Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Q3 2026 Earnings Presentation
22 slides from Palvella Therapeutics's earnings presentation, Nov 5, 2026
Slide 1: Q3 2026 Financial Results & Corporate Update
First-in-disease therapies for patients with rare diseases Q3 2026 Financial Results & Corporate Update November 5, 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: QTORIN Rapamycin: Microcystic LM Opens the Door to Many mTOR-driven Diseases
QTORIN™ Rapamycin: Microcystic LM Opens the Door to Many mTOR-driven Diseases October 2026 NDA Accepted with Priority Review 1H 2027 Standalone U.S. Launch if Approved >30,000 estimated diagnosed U.S. patients1 Potentially first FDA approved mLM therapy February 28, 2027 PDUFA Target Action Date 202
Slide 4: What Makes Palvella Stand Apart
What Makes Palvella Stand Apart Repeatably unlocking multi-billion dollar market opportunities in previously untreated orphan diseases Zero to One: First-in-Disease Focus Rare Diseases with Clear Disease Biology Leveraging Existing Human Proof-of-Concept and Safety Data Innovative QTORIN™ Platform:
Slide 5: Shaping an Exceptional Launch for mLM Patients
Shaping an Exceptional Launch for mLM Patients
Slide 6: Keys to a Successful Orphan Launch
Ashley Kline Chief Commercial Officer Led U.S. launch of Oxervate® a topical medicine for neurotrophic keratitis, a rare eye disease Oxervate® reached >$1 billion in U.S. sales seven years after launch Joined Palvella ~18 months ago; prior roles at Dompé, Genentech, and Bain Consulting Proven Orphan
Slide 7: New Market Research Reinforces Potential for Strong Orphan Launch
>70% of surveyed physicians expect to begin prescribing within 3 months of approval Physician Survey (~150 treating U.S. physicians) Product profile supported by compelling clinical evidence package Responses weighted by patient volume and adjusted for overstatement. Physicians evaluated a blinded p
Slide 8: Proven Launch Leaders Recruited to Serve the mLM Community
Proven Launch Leaders Recruited to Serve the mLM Community Anticipated launch in 1H 2027 >70 field-based team members at launch Sales Focused on winners from first-in-indication launches Field Marketing HCP education beyond sales call points In-House Patient Team Personalized support for patients Me
Slide 9: Opportunity to be the First FDA-approved Therapy for Microcystic LM Patients
Broad applicability potential: pediatric and adult, all severities, treatment-naïve and experienced Long-term chronic administration anticipated Position as first-line and establish as the new standard of care QTORIN™ Rapamycin: Opportunity to be the First FDA-approved Therapy for Estimated >30K Mic
Slide 10: Rare Disease Pipeline Updates
Rare Disease Pipeline Updates
Slide 11: FDA Rare Disease Framework Public Meeting (October 2026)
FDA Rare Disease Framework Public Meeting (October 2026) FDA PUBLIC MEETING Rare Disease Framework Initiative Public Meeting October 28, 2026 FDA White Oak Campus, Silver Spring, MD Hosted by the Reagan-Udall Foundation for the FDA “ “Dermatology has more rare diseases than any other specialty.” Dr.
Slide 12: Deep Pipeline in Serious, Rare Skin Diseases and Vascular Anomalies
Deep Pipeline in Serious, Rare Skin Diseases and Vascular Anomalies Phase 3 PRODUCT CANDIDATE DISEASE ESTIMATED U.S. PREVALENCE1 CLINICAL STATUS Preclinical Phase 1 Phase 2 UPCOMING MILESTONES QTORIN™ Rapamycin 3.9% anhydrous gel Microcystic Lymphatic Malformations PDUFA: Feb 28, 2027 Clinically Sig
Slide 13: Why the Microcystic LM Field is Moving Toward Early Intervention
Why the Microcystic LM Field is Moving Toward Early Intervention THE BIOLOGY Somatic PIK3CA mutations keep the growth pathway always “on”1 LM is proliferative and progressive, not a static lesion Luks et al., J Pediatr 2015 (Boston Children’s)1 85% Boston Children’s2 of childhood LMs progressed by a
Slide 14: Cutaneous Venous Malformations
No FDA-approved therapies Fast Track Designation Granted Pipeline-in-a-product: sNDA planned > 75k patients ESTIMATED DIAGNOSED IN THE U.S. Phase 3 initiation on track for Q4 2026 Significant treater overlap with microcystic lymphatic malformations Positive Phase 2 TOIVA data with 73% of patients im
Slide 15: Clinically Significant Angiokeratomas
Topline results (n=~15) expected in 2H 2027 Phase 2 LOTU study is a 12-week, single-arm, baseline-controlled clinical trial evaluating QTORIN™ rapamycin topically once daily Strong enrollment momentum and site engagement Highly symptomatic with bleeding and painful clots; sensitive location limits d
Slide 16: QTORIN pitavastatin: First pathogenesis-directed therapy
QTORIN™ pitavastatin: First pathogenesis-directed therapy Milestones: No FDA-approved therapies Current options: Laser, surgery, and off-label topical chemo agents & mevalonate pathway inhibitors POTENTIAL TO BE FIRST FDA-APPROVED THERAPY AND STANDARD OF CARE Disseminated Superficial Actinic Poroker
Slide 17: Finance & Cash Update
Finance & Cash Update palvella THERAPEUTICS
Slide 18: U.S. Launch and Rare Disease Pipeline Well-Funded with $236.7 Million
Key strategic priorities accelerating: NDA accepted with Priority Review; PDUFA date of February 28, 2027 Commercial and medical affairs expansion Major QTORIN™ pipeline and platform value drivers $236.7 million 9/30/26 cash Cash, cash equivalents and short-term investments U.S. Launch and Rare Dise
Slide 19: Closing Remarks
Closing Remarks palvella THERAPEUTICS
Slide 20: Driving the Next Wave of Innovation in Under-Appreciated Rare Skin Diseases
Palvella: Driving the Next Wave of Innovation in Under-Appreciated Rare Skin Diseases and Vascular Malformations 1. Clarity Pharma, Trinity Life Sciences, MedaCorp, and ZS Associates. 2. MedaCorp market research. Diagnosed US Prevalence (Estimated)1 Number of FDA Approved Therapies Physicians Who Wo
Slide 21: PALVELLA: To Serve
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 22: Thank You
Striving to be first for rare disease patients Thank You
Palvella Therapeutics — Q3 2026 Earnings Presentation on 6ix