Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Investor Presentation (March 2025)
37 slides from Palvella Therapeutics's investor presentation, Mar 4, 2025
Slide 1: Palvella Therapeutics
palvella THERAPEUTICS
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 therapeutics company focused on rare genetic skin diseases
Building the leading therapeutics company focused on rare genetic skin diseases Palvella debuted asa publicly listed company (NASDAQ:PVLA) in December 2024
Slide 4: What Sets Palvella Apart
What Sets Palvella Apart O* Q 8 iad 00 First-in-Disease Rare Disease Capital Late-stage Pipeline Therapies Expertise Efficiency and Platform + Exclusively focused on * Team with expertise in * Disciplined approach + Lead product developing rare disease drug to operating business candidate, QTORIN™ t
Slide 5: QTORIN Platform: Focused on Rare Genetic Skin Diseases
QTORIN™ Platform: Focused on Rare Genetic Skin Diseases Designing product candidates with transformational clinical impact in rare diseases... Tunable Anhydrous Manufacturing Formulations ; . Applying established QTORIN™ manufacturing to additional novel product candidates High active concentrations
Slide 6: Multiple High-Impact Milestones Over Next Quarters
Multiple High-Impact Milestones Over Next 4 Quarters Additional mTOR-driven indication for QTORIN™ Rapamycin (2H:25) mn EA | Phase 3 SELVA data in Phase 2 TOIVA datain microcystic LMs (Q1:26) cutaneous VMs (Q4:25) N y A ~OforIN™ palvella
Slide 7: Well-Capitalized Through Multiple Inflection Points with Funding from Leading Healthcare-Dedicated Investors
Well-Capitalized Through Multiple Inflection Points with Funding from Leading Healthcare-Dedicated Investors Strong Cash Position Oversubscribed PIPE Financing (Dec. 2024) PIPE financing of $78.9 million concurrent BVF FRAZIER with reverse merger PARTNERS LP BIEE SGIERSES Cash position expected to f
Slide 8: Our Lead Product Candidate
® pee ° . OUR LEAD PRODUCT CANDIDATE e ee QTORIN™ 3.9% -. RAPAMYCIN *o5 ee ANHYDROUS GEL THERAPEUTICS
Slide 9: Broad Potential for mTOR Inhibition in Rare Skin Diseases
Broad Potential for mTOR Inhibition in Rare Skin Diseases mTOR is a key driver for genetic skin diseases Rena cony Subsequent Vascular Tumors? a Microcystic LMs* Additional Potential Lead ar ae Indications Indications Capillary Malformations! Venous Malformations? Cutaneous Sarcoidosis® 5 1.Adamset
Slide 10: Oral Systemic Rapamycin Limitations Restrict Use in Genetic Skin Diseases
Oral Systemic Rapamycin Limitations Restrict Use in Genetic Skin Diseases Strong immunosuppressive activity poses significant risks to patients with . localized cutaneous disease! Systemic Rapamycin Oral Rapamycin 10, Systemic toxicities is NOT including stomatitis, hypertriglyceridemia, FDA-approve
Slide 11: Significant Barriers to Commercially Viable Topical Rapamycin Formulation
Significant Barriers to Commercially Viable Topical Rapamycin Formulation VEX Poor Solubility Restricted Skin Penetration Chemically Unstable Leads to technical challenges in Due to rapamycin’s high Sensitive molecule that is achieving optimal concentrations molecular weight (significantly susceptib
Slide 12: Our Breakthrough Innovation: QTORIN 3.9% Rapamycin Anhydrous Gel
Our Breakthrough Innovation: QTORIN™ 3.9% Rapamycin Anhydrous Gel OPTIMIZED CONCENTRATION QTORIN synergistic solubility results in 3.9% concentration DERMAL ENGAGEMENT ar F rapamycin concentration i m3 QToRIN™ Stable at room temperature in dermis exceeds IC90 ea for >2 years for mTOR inhibition’ | —
Slide 13: QTORIN Rapamycin: On Target, In Tissue
QTORIN™ Rapamycin: On Target, In Tissue Target: mTOR Tissue: Dermis ne expression * Monogenic somatic mutations leads * QTORIN™ delivers pharmacologically to overactivated PI3K/mTOR signaling active levels of rapamycin transepidermally to diseased tissue 6 QTORIN' 3.9% rapamycin anhydrous gets for i
