Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Investor Presentation (November 2025)
23 slides from Palvella Therapeutics's investor presentation, Nov 5, 2025
Slide 1: QTORIN Pitavastatin: Developing the First Pathogenesis-directed Therapy for DSAP
1 First - in - disease therapies for patients with rare skin diseases QTORIN Pitavastatin: Developing the First Pathogenesis - directed Therapy for Disseminated Superficial Actinic Porokeratosis November 5, 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: QTORIN Pitavastatin Program: Today's Attendees
3 QTORIN Pitavastatin Program: Today’s Attendees Keith Choate , MD, PhD • Aaron B. and Marguerite Lerner Professor and Chair of Dermatology • Professor of Genetics and Pathology • Associate Dean for Physician - Scientist Development • President of the Pediatric Dermatology Research Alliance ( PeDRA
Slide 4: Multiple High-Impact Milestones Between Now and End of Q1 2026
4 Multiple High - Impact Milestones Between Now and End of Q1 2026 SEPTEMBER 2025 TODAY x Serious, rare, no FDA - approved therapies x Commercially attractive Third Planned Indication for QTORIN Rapamycin: Clinically Significant Angiokeratomas 1 Phase 2 Topline Data in Cutaneous VMs 3 x 16 subjects
Slide 5: Disease Overview & Scientific Rationale
5 Disease Overview & Scientific Rationale Dr. Keith Choate, Yale School of Medicine QTORIN Pitavastatin for Disseminated Superficial Actinic Porokeratosis
Slide 6: DSAP is a Serious Disease with No FDA-Approved Therapy
6 Disseminated Superficial Actinic Porokeratosis (DSAP) is a Serious Disease with No FDA - Approved Therapy Keith Choate , MD, PhD High impact to patient quality of life with meaningful risk of malignant transformation to squamous cell carcinomas Image sources: Sim et al, Ann Dermatol, (2018) and Dr
Slide 7: DSAP: Progressing to the First Potential Pathogenesis-directed Therapy
7 Disseminated Superficial Actinic Porokeratosis (DSAP): Progressing to the First Potential Pathogenesis - directed Therapy 2011 Amy Paller, MD Discovery that mutations in the mevalonate pathway are responsible for many genetic skin diseases Member of Palvella MSAB 2019 - 2020 Keith Choate, MD, PhD
Slide 8: Clear Biology: Targeting the Causal Mevalonate Pathway
8 Clear Biology: Targeting the Causal Mevalonate Pathway Target: Mevalonate Pathway Tissue: Epidermis & Dermis Site of pathogenesis - directed therapy An on - target, in - tissue approach could result in significant clinical improvement *HMGCR = 3 - hydroxy - 3 - methylglutaryl - coenzyme A reductas
Slide 9: Unmet Need for First FDA-approved Topical Mevalonate Pathway Inhibitor for DSAP
9 Unmet Need for First FDA - approved Topical Mevalonate Pathway Inhibitor for DSAP Proof - of - concept study, demonstrating a plausible mechanistic approach Significant need for an FDA - approved topical mevalonate pathway inhibitor >20 subsequent supportive studies of off - label use of topical s
Slide 10: QTORIN Pitavastatin Formulation Development
10 QTORIN Pitavastatin Formulation Development Dr. David Osborne, Chief Innovation Officer QTORIN Pitavastatin for Disseminated Superficial Actinic Porokeratosis
Slide 11: QTORIN Platform: Application to Mevalonate Pathway Inhibitors
11 • Recognized formulation expert with 35+ years of industry - leading experience in topical drug development • Founding scientist and CTO at Arcutis Biotherapeutics (NASDAQ: ARQT) • Contributed to the development of three dozen topical therapies, incl. ZORYVE® (roflumilast) cream and foam David Os
