Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Pieris Pharmaceuticals — Merger or Acquisition (July 2024)
35 slides from Palvella Therapeutics's merger presentation, Jul 24, 2024
Slide 1: Palvella Therapeutics
palvella THERAPEUTICS
Slide 2: Forward Looking Statements
Forward Looking Statements Certain statements in this Presentation may be considered forward-ooking, Forward-Looking statements generally relateto future events or Patella Therapeutics, Inc.'s (the*Company") futuretinancit or operating performance. For example, statements regarding anticipated growt
Slide 3: Building the leading therapeutics company focused on rare genetic skin diseases
Building the leading therapeutics company focused on rare genetic skin diseases Petrichor Ligand:
Slide 4: What Sets Palvella Apart
What Sets Palvella Apart ae First-in-Disease Therapies Exclusively focused ‘on developing transformational therapies for rare diseases with no FDA approved treatments 9NQ ARare Team Team with proven history in building successful rare disease companies, including Insmed ® 4 Capital Efficiency Lean s
Slide 5: QTORIN Rapamycin: Landmark Phase 2 Study Sets Stage for Potential U.S. Launch
QTORIN™ Rapamycin: Landmark Phase 3 Study Sets Stage for Potential U.S. Launch Highly statistically significant Phase 2 results in Microcystic LMs 100% of patients (n=12) “much” or “very much” improved with QTORIN™ rapamycin on Clinician Global Impression of Change (p<0.0001) Phase 3 designed for su
Slide 6: Our Lead Innovation
OUR LEAD INNOVATION g QTORIN™ 3.9% RAPAMYCIN ANHYDROUS GEL
Slide 7: mTOR inhibition in Rare Skin Diseases: Broad Potential
mTOR inhibition in Rare Skin Diseases: Broad Potential Scientific data supports potential in many rare skin diseases JClinsight. (2026). 8. Adams ot. a, Padlatrics. (2016). 4. Red et, Lancet palvelia
Slide 8: Systemic Rapamycin Limitations Restrict Use in Genetic Skin Diseases
Systemic Rapamycin Limitations Restrict Use in Genetic Skin Diseases e Systemic Rapamycin Strong immunosuppressive activity poses significant risks to patients with localized cutaneous disease’ Systemic toxicities including stomatitis, hypertriglyceridemia, hypercholesterolemia, Gl distress, periphe
Slide 9: Our Innovation: QTORIN Platform
Our Innovation: QTORIN™ Platform Reproducible platform for generation of novel topical product candidates for rare diseases High payload capacity optimizes potential for therapeutic activity: Accommodates high API concentrations, i.e., 3.9% rapamycin Delivery to dermis with limited systemic absorpti
Slide 10: QTORIN 3.9% Rapamycin Anhydrous Gel
OTORIN™ 3.9% Rapamycin Anhydrous Gel RAPAMYCIN Direct mechanistic engagement of causal mTOR pathway rorin™ 1000x higher rapamycin levels at site of disease vs. systemic rapamycin’ TOPICAL Lil ited-to-undetectable systemic absorption? atone" 49% rapanyen ‘nhyarous gel Granted U.S. patents through at
Slide 11: QTORIN 3.9% Rapamycin for Microcystic Lymphatic Malformations and Cutaneous Venous Malformations
QTORIN™ 3.9% RAPAMYCIN FOR Microcystic Lymphatic Malformations and Cutaneous Venous Malformations
Slide 12: Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong
Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong Early onset: Presentat birth and significant impact to adolescents Lymphorrhea: Persistent discharge of lymphatic fluid through skin layers Genetics & Pathophysiology: ‘Somatic PIKSCA mutations cause hyperactivated PI3K/mTOR De
Slide 13: No Spontaneous Regression Well-Established in Micro LMs
