Slide 1: A Phase 2 Trial of Belzupacap Sarotalocan (AU-011) for Choroidal Melanoma
A Phase 2 Trial of Belzupacap Sarotalocan (AU-011) A First-in-Class Targeted Therapy for Choroidal Melanoma via Suprachoroidal Administration AAO 2022 October 2, 2022 Ivana K. Kim, MD, MBA On Behalf of the AU-011 Investigator Group AU-011-202, NCT04417530 Co-Director Ocular Melanoma Center Massachus
Slide 2: Targeted Oncology Platform: Virus-Like Drug Conjugates (VDCs)
Targeted Oncology Platform: Virus-Like Drug Conjugates (VDCs) Virus-Like Particles Conjugated to a Cytotoxic Payload to form the VDC VDCs can Recognize Tumor Associated HSPGs* Virus-Like Particle (VLP) Virus-Like Drug Conjugate (VDC) Cx Cytotoxic Drug Kines et al; International Journal of Cancer, 13
Slide 3: Belzupacap Sarotalocan (AU-011) is a VDC with a Novel Dual Mechanism of Action
Belzupacap Sarotalocan (AU-011) is a VDC with a Novel Dual Mechanism of Action Light Activated Drug Belzupacap Sarotalocan Belzupacap sarotalocan is a novel VDC that consists of a VLP conjugated to ~200 molecules of phthalocyanine dye Kines et al; Cancer Immunology Research, May 2021
Slide 4: Phase 2 Trial of Belzupacap Sarotalocan via Suprachoroidal Administration Dose Escalation Study Design
Phase 2 Trial of Belzupacap Sarotalocan via Suprachoroidal Administration Dose Escalation Study Design Single Dose Cohorts (Completed) Multiple Dose Cohorts 20 μg x 1 Laser 40 μg x 1 Laser 40 μg x 2 Lasers 40 μg x 2 Lasers QWx2 40 μg x 2 Lasers QWx3 Up to 3 cycles Cohort 1 (n=1) Cohort 2 (n=3*) Coho
Slide 5: Therapeutic Regimen is Completed in Treatment Cycles
Therapeutic Regimen is Completed in 3 Treatment Cycles WEEK 0 1 2 3 4 5 6 7 8 9 10 Cycle 1 Cycle 2 Cycle 3 4-6 Hours Waiting Period Belzupacap Sarotalocan Injections Laser #1 30 Minutes Waiting Period Laser #2 One treatment consists of two suprachoroidal injections of belzupacap sarotalocan, followe
Slide 6: Patient Population Representative of Early-Stage Disease
Patient Population Representative of Early-Stage Disease Indeterminate Lesions and Small Choroidal Melanoma Small Tumors with Documented Growth Tumor thickness ≥0.5 mm and ≤2.5 mm Largest Basal Diameter (LBD) ≤10 mm Documented tumor growth within 2 years of screening Tumor growth rate ≥0.2mm/year
Slide 7: Tumor Control Rates at 6 Months of Follow Up Demonstrate Dose Response
Tumor Control Rates at 6 Months of Follow Up Demonstrate Dose Response Tumor Progression: change from baseline in thickness ≥0.5mm; or in LBD ≥1.5mm confirmed by at least one repeat assessment 19-Aug-2022 cutoff, interim data Populations Total Patients (n) Tumor Control Rate Average Follow-up (month
Slide 8: Early Analysis of Tumor Control with 3 Cycle Regimen
Early Analysis of Tumor Control with 3 Cycle Regimen Change from Baseline in Tumor Thickness Over 12 Months Ongoing Phase 2 SC trial (AU-011-202), post-SOC data not included *1 subject without post-baseline tumor thickness data not included in plot Population Total Patients (n) Tumor Control Rate (%
Slide 9: Early Analysis of Tumor Growth Rate with 3 Cycle Regimen
n Historical Growth Rate (mm/yr) AU-011 Growth Rate (mm/yr) Growth Rate Reduction (mm/yr) p-value Average Follow up (months) Active Growth and Highest Dose/Regimens* 3 Cycles (40µg-80µg) 40µg (n=2) 80µg (n=7) 9 0.463 0.166 -0.296 0.0007 6 Tumor thickness growth rates/ slopes estimated using MMRM Red
Slide 10: Early Analysis of Visual Acuity Preservation Rate of 89% at the Highest Dose Regimen
Populations Total Patients (n) Vision Failures** (n) Vision Preservation Rate Mean Change from Baseline at Last Visit (letters) Average Follow-up (months) All Dose Cohorts All Treated Patients 20 2 90% -3.3 8 High Risk for Vision Loss 15 2 87% -4.5 7 Highest Doses/Regimens * 2 Cycles (40µg) 1 0 100%
Slide 11: Ongoing Safety Evaluation Continues to Be Favorable with No Related SAEs/DLTs Observed to Date
Ongoing Safety Evaluation Continues to Be Favorable with No Related SAEs/DLTs Observed to Date 19-Aug-2022 data cutoff, interim data Table presents percentage of subjects with AEs related to AU-011 or laser by severity and overall; subjects with more than 1 AE are counted in the highest severity gro
Slide 12: Ongoing Ph2 Trial of Suprachoroidal Administration Provides Additional Safety and Efficacy Data
Ongoing Ph 2 Trial of Suprachoroidal Administration Provides Additional Safety and Efficacy Data Supports Potential Treatment of Early-Stage Disease Statistically significant reduction in early analysis of tumor growth rates (p=0.0007) Early outcomes have shown high tumor control rate (89%) with app
Slide 13: Clinical Investigators & Participating Sites
Clinical Investigators & Participating Sites Belzupacap Sarotalocan Ocular Oncology Investigator Group Dr. Carol Shields Philadelphia, PA Dr. Ivana Kim Boston, MA Dr. Tara McCannel Los Angeles, CA Dr. Abdhish Bhavsar Minneapolis, MN Dr. Antonio Capone Jr. Royal Oak, MI Dr. Amy Schefler Houston, TX D