Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Investor Presentation (January 2025)
38 slides from Palvella Therapeutics's investor presentation, Jan 13, 2025
Slide 1: Palvella Therapeutics
palvella THERAPEUTICS
Slide 2: Forward Looking Statements
Forward Looking Statements 1965, Foror "could" "would" "shoul,” "expect" “intend,” “plan,” “objective,” “anticipete,” “belive,” “estimste,” “predict,” “potential,” “continue,” “ongoing.” ‘Company's ‘he abiityof the Company to cefenaitsintaloctual propery. TheCompeny Undertake no duty to update these
Slide 3: Building the leading therapeutics company focused on rare genetic skin diseases
Building the leading therapeutics company focused on mony (NASDAQ:PVLA) rare genetic skin diseases toeasat 2024
Slide 4: What Sets Palvella Apart: Building The Leader in Rare Genetic Skin Diseases
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 cand
Slide 5: Well-Capitalized Through Multiple Inflection Points with Funding
Well-Capitalized Through Multiple Inflection Points with Funding from Syndicate of Leading Healthcare-Dedicated Investors Palvella closed reverse merger in December 2024 and debuted as publicly listed company (NASDAQ:PVLA) Funded with approximately $78.9 million of cash at close of reverse merger an
Slide 6: Building On Milestones Achieved in Q4:2024
Building On Milestones Achieved in Q4:2024 to Drive Sustained Momentum in 2025 and Beyond Notable Q4:24 Developments Potential 2025+ Catalysts Initiated dosing in Phase 3 SELVA trial in microcystic LMs and Phase 2 TOIVA trial in cutaneous VMs Complete enrollment in Phase 3 SELVA and Phase 2 TOIVA tr
Slide 7: Our Lead Product Candidate
OUR LEAD PRODUCT CANDIDATE q QTORIN™ 3.9% RAPAMYCIN ANHYDROUS GEL
Slide 8: Our Breakthrough Innovation: QTORIN Platform
Our Breakthrough Innovation: QTORIN™ Platform Reproducible platform for generation of novel topical product candidates for rare diseases... Targeted Anhydrous Formulations Manufacturing Applying established QTORIN ‘manufacturing to additional novel product candidates High payload capacity optimizes
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 Refractory Vascular Tumors? Microcystic LMs* indoations Capittary Malformations! Cutaneous Sarcoidosis! Venous Malformations? 2010, 2 Sant a, line 2029). 2,rerbereret ast Demat 201) 4 Mars a} 201
Slide 10: Systemic Rapamycin Limitations Restrict Use in Genetic Skin Diseases
Systemic Rapamycin Limitations Restrict Use in Genetic Skin Diseases Strong immunosuppressive activity poses significant risks to patients with localized cutaneous disease’ Systemic Rapamycin Systemic toxicities including stomatitis, hypertriglyceridemia, hypercholesterolemia, GI distress, periphera
Slide 11: QTORIN 3.9% Rapamycin Anhydrous Gel
QTORIN™ 3.9% Rapamycin Anhydrous Gel RAPAMYCIN, Direct mechanistic é engagement of causal RS mTOR pathway Torin 1000x higher rapamycin Ee “ roan levels at site of disease ‘ase! vs. systemic rapamycin? =< TOPICAL Limited-to-undetectable systemic absorption? Granted U.S. patents through at least 2038
Slide 12: QTORIN 3.9% Rapamycin: Microcystic Lymphatic Malformations
QTORIN™ 3.9% RAPAMYCIN ° Microcystic Lymphatic Malformations
Slide 13: Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong
Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong Early onset: Present at birth and significant impact to adolescents Genetics & Lymphorrhea: Persistent discharge Pathophysiology: of lymphatic fluid through skin layers Deep infections: Cellulitis and other serious infections P
