial Work Validates Multibillion-Dollar U.S. Market
24 Microcystic LMs: Pre - Commercial Work Validates Attractive Multibillion - Dollar Uncontested U.S. Market Dynamics 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 effec
Slide 25: Physician Market Research: Potential to be First Line Therapy
25 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 pa
Slide 26: Highly Concentrated Patient Population: ~400 High Volume Centers
26 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 SEGMENT
Slide 27: Payor Research (Q2 2025): Low Likelihood of Step-edits
27 Payor Research (Q2 2025): High Unmet Need Rare Disease with Low Likelihood of Step - edits 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 specialists to decide what is best. – Nationa
Slide 28: QTORIN Rapamycin Pricing: Prior First-in-Disease Launches
28 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 pr
Slide 29: Strong Medical Affairs Presence in 2026 and Beyond
29 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
Slide 30: QTORIN 3.9% Rapamycin for Cutaneous Venous Malformations
30 QTORIN 3.9% RAPAMYCIN Cutaneous Venous Malformations FOR
Slide 31: Cutaneous Venous Malformations: Serious, High Unmet Need
31 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 impacting the skin Dysfunctional venous architecture leads to bleeding, thrombosis, ulcer
Slide 32: QTORIN Rapamycin Targets Cutaneous Venous Malformations
32 Epid ermis Dermis Subcutaneous / Internal VM Cutaneous VM Bleeding, thrombosis, ulceration, disfigurement, and proliferation can significantly impact patient QoL QTORIN Rapamycin Targets Cutaneous Venous Malformations: On Target, In Tissue Current unmet need for targeted, localized therapy for Cu
Slide 33: Data Supporting Rapamycin Use in TIE2 and PIK3CA Mutations
33 “A sustained benefit of sirolimus was observed in 84% of TIE2 - mutated and 83% of the PIK3CA - mutated patients.” Data Supporting Rapamycin Use in TIE2 and PIK3CA Mutations 1. Data on file. TIE2 VENOUS MALFORMATION MODEL Preclinical Evidence Boscolo 2015 ORAL RAPAMYCIN VASE Clinical Study Seront
Slide 34: Phase 2 TOIVA Study in cVMs: Full Enrollment Anticipated Sept '25
34 Phase 2 TOIVA Study in cVMs: Full Enrollment Anticipated Sept ‘25 Single - arm, baseline - controlled, QD dose, age 6+, 12 weeks, n=~15 No Statistical Hierarchy of Endpoints Safety o Safety and tolerability Efficacy o Cutaneous venous malformation – investigators’ global assessment (7 - point cli
Slide 35: Market Research in Cutaneous VMs Indicates First Line Potential
35 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: la
Slide 36: QTORIN Platform: Additional Rare Skin Diseases
36 QTORIN PLATFORM Additional Rare Skin Diseases FOR
Slide 37: Key Value Drivers from Pipeline Programs in Second Half of 2025
37 Key Value Drivers from Pipeline Programs in Second Half of 2025 Additional mTOR - driven indication for QTORIN Rapamycin (Sept ‘25) New QTORIN Program (Q4:25) QTORIN QTORIN PLATFORM QTORIN 3.9% rapamycin anhydrous gel is for investigational use only and has not been approved or cleared by the FDA
Slide 38: Finance & IP
Finance & IP palvella THERAP EUTICS
Slide 39: Well-Capitalized Through Multiple Inflection Points
39 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 ~2 years Runway into 2H 2027 $70.4 mill
Slide 40: Strong Barriers Through Multi-Layered Exclusivity Strategy
40 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
Slide 41: What Sets Palvella Apart
41 What Sets Palvella Apart: Building The Leader in Rare Genetic Skin Diseases Late - stage rare disease pipeline and QTORIN platform QTORIN rapamycin in two ongoing studies: Phase 3 (microcystic lymphatic malformations) and Phase 2 (cutaneous venous malformations), with additional QTORIN product ca
Slide 42: Thank You
42 Striving to be first for rare disease patients Thank You
Slide 43: No Spontaneous Regression Well-Established in Microcystic LMs
43 A 34 - year, 28 - subject study confirmed no spontaneous regression No Spontaneous Regression Well - Established in Microcystic LMs * Consistent with well - established history of PI3K Related Overgrowth Spectrum, which includes microcystic LM **Kato M et al., Plast Reconstr Surg Glob Open. 2017
Slide 44: QTORIN Rapamycin: Pipeline-in-a-Product
44 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
Slide 45: Microcystic LMs: Multiple Studies Indicate >30k Diagnosed U.S. Patients
45 Microcystic LMs: Multiple Studies Indicate > 30k Diagnosed U.S. Patients *Includes microcystic LM and mixed LM (patients with both microcystic and macrocystic manifestations). 1. Claims analysis conducted by Trinity Life Sciences (June 2024). 1. Physician Survey published 2022 2. Claims Analysis
Slide 46: Regulatory: Real-world Evidence to Support NDA Submission
46 Regulatory : Real - world Evidence to Support NDA Submission Sirolimus in th e Treatment of Microcystic Lymphatic Malformations: A Systematic Review Joyce M.C. Teng, MD, PHD, 1 Adrienne Hammill, MD, PHD, 2 Jeff Martini, PHD, 3 and James Treat, MD 4 LYMPHATIC RESEARCH AND BIOLOGY ©Mary Ann Liebert
Slide 47: Venous Malformation Cases with Cutaneous Involvement
47 Multiple Studies Indicate At Least Half of Venous Malformation Cases Have Cutaneous Involvement ~80% of 1,517 cases ~47% of 2,075 cases ~65% of 118 cases Percentage of venous malformation cases with cutaneous involvement
Slide 48: Palvella Capitalization Detail
48 Palvella Capitalization Detail 1. Includes 0.2 million of BVF Partners converted preferred shares. 2. Based on closing price of $50.13 as of 8/25/25. Does n ot include dilutive impact from 2.7 million options outstanding with a weighted average exercise price of $13.56. Basic Shares Outstanding 1
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