Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — AAO Retina Subspecialty Days 2019
24 slides from Regenxbio's conference presentation, Oct 15, 2019
Slide 1: Key Takeaways from the RGX-314 Phase I/IIa Clinical Trial for Wet AMD (Cohorts 1-5)
Exhibit 99.1 Key Takeaways from the RGX-314 Phase I/IIa Clinical Trial for Wet AMD (Cohorts 1-5) Jeffrey Heier, MD Peter Campochiaro, MD, Allen Ho, MD, Albert Maguire, MD, David M. Brown, MD, Robert Avery, MD, Dante Pieramici, MD, Szilard Kiss, MD, Arshad Khanani, MD, Charles Wykoff, MD Samir Patel,
Slide 2: Disclosures
Disclosures Research grants: Aerie, Aerpio, Apellis, Clearside, Daiichi, Genentech/Roche, Graybug, Gyroscope, Hemera, Janssen R&D, KalVista, Kanghong, Novartis, Ophthotech, Optos, Optovue, Regeneron, Regenxbio, Stealth Biotherapeutics, Thrombogenics, Tyrogenex Scientific Advisor: 4DMT, Adverum, Aeri
Slide 3: RGX-314 Uses a Novel AAV8 Vector to Deliver an anti-VEGF Fab Gene
RGX-314 Uses a Novel AAV8 Vector to Deliver an anti-VEGF Fab Gene Encoding NAV AAV8 Vector for anti-VEGF fab Cell AAV8 AAV2 anti-VEGF fab RPE Protein RNA Nucleus 1 More Efficient Gene Delivery to the RPE RGX-314 is Designed to Deliver a Gene Encoding for an anti-VEGF fab Protein 1. Vandenberghe et a
Slide 4: RGX-314 Phase I/IIa wAMD Study Has Fully Enrolled 5 Dose Cohorts
RGX-314 Phase I/IIa wAMD Study Has Fully Enrolled 5 Dose Cohorts Baseline assessment Treatment evaluation Follow up anti–VEGF SD–OCT Anti–VEGF PRN Rescue Injection Criteria injection assessment 0 1 2 6 10 14 18 22 26 54 106 Weeks RGX–314 Safety endpoint administration Secondary endpoints Previously
Slide 5: Anti-VEGF Retreatment Allowed for Any Fluid or Disease Activity
Anti-VEGF Retreatment Allowed for Any Fluid or Disease Activity Anti-VEGF may be given beginning 4 weeks post-treatment and PRN every 4 weeks thereafter per investigator’s discretion if one or more of the criteria apply: CNV-related Vision loss of ≥5 increased, New ocular letters associated w/ new,
Slide 6: Subjects Enrolled in the Phase I/IIa Trial Were Chronically Treated
Subjects Enrolled in the Phase I/IIa Trial Were Chronically Treated Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Total Variable (n=6) (n=6) (n=6) (n=12) (n=12) (n=42) Mean Age (Years) 78.2 78.0 80.0 80.3 81.6 80.0 Baseline BCVA 53.7 50.7 54.7 61.3 54.3 55.7 (Snellen equivalents) (20/100) (20/100) (2
Slide 7: RGX-314 Has Been Well Tolerated in All Cohorts
RGX-314 Has Been Well Tolerated in All Cohorts § RGX–314 was well–tolerated (n=42) § No drug-related SAEs § Most AEs were assessed as mild (Grade 1 – 79%) § No observed clinically determined immune responses, drug-related ocular inflammation, or any post-surgical inflammation beyond what is expected
Slide 8: Dose Dependent Increase in RGX-314 Protein Observed Across Cohorts
Dose Dependent Increase in RGX-314 Protein Observed Across Cohorts As Measured from Aqueous Samples by ECL 1 Month post-RGX-314 500 376.0 ng/ml 400 2 249.4 ng/ml 160.2 ng/ml 300 200 100 12.8 ng/ml 2.5 ng/ml 0 Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 9 10 10 11 11 3 x 10 GC/eye 1 x 10 GC/eye 6 x
Slide 9: Cohort 3: Sustained Visual and Anatomic Outcomes over 1.5 years
Cohort 3: Sustained Visual and Anatomic Outcomes over 1.5 years Best Corrected Visual Acuity (BCVA) 70 70 70 +1 letters 60 60 60 -9 letters 50 +9 letters 50 50 40 40 40 30 30 30 Time Time Time Central Retinal Thickness (CRT) on Heidelberg SD-OCT 600 600 600 +45 µm 500 500 500 -26 µm 400 400 400 -40
Slide 10: Cohort 3: Injection-free Subjects Continue to Do Well Over 1.5 Years
Cohort 3: Injection-free Subjects Continue to Do Well Over 1.5 Years Anti-VEGF Injection-free Subjects (n= 3 of 6) Sustained RGX-314 Protein Levels Over 1 Year Best Corrected Visual Acuity (BCVA) +11 letters 70 1000 274.9 260.5 236.2 60 ng/ml ng/ml ng/ml 50 40 D1 M1 M6 1 Yr 1.5 100 Yrs Time Central
Slide 11: Cohort 4: Visual and Anatomic Outcomes
Cohort 4: Visual and Anatomic Outcomes Best Corrected Visual Acuity (BCVA) 80 § Stable to improved vision and OCT +2 Letters 70 on average 60 50 § 42% (5 of 12) injection-free at 6 40 months D1 M1 M2 M3 M4 M5 M6 Time § 2 patients with incomplete response Central Retinal Thickness (CRT) on Heidelberg
Slide 12: Cohort 5: Visual and Anatomic Outcomes
