Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — FY2025 Earnings Presentation
25 slides from Palvella Therapeutics's earnings presentation, Mar 31, 2026
Slide 1: Full Year 2025 Financial Results & Corporate Update
First-in-disease therapies for patients with rare diseases Full Year 2025 Financial Results & Corporate Update March 31, 2026
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 rare disease biopharma company
PALVELLA (pɑlʋelːɑ, Finnish): TO SERVE Building the leading rare disease biopharma company focused on developing and commercializing first-in-disease therapies for serious, rare skin diseases and vascular malformations
Slide 4: What Makes Palvella Stand Apart
What Makes Palvella Stand Apart Repeatably unlocking multi-billion dollar market opportunities in previously untreated orphan diseases …through: First-in-Disease Focus Rare Diseases with Clear Disease Biology Leveraging Existing Human Proof-of-Concept Data Innovative QTORIN™ Platform: On Target, In
Slide 5: Multiple High-Impact Milestones Over Next 12+ Months
Multiple High-Impact Milestones Over Next 12+ Months: Anticipated U.S. Commercialization + Pipeline Expansion to Six Rare Diseases QTORIN™ 3.9% rapamycin anhydrous gel and QTORIN™ pitavastatin are for investigational use only and has not been approved or cleared by the FDA or by any other regulatory
Slide 6: QTORIN 3.9% Rapamycin: Microcystic Lymphatic Malformations
QTORIN™ 3.9% RAPAMYCIN Microcystic Lymphatic Malformations FOR
Slide 7: QTORIN Rapamycin in Microcystic LMs: NDA On Track for 2H 2026
QTORIN™ Rapamycin in Microcystic Lymphatic Malformations: NDA On Track for 2H 2026 with Potential Approval in 1H 2027 Potential to be first-line and standard of care for >30,000 U.S. diagnosed patients QTORIN™ 3.9% rapamycin anhydrous gel is for investigational use only and has not been approved or
Slide 8: Upside Target Clinical Profile vs SELVA Outcome
Upside Target Clinical Profile SELVA Outcome Statistically significant primary endpoint with mean mLM-IGA of ≥+1.5 at Week 24 Statistical significance on independent, blinded key secondary endpoint Safety profile: well-tolerated and similar to previous clinical trials Highly statistically significan
Slide 9: Phase 3 SELVA data highly statistically significant and clinically meaningful
Phase 3 SELVA data highly statistically significant and clinically meaningful, confirming positive Phase 2 results 1 Intent to seek broad label: age 3 and above based on clinical rationale for earlier intervention 2 Previously granted Breakthrough, Fast Track, and Orphan Designations; 505(b)(2) subm
Slide 10: Characteristics of an Attractive Potential Orphan Launch
QTORIN™ Rapamycin in Microcystic LMs: Characteristics of an Attractive Potential Orphan Launch Serious, rare disease with high unmet medical need and no FDA-approved therapies Enthusiasm from key opinion leaders to drive adoption Anticipated orphan pricing Positive Phase 3 data that support a favora
Slide 11: Significant Pre-Launch Investment Underway for Potential 1H 2027 Launch
Microcystic LMs: Significant Pre-Launch Investment Underway for Potential 1H 2027 Launch Leadership Medical Affairs Patient Identification MSL hiring underway Recruited Ashley Kline as Chief Commercial Officer (May 2025) Recruited Jen McDonough as SVP Market Access; previously helped lead launch of
Slide 12: QTORIN 3.9% Rapamycin: Cutaneous Venous Malformations
QTORIN™ 3.9% RAPAMYCIN Cutaneous Venous Malformations FOR
Slide 13: Cutaneous Venous Malformations: 73% of Patients Improved in Phase 2
Cutaneous Venous Malformations: 73% of Patients Improved in Phase 2 No FDA-approved therapies Pipeline-in-a-product: sNDA planned > 75k patients ESTIMATED DIAGNOSED IN THE U.S.1 POSITIVE PHASE 2 DATA IN DEC 2025 DENISE ADAMS, MD Children’s Hospital of Philadelphia Presented at BTD Preliminary Advice
Slide 14: QTORIN 3.9% Rapamycin: Clinically Significant Angiokeratomas
QTORIN™ 3.9% RAPAMYCIN Clinically Significant Angiokeratomas FOR
Slide 15: Clinically Significant Angiokeratomas: Superficial Lymphatic Malformations
> 50k patients ESTIMATED DIAGNOSED IN THE U.S.1 Clinically Significant Angiokeratomas: Superficial Lymphatic Malformations PHASE 2 TRIAL INITIATION EXPECTED IN Q2, EARLIER THAN PRIOR GUIDANCE OF 2H 2026 JAMES TREAT, MD Children’s Hospital of Philadelphia Member of Palvella MSAB Angiokeratomas share
Slide 16: QTORIN Rapamycin: Fourth Indication Announcement in 2H 2026
Microcystic LMs Cutaneous VMs Clinically Significant Angiokeratomas Additional Potential Future Indications2 TODAY FUTURE Announced September 2025 Targeting announcement in 2026 and beyond = 5k 30k+ 75k+ 200k+ 50k+1 QTORIN™ Rapamycin: Fourth Indication Announcement in 2H 2026 1. Clarity Pharma resea
Slide 17: QTORIN Pitavastatin: Disseminated Superficial Actinic Porokeratosis
QTORIN™ PITAVASTATIN Disseminated Superficial Actinic Porokeratosis FOR
Slide 18: DSAP: Chronic, Pre-Cancerous, and Progressive
> 50k patients ESTIMATED DIAGNOSED IN THE U.S.1 Disseminated Superficial Actinic Porokeratosis (DSAP): Chronic, Pre-Cancerous, and Progressive No FDA-approved therapies Current options: Laser, surgery, and off-label topical chemo agents & mevalonate pathway inhibitors QTORIN™ PITAVASTATIN PHASE 2 IN
Slide 19: Our Platform: QTORIN
OUR PLATFORM QTORIN™ THERAPEUTICS
Slide 20: QTORIN: New Product Development Engine
QTORIN™: New Product Development Engine VALIDATION OF QTORIN™ PLATFORM Demonstrated by two positive clinical study readouts (Phase 3 SELVA trial and Phase 2 TOIVA trial results) PLANNING TO PURSUE PLATFORM DESIGNATION We expect to apply for FDA’s Platform Technology Designation Program following QTO
Slide 21: Finance
Finance THERAPEUTICS
Slide 22: Well-Capitalized Following $230mm Oversubscribed Feb. 2026 Financing
Well-Capitalized with High-Quality Investor Participation in $230mm Oversubscribed Feb. 2026 Financing Following Positive SELVA Data Potential to Fund Through: QTORIN™ + 505(b)(2) + rare disease focus offers potential for attractive ROI NDA filing, FDA approval, and, if approved, U.S. launch for QTO
Slide 23: Closing Remarks
Closing Remarks Wes Kaupinen Founder and CEO
Slide 24: What Sets Palvella Apart: Building The Leader in Rare Skin Diseases
What Sets Palvella Apart: Building The Leader in Rare Skin Diseases and Vascular Malformations Positive Phase 3 data in microcystic lymphatic malformations, focused on NDA submission and potential U.S. commercial launch QTORIN™ rapamycin: potential to be first approved therapy and SOC in U.S. for mi
Slide 25: Thank You
Striving to be first for rare disease patients Thank You