Slide 14: QTORIN 3.9% Rapamycin Microcystic Lymphatic Malformations
QTORIN™ 3.9% RAPAMYCIN 2 Microcystic Lymphatic Malformations THERAPEUTICS
Slide 15: Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong
Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong Early onset: Present at birth and significant impact to adolescents Lymphorrhea: Persistent discharge of lymphatic fluid through skin layers Genetics & Pathophysiology: Somatic PIK3CA mutations cause hyperactivated PI3K/mTOR De
Slide 16: QTORIN Rapamycin: Phase 2 Study in Microcystic LMs
QTORIN™ Rapamycin: Phase 2 Study in Microcystic LMs n=12; QD dose @3:3s5@ James Treat, MD Joyce Teng, MD, PhD Steve Kempers, MD Milton Waner, MD Alison Small, MD cular CH | ] Stanford hace School of LUBE institute Bast MEDICINE Baseline Single arm, QTORIN™ rapamycin treatment (QD) (4 weeks) (12 week
Slide 17: Phase 2: Clinically Meaningful, Statistically Significant Improvements
Phase 2: Clinically Meaningful, Statistically Significant Improvements Very Much Improved Much Improved Minimal Improvement No Change Minimally Worse Much Worse ~ VeryMuch ~ Worse 17 Mean CGI-C, + 2.42 (p<0.0001) Week4 Week8 Week 12 Week4 Week8 Week 12 Statistically significant across key clinician-
Slide 18: Phase 2 Results: Visible Improvement
Phase 2 Results: Visible Improvement Baseline palvella nly and has not been approved or cleared by the FDA or by any other res
Slide 19: Phase 2 Results: Visible Improvement
Phase 2 Results: Visible Improvement Baseline palvella for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency. hed for any use.
Slide 20: Microcystic Lymphatic Malformation: Phase 2 Safety
Microcystic Lymphatic Malformation: Phase 2 All Treatment-Related Adverse Events ow blood levels of rapamycin detected insome patients: 120.98 pg/mL (mean) QTORIN™ rapamycin had favorable safety profile and was well tolerated TREATMENT RELATED AES BEDE ANY Gnace EVENTS 2) All Treatment Related Adver
Slide 21: Phase 2 Study Results Published in Journal of Vascular Anomalies (JoVA)
Phase 2 Study Results Published in Journal of Vascular Anomalies (JoVA) nemttnemte on Journal of Phase 2 study of the safety and efficacy of Vascular QTORIN rapamycin in the treatment of microcystic Anomalies lymphatic malformations James Treat, Jetrey Martin?, Jason T. Connor, Alison Small, Tracy F
Slide 22: SELVA Phase 3 Study: Single-Arm, Baseline-Controlled
SELVA Phase 3 Study: Single-Arm, Baseline-Controlled n=40; QD dose, age 3+") MAYO y UNC CLINIC tea, {Sally Cohen-Cutler, MD) (Kiersten Ricci, MD) HEALTH. Gy z me Cues (Elizabeth Nieman, MD) (lonela lacobas, MD) Stanford ‘DTIC (megha Tollefson, mp) PennState MEDICINE id [STUDY CENTER] Health UoyceTen
Slide 23: Phase 3 Study Design Mimics Phase 2 Study
Phase 3 Study Design Mimics Phase 2 Study Phase 3 trial design based on statistical significance with n=12 in Phase 2 h D Bhased Phase 3 primary nD =O endpoint (mLM-IGA) (p<0.0001) (for NDA safety database) eS Phase 2 CGI-C endpoint CGI-C, a 7-point clinician mLM-IGA, a 7-point clinician ° qemu eeee
Slide 24: FDA Orphan Products Grant Recipient: Announced November 2024
FDA Orphan Products Grant Recipient: Announced November 2024 Based on scientific and technical merit as determined by rare disease and regulatory experts Out of 51 grant applications received by the FDA Orphan Products Grants Program in fiscal year 2024, Palvella’s clinical trial was one of seven ne
Slide 25: Regulatory Overview: NDA Submission Planned for 2026
Regulatory Overview: NDA Submission Planned for 2026" g Y ZN Phase 3 study in Seeking full FDA approval Leveraging 505(b)(2) microcystic LMs ongoing; based on clinical endpoints pathway and real- data expected in Q1 2026 utilized in prospective Phase world clinical 2 and Phase 3 studies evidence of