Cohort 5: Visual and Anatomic Outcomes Best Corrected Visual Acuity (BCVA) 80 § Stable to improved vision and OCT +4 Letters 70 on average 60 50 § 75% (9 of 12) injection free at 5-6 40 months D1 M1 M2 M3 M4 M5 M6 Time § Highest clinical response observed Central Retinal Thickness (CRT) on Heidelber
Slide 13: Cohort 4 and Cohort 5: Anti-VEGF Injection-free Subjects
Cohort 4 and Cohort 5: Anti-VEGF Injection-free Subjects Best Corrected Visual Acuity (BCVA) 80 80 +5 Letters +2 Letters 70 70 60 60 50 50 40 40 D1 M1 M2 M3 M4 M5 M6 D1 M1 M2 M3 M4 M5 M6 Time Time Central Retinal Thickness (CRT) on Heidelberg SD-OCT* 600 600 500 500 -80 μm -61 μm 400 400 300 300 200
Slide 14: Cohort 5: Injections Pre and Post RGX-314 (n=12)
Cohort 5: Injections Pre and Post RGX-314 (n=12) 1 2 3 4 5 6 7 8 9 10 11 12 1 Year 0 Month Month Month Month Month Month Time prior to treatment with RGX-314 1 2 3 4 5 6 RGX-314 (n=12) (n=11) (n=5) Months since RGX-314 AFLIBERCEPT BEVACIZUMAB Visit With No Injection RANIBIZUMAB § Subject #1 disconti
Slide 15: Cohort 5 Case A: Subject History Prior to RGX-314
Cohort 5 11 2.5x10 GC/eye Case A: Subject History Prior to RGX-314 09MAY2018 08AUG2018 31OCT2018 30JAN2019 06JUN2018 05SEP2018 05DEC2018 27FEB2019 11JUL2018 03OCT2018 02JAN2019 27MAR2019 15Cohort 5 11 2.5x10 GC/eye Case A: Subject History Prior to RGX-314 09MAY2018 08AUG2018 31OCT2018 30JAN2019 06JU
Slide 16: Cohort 5 Case A: 13 Injections in Year Prior with 0 Rescue Injections
Cohort 5 Day 1 – VA 73 letters 1 Month 4 Months 11 2.5x10 GC/eye Case A: 13 Injections in Year Prior with 0 Rescue Injections after Week 1 – OCT response 2 Months 5 Months – VA 75 (+2 letters) RGX-314 Age: 87 Total prior anti-VEGF hx: 40 Last year anti-VEGF: 13 Rescue Inj in Study: 0 RGX-314 1 Week
Slide 17: Cohort 5 Case B: 8 Injections in Year Prior with 0 Rescue Injections
Cohort 5 Day 1 – VA 46 letters (20/125) 1 Month 4 Months 11 2.5x10 GC/eye Case B: 8 Injections in Year Prior with 0 Rescue Injections after Week 1 – OCT response 2 Months 5 Months RGX-314 Age: 86 Total prior anti-VEGF hx: 20 Last year anti-VEGF: 8 Rescue Inj in Study: 0 RGX-314 Week 1 post-RGX-314 3
Slide 18: RGX-314 Program Next Steps
RGX-314 Program Next Steps wAMD moving Diabetic to Phase IIb Retinopathy Study by the IND by end of the year end of the year RGX-314 Gene Therapy Expanding to evaluate SCS delivery using Clearside’s proprietary, in-office SCS Microinjector 18RGX-314 Program Next Steps wAMD moving Diabetic to Phase
Slide 19: Key takeaways from the RGX-314 Phase I/IIa wAMD Clinical Trial
Key takeaways from the RGX-314 Phase I/IIa wAMD Clinical Trial § RGX-314 Phase I/IIa wAMD study has fully enrolled 42 patients in 5 dose cohorts § Patients enrolled were severe wAMD requiring frequent anti-VEGF injections § Subretinal RGX-314 was well tolerated in 5 dose Cohorts § Dose dependent inc
Slide 20: Acknowledgments
Acknowledgments § Robert Avery, MD (Santa Barbara, CA) § David Brown, MD (Houston, TX) University of § Peter Campochiaro, MD (Baltimore, MD) Ophthalmic Pennsylvania, Philadelphia, PA Consultants § Jorge Calzada, MD (Memphis. TN) of Boston, Boston, MA § Jeff Heier, MD (Boston, MA) Wills Eye Sierra Ey
Slide 21: Supplemental Information
Supplemental InformationSupplemental Information
Slide 22: Mean Change in Annualized Injection Rate Pre and Post RGX-314
Mean Change in Annualized Injection Rate Pre and Post RGX-314: >80% Reduction in Cohort 5 Comparison of injection rate PRIOR and POST RGX-314 12 10.5 10.5 11 10.2 9.9 9.9 9.6 10 9.0 9 7.8 8 6.8 7 6 4.4 5 4 2.7 2.6 3 1.6 2 1 0 Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 9 10 10 11 11 3 x 10 GC/eye 1
Slide 23: RGX-314: Standardized Automated Subretinal Delivery Procedure
RGX-314: Standardized Automated Subretinal Delivery Procedure Step 1 – Vitrectomy Step 2 – Subretinal Injection MedOne MicroDose Syringe Performed Under Local Anaesthesia in the OR § Standard small gauge vitrectomy to perform § Inject 250μl to create subretinal bleb § Can create another bleb area a
Slide 24: Cohort 5 Case C: 12 Injections in Year Prior with 0 Rescue Injections
Cohort 5 Day 1 – VA 73 letters 1 Month 4 Months Day 1 – VA 73 letters Week 6 – VA 76 letters Week 18 – VA 72 letters 11 2.5x10 GC/eye Case C: 12 Injections in Year Prior with 0 Rescue Injections Week 1 – OCT response 2 Months 5 Months Week 1 – VA 72 letters Week 10 – VA 74 letters Week 22 – VA 76 le