Pharma Update
SPK-8011 in hem A: Durable FVIII activity observed up to 5 years
FVIII activity accompanied by durable clinical benefit in ABR, joint health and quality of life
Study design
5 1011 vg/kg (n=2)
1 1012 vg/kg (n=3)
2 1012 vg/kg (n=9)
1.5 1012 vg/kg (n=10)
1-hour
outpatient
IV infusion*
52 (+2) weeks
4-year long-term
follow-up
period
(n=24)
follow up study:
visits every 3-6
months
(n=18)
Mean FVIII activity OSA (%)
Roche
FDA BTD
Stable FVIII activity levels and improved QoL over long-term follow-up
Mean FVIII activity OSA
50
1.5x1012 vg/kg
5x1011 vg/kg
1x1012 vg/kg
2x1012 vg/kg
35
30
15
10
4422250
5
0
>52-104
>104-156
>156-208
>208
Time after vector administration (weeks)
Mean (SD) change in
Haem-A-QoL TS
Change in Haem-A-QoL
total score from baseline
Year 1 Year 2
Year 3
5050522
-30
-12.3
(16.3)
-11.3
-13.4
(15.8) (13.0)
n=16
n=15
n=13
Efficacy and safety of single SPK-8011* infusion:
•
16 participants with ≥1 year follow-up showed no apparent decrease in FVIII activity over time, with a majority expressing in the mild HA range
•
82-99% reductions in ABR and substantial reductions in FVIII consumption, no major safety signals
•
•
Clinically meaningful improvements in joint health by total HJHS score at Year 2 and 3 and in total Haem-A-QoL at Years 1, 2, and 3
Ph III study to open enrolment in 2H 2023
© dirloctocogene samoparvovec; Croteau et al ASH 2022; Data cut-off: Oct 4 2022; analysis includes participants with >52 weeks of follow up (n=16). Grey shaded area indicates the mild range of hemophilia A; FVIII assessments
obtained within 5 days of administration of a FVIII infusion were excluded; OSA-one-stage assay; FVIII:C=FVIII coagulant activity; HA=hemophilia A; QoL-Quality of life; ABR=Annualized bleeding rate; M.V. Ragni et al., ISTH 2023,
B. Samelson-Jones et al., ISTH 2023
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