First-in-disease therapies for patients with rare diseases Q3 2026 Financial Results & Corporate Update November 5, 2026
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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 2029+ Additional Potential Approvals in mTOR-Driven Diseases >300,000 U.S. patients across diseases1 1. Clarity Pharma, Trinity Life Sciences, MedaCorp, and ZS Associates.
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: Durable IP Generation Veteran team executing rare disease model designed to reduce time and capital to FDA approval 1 2 3 4
Shaping an Exceptional Launch for mLM Patients
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 Launch Leadership, Applied With Urgency Significant progress across crucial keys to launch success Strong Prescribing Intent >70% plan to prescribe within 3 months of approval Clear Path to Coverage Payer research has confirmed pricing and access assumptions Precision Patient-Finding Multiple patient identification approaches deployed Keys to a Successful 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 product profile. Source: Palvella-sponsored market research (survey of 150 U.S. physicians; interviews with 14 physicians and 10 payers), April–June 2026. >75% would start several patients at once, then expand Payer Survey (10 U.S. payers) mLM is recognized as a serious, genetic vascular anomaly Clinical diagnosis with no step-through anticipated Favorable coverage anticipated at orphan pricing corridor New Market Research Reinforces Potential for Strong Orphan Launch Post-SELVA market research further substantiates previous launch positioning
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 Medical Affairs Deep discussions with providers on our science Precision Digital Effort Physician National Prescriber Identification (NPI)-targeted strategy informed by claims data Leadership team brings deep expertise in both rare disease and dermatology
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 Microcystic LM Patients in the U.S. QTORIN™ 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. QTORIN™ 3.9% Rapamycin Microcystic Lymphatic Malformations
Rare Disease Pipeline Updates
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. Matthew Zirwas, Board-certified dermatologist Public comment, October 28, 2026
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 Significant Angiokeratomas Phase 2 data expected 2H 2027 Cutaneous Venous Malformations >75,000 Phase 3 trial initiation expected Q4 2026 QTORIN™ Pitavastatin Disseminated Superficial Actinic Porokeratosis (DSAP) PDUFA: Feb 28, 2027 Phase 2 Completed Phase 2 Ongoing Phase 2 Ready >30,000 FDA DESIGNATIONS Breakthrough Therapy Fast Track Orphan Fast Track Phase 2 trial initiation expected Q4 2026 >50,000 >50,000 1. Clarity Pharma, Trinity Life Sciences, MedaCorp, and ZS Associates. Announcement expected Q4 2026 Third QTORIN™ Program Fourth QTORIN™ rapamycin indication Phase 2 Ready TBD Announcement expected Q4 2026 Serious, rare disease TBD Fast Track Planned Planned Planned Announcement expected 2027+ Additional New QTORIN™ Programs Serious, rare diseases TBD Planned
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 adulthood 2.8x Stanford3 higher odds of progression without treatment “Early intervention should be considered…because of their tendency to progress.”2 1. Luks VL et al. J Pediatr. 2015;166:1048–54. 2. Hassanein AH et al. J Craniofac Surg. 2012;23(1):149–52. 3. Patel V et al. J Clin Exp Dermatol Res. 2017;8(6):424.
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 improved on cVM-IGA at Week 12 24-week data presented at PeDRA October 2026 Additional improvement between Week 12 and Week 24 on multiple endpoints Data demonstrated reduction in disease severity and bleeding in pediatric patients after treatment Cutaneous Venous Malformations Near-term Phase 3 initiation on track QTORIN™ RAPAMYCIN HAS POTENTIAL TO BE FIRST APPROVED THERAPY
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 destructive interventional treatments Clinically Significant Angiokeratomas Superficial lymphatic malformations UNDER-RECOGNIZED MARKET OPPORTUNITY Market research (n=50 physicians): 96% would incorporate QTORIN™ rapamycin into their practice > 50k patients ESTIMATED DIAGNOSED IN THE U.S. No FDA-approved therapies Fast Track Designation Granted Pipeline-in-a-product: sNDA planned
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 Porokeratosis Chronic, pre-cancerous, and progressive FALL CLINICAL DERMATOLOGY CONFERENCE® New claims analysis
October 2026 ~155K diagnosed U.S. DSAP patients First real-world claims analysis-based characterization of the diagnosed U.S. DSAP population Formulation development completed Q4 2026: Phase 2 initiation anticipated Licensed Yale U.S. patent and filed IP Multiple IND-enabling tox studies completed Yale collaboration: pharmacodynamic proof of principle with pitavastatin reversing hyperproliferation in a human porokeratosis cell model
Finance & Cash Update
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 Disease Pipeline are Well-Funded with $236.7 Million on Balance Sheet as of 9/30/26
Closing Remarks
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 Would Consider QTORIN™ Rapamycin / Pitavastatin as First-line Therapy2 30k+ Microcystic LMs 75k+ Cutaneous VMs 50k+ Clinically Significant Angiokeratomas 50k+ Disseminated Superficial Actinic Porokeratosis None > 80%
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
Striving to be first for rare disease patients Thank You