No Spontaneous Regression Well-Established in Micro LMs Types Subtotal Sex (F:M) Age (y), mean + SD Maximum diameter (cm), mean + SD Spontaneous regression Positive 0 Negative 28 * Consistent with well-established history of PI3K Related Overgrowth Spectrum, which includes microcysticLM CONFDENTIAL|
Slide 14: QTORIN Rapamycin: On Target, In Tissue
QTORIN™ Rapamycin: On Target, In Tissue + Monogenic somatic mutations leads + QTORIN™ delivers pharmacologically to overactivated PI3K/mTOR signaling active levels of rapamycin transepidermally to diseased tissue SONFIDENTIAL | 14 palvelia
Slide 15: QTORIN Rapamycin: Phase 2 Study in Microcystic LM
QTORIN™ Rapamycin: Phase 2 Study in Microcystic LM n=12; QD dose @3:35@ James Treat,M.D. —_Joyce Teng, M.D., Ph.D. ‘Steve Kempers, M.D. Milton Waner, M.D. Alison Small, M.D. Children's Hospital mm Shir ‘School of or pniagelphis Stanford kin Care %Y MEDICINE Study Objectives: Safety and efficacy Resu
Slide 16: Phase 2: Clinically Meaningful, Statistically Significant Improvements
Phase 2: Clinically Meaningful, Statistically Significant Improvements 4 cGlc + 2.42 (p<0.0001) as Se) 3 H 3 H * 22 * } 5 H * Bis i t 8 H 4 H E H * #7 i = os t | ° Week 4 ‘Week 8 | Week 12! Week 4 Week 8 Week 12 Clinician Global Patient Global Impression of Change Impression of Change CONFIDENTIAL |
Slide 17: Phase 2: Clinically Meaningful, Statistically Significant Improvements
Phase 2: Clinically Meaningful, Statistically Significant Improvements Very Much Improved ‘Much Improved Minimat Improvement No Change Minimatty Worse Much Worse Very Much Worse ‘CONFIDENTIAL | 17 Patient # Clinician Global Impression of Change (week 12) n=12 1 Improved palvella
Slide 18: Phase 2 Results: Visible Improvement
Phase 2 Results: Visible Improvement Baseline Week 12 palve
Slide 19: Phase 2 Results: Visible Improvement
Phase 2 Results: Visible Improvement palvelia
Slide 20: Microcystic Lymphatic Malformation: Phase 2 Safety
Microcystic Lymphatic Malformation: Phase 2 All Treatment-Related Adverse Events TREATMENTRELATED AES. RELATED ANY GRADE EVENTS Application site pain Application site pruritus Application site discharge Application site erythema Application site paraesthesia Nodule Eczema Skin exfoliation Diarrhea H
Slide 21: 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 n=12 n=40 Size VUOICE (Alpelisib) for (for NDA safety database) pusck a ae Ueiaune CGI-C, a 7-point clinician mLM-IGA, a7-point clinician Overgrowth Spectrum aireromuritiuies
Slide 22: 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 24 weeks 24+ weeks ve Day 1 Baseline Primary Efficacy Photot Photo? Photo 3 Baseline (= 8 weeks) Primary Efficacy + mLM-IGA, a 7-point clinician change scale Datalanticipated Key Seconda
Slide 23: Real world evidence and OUS treatment guidelines
Real world evidence and OUS treatment guidelines Onphaet uma ohare Dasen 209) 810 Tena icn ayes oo bey ‘Sirolimus inthe Treatment of Mirooystic Lymphatic Malformations: ‘Systematic Review soy tng 0, Pa Aenean MP et PH? “Micro LMs represent therapeutically challenging congenital vascular lesions. The
Slide 24: FDA Status: NDA Submission Planned for 2026
FDA Status: NDA Submission Planned for 2026' Completed Breakthrough Therapy Designation and End of Phase 2 meetings with FDA Seeking full FDA approval based on clinical endpoints od utilized in prospective Phase 2 and Phase 3 studies Breakthrough 505(b)(2) pathway leveraging prior FDA approvals of r
Slide 25: Market research (May 2024): Strong uptake in U.S. anticipated
