Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — Technical Presentation (2022)
16 slides from Biomarin Pharmaceutical's technical presentation, 2022
Slide 1: Health-Related Quality of Life Over 2 Years Following Valoctocogene Roxaparvovec Gene Transfer
Health-Related Quality of Life Over 2 Years Following Valoctocogene Roxaparvovec Adeno-Associated Virus Gene Transfer For Severe Hemophilia A: Results From GENEr8-1 Amy L Dunn1, Andrew D Leavitt2, Flora Peyvandi3,4, Hervé Chambost5, Erin Cockrell6, Rashid Kazmi7, Robert Klamroth8, Gillian Lowe9, Joh
Slide 2: Disclosures for: Amy L Dunn
Disclosures for: AMY L DUNN Conflict Disclosure - if conflict of interest exists Sanofi, Takeda, Freeline, BioMarin Research Support Pharmaceutical Inc, and ATHN Service on the board of the World Federation of Director, Officer, Employee Hemophilia, USA Shareholder Honoraria Advisory Committee BioMa
Slide 3: Valoctocogene roxaparvovec gene transfer for severe HA
Valoctocogene roxaparvovec gene transfer for severe HA AAV5 capsid hFVIII-SQ expression cassette Hepatocytes FVIII 3 AAV5, adeno-associated virus serotype 5; hFVIII-SQ, B-domain deleted human factor VIII; FVIII, factor VIII; HA, hemophilia A.
Slide 4: GENEr8-1 phase 3 study
GENEr8-1 phase 3 study1 • 134 adult men with severe HA Baseline Characteristics mITT (N = 132) Age at enrollment, years (mean ± SD) 31.4 ± 10.1 o No inhibitors Male sex 132 (100) o No detectable antibodies to AAV5 capsid Race and ethnicitya White 94 (71.2) • 6x1013 vg/kg valoctocogene roxaparvovec I
Slide 5: Quality of life questionnaires
Quality of life questionnaires 1 3 WPAI+CIQ:HS EQ-5D-5L EuroQol 5 Dimension 5 Level Work Productivity and Activity Impairment plus Classroom Impairment Questions: Hemophilia Specific 2 4 Haemo-QOL-A HAL Haemophilia-specific Quality of Life Haemophilia Activities List Questionnaire for Adults 5
Slide 6: EQ-5D-5L
EQ-5D-5L Best health you can imagine mobility self-care 5 dimensions usual activities pain/discomfort anxiety/depression no problems slight problems 5 levels moderate problems severe problems extreme problems Worst health you can imagine 6
Slide 7: EQ-5D-5L VAS and Utility Index scores
EQ-5D-5L VAS and Utility Index scores CFB data are based on participants with data at both time points. 7 CFB, change from baseline; CI, confidence interval; CID, clinically important difference; EQ-5D-5L, EuroQol 5 Dimension 5 Level; SD, standard deviation; VAS, visual analogue scale.
Slide 8: Haemo-QOL-A domains
Haemo-QOL-A domains Physical Role Consequences Functioning Functioning of Bleeding Emotional Treatment Worry Impact Concern 8
Slide 9: Haemo-QOL-A Total Score
Haemo-QOL-A Total Score ***P <0.001 and ****P <0.0001 were based on two-sided t-test of CFB vs 0 without controlling for multiplicity.####P <0.0001 based on two-sided t-test of CFB vs 0 performed as part of a hierarchical testing sequence controlling overall Type 1 error. CFB data are based on parti
Slide 10: Haemo-QOL-A Domain Scores
Haemo-QOL-A Domain Scores *P <0.05; ***P <0.001 and ****P <0.0001 were based on two-sided t-test of CFB vs 0 without controlling for multiplicity.####P <0.0001 based on two-sided t-test of CFB vs 0 performed as part of a hierarchical testing sequence controlling overall Type 1 error. CFB data are ba
Slide 11: Post hoc analysis by pre-existing problem joints
Post hoc analysis by pre-existing problem joints Change in Haemo-QOL-A domain scores were grouped by number of problem joints at baseline. Not all participants completed all domains. CFB data are based on participants with data at both time points. For the “0 problem joints “ group with at baseline,
Slide 12: Post hoc analysis by changes in bleeding rate
Post hoc analysis by changes in bleeding rate Change in Haemo-QOL-A domain scores were grouped by whether annualized rate of treated bleeds improved, worsened, or did not change from baseline to the data cutoff date. All participants with no change in bleed rate had rates of zero before and after ge
Slide 13: WPAI+CIQ:HS scores
WPAI+CIQ:HS scores Data presented as mean (95% CI). CFB data are based on participants with data at both time points. 13 CFB, change from baseline; CI, confidence interval; WPAI+CIQ:HS, Work Productivity and Activity Impairment plus Classroom Impairment Questions: Hemophilia-Specific.
Slide 14: HAL summary and domain scores
HAL summary and domain scores Data presented as mean (95% CI). CFB data are based on participants with data at both time points. 14 CFB, change from baseline; CI, confidence interval; HAL, Haemophilia Activities List.
Slide 15: Conclusions
Conclusions Improvements in health-related quality of life were maintained through 2 years post gene transfer An annualized treated bleed rate of zero on FVIII prophylaxis did not preclude appreciable, meaningful quality of life improvements for participants after gene transfer Participants with
Slide 16: Acknowledgments
Acknowledgments • Thank you to all the trial participants, their families, study site personnel, and the GENEr8-1 Trial Group • Funding for this study was provided by BioMarin Pharmaceutical Inc • Medical writing support was provided by Prachi Patil, MS, of AlphaBioCom, and funded by BioMarin Pharma
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