Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — FY2024 Earnings Presentation
27 slides from Palvella Therapeutics's earnings presentation, Mar 31, 2025
Slide 1: Full Year 2024 Financial Results & Corporate Update
First-in-disease therapies for patients with rare genetic skin diseases Full Year 2024 Financial Results & Corporate Update March 31, 2025
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 case
Slide 3: Vision: Build the leading therapeutics company focused on rare genetic skin diseases
Vision: Build the leading therapeutics company focused on rare genetic skin diseases Palvella debuted as a publicly listed company (NASDAQ:PVLA) in December 2024
Slide 4: What Sets Palvella Apart
What Sets Palvella Apart Our Mission is to Serve Patients with Rare Diseases First-in-Disease Therapies Exclusively focused on developing transformational therapies for rare diseases with no FDA approved treatments Rare Disease Expertise Team with expertise in rare disease drug development, includin
Slide 5: Our Breakthrough Innovation: QTORIN 3.9% Rapamycin Anhydrous Gel
QTORINTM 3.9% rapamycin anhydrous gel API Our Breakthrough Innovation: QTORINTM 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 tradi
Slide 6: Multiple High-Impact Milestones Over Next Quarters
Multiple High-Impact Milestones Over Next 4 Quarters Phase 3 SELVA data in microcystic LMs (Q1:26) Phase 2 TOIVA data in cutaneous VMs (Q4:25) Additional mTOR-driven indication for QTORINTM Rapamycin (2H:25) New QTORINTM Program (2H:25) QTORINTM QTORINTM PLATFORM QTORINTM 3.9% rapamycin anhydrous ge
Slide 7: QTORIN 3.9% Rapamycin: Microcystic Lymphatic Malformations
QTORINTM 3.9% RAPAMYCIN Microcystic Lymphatic Malformations FOR
Slide 8: Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong
Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong Genetics & Pathophysiology: Somatic PIK3CA mutations cause hyperactivated PI3K/mTOR Lymphorrhea: Persistent discharge of lymphatic fluid through skin layers Deep infections: Cellulitis and other serious infections Proliferation
Slide 9: Phase 3 SELVA Trial in Microcystic LM: Enrollment On Track
Phase 3 SELVA Trial in Microcystic LM: Enrollment On Track n=40; single-arm, baseline-controlled, QD dose, age 3+(1) (Mike Kelly, MD, PhD) (Kiersten Ricci, MD) (Megha Tollefson, MD) (Sally Cohen-Cutler, MD) (Elizabeth Nieman, MD) (Ionela Iacobas, MD) (Andrea Zaenglein, MD) (Steve Kempers, MD) Primar
Slide 10: 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 11: Phase 2 Results: Visible Improvement (Underarm)
Week 12 Phase 2 Results: Visible Improvement Baseline Patient CGI-C: Very Much Improved (+3). QTORINTM 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 a
Slide 12: Phase 2 Results: Visible Improvement (Wrist)
Week 12 Baseline Phase 2 Results: Visible Improvement Wrist Wrist Patient CGI-C: Very Much Improved (+3). QTORINTM 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 estab
Slide 13: Phase 2 Study Results Published in Journal of Vascular Anomalies
Phase 2 Study Results Published in Journal of Vascular Anomalies (JoVA) “Efficacy from this phase 2 study showed a robust clinical response as measured from both the clinicians’ and patients’ perspective. All 12 patients in the study demonstrated clinical and statistical improvements across a variet
Slide 14: Regulatory Overview: NDA Submission Planned for 2026
Rolling NDA submission with potential for six-month priority review planned for 2026 Regulatory Overview: NDA Submission Planned for 20261 Breakthrough Therapy Designation Fast Track Designation Orphan Drug Designation Phase 3 study in microcystic LMs ongoing; data expected in Q1 2026 Seeking full F
Slide 15: FDA Orphan Products Grant Recipient
FDA Orphan Products Grant Recipient: Announced November 2024 Based on scientific and technical merit as determined by rare disease and regulatory experts Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical developme
Slide 16: Microcystic LMs: Large U.S. Market Opportunity
Microcystic LMs: Large U.S. Market Opportunity *Includes microcystic LM and mixed LM (patients with both microcystic and macrocystic manifestations) Study conducted in partnership with Trinity Life Sciences Estimated microcystic/mixed US prevalence* 79.9K 1. Physician Survey published 2022 MULTIPLE
Slide 17: QTORIN Rapamycin: Potential to be First Line, Standard of Care Therapy
Would you incorporate Product X into your clinical practice? Would you consider this as a first-line therapy for these patients? Do you see advantages to Product X compared to orally administered mTOR or PI3K inhibitors? 98% 96% Survey of 52 high-volume dermatologists and hematologists (average of 1
Slide 18: Streamlined and Efficient Commercial Strategy Targeting Centers of Excellence
Streamlined and Efficient Commercial Strategy Targeting Concentrated Centers of Excellence (CoEs) 142 established Vascular Anomaly Centers across the U.S. Second indication (cutaneous VMs) treated at same CoEs – able to leverage synergies with Microcystic LMs Ideal for self-commercialization with fo
Slide 19: QTORIN 3.9% Rapamycin: Cutaneous Venous Malformations
QTORINTM 3.9% RAPAMYCIN Cutaneous Venous Malformations FOR
Slide 20: Cutaneous Venous Malformations: Serious, High Unmet Need
Cutaneous Venous Malformations: Serious, High Unmet Need Genetics & Pathophysiology: TIE2 or PIK3CA over-activating mutations; part of the PI3K/AKT/mTOR pathway Dysregulated growth of malformed veins impacting the skin Functional impairment Hemorrhaging Deformity as lesions continue to grow Leads to
Slide 21: Rapamycin and the treatment of venous malformations
Rapamycin and the treatment of venous malformations Summary Targeted molecular therapies are becoming interesting additions to the management of vascular anomalies, and rapamycin is establishing itself as the gold standard for venous malformations. 1 2 “Rapamycin is the first targeted therapy that i
Slide 22: Data Supporting Rapamycin Use in TIE2 and PIK3CA Mutations
“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. VENOUS MALFOMATION Pathophysiology Queisser 2021 TIE2 VENOUS MALFORMATION MODEL Preclinical Evidence Boscolo 2015
Slide 23: Cutaneous Venous Malformations Phase 2 TOIVA Study
Cutaneous Venous Malformations Phase 2 TOIVA Study n=~15; QD dose Safety Safety and tolerability Efficacy Cutaneous venous malformation – investigators’ global assessment (7-point clinician change scale) Cutaneous venous malformation - multicomponent static scale Other clinician and patient-reported
Slide 24: Market Research in Cutaneous VMs
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 25: Financial Overview
Financial Overview THERAPEUTIC
Slide 26: 2024 Financial Highlights and 2025 Outlook
2024 Financial Highlights and 2025 Outlook $83.6 million Cash at 12/31/2024 2+ years Runway into 2H 2027 $14.1 million R&D + G&A spend in 2024 >$55 million Projected cash at year end
Slide 27: Q&A
Q&A Striving to be first for rare disease patients