Market research (May 2024): Strong uptake in U.S. anticipated Percent of my %Yes microcystic LM Would you incorporate 99 patients | would Product X into your clinical prescribe Product X: Practice? “it would be a first oo choice medical Would you consider this as 3 ieranyi a first-line therapy . ¥;
Slide 26: Favorable Market Dynamics Enables Potential for Self-Commercialization
Favorable Market Dynamics Enables Potential for Self-Commercialization ONFIDENTIA First and only market position ina serious disease with no FDA-approved therapies Relatively small number of US vascular anomaly centers (n=142), mostly within academic medical centers, streamline our commercial and me
Slide 27: Cutaneous Venous Malformations: Serious, High Unmet Need
Cutaneous Venous Malformations: Serious, High Unmet Need Dysregulated growth of malformed veins impacting the skin Genetics & Pathophysiology: TIE2 or PIK3CA over activating mutations; partof the PISK/AKT/MTOR pathway Hemorrhaging Functional impairment Deformity as lesions continue to grow 1. Primar
Slide 28: Substantial body of research supporting rapamycin's potential in VM
Substantial body of research supporting rapamycin’s potentialin VM led to FDA Fast Track Designation for QTORIN™ rapamycin 8 VASCUMGRLOEDTED OY UESARLELA AE High potential of rapamycin peered tne reatmeee. se ees eae ese “Rapamycin is the first targeted therapy that improves considerably the QoL of
Slide 29: Cutaneous Venous Malformation Phase 2 Study
Cutaneous Venous Malformation Phase 2 Study n=~15; QD dose Efficacy Evaluation Period VE; Photo 1 12.weeks Baseline (= 4 weeks) Primary Efficacy + Cutaneous venous malformation — investigators’ global assessment (7-point Data anticipated clinician change scale) Q4 2025 ‘Secondary Endpoints + Cutaneo
Slide 30: QTORIN rapamycin: > $1B peak sales potential in five years
QTORIN™ rapamycin: > $1B peak sales potentialin five years" + Recent (June 2024) claims data analysis confirms significant commercial opportunity in both diseases Microcystic Lymphatic Malformations Cutaneous Venous Malformations 38K+with Microcystic disease? don Wiyeuaneousve matformatio are at pea
Slide 31: Pipeline in a Product for mTOR-driven skin diseases
Pipeline in a Product for mTOR-driven skin diseases Potential 2029 U.S. launch anticipated, i Refractory Potential 2027 U.S. Ifapproved Capillary launch anticipated, Malformations if approved 15k + patients? Microcystic Cutaneous Refractory Lymphatic Venous Vascular Malformations Tumors Malformation
Slide 32: QTORIN Platform for Additional Rare Genetic Skin Diseases
QTORIN™ PLATFORM FOR . Additional Rare Genetic Skin Diseases
Slide 33: QTORIN platform has broad potential across rare dermatological diseases
QTORIN™ platform has broad potential across rare dermatological diseases QTORIN™ Rapamycin + Microcystic lymphatic Malformations + Venous Matformat + Additional mTOR-driven indications Additional QTORIN™-enabled products + Multiple rare genodermatoses and molecules under evaluation “We have begun to
Slide 34: $30M PIPE: Sufficient Capital Runway into Q4 2026
$30M PIPE: Sufficient Capital Runway into Q4 2026 we Ph.tData Potential FDA 4" tt econ all Patient Dosed JToRIN™ O * vm TPht Ph. lt Data Patient Dosed Corporate Reverse
Slide 35: QTORIN Rapamycin: Landmark Phase 2 Study Sets Stage for Potential U.S. Launch
QTORIN™ Rapamycin: Landmark Phase 3 Study Sets Stage for Potential U.S. Launch Highly statistically significant Phase 2 results in Microcystic LMs 100% of patients (n=12) “much” or “very much” improved with QTORIN™ rapamycin on Clinician Global Impression of Change (p<0.0001) Phase 3 designed for su
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