Slide 26: Market Research in Microcystic LMs (May 2024)
Market Research in Microcystic LMs (May 2024): Strongly indicates QTORIN™ rapamycin’s potential as first line therapy Product X: topical 3.9% rapamycin gel % 98% 9 8 (0) (+) “It would be a first choice medical » ». therapy” would incorporate would consider Product X Product X into clinical as a firs
Slide 27: Streamlined and Efficient Commercial Strategy Targeting Centers of Excellence
Streamlined and Efficient Commercial Strategy Targeting Concentrated Centers of Excellence (CoEs) 27 142 established Vascular Anomaly Centers across the U.S. Ideal for self-commercialization with focused sales force and medical affairs teams Second indication (cutaneous VMs) treated at same CoEs - a
Slide 28: QTORIN 3.9% Rapamycin for Cutaneous Venous Malformations
QTORIN™ 3.9% RAPAMYCIN FOR ° Cutaneous Venous Malformations THERAPEUTICS
Slide 29: Cutaneous Venous Malformations: Serious, High Unmet Need
Cutaneous Venous Malformations: Serious, High Unmet Need veins impacting the skin Genetics & Pathophysiology: TIE2 or PIK3CA over- activating mutations; part of the PI3K/AKT/mTOR pathway Hemorrhaging Functional impairment Deformity as lesions continue to grow 29 1. Primary prospective research condu
Slide 30: Substantial Body of Research Supporting Rapamycin's Potential in VM Led to FDA Fast Track Designation
Substantial Body of Resear ch Supporting Rapamycin’s Potential in VM Led to FDA Fast Track Designation for QTORIN™ Rapamycin yet GAS. vascu.aR BOLOGY EOTED BY M LUBA FLELAARESFE Rapamycin and the treatment of venous malformations Seront, Emmanuel*®; Van Damme, An ®=; Boon, Laurence M* Vikkula, Miikk
Slide 31: Cutaneous Venous Malformations Phase 2 TOIVA Study
Cutaneous Venous Malformations Phase 2 TOIVA Study n=~15; QD dose Study objectives: evaluate safety and tolerability (incl. determining systemic concentration of rapamycin) and evaluate efficacy across multiple endpoints (no statistical hierarchy) Efficacy Evaluation Period Treatment Extension Perio
Slide 32: Market Research in Cutaneous VMs (Sept 2024)
Market Research in Cutaneous VMs (Sept 2024): Strongly indicates QTORIN™ rapamycin’s potential as first line therapy Product X: topical 3.9% r. mycin gel 9 9 0 % 8 6 % “This product would be life changing for current patients with limited » » treatment options” would consider Product X would conside
Slide 33: QTORIN Rapamycin: >$1bn Sales Potential
QTORIN™ Rapamycin: >$1bn Sales Potential + Claims data analysis confirms significant commercial opportunity in both diseases Microcystic Lymphatic Malformations Cutaneous Venous Malformations = 5 >30K with microcystic disease’ + Zomowinh cutaneous venous malformations? phan pricing anticipated -dise
Slide 34: QTORIN Platform has Broad Potential Across Rare Dermatological Diseases
QTORIN™ Platform has Broad Potential Across Rare Dermatological Diseases QTORIN™ Rapamycin * Microcystic Lymphatic Malformations * Venous Malformations * Additional mTOR-driven indications PLATFORM Additional QTORIN™-enabled products + Multiple rare genodermatoses and molecules under evaluation “We
Slide 35: Thank You
- Thank You [ a >, Striving tobe first for rare disease patients palvella THERAPEUTICS
Slide 36: No Spontaneous Regression Well-Established in Microcystic LMs
No Spontaneous Regression Well-Established in Microcystic LMs ORIGINAL ARTICLE 8 Reconstructive Regression of Lymphangiomas A34-year, 28-subject study confirmed no spontaneous regression Types Microcystic Subtotal 28 Sex (F:M) 14:14 Age (y), mean + SD 0.89+41.4 Maximum diameter (cm), mean + SD — Spo
Slide 37: Palvella Capitalization Detail
Palvella Capitalization Detail Number of Shares (millions) AA 25 13.7 ~$265mm palvel snot include dutiveimpactfrom 2.5 mil
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