Slide 12: Developing the First Pathogenesis-directed Therapy For DSAP
12 Developing the First Pathogenesis - directed Therapy For DSAP Molecule: optimal potency, skin PK, and stability Topical formulation leveraging QTORIN platform high active payload, dermal penetration, limited systemic absorption, chemical stability QTORIN product candidate Targeted dermal penetrat
Slide 13: QTORIN Pitavastatin: On Target, In Tissue
13 Using QTORIN , we considered and tested a wide range of mevalonate pathway inhibitors QTORIN Pitavastatin: On Target, In Tissue QTORIN PITAVASTATIN Stability Optimal Skin PK Potency Molecule Pitavastatin Mev. Inhibitor 2 Mev. Inhibitor 3 Mev. Inhibitor 4 Mev. Inhibitor 5 Mev. Inhibitor 6 Me
Slide 14: Clinical Development Plan
14 Clinical Development Plan Dr. Jeff Martini, Chief Scientific Officer QTORIN Pitavastatin for Disseminated Superficial Actinic Porokeratosis
Slide 15: DSAP: Chronic, Pre-Cancerous, and Progressive
15 > 50k patients ESTIMATED IN THE U.S. 1 Disseminated Superficial Actinic Porokeratosis (DSAP): Chronic, Pre - Cancerous, and Progressive Persistent and extensive: Clonal proliferation of abnormal keratinocytes leads to increased number and size of lesions Risk of malignant transformation: Premalig
Slide 16: QTORIN Pitavastatin Clinical Pathway: Planned Initiation of Phase 2 in 2H 2026
16 QTORIN Pitavastatin Clinical Pathway: Planned Initiation of Phase 2 in 2H 2026 FDA meeting planned 1H 2026 • Discuss proposed Phase 2 study design • Discuss eligibility for expedited programs (Fast Track Designation) QTORIN Pitavastatin: From Concept to Clinic • Targeting <2.5 years to Phase 2 PO
Slide 17: QTORIN Pitavastatin: Future Expansion Opportunities
17 QTORIN Pitavastatin: Future Expansion Opportunities First Clinical Trial: DSAP Patients Subsequent Studies: Expand into Other Porokeratosis Subtypes DSAP: 50 k+ U.S. pts Clarity Pharma (September 2025), n=277 physicians surveyed, MedaCorp Survey (August 2025), n=55 physicians surveyed. Porokerato
Slide 18: Commercial Opportunity
18 Commercial Opportunity Matt Korenberg Chief Financial Officer QTORIN Pitavastatin for Disseminated Superficial Actinic Porokeratosis
Slide 19: Attractive U.S. Commercial Opportunity for DSAP
19 Attractive U.S. Commercial Opportunity for DSAP anticipated based on disease severity and lack of FDA - approved therapies Orphan Pricing diagnosed DSAP patients in the U.S. based on two sources (Clarity Pharma, n=277 physicians; Zagoras et al, 2023) and confirmed by KOL calls >50k Market researc
Slide 20: Key Takeaways
20 Key Takeaways Wes Kaupinen Founder & Chief Executive Officer QTORIN Pitavastatin for Disseminated Superficial Actinic Porokeratosis
Slide 21: QTORIN Pitavastatin for DSAP: Key Program Milestones Achieved
21 QTORIN Pitavastatin for DSAP: Key Program Milestones Achieved x Strong KOL support - Dr. Keith Choate, Dr. Amy Paller, Dr. Dirk Elston, Dr. Jim Treat, Dr. John Doux, and others x Known genetics and c lear biology confirmed x Analyzed published case studies of off - label statin use, confirming pr
Slide 22: QTORIN Pitavastatin (DSAP): Aligned With Palvella's Pipeline Strategy
22 QTORIN Rapamycin Rare Serious Strong Scientific & Biologic Rationale Commercially Attractive No FDA - approved Therapies Chronic, extensive lesions, malignant transformation >50k U.S. patients Mutations in mevalonate pathway lead to accumulation of toxic intermediates None Microcystic LMs, Cutane
Slide 23: Thank You
23 Striving to be first for rare disease patients Thank You
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