Regenxbio Inc. · NASDAQ:RGNX

Regenxbio — Technical Presentation (October 2024)

17 slides from Regenxbio's technical presentation, Oct 19, 2024

  1. Slide 1: Subretinal Delivery of Investigational ABBV-RGX-314 as a Gene Therapy for nAMD

    Subretinal Delivery of Investigational ABBV-RGX-314 as a Gene Therapy for nAMD: First Time Results of a Fellow Eye Bilateral Dosing Study Arshad M. Khanani, MD, MA, FASRS Managing Partner, Director of Clinical Research, Director of Fellowship, Sierra Eye Associates Clinical Professor, University of

  2. Slide 2: Disclosures

    Disclosures 4D Molecular Therapeutics: C, G Frontera Therapeutics: C Opthea: C, G AbbVie: C Genentech, Inc.: C, G Oxular: G Adverum Biotechnologies : C, G Gyroscope: C, G Oxurion: C, G Aerie: C iLumen: C Perfuse: C Aldebaran: C Iveric Bio: C, G, S PolyPhotonix: C, O Alexion: G Janssen: C Protagonist

  3. Slide 3: Ocular Gene Therapy Has the Potential to Close the Gap Between Trials and Real-World Outcomes

    Ocular Gene Therapy Has the Potential to Close the Gap Between Randomized Clinical Trials and Real-World Outcomes Visual Acuity Over Time in nAMD 12 Clinical Trial Data1 Real-World Data2 10 Visual Acuity Letter Change 8 6 4 Sustained Vision Gain 2 0 -2 -4 Baseline Year 1 Year 2 Year 3 Year 4 Year 5

  4. Slide 4: The Potential of Bilateral Dosing of Gene Therapy in nAMD

    The Potential of Bilateral Dosing of Gene Therapy in nAMD nAMD is frequently a bilateral disease and the ability to treat both eyes with gene therapy is important • Two other subretinal AAV gene therapies have treated IRD patients bilaterally1,2 • After exposure to AAVs, antibodies are formed, which

  5. Slide 5: ABBV-RGX-314 for Treatment of Neovascular Age-related Macular Degeneration (nAMD)

    ABBV-RGX-314 for Treatment of Neovascular Age-related Macular Degeneration (nAMD) A B B V - R G X - 3 1 4 P R O D U C T C A N D I D AT E ROUTES OF Subretinal OR Suprachoroidal N A V ® V E C T O R AAV8 A D M I N I S T R AT I O N (nAMD) (nAMD/DR) G E N E Anti-VEGF fab MECHANISM Reducing leaky blood ve

  6. Slide 6: Current Program Status for ABBV-RGX-314

    Current Program Status for ABBV-RGX-314 Phase 1/2a trial is complete LO N G - T E R M F O L LOW - U P S T U DY Subretinal Subretinal ABBV-RGX-314 for nAMD Phase 2 pharmacodynamic trial is ongoing in patients from parent trials Phase 2b/3 pivotal trials are ongoing F EL LOW EY E S U B - ST UDY Invest

  7. Slide 7: Subretinal Program: Interim Results of Previous Studies

    SUBRETINAL PROGRAM: Interim Results of Previous Studies ABBV-RGX-314 for nAMD OV E R A L L SA F E T Y ABBV-RGX-314 has been well tolerated across Phase I/IIa (up to 4 years)1 and Phase II Pharmacodynamic studies (at 6 months)2 at doses similar to pivotal study EFFICACY ENDPOINTS With a single inject

  8. Slide 8: First Ever Bilateral Gene Therapy Clinical Trial for Treatment of nAMD

    First Ever Bilateral Gene Therapy Clinical Trial for Treatment of nAMD Fellow Eye Sub-Study of Subretinal ABBV-RGX-314 2nd Eye Treatment Evaluation (n=20) 1st Eye treated: Anti-VEGF Injections Anti-VEGF PRN Retreatment Criteria PHASE 1/2a STUDY n=3 W -2 D1 W2 3 Mo 6 Mo 9 Mo 1 Year PHASE 2 PHARMACODY

  9. Slide 9: Fellow Eye Sub-Study (FESS): Open Label Patient Population

    Fellow Eye Sub-Study (FESS): Open Label Patient Population FESS BASELINE DEMOGRAPHICS I N J E C T I O N S I N Y E A R P R I O R T O T R E AT M E N T (Open Label Patients Only) ABBV- Ranibizumab Aflibercept Faricimab RGX- 314 2nd Study Eye (n=10) Mean age (years) 77.6 72.1 BCVA (letters) (~20/40) OCT

