Study did not meet primary endpoint nor notable secondary endpoints – no benefit observed in either treatment arm versus shamAdditional analyses to be conducted and presented at a future medical congressSepofarsen continues to be generally well tolerated
Foundation Fighting Blindness and ProQR enter into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene.
Reported rapid, significant and durable improvements in vision at twelve months
Concordant improvement in key secondary outcome measures
The target registration dose of sepofarsen was well-tolerated with a favorable benefit/risk profile
Strengthens confidence