Regenxbio Inc. · NASDAQ:RGNX

Regenxbio — ASRS 2023

13 slides from Regenxbio's conference presentation, Jul 30, 2023

  1. Slide 1: Suprachoroidal Delivery of Investigational ABBV-RGX-314 for Neovascular AMD: Phase II AAVIATE Results

    1 Suprachoroidal Delivery of Investigational ABBV-RGX-314 for Neovascular AMD: Results from the Phase II AAVIATE® Study David Boyer, MD Adjunct Clinical Professor Ophthalmology Keck School of Medicine Partner Retina Vitreous Associates Medical Group ASRS 30 July 2023

  2. Slide 2: Disclosures

    Disclosures  Consultant: 4DMT, Adverum Biotech, AiViva, Alcon, Aldeyra, Alimera, Alkahest, Allegro, Allergan, Allgenesis, Amydis, Annexon, Apellis, AGTC, AsclepiX, Ashvattha, Aviceda, Bausch + Lomb, Bayer, Biovisics, Boehringer Ingelheim, Cell Care, Chengdu Kanghong, Clearside Bio, Curacle, Delsite

  3. Slide 3: AAVIATE: ABBV-RGX-314 Suprachoroidal Phase II Clinical Trial in nAMD

    AAVIATE®: ABBV-RGX-314 Suprachoroidal (SCS) 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 ABBV-RGX

  4. Slide 4: AAVIATE: ABBV-RGX-314 Phase II Clinical Trial in nAMD

    AAVIATE®: ABBV-RGX-314 Phase II Clinical Trial in nAMD Primary Objective Route of Administration  To evaluate the mean change in BCVA for ABBV-RGX-314  In-office SCS Microinjector™ delivers ABBV-RGX-314 to the compared with ranibizumab monthly injection at Month 9 suprachoroidal space Secondary Ob

  5. Slide 5: AAVIATE Baseline Characteristics (Cohorts 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

  6. Slide 6: AAVIATE Interim Safety Summary

    AAVIATE® Interim Safety Summary  ABBV-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

  7. Slide 7: Cohorts 1–4: Mean BCVA and CRT Through Month 6

    Cohorts 1–4: Mean BCVA and CRT from Day 1 (Screening) 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 ABBV-RGX-314 Cohort 1 75 -1.7 letters (n=19) 70 ABBV-RGX-314 Cohort 3 -0.07 Letters (n=15) 65 ABBV-RG

  8. Slide 8: Mean Change in Annualized Injection Rate Pre and Post ABBV-RGX-314

    Mean Change in Annualized Injection Rate PRE and POST ABBV-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 ABBV-RGX-314 6 Month ABBV-RGX-314 6 Month ABBV-RGX-314 6 Month ABBV-RGX-314 6 Mo

  9. Slide 9: 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 ABBV-RGX-314 Time BCVA Primary Secondary Assess

  10. Slide 10: Dose Level 3 with Short-course Prophylactic Ocular Steroids: Interim Safety

    AAVIATE® Dose Level 3 with Short-course Prophylactic Ocular Steroids: Interim Safety ABBV-RGX–314 has been well–tolerated in Dose Level 3  No study drug-related SAEs for AAVIATE (n=55)  No cases of chorioretinitis, vasculitis, occlusion, or hypotony No PPX Steroid w/PPX 26 Weeks 6–26 Weeks Follow-

  11. Slide 11: Cohort with Short-course Prophylactic Ocular Steroids: IOI

    AAVIATE® Cohort 6 with Short-course Prophylactic Ocular Steroids: IOI (AC Cells, AC Flare, Vit Cells, Vit Haze) ABBV-RGX-314 SUBJECT Dosing D2 W1 W2 W4 W6 W8 W10 W12 W14 W16 W18 W20 W22 W24 W26 COHORT 5 Periocular Steroid Patient 1 0 0 0 0 0 – 0 0 0 Patient 2 0 0 0 0 0 0 0 0 0 Patient 3 0 0 0 0 0 0

  12. Slide 12: Summary of Interim Results from the Phase II AAVIATE nAMD Study

    Summary of Interim Results from the Phase II AAVIATE® nAMD Study ABBV-RGX-314 Cohorts 1-5 (n=85): Safety  Suprachoroidal ABBV-RGX-314 has been well-tolerated ABBV-RGX-314 Cohorts 1-4 (n=65): 6 Month Results  ABBV-RGX-314 treated patients had stable vision and retinal thickness, with a meaningful r

  13. Slide 13: Thank You

    13 Thank You 13

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