Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — WFH Comprehensive Care Summit 2025
17 slides from Biomarin Pharmaceutical's conference presentation, April 2025
Slide 1: Efficacy and safety of valoctocogene roxaparvovec for severe hemophilia A
Efficacy and safety of SPEAKER Johnny Mahlangu valoctocogene roxaparvovec Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and for severe hemophilia A NHLS, Johannesburg, South Africa 5 years after gene transfer in GENEr8-1 ORGANI
Slide 2: Disclosures for: Prof. Johnny Mahlangu
Disclosures for: Prof. Johnny Mahlangu Conflict Disclosure - if conflict of interest exists BioMarin Pharmaceutical Inc., Novo Nordisk, Pfizer, Research Support Roche, Sanofi, Spark Therapeutics, and Vega Therapeutics Director, Officer, Employee Shareholder Honoraria (Speaker Bureau) Novo Nordisk, P
Slide 3: Valoctocogene roxaparvovec for severe hemophilia A
Valoctocogene roxaparvovec for severe hemophilia A Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is a liver-directed gene therapy that transfers a FVIII coding sequence to enable FVIII production in people with severe hemophilia A (FVIII ≤1 IU/dL)1-4 As previously shown, participants who received 6x10
Slide 4: Study design
Study design Eligibility Endpoints • Adult men with severe hemophilia A (FVIII ≤1 IU/dL) • FVIII activity • Previously receiving FVIII prophylaxis • Change from baseline • No history of FVIII inhibitors or anti-AAV5 • Annualized bleeding rate antibodies • Annualized FVIII infusion rate • No signific
Slide 5: Participant disposition
Participant disposition ITT population All participants infused N = 134 Rollover from BMN 270-902 Directly enrolled Directly enrolled HIV-negative, N = 112 HIV-negative, n = 20 HIV-positive, n = 2 mITT population HIV-negative N = 132 Discontinued from study, n = 6 • Lost to follow-up, n = 3 • Death,
Slide 6: Baseline characteristics
Baseline characteristics Rollover population mITT ITT Baseline characteristics N = 112 N = 132 N = 134 Age, years, mean (range) 31.8 (19–70) 31.4 (18–70) 31.7 (18–70) Race, n (%) White 78 (69.6) 94 (71.2) 96 (71.6) Asian 17 (15.2) 19 (14.4) 19 (14.2) Black or African American 14 (12.5) 15 (11.4) 15
Slide 7: No new safety signals in year 5
No new safety signals in year 5 ITT population (N = 134) Participants, n (%) In year 5 AEs in year 5 ITT (N = 129) AEs 102 (79.1) • No new safety signals SAEs 4 (3.1) • No grade 3 or higher ALT Treatment-related AEsa 5 (3.9) elevations occurred Treatment-related SAEs 0 (0.0) AEs grade ≥3 6 (4.7) • N
Slide 8: ALT elevation and glucocorticoid use
ALT elevation and glucocorticoid use ITT population (N = 134) In year 5 • 63 (48.8%) participants had an ALT elevation >1.5x baseline • 23 (17.8%) participants had an ALT Participants, n (%) elevation >ULN Events in year 5 (N = 129) ALT elevation >ULN, n (%) 23 (17.8) • No participants used glucocor
Slide 9: FVIII activity across the trial (CSA)
FVIII activity across the trial (CSA) mITT population (N = 132) 100 mITT population Week 208 Mean ± SE: 15.9 ± 2.5 IU/dL Mean ± SE FVIII activity (IU/dL) 80 Median (Q1, Q3): 6.5 (2.8, 17.7) IU/dL Week 156 60 Mean ± SE: 18.4 ± 2.7 IU/dL Median (Q1, Q3): 8.3 (3.0, 17.2) IU/dL Week 260 Mean ± SE: 13.7
Slide 10: FVIII activity (CSA) at the end of year
FVIII activity (CSA) at the end of year 5 mITT population (N = 132) 100% 10.6 7.6 8.3 90% 15.9 80% 37.1 58.3% of participants 70% remain in the mild to Participants, % 51.5 50.0 60% 56.1 non-hemophilic range 58.3 50% 40% 50.8 12.1 14.4 30% 9.1 Median FVIII activity per CSA 20% 10.6 24.2 26.5 29.5 10
Slide 11: FVIII activity across the trial (OSA)
FVIII activity across the trial (OSA) mITT population (N = 132) 100 mITT population Week 208 Mean ± SE: 26.7 ± 4.0 IU/dL Mean ± SE FVIII activity (IU/dL) Median (Q1, Q3): 13.2 (5.3, 29.1) IU/dL 80 Week 156 Mean ± SE: 28.2 ± 3.6 IU/dL 60 Median (Q1, Q3): 16.0 (5.3, 30.4) IU/dL Week 260 Mean ± SE: 24.
Slide 12: FVIII activity (OSA) at the end of year
FVIII activity (OSA) at the end of year 5 mITT population (N = 132) 100% 90% 18.9 16.7 15.2 27.3 80% 50.8 70% 73.5% of participants Participants, % 60% remain in the mild to 58.3 50% 60.6 61.4 non-hemophilic range 56.8 40% 30% 41.7 Median FVIII activity per OSA 20% 13.6 11.4 10.6 10% 9.8 6.1 9.1 11.
Slide 13: Durable hemostatic efficacy across years
Durable hemostatic efficacy across 5 years Rollover population (N = 112) ABR for treated bleeds decreased >80% from Annualized FVIII infusion rate decreased >90% baseline during the post-prophylaxis period from baseline during the post-prophylaxis period 15 88.1% reduction 250 93.3% reduction AFR (i
Slide 14: Most participants continue to remain off prophylaxis at year 5
Most participants continue to remain off prophylaxis at year 5 ITT population (N = 134) 81.3% of participants remain off prophylaxis at the end of year 5 (25/134 have resumed) 1.0 Prophylactic treatment-free probability 0.9 0.8 0.7 0.6 Only 1 additional 0.5 participant resumed 0.4 prophylaxis in yea
Slide 15: Conclusions
Conclusions Valoctocogene roxaparvovec provided stable, durable, and clinically significant efficacy with a reassuring clinical safety profile at 5 years Durable hemostatic efficacy • The rate of treated bleeds and FVIII infusions was reduced compared with FVIII prophylaxis 5 years after infusion, c
Slide 16: Acknowledgements
Acknowledgements • Thank you to all co-authors: • Thank you to all trial participants, their Andrew D. Leavitt, Priyanka Raheja, families, study-site personnel, and investigators Emily Symington, Doris V. Quon, Adam Giermasz, Gili Kenet, Gillian Lowe, • Funding for this study was provided by BioMari
Slide 17: Thank you!
Ay WFH COMPREHENSIVE $ CARE SUMMIT Thank you!
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