  10. Slide 10: Safety Summary for 2nd Eyes through Month 9

    Safety Summary for 2nd Eyes through Month 9 • ABBV-RGX-314 was well-tolerated by all open-label subjects (n=10) • No drug-related SAEs • No cases of intraocular inflammation, chorioretinitis, vasculitis, occlusion, or hypotony were observed • Common AEs1 in the treated fellow eye included: Retinal p

  11. Slide 11: 78% of Treated 2nd Eyes Were Injection-free through Month 9

    78% of Treated 2nd Eyes Were Injection-free through 9 Months ABBV- Number (%) of Y E A R P R I O R T O T R E AT M E N T RGX-314 TIME SINCE ABBV-RGX-314 Injection-free Subjects 7/9 (78%) -1 Year Day 1 Month 9 Per-Protocol Ranibizumab Ranibizumab Aflibercept Faricimab Visit with no injection 100% of T

  12. Slide 12: Subjects Had Meaningful Reduction in Anti-VEGF Injection Burden in 2nd Eyes

    Subjects Had Meaningful Reduction in Anti-VEGF Injection Burden in 2nd Eyes Treated Annualized Injection Rate Based on Month 9 Data 12 10 Annualized Injections 8 9.0 6 -97.0% 4 2 Pre 0.3 ABBV-RGX-314 0 Post FELLOW EYE SUB-STUDY (n=10) PRE ABBV-RGX-314 Mean Annualized Rate POST ABBV-RGX-314 Mean Annu

  13. Slide 13: Treated 2nd Eyes Demonstrated Sustained BCVA and CRT through Month 9

    Treated 2nd Eyes Demonstrated Sustained BCVA and CRT through Month 9 M EA N B EST CO R R EC T E D V I S UA L AC U I T Y (B C VA ) 9 5 % C I AB B V- 85 RGX -314 80 Letters Read 75 +1.1 70 Letters 65 60 55 W-2 W2 M1 M2 M3 M5 M7 M9 anti–VEGF Time MEAN CENTRAL RETINAL THICKNESS (CRT) 95% CI 500 AB B V -

  14. Slide 14: Treated 2nd Eyes Produce Similar Levels of Aqueous Protein as 1st Eyes

    Treated 2nd Eyes Produce Similar Levels of Aqueous Protein as 1st Eyes after Bilateral Treatment with ABBV-RGX-314 P R O T E I N L E V E L S (Dose: 1.3x1011 GC/eye ) 1,000 Treated 1st Eyes Treated 2nd Eyes Protein Concentration 800 600 (ng/ml) 474.0 ng/mL 474.3 ng/mL 400 200 0 PHASE 2 PD STUDY FELLO

  15. Slide 15: Patient Case: Pivotal Doses Administered in Both Eyes

    Patient Case: Pivotal Doses Administered in Both Eyes Injection-free in Both Eyes 1st Eye: OD 2nd Eye: OS Low Dose High Dose 6.4x1010 GC/eye 1.3x1011 GC/eye + 6 Letters +4 Letters 76 letters 84 letters 82 letters 80 letters 84 letters Baseline 1 Year Baseline Month 9 Received 9 injections in the pri

  16. Slide 16: 2nd Eyes Treated Achieved Similar Safety and Efficacy Outcomes to 1st Eyes

    2nd Eyes Treated Achieved Similar Safety and Efficacy Outcomes to 1st Eyes Treated with ABBV-RGX-314 9 Month Results of ABBV-RGX-314 Subretinal 2nd Eye Treatment Meaningful reduction in Well-tolerated anti-VEGF injections in patients with high treatment burden Sustained vision and anatomy • 78% Inje

  17. Slide 17: Acknowledgements

    Acknowledgements ▪ Ashkan Abbey, MD (Dallas, TX) ▪ Suhail Alam, MD (Phoenix, AZ) ▪ Peter Campochiaro, MD (Baltimore, MD) ▪ David Eichenbaum, MD (Clearwater. FL) Wills Eye Institute, Philadelphia, PA ▪ Sarah Haug, MD PhD (Durango, CO) ▪ Allen Ho, MD (Philadelphia, PA) Sierra Eye Associates Southwest

REGENXBIO — Technical Presentation (October 2024) on 6ix