Slide 14: No Spontaneous Regression Well-Established in Microcystic LMs
No Spontaneous Regression Well-Established in Microcystic LMs A 34-year, 28-subject study confirmed no spontaneous regression ‘Types Microcystic Subtotal 28 Sex (F:M) 144 Age (y), mean + SD 0.8914 Maximum diameter (em), mean + SD — Spontaneous regression Positive 0 Negative 28 * consistent with well
Slide 15: QTORIN Rapamycin: On Target, In Tissue
QTORIN™ Rapamycin: On Target, In Tissue Target: mTOR Tissue: Dermis + Monogenic somatic mutations leads + QTORIN™ delivers pharmacologically to overactivated PI3K/mTOR signaling active levels of rapamycin transepidermally to diseased tissue roninns, palvella
Slide 16: Real World Evidence and OUS Treatment Guidelines
Real World Evidence and OUS Treatment Guidelines Tasislagsa neous oy ‘Sli inthe rt npg M0; Ane. Mr? “micro LMs represent therapeutically challenging congenital vascular lesions. There is no universally accepted gold standard of care and there are no FDA ‘approved therapies...this review examines cli
Slide 17: QTORIN Rapamycin: Phase 2 Study in Microcystic LMs
QTORIN™ Rapamycin: Phase 2 Study in Microcystic LMs n=12; QD dose 3 > 3 James Treat, M.D. Joyce Teng, M.D., Ph.D. ‘Steve Kempers, M.D. Milton ‘Waner, M.D. Alison Small, M.D. ildren's Hospital an Shir ‘School of Chtgrens Hospital i) Stanford kin, Care ‘6¥ MEDICINE Baseline (4week Study Objectives: Sa
Slide 18: Phase 2: Clinically Meaningful, Statistically Significant Improvements
Phase 2: Clinically Meaningful, Statistically Significant Improvements ory Much Improved Much Improved Minimal Improvernent No Change Minimally ‘Worse Much Worse Very Much ‘Were a Hi H H 4 H CGI-C, +2.42 (p<0.0001) Week 4 Week Weak12 Week4 Weak ® Week 12 Clinician Globat Impression of Change + =pval
Slide 19: Phase 2 Results: Visible Improvement
Phase 2 Results: Visible Improvement Week 12 ne FDA orbyany other regulatory agency. palvella
Slide 20: Phase 2 Results: Visible Improvement
Phase 2 Results: Visible Improvement Baseline Week 12 palvella
Slide 21: Microcystic Lymphatic Malformation: Phase 2 Safety
Microcystic Lymphatic Malformation: Phase 2 All Treatment-Related Adverse Events Low blood levels of rapamycin detected in some patients: 120.98 pg/mL (mean) Application site pain 3 (25) Application site pruritus 3 (25) Application site discharge 1(8.3) Application site paraesthesia 1 (8.3) Nodule 1
Slide 22: Phase 2 Study Results Published in Journal of Vascular Anomalies (JoVA)
Phase 2 Study Results Published in Journal of Vascular Anomalies (JoVA) Journal of VaScular Anomalies imphatie ‘malformations, bres tt Mate Jo Conor lo Sa, ar, Mtn ne Je Teg “Efficacy from this phase 2 study showed a robust clinical response as measured from both the clinicians’ and patients’ perspe
Slide 23: SELVA Phase 3 Study: Single-Arm, Baseline-Controlled
SELVA Phase 3 Study: Single-Arm, Baseline-Controlled n=40; QD dose Efficacy Evaluation Period ‘Treatment Extension 2aweeks 24+ weeks we Day 1 Basetine Photo 2 Primary Eticacy Photo Ezititeriew with with poricipant participant Primary Efficacy * mLM-IGA, a 7-point clinician change scale aoun ia a Dat
Slide 24: Phase 3 Pivotal Study Design Mimics Phase 2 Study
Phase 3 Pivotal Study Design Mimics Phase 2 Study Phase 3 trial design based on statistical significance with n=12 in Phase 2 sise 12 n=40 (for NDA safety database) Kecetnme CGI-C, a7-point clinician mLM-IGA, @ 7-point clinician eet ene change scale (p<0.0001)' change scale (primary)? ‘Study duratio
Slide 25: 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 26: Regulatory Overview: NDA Submission Planned for 2026
