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
Agreement provides ProQR with initial payment of €12.5M and up to €135M in further payments, as well as potential additional earn outs based on commercial sales in the US and EU
Divestment of sepofarsen and ultevursen supports ProQR’s strategic focus on the Axiomer®&
Top-line results expected H1 2022
Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan.
LEIDEN, Netherlands & CAMBRIDGE, Mass., April 23, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for
Call on July 20 will include a snapshot from the ongoing InSight open-