Palvella Therapeutics, Inc. · NASDAQ:PVLA
Palvella Therapeutics — Technical Presentation (February 2026)
26 slides from Palvella Therapeutics's technical presentation, Feb 24, 2026
Slide 1: Phase 3 SELVA Topline Data in Microcystic Lymphatic Malformations
First-in-disease therapies for patients with rare diseases Phase 3 SELVA Topline Data in Microcystic Lymphatic Malformations February 24, 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: Phase 3 SELVA Topline Data: Today's Presenters
Phase 3 SELVA Topline Data: Today’s Presenters Bohan Wei VP Corporate Development & New Product Planning Wes Kaupinen Founder and CEO Jeff Martini, PhD Chief Scientific Officer Matt Korenberg Chief Financial Officer Michael Kelly, MD, PhD Pediatric hematologist-oncologist at Cleveland Clinic Executi
Slide 4: Opening Remarks
Opening Remarks Wes Kaupinen Founder and CEO QTORIN™ Rapamycin for Microcystic Lymphatic Malformations
Slide 5: 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 6: A Clear Path Forward for Patients
mLM-IGA = Microcystic Lymphatic Malformation Investigator Global Assessment. Note: Data analyzed per statistical analysis plan; non-completer data imputed per statistical analysis plan for primary endpoint; endpoints tested according to pre-specified hierarchical testing procedure; statistical signi
Slide 7: Phase 3 SELVA Topline Results
Phase 3 SELVA Topline Results Dr. Jeff Martini Chief Scientific Officer QTORIN™ Rapamycin for Microcystic Lymphatic Malformations
Slide 8: Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong
Microcystic Lymphatic Malformations: Serious, Debilitating, and Lifelong Genetics & Biology: Monogenic somatic PIK3CA mutations cause hyperactivated PI3K/mTOR Lymphorrhea: Persistent discharge of lymphatic fluid through skin layers Deep infections: Recurrent cellulitis and serious soft tissue infect
Slide 9: QTORIN: Targeted mTOR Inhibition with Dermal Target Engagement
QTORIN™: Targeted mTOR Inhibition with Dermal Target Engagement 3.9% concentration enabled through synergistic multi-excipient anhydrous solubilization system Elevated thermodynamic activity drives rapamycin vehicle release and skin bioavailability Deep dermal penetration & sustained dermal retentio
Slide 10: QTORIN Rapamycin Exceeded Upside Case Profile
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 11: Phase 3 SELVA Trial Design
Phase 3 SELVA Trial Design Single-arm, baseline-controlled, QD dose Primary Endpoint: mLM-IGA (Investigator Global Assessment) Key Secondary Endpoint: Blinded mLM Multi-Component Static Scale (mLM-MCSS): independent, blinded review of randomized Baseline and Week 24 photos evaluating three key signs
Slide 12: Phase 3 SELVA: Baseline Characteristics
Phase 3 SELVA: Baseline Characteristics ITT Population (n=49)1 Age, Mean [Range] 19.4 [6-57] Sex M:F 24:25 Prior Medical Interventions for mLM2 34 (69%) Laser 17 (35%) Sclerotherapy 14 (29%) Surgery 13 (27%) Topical Sirolimus [Rapamycin] 13 (27%) Oral Sirolimus [Rapamycin] 2 (4%) 1. 51 participants
Slide 13: SELVA: Primary and Key Secondary Endpoints Achieved
SELVA: Primary and Key Secondary Endpoints Achieved Mean Change at Week 24 (95% CI) p-value Primary Endpoint: Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA)* +2.13 (1.88, 2.38) p<0.001 Key Secondary Endpoint: Blinded Microcystic Lymphatic Malformation Multi-Component Sta
Slide 14: SELVA: All Additional Secondary Endpoints Achieved
SELVA: All Additional Secondary Endpoints Achieved Mean Change at Week 24 (95% CI) p-value Patient Global Impression of Change* +1.9 (1.66, 2.16) p<0.001 Live mLM-MCSS** -4.6 (-5.20, -3.92) p<0.001 Clinician Global Impression of Severity*** -1.7 (-1.91, -1.39) p<0.001 Patient Global Impression of Se
Slide 15: Phase 3 Results: Well-Tolerated and Favorable Safety Profile
Phase 3 Results: Well-Tolerated and Favorable Safety Profile Rapamycin levels were below 2 ng/mL2 in systemic circulation on for all participants at all timepoints in the study Note: n=50 participants in efficacy evaluation evaluated for safety. 1. Denotes related or possibly related. 2. Validated a
Slide 16: KOL Perspective
KOL Perspective Dr. Michael Kelly Cleveland Clinic QTORIN™ Rapamycin for Microcystic Lymphatic Malformations
Slide 17: Phase 3 Results: Age 10, Female
Phase 3 Results: Age 10, Female Note: Selective photos. Back
Slide 18: Phase 3 Results: Age 10, Female, mLM-IGA +2
Week 24 Baseline Phase 3 Results: Age 10, Female, mLM-IGA: +2 “Much Improved” Note: Selective photos. Back Back
Slide 19: Phase 3 Results: Age 10, Male
Phase 3 Results: Age 10, Male Note: Selective photos. Back
Slide 20: Phase 3 Results: Age 10, Male, mLM-IGA +2
Week 24 Baseline Phase 3 Results: Age 10, Male, mLM-IGA: +2 “Much Improved” Note: Selective photos. Thigh Thigh
Slide 21: Phase 3 Results: Age 14, Female, mLM-IGA +3
Week 24 Baseline Phase 3 Results: Age 14, Female, mLM-IGA: +3 “Very Much Improved” Note: Selective photos. Neck Neck
Slide 22: Phase 3 Results: Age 7, Female, mLM-IGA +3
Week 24 Baseline Phase 3 Results: Age 7, Female, mLM-IGA: +3 “Very Much Improved” Note: Selective photos. Elbow Elbow
Slide 23: QTORIN Rapamycin Has Potential to be First-Line and Standard of Care
QTORIN™ Rapamycin Has the Potential to be First-Line and Standard of Care in Microcystic Lymphatic Malformations Targets underlying pathobiology 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. The
Slide 24: Summary
Summary Wes Kaupinen Founder and CEO QTORIN™ Rapamycin for Microcystic Lymphatic Malformations
Slide 25: Palvella: Leading the Way in Rare Skin Diseases and Vascular Malformations
Palvella: Leading the Way in Addressing Rare Skin Diseases and Vascular Malformations Planned NDA submission in 2H:2026 Potential first FDA-approved therapy in mLM; U.S. launch prep accelerating Pipeline-in-a-product: Broad potential to address mTOR-driven diseases Platform: QTORIN™ pitavastatin and
Slide 26: Thank You
Striving to be first for rare disease patients Thank You
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