Regulatory Overview: NDA Submission Planned for 2026! Phase 3 study in Seeking full FDA approval 505(b)(2) pathway microcystic LMs ongoing; based on clinical endpoints leveraging prior FDA data expected in Q1 2026 utilized in prospective Phase approvals of 2and Phase 3 studies rapamycin Rolling NDA
Slide 27: Market Research (May 2024): Strong Uptake in U.S. Anticipated
Market Research (May 2024): Strong Uptake in U.S. Anticipated Percent of my microcystic LM Would you incorporate patients | would Product X into your clinical prescribe Product X: practice? Would you consider this as a first-line therapy 7 5 % for these patients? Do you see advantages of topical adm
Slide 28: Favorable Market Dynamics Enable Potential for Self-Commercialization
Favorable Market Dynamics Enable Potential for Self-Commercialization 1 2 3 Relatively small number of US Well-defined disease with clear First and only market postion vascular anomaly centers diagnostic paramet ina serious disease with (n=142), mostly within including alignment from the FDA-approve
Slide 29: QTORIN Rapamycin: >$1bn Sales Potential in Five Years
QTORIN™ Rapamycin: >$1bn Sales Potential in Five Years! + Claims data analysis confirms significant commercial opportunity in both diseases Orphan pricing anti id isease launches and recent topical orphan launches both support orphan pricing
Slide 30: QTORIN 3.9% Rapamycin: Cutaneous Venous Malformations
QTORIN™ 3.9% RAPAMYCIN FOR . Cutaneous Venous Malformations
Slide 31: Cutaneous Venous Malformations: Serious, High Unmet Need
; > 75k patients Cutaneous Venous Malformations: ESTIMATED DIAGNOSED INTHE Serious, High Unmet Need Dysregulated growth of malformed veins impacting the skin Genetics & Hemorrhaging Pathophysiology: TIE2 or PIK3CA over- activating mutations; \ y eee iia Functional impairment Leads to physical & func
Slide 32: Substantial Body of Research Supporting Rapamycin's Potential in VM
Substantial Body of Research Supporting Rapamycin’s Potential in VM Led to FDA Fast Track Designation for QTORIN™ Rapamycin - Summary Takeaways CFS sewn setoct emer Ua REA ARSE C+) High potential of rapamycin Rapamycin and the treatment of venous malformations ‘Serort, Emmanuel; Van Damme, An °*; Bo
Slide 33: Cutaneous Venous Malformations Phase 2 Study
Cutaneous Venous Malformations Phase 2 Study n=~15; QD dose VE; Photo 1 Baseline Efficacy Evaluation Period oe Eatenterview with with patipant artcipant Safety + Safety and tolerability Efficacy Data anticipated clinician change scale) Q4 2025 Guaneous eros matoratonmuttomponent ati sale 1 and patie
Slide 34: cVM Market Research (Sept 2024): Potential to be First Line Therapy
cVM Market Research (Sept 2024): Potential to be First Line Therapy Product X: Topical 3.9% rapamycin gel Yes 9 Percent of my cutaneous VM patients Would you consider Product | would prescribe | mTOR and “Patients deserve Product X: this novel treatment” Would you consider this as a g fe) first-line
Slide 35: 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 OTORIN™> PLATFORM Additional QTORIN™-enabled products + Multiple rare genodermatoses and y molecules under eval
Slide 36: What Sets Palvella Apart: Building The Leader in Rare Genetic Skin Diseases
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 cand
Slide 37: Thank You
~~. Thank You for rare disease patients palvella
Slide 38: Palvella Senior Leadership Team
Palvella Senior Leadership Team 269 Matinew Kornberg Jetta, Ph Kathy Goin
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