Slide 1: QTORIN 3.9% Rapamycin Anhydrous Gel: Statistically Significant, Clinically Meaningful Improvement
QTORIN 3.9% Rapamycin Anhydrous Gel: Statistically Significant, Clinically Meaningful Improvement in Microcystic Lymphatic Malformations (Phase 3 SELVA Study) and Cutaneous Venous Malformations (Phase 2 TOIVA Study) Presentation at ISSVA World Congress 2026 Dr. James Treat, Children’s Hospital of Ph
Slide 2: Conflict Statement
• Royalties: UpToDate (Tinea Capitis and Pityriasis Conflict Lichenoides et Varioliformis Acuta) and Andrews Statement Diseases of the Skin • Advisory Board: Incyte, Arcutis • I provide consulting services for Arcutis and Palvella and Nektar. • Many therapies in Pediatric dermatology are off label 2
Slide 3: Agenda
Agenda Phase 3 SELVA Study: QTORIN rapamycin for treatment of microcystic lymphatic malformations (mLMs) 1. SELVA Phase 3 Topline Results: Statistically Significant Improvement Across All Pre-specified Endpoints 2. Early Intervention and Chronic Disease Management: Results from Pediatric Patients Ag
Slide 4: Agenda (cont.)
Agenda (cont.) Phase 2 TOIVA Study: QTORIN rapamycin for treatment of cutaneous venous malformations (cVMs) James R. Treat, MD 1. TOIVA Phase 2 Topline Results: Statistically Significant Improvement Professor of Clinical Pediatrics and 2. cVM Clinical Signs: Height/Engorgement and Appearance Dermato
Slide 5: Background
Background • QTORIN rapamycin 3.9% anhydrous gel (QTORIN rapamycin) is an investigational therapy in development for microcystic LMs, cutaneous VMs, clinically significant angiokeratomas, and other skin diseases driven by the mammalian target of rapamycin (mTOR) pathway • Microcystic LMs typically d
Slide 6: SELVA Phase 3 Topline Results: Statistically Significant Improvement Across All Prespecified Endpoints
1 SELVA Phase 3 Topline Results: Statistically Significant Improvement Across All Prespecified Endpoints Mean Change at Week 24 p-value (95% CI) Primary Endpoint: mLM-IGA* +2.13 (1.88, 2.38) p<0.001 Key Secondary Endpoint: Blinded mLM-MCSS*** -3.4 (-4.34, -2.38) p<0.001 Secondary Endpoint: Live mLM-
Slide 7: Early Intervention and Chronic Disease Management: Results from Pediatric Patients Aged 6-11 Years
2 Early Intervention and Chronic Disease Management: Results from Pediatric Patients Aged 6-11 Years 100% mLM-IGA (6-11 year old cohort, were “Much Improved” (+2) or n=13) “Very Much Improved” (+3) Very Much Mean: +2.46 3 Improved (p<0.001) of the 6-11 y/o ALL rolled over into Much cohort at Day 168
Slide 8: Rapid Onset and Large Treatment Effect in Leaking and Bleeding
3 Rapid Onset and Large Treatment Effect in Leaking and Bleeding – Two of the Most Burdensome Signs of Microcystic LMs mLM-IGA Moderate or Worse Leaking/Bleeding at Baseline (n=23) Very Much Mean: +2.48 Improved 3 (p<0.001) Much 2 Improved Minimally 1 Improved No Change 0 Day 60 Day 90 Day 168 Minim
Slide 9: Treatment Satisfaction Questionnaire for Medication (TSQM) and Patient Qualitative Interviews
4 Treatment Satisfaction Questionnaire for Medication (TSQM) and Patient Qualitative Interviews Support the Clinical Meaningfulness of QTORIN Rapamycin’s Treatment Effect Treatment Satisfaction Questionnaire for Patient Qualitative Interviews Medication (TSQM) Pre-specified patient interview sub-stu
Slide 10: Blinded Independent Review Demonstrated Stability During Run-In and Marked Improvement on Treatment
5 Phase 3 SELVA Study for Treatment of Microcystic LMs: Blinded Independent Review Demonstrated Stability During Run-In and Marked Improvement on Treatment mLM-MCSS: Sum of 3 static severity scales: Height, Leaking/Bleeding, Vesicle Appearance: Each scale rated 1 to 5; total score 3-15; Negative val
Slide 11: Phase 3 SELVA Conclusion: Well-Tolerated and Favorable Safety and Efficacy Profile
Phase 3 SELVA Conclusion: Well-Tolerated and Favorable Safety and Efficacy Profile Number of Participants (%) • Overall, QTORIN rapamycin Any Treatment-Emergent Adverse Event 35 (70%) was well-tolerated Severe (not related to study drug) 1 (2%) • All treatment-related AEs Serious (not related to stu
Slide 12: TOIVA Phase 2 Topline Results: Statistically Significant Improvement
1 TOIVA Phase 2 Topline Results: Statistically Significant Improvement Single-arm, baseline-controlled, daily dose, age 6+ Overall cVM-IGA at Week 12 100% • Overall cVM-IGA: 7-point clinician-assessed dynamic change scale ranging from “Very Much Worse” (-3) to “Very Much Improved” (+3) Percentage of
Slide 13: cVM Clinical Signs: Height/Engorgement and Appearance Results Through 24 Weeks
2 cVM Clinical Signs: Height/Engorgement and Appearance Results Through 24 Weeks Today, presenting new 24-week data on two of the key clinical signs measured in the TOIVA trial: height/engorgement and appearance • Data on bleeding, another key clinical sign in cutaneous venous malformations, were re
Slide 14: Thank You
Thank You Striving to be first for rare disease patients 14