Biomarin Pharmaceutical Inc. · NASDAQ:BMRN

Biomarin Pharmaceutical — 13th BIC International Conference (September 2025)

14 slides from Biomarin Pharmaceutical's conference presentation, September 2025

  1. Slide 1: Safety and efficacy of valoctocogene roxaparvovec in participants with active or prior FVIII inhibitors

    Safety and efficacy of valoctocogene roxaparvovec in participants with active or prior FVIII inhibitors: Results from a phase 1/2 trial Konstantia-Maria Chavele1, Shyh-Shin Chiou2, Sheng-Chieh Chou3, Kaan Kavakli4, Gili Kenet5, Margareth C. Ozelo6, Young-Shil Park7, Jiaan-Der Wang8, Guy Young9 1BioM

  2. Slide 2: Disclosures

    Disclosures I am an employee and stockholder of BioMarin Pharmaceutical Inc. 2

  3. Slide 3: Valoctocogene roxaparvovec for severe hemophilia A

    Valoctocogene roxaparvovec for severe hemophilia A Valoctocogene Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) FVIII gene Vector roxaparvovec is a liver-directed gene therapy that transfers a B-domain-deleted FVIII coding sequence to enable FVIII production in people with severe hemophilia A (FVIII ≤1

  4. Slide 4: Preclinical studies provide strong evidence of gene therapy-mediated ITI

    Preclinical studies provide strong evidence of gene therapy-mediated ITI1–3 Canine model of HA 70 5 cFVIII antigen (ng/mL) 60 Antigen cFVIII activity (%) 4 50 Activity Inhibitors develop in 25%–40% 40 3 of individuals with HA receiving 30 2 replacement therapy1,4 20 1 10 0 0 0 10 20 30 40 50 100 150

  5. Slide 5: GENEr8-INH study design

    GENEr8-INH study design Primary objective: To assess the safety of a single IV administration of valoctocogene roxaparvovec for individuals with severe HA and active (part A) or prior (part B) FVIII inhibitors Part A: Active inhibitor population (n = 3) • Primary outcome: Participant 1 12-wk minimum

  6. Slide 6: Participant demographics

    Participant demographics Participants completed between 44.7 and 122.7 weeks of follow-up and all remained on study Part A Part B Parts A and B combined (n = 3) (n = 7) (N = 10) Age, years, mean (SD) 25.3 (5.7) 32.0 (12.7) 30.0 (11.2) Age at enrollment, n (%) 18 to <30 years 2 (66.7) 4 (57.1) 6 (60.

  7. Slide 7: Safety signals are consistent with results from GENEr8-1

    Safety signals are consistent with results from GENEr8-1 Part A and B combined safety outcomes Part A participants Part B participants Combined Safety outcomes (n = 3) (n = 7) (N = 10) AEs, n (%) 3 (100.0) 7 (100.0) 10 (100.0) SAEs, n (%) 0 1 (14.3) 1 (10.0) Treatment-related AEs, n (%) 2 (66.7) 6 (

  8. Slide 8: A: Active inhibitors - 30-year-old male receiving emicizumab

    Part A: Active inhibitors 30-year-old male receiving emicizumab Inhibitors FVIII activity (chromogenic) ALT FVIII antigen 380 200 360 340 FVIII activity (chromogenic, IU/dL) 320 300 280 150 FVIII antigen (ng/mL), 260 240 Inhibitors (BU) ALT (U/L), 220 200 100 180 160 140 120 100 50 80 60 40 20 0 0 W

  9. Slide 9: A: Active inhibitors - 27-year-old male receiving emicizumab

    Part A: Active inhibitors 27-year-old male receiving emicizumab Inhibitors FVIII activity (chromogenic) ALT FVIII antigen 30 200 FVIII activity (chromogenic, IU/dL) 25 150 FVIII antigen (ng/mL), 20 Inhibitors (BU) ALT (U/L), 15 100 10 50 5 0 0 Weeks Prophylactic GCs W2 to W22 ALT normal range, 5–48

  10. Slide 10: A: Active inhibitors - 19-year-old male receiving anti-inhibitor coagulant complex

    Part A: Active inhibitors 19-year-old male receiving anti-inhibitor coagulant complex Inhibitors FVIII activity (chromogenic) ALT FVIII antigen 30 200 FVIII activity (chromogenic, IU/dL) 25 150 FVIII antigen (ng/mL), 20 Inhibitors (BU) ALT (U/L), 15 100 10 50 5 0 0 Weeks Reactive GCs W8 to W14 ALT n

  11. Slide 11: B: Prior inhibitors - efficacy outcomes consistent with GENEr8-1

    Part B: Prior inhibitors Efficacy outcomes in participants with prior inhibitors are consistent with the results of GENEr8-1 Chromogenic assay (n = 7) One-stage assay (n = 7) 300 300 250 250 FVIII activity (IU/dL) FVIII activity (IU/dL) 200 200 150 150 100 100 50 50 0 0 Weeks Weeks FVIII activity va

  12. Slide 12: B: Prior inhibitors - efficacy outcomes consistent with GENEr8-1

    Part B: Prior inhibitors Efficacy outcomes in participants with prior inhibitors are consistent with the results of GENEr8-1 GENEr8-1 trial GENEr8-1 trial Efficacy outcomes Weeks 25–28 Weeks 49–52 Week 26 Weeks 49–52 n 7 112 5 112 FVIII activity, IU/dL, mean (SD) n = 132 n = 132 Chromogenic assay 50

  13. Slide 13: Conclusions

    Conclusions Valoctocogene roxaparvovec safety was similar for participants with or without inhibitors and had promising efficacy FVIII protein Participants showed a similar safety profile to the GENEr8-1 trial regardless of inhibitor status Prior inhibitor status was not associated with a relapse of

  14. Slide 14: Acknowledgments

    Acknowledgments Thank you to all the trial participants, their families, study-site personnel, and investigators • Funding for this study was provided by BioMarin Pharmaceutical Inc. • Project management support was provided by Gillian Clague, CMPP, of BioMarin Pharmaceutical Inc. • Medical writing

BioMarin Pharmaceutical — 13th BIC International Conference (September 2025) on 6ix