Slide 1: Health-Related Quality of Life Outcomes 4 Years After Treatment with Valoctocogene Roxaparvovec
HEALTH-RELATED QUALITY OF LIFE OUTCOMES 4 YEARS AFTER TREATMENT WITH VALOCTOCOGENE ROXAPARVOVEC Bella Madan1, Margareth C. Ozelo2, Gili Kenet3, Sheng-Chieh Chou4, Steven W. Pipe5, Andrew D. Leavitt6, Francisco P. Careta7, Patrick James Lynch8, Simon Fletcher9, Dor Goshen10, Andres Ruiz11, Ebony Dash
Slide 2: Disclosures
Disclosures • I have acted as a speaker or member of a speaker bureau for Bayer, BioMarin, CSL Behring, Roche, Takeda and Viatris Pharmaceuticals, and as a consultant or ad hoc speaker/consultant for Bayer, BioMarin, Pfizer, Takeda and Viatris Pharmaceuticals 2
Slide 3: Hemophilia A
Hemophilia A People with hemophilia A lack the Hemophilia A can negatively affect blood clotting protein FVIII because health-related quality of life (HRQOL) the gene is faulty Constant Low factor VIII (FVIII) levels can worry result in excessive bleeding or bleeding with no apparent cause Current t
Slide 4: Valoctocogene roxaparvovec and HRQOL outcomes
Valoctocogene roxaparvovec and HRQOL outcomes Valoctocogene roxaparvovec is a gene Potential to reduce disease burden therapy for severe hemophilia A that of hemophilia A4 delivers functional genetic instructions for producing FVIII Valoctocogene roxaparvovec provides protection from bleeding with a
Slide 5: GENEr8-1 study design
GENEr8-1 study design Eligibility: 134 participants enrolled and received an infusion of valoctocogene roxaparvovec • Adult men with severe hemophilia A (FVIII ≤1 IU/dL) The modified intention-to-treat (mITT) population • Previously receiving FVIII prophylaxis included the 132 participants who were
Slide 6: Haemo-QOL-A measures HRQOL in people with hemophilia
Haemo-QOL-A measures HRQOL in people with hemophilia Rating Scale Domains Examples (converted to 0–100) Physical 9 items “It is easy for me to lift 0 None of Functioning heavy objects” the time “I feel like a burden to 1 A little of Role Functioning 11 items my family” the time Consequences of 7 ite
Slide 7: Valoctocogene roxaparvovec improved Haemo-QOL-A Total Score across 4 years
Valoctocogene roxaparvovec improved Haemo-QOL-A Total Score across 4 years mITT population The improvements at the end of each year were deemed clinically meaningful1 16 Mean improvement from baseline 14 12 7.3 6.8 6.5 10 6.3 8 6 4 2 Y1 Y2 Y3 Y4 0 = 127 122 113 103 1. Quinn J, et al. Patient Relat O
Slide 8: Consistent improvements for Haemo-QOL-A domain scores
Consistent improvements for Haemo-QOL-A domain scores mITT population 16 10.5 10.7 9.7 9.2 14 7.6 7.2 7.5 12 6.0 6.1 5.3 6.2 10 4.8 8 6 Mean improvement from baseline 4 2 0 Y1 Y2 Y3 Y4 Y1 Y2 Y3 Y4 Y1 Y2 Y3 Y4 Physical Functioning Role Functioning Consequences of Bleeding = 130 125 114 106 129 124 11
Slide 9: HRQOL improvements were partly independent of FVIII activity
HRQOL improvements were partly independent of FVIII activity mITT population Improvement in Haemo-QOL-A Total Score at the end of year 4 was deemed clinically meaningful for participants with FVIII levels below 5%1 16 14 7.6 Mean change from baseline 12 6.3 10 6.2 8 6 4 2 0 1 2 3 FVIII levels corres
Slide 10: HAL measures self-reported functional ability for people with hemophilia
HAL measures self-reported functional ability for people with hemophilia In the previous month, did you have any difficulty due to Domains hemophilia with… Rating Scale Lying/Sitting/ “Standing for long 1 Impossible 8 items Kneeling/Standing periods of time” Function of the “Walking on an Always Leg
Slide 11: HAL Summary Score improved over years
HAL Summary Score improved over 4 years mITT population 8 4.7 4.0 3.0 Mean change from baseline 6 2.4 4 2 0 Year 1 Year 2 Year 3 Year 4 = 124 122 113 104 Results are based on available data at each time point. Error bars represent 95% confidence intervals. Data after participants resumed prophylaxis
Slide 12: The WPAI+CIQ:HS measures impairment at work and school due to hemophilia
The WPAI+CIQ:HS measures impairment at work and school due to hemophilia “In general, how many hours per week do you usually work/attend classes?” 0 Hemophilia had no effect on my work/classwork “During the past seven days, how many hours did you miss from work/class or school because of problems as
Slide 13: WPAI+CIQ:HS activity impairment was reduced over years
WPAI+CIQ:HS activity impairment was reduced over 4 years mITT population 0 Mean percent change from baseline -2 -4 -6 -8 -10 −5.2 −5.1 −5.4 -12 −7.6 -14 Year 1 Year 2 Year 3 Year 4 = 115 117 99 98 Results are based on available data at each time point. Error bars represent 95% confidence intervals.
Slide 14: Conclusions
Conclusions Haemo-QOL-A HAL • Valoctocogene roxaparvovec • Participants reported improved ability to provides clinically meaningful HRQOL perform daily activities over 4 years improvements over 4 years • The meaningful improvements also apply to participants with FVIII activity below 5% at year 4 WP
Slide 15: Acknowledgments
Acknowledgments • Thank you to all trial participants, their families, study site personnel, and investigators • Funding for this study was provided by BioMarin Pharmaceutical Inc. • Medical writing support was provided by Amin Ghane, PhD, of AlphaBioCom, a Red Nucleus company, and funded by BioMari