Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — Technical Presentation (October 2022)
19 slides from Regenxbio's technical presentation, Oct 3, 2022
Slide 1: Suprachoroidal Delivery of RGX-314 for nAMD: AAVIATE Study
1 Suprachoroidal Delivery of RGX-314 for nAMD: AAVIATE® Study 10 | 3 | 2022
Slide 2: Agenda
Agenda • Welcome & Introductions • Interim AAVIATE Study Update • Q&A Ken Mills Steve Pakola, MD Arshad Khanani, MD, MA, FASRS, President and CEO Chief Medical Officer, Director of Clinical Research, REGENXBIO Inc. REGENXBIO Inc. Sierra Eye Associates 2
Slide 3: Forward-looking statements
Forward-looking statements This presentation includes “forward-looking statements,” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are gen
Slide 4: Ocular gene therapy delivery methods
Ocular gene therapy delivery methods Comparative profiles Delivery Space Considerations Suprachoroidal Space (SCS)1 • Targeted access and broad transduction of the retinal cells observed in preclinical studies • Compartmentalized AAV delivery • Minimal exposure to the vitreous and anterior segment S
Slide 5: RGX-314 for Treatment of Neovascular Age-related Macular Degeneration (nAMD)
RGX–314 for Treatment of Neovascular Age-related Macular Degeneration (nAMD) RGX–314 PRODUCT CANDIDATE Vector: AAV8 Gene: anti-VEGF fab Mechanism of action: Reducing leaky blood vessel formation Route of administration: by giving ocular cells the ability to produce an anti-VEGF fab Subretinal (nAMD)
Slide 6: AAVIATE: RGX-314 Phase II Clinical Trial in nAMD
AAVIATE®: RGX-314 Phase II Clinical Trial in nAMD Baseline Assessment Treatment Evaluation Long Term Follow Up anti–VEGF SD–OCT Injection Assessment by CRC Anti–VEGF PRN Retreatment Criteria Previously Treated nAMD screening D1 W1 Wk2 3 Mo 6 Mo 9 Mo 1 Year Subjects RGX–314 SCS Time BCVA Primary Seco
Slide 7: AAVIATE: RGX-314 Phase II Clinical Trial in nAMD
AAVIATE®: RGX-314 Phase II Clinical Trial in nAMD Primary Objective Route of Administration To evaluate the mean change in BCVA for RGX-314 compared In-office SCS Microinjector™ delivers RGX-314 to the with ranibizumab monthly injection at Month 9 suprachoroidal space Secondary Objectives Key In
Slide 8: AAVIATE Baseline Characteristics (Cohort 1 to 5)
AAVIATE Baseline Characteristics (Cohort 1 to 5) Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Control Dose 2 Dose 2 Dose 3 Dose 3 Total Ranibizumab Dose NAb- 1 NAb- NAb+ NAb- NAb+ (N=95) (N=10) (N=15) (N=15) (N=20) (N=15) (N=20) Variable Mean Age (Years) 75.9 74.0 77.9 72.6 79.7 75.0 75.6 Screening
Slide 9: AAVIATE Safety Summary
AAVIATE® Safety Summary RGX–314 was well–tolerated in Cohorts 1–5 (n=85) with follow-up ranging from 1–12 months post dosing 15 SAEs: None considered drug-related No cases of chorioretinal vasculitis or occlusion, or hypotony were observed Cohort 1 Cohort 2 Cohort 3 Cohort 4 Dose 1 Dose 2 Dose
Slide 10: Cohorts 1-4: Mean BCVA Through Month 6
Cohorts 1-4: Mean BCVA Through Month 6 Best Corrected Visual Acuity (BCVA) 95% CI 85 +4.0 Letters (n=10) Ranibizumab 80 From Screening -2.8 Letters (n=14) Letters Read RGX-314 Cohort 1 75 -1.7 letters (n=19) 70 RGX-314 Cohort 3 -0.07 Letters (n=15) 65 RGX-314 Cohort 2 60 -2.5 Letters (n=15) D1 W1 W2
Slide 11: Cohorts 1-4: Mean CRT from Day 1 Through Month 6
Cohorts 1–4: Mean CRT from Day 1 (Screening) Through Month 6 Central Retinal Thickness (CRT) 95%CI 500 4.6 µm (n=19) RGX-314 Cohort 3 400 -2.5 µm (n=14) RGX-314 Cohort 1 300 -2.9 µm (n=15) μm RGX-314 Cohort 4 200 -33 µm (n=15) RGX-314 Cohort 2 100 -12.3 µm (n=10) Ranibizumab 0 D1 W1 W2 Time Post-Ran
Slide 12: Cohort 1 (Dose 1): Injections Pre and Post RGX-314
Cohort 1 (Dose 1): Injections Pre and Post RGX-314 (n=15) – 6 Month Data Change in Annualized Injection Rate Cohort 1 – Nab - Subject -75.9% 4/14 (29%) injection -free -1 Year Time Prior to Treatment with RGX-314 D1 RGX-314 Months Since RGX-314 Month 6 RANIBIZUMAB AFLIBERCEPT BEVACIZUMAB BROLUCIZUMA
Slide 13: Cohort 2 and Cohort 3 (Dose 2): Injections Pre and Post RGX-314
Cohort 2 and 3 (Dose 2): Injections Pre and Post RGX-314 (n=35) – 6 Month Data Change in Annualized Injection Rate Cohort 2 – Nab- -71.8% 6/15 (40%) Subject injection -free -63.8% Cohort 3 - Nab+ 7/20 (35%) injection -free -1 Year Time Prior to Treatment with RGX-314 D1 RGX-314 Months Since RGX-314
Slide 14: Cohort 4 (Dose 3): Injections Pre and Post RGX-314
Cohort 4 (Dose 3): Injections Pre and Post RGX-314 (n=15) – 6 Month Data Change in Annualized Injection Rate -84.7% Cohort 4 – Nab - Subject 10/15 (67%) injection -free -1 Year Time Prior to Treatment with RGX-314 D1 RGX-314 Months Since RGX-314 Month 6 RANIBIZUMAB AFLIBERCEPT BEVACIZUMAB BROLUCIZUM
Slide 15: Mean Change in Annualized Injection Rate in Cohorts 1-4
Mean Change in Annualized Injection Rate PRE and POST RGX-314 in Cohorts 1–4 Annualized Injection Rate 12 10 Annualized Injections 9.7 9.3 8 8.7 8.9 6 4 -63.8% -75.9% -71.8% 3.0 2 2.5 -84.7% 2.2 Pre Pre Pre Pre 1.3 RGX-314 6 Month RGX-314 6 Month RGX-314 6 Month RGX-314 6 Month 0 Cohort 1 Cohort 2 C
Slide 16: Cohorts 1-4: Subjects with No Anti-VEGF Injections over 6 Months
Cohorts 1-4: Subjects with No Anti-VEGF Injections over 6 Months Mean BCVA and CRT from Day 1 (Screening) Best Corrected Visual Acuity (BCVA) 95% CI 90 +1.0 letters (n=6) RGX-314 Cohort 2 80 +4.0 Letters (n=10) Letters Read Ranibizumab 70 +2.3 letters (n=6) RGX-314 Cohort 3 60 +1.3 letters (n=4) RGX
Slide 17: AAVIATE: Study Design with Addition of Cohort 6
AAVIATE®: Study Design with Addition of Cohort 6 Baseline Assessment Treatment Evaluation Long Term Follow Up anti–VEGF Injection Anti–VEGF PRN Retreatment Criteria Previously screening Treated nAMD W-4 D1 W1 W2 W4 3 Mo 6 Mo 9 Mo 1 Year Subjects SD–OCT RGX–314 Time BCVA Primary Secondary Assessment
Slide 18: Summary of Results from the Phase II AAVIATE nAMD Study
Summary of Results from the Phase II AAVIATE® nAMD Study RGX-314 Cohorts 1-5 (n=85): Safety Suprachoroidal RGX-314 has been well-tolerated RGX-314 Cohorts 1-4 (n=65): 6 Month Results RGX-314 treated patients had stable vision and retinal thickness, with a meaningful reduction in treatment burden
Slide 19: Thank You
€3 R=G=NxBIO Thank You