Half-Year 2022 Financial and Clinical Trials Update
Hemophilia A
Unique gene therapy platform
Molecule
Indication
Phase/study
# of patients
Design
Phase I
N=100
SPK-8011
(RG6357)
Hemophilia A
Phase I/II
N=30
◉
Gene transfer, dose-finding safety, tolerability,
and efficacy study of SPK-8011
■Long term follow up study of patients who
have received SPK-8011 in any prior Spark-
sponsored SPK-8011 study
Safety
Primary endpoint
Ongoing
Status
CT Identifier
NCT03432520
• Safety and changes from baseline in FVIII
activity levels at week 52
"
Spark
THERAPEUTICS
TM
SPK-8016
(RG6358)
Hemophilia A with inhibitors to Factor VIII
Roche
"
Phase I/II
N=30
Gene transfer, dose-finding safety, tolerability, and
efficacy study of SPK-8016 in individuals with FVIII
inhibitors
Safety; peak and steady state FVIII activity levels at
week 52
☐
FPI Q1 2017
■ Updated data presented at ISTH 2020 and 2021
Recruitment completed Q1 2021
◉
FPI Q1 2019
Data published in NEJM 2021; 385:1961-1973
ISTH=International Society on Thrombosis and Haemostasis; NEJM-New England Journal of Medicine
NCT03003533
NCT03734588
161
HemophiliaView entire presentation