Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — AAO 2022
16 slides from Regenxbio's conference presentation, 2022
Slide 1: Gene Therapy for Neovascular AMD: Subretinal RGX-314 Long-Term Follow-Up Results
Gene Therapy for Neovascular AMD: Subretinal RGX-314: Phase I/IIa Long-Term Follow-Up Results up to 4 Years Peter A. Campochiaro The Wilmer Eye Institute Johns Hopkins University School of Medicine AAO 2022
Slide 2: Financial Disclosure
Financial Disclosure I have the following financial interests or relationships to disclose: Allegro: Stock Options – Public or Private Corp Graybug Vision: Stock Options – Public or Private Corp AsclipiX: Consultant/Advisor, Grant Support Gyroscope: Consultant/Advisor Ashvattha Therapeutics: Consult
Slide 3: Vision outcomes in nAMD decrease over time despite anti-VEGF therapy
Vision outcomes in nAMD decrease over time despite anti-VEGF therapy Mean VA Change after RCTs1 Mean VA Change in Real World Practice2 15 8 OL Extension Period RCT Period 6 10 Change in BCVA (Letters) Change in BCVA (Letters) 4 5 2 0 0 -5 -2 -10 -4 0 1 2 3 4 5 6 7 8 0 1 2 3 4 5 Time (Years) Time (Ye
Slide 4: Gene Therapy for nAMD
Gene Therapy for nAMD Different Transgenes and Delivery Routes Being Tested Most Advanced with Regard to Development is Subretinal Injection of RGX-314 RGX-314 Subretinal Injection AAV8 vector containing an expression construct coding for Fab similar to ranibizumab 4
Slide 5: RGX-314 Phase I/IIa nAMD Study is Complete
RGX-314 Phase I/IIa nAMD Study is Complete Long-Term Follow up Baseline assessment Treatment evaluation up to 5 Years* anti–VEGF SD–OCT injection assessment Anti–VEGF PRN Rescue Injection Criteria Previously Treated Patients 0 1 2 6 10 14 18 22 26 54 106 Requiring Weeks Frequent RGX–314 Subretinal I
Slide 6: RGX-314 Phase I/IIa nAMD Study Outcomes
RGX-314 Phase I/IIa nAMD Study Outcomes ▪ Dose dependent increases in treatment effect were observed in the RGX-314 Phase I/IIa study ▪ After study completion, patients were encouraged to enroll in a Long-Term Follow-up study for a total of 5 years of follow-up post RGX-314 administration – Doses si
Slide 7: Long-Term Follow-Up (LTFU) Study of Phase I/IIa Patients
Long-Term Follow-Up (LTFU) Study of Phase I/IIa Patients* Baseline Assessment Treatment Evaluation Follow-up Long-Term Follow-up Anti–VEGF PRN Rescue Injection Criteria Long-term Safety & Efficacy of RGX-314 0 1 2 2.5 3 4 5 Year RGX–314 Subretinal Administration Years post-RGX-314 ▪ Long Term Follow
Slide 8: RGX-314 Long-Term Follow-Up Study: Safety
RGX-314 Long-Term Follow-Up Study: Safety ▪ RGX–314 continues to be generally well-tolerated in the long-term follow-up study (n=37*) with 2.5–5 years of follow-up ▪ 9 SAEs were reported in 4 patients, and none were considered drug-related ▪ Drug-related ocular AEs: – Cohort 1–4: no new events – Coh
Slide 9: Long-Term Follow-Up: Mean BCVA for Cohorts 3 and 4
Long-Term Follow-Up: Mean BCVA for Cohorts 3 and 4 Best Corrected Visual Acuity (BCVA) +14 Letters 80 Cohort 3 +12 Letters 2 Years Cohort 3 4 Years 70 60 Letters Read -5 Letters -1 Letters Cohort 4* 50 Cohort 4* 3 Years 2 Years 40 30 20 D1 M1 M6 1 Yr 1.5 Yrs 2 Yrs 2.5 Yrs 3 Yrs 4 Yrs Time Cohort 3 C
Slide 10: Individual Patients: Cohort Injections and Change in BCVA Over Years
Cohort 3 Injections and Change in BCVA Over 4 Years Long-term Follow-up Study* -2 letters** +36 letters Individual Patients +16 letters 0 letters +22 letters +1 letters 1 Year 0 1 2 3 4 RGX-314 Year Years Years Years Time Prior to Treatment Time Since RGX-314 with RGX-314 RANIBIZUMAB AFLIBERCEPT BEV
Slide 11: Individual Patients: Cohort Injections and Change in BCVA Over Years
Cohort 4 Injections and Change in BCVA Over 3 Years Long-term Follow-up Study* RGX-314 Patients that enrolled and had a visit in the LTFU Study -52 letters** -18 letters +16 letters Individual Patients -13 letters +8 letters +4 letters +1 letters -10 letters +14 letters -2 letters 1 Year 0 1 Year 2
Slide 12: Key Learning: Exclude Subjects with Uncontrolled and End-Stage Disease in Pivotal Trials
Key Learning: Exclude Subjects with Uncontrolled and End-Stage Disease in Pivotal Trials RGX-314 Phase I/IIa: Characteristics of Patients to be Excluded in Pivotal Trials Baseline Week 1- OCT response Week 3 Patients with Very Large Central Retinal Thickness (CRT) Baseline Subfoveal Fibrosis, Subfov
Slide 13: Mean Change in Annualized Injection Rate PRE and POST RGX-314
Mean Change in Annualized Injection Rate PRE and POST RGX-314 in Cohorts 1–5 Over 4 Years Annualized Injection Rate* Lower RGX-314 Doses 12 10.3 -5.2% 10.5 -3.6% 10.2 9.6 9.9 10 9.5 9.3 9.4 9.3 9.1 Annualized Injections 8 6.8 6 -58.4% 4.4 4.4 4 -67.0% -78.1% 2.8 2.4 2.4 2.0 2 Pre Pre Pre Pre Pre RGX
Slide 14: Mean Change in BCVA for Cohort 3 and 4 Compared to Lower Doses
Mean Change in BCVA for Cohort 3 and 4 Compared to Lower Doses (Cohort 1 & 2 combined) Cohorts 1 and 2 lost vision despite regular injections +14 Letters 80 Cohort 3 +12 Letters 2 Years Cohort 3 70 4 Years 60 -5 Letters Letters Read -1 Letters Cohort 4* Cohort 4* 3 Years 2 Years 50 40 -4 Letters Coh
Slide 15: Interim Conclusions from the RGX-314 Subretinal Long-Term Follow-Up Study
Interim Conclusions from the RGX-314 Subretinal Long-Term Follow-Up Study ▪ Subretinal RGX-314 continues to be generally well-tolerated in a Long-Term Follow-Up Study (n=37*) and will continue to be followed for a total of 5 years ▪ No new drug-related ocular AEs were reported in the last year of fo
Slide 16: RGX-314 Subretinal nAMD Phase I/IIa – LTFU Study Group
RGX-314 Subretinal nAMD Phase I/IIa – LTFU Study Group ▪ Robert Avery, MD (Santa Barbara, CA) University of ▪ David Brown, MD (Houston, TX) Pennsylvania, Ophthalmic Philadelphia, PA Consultants ▪ Peter Campochiaro, MD (Baltimore, MD) of Boston, Boston, MA ▪ Stephen Huddleston, MD (Memphis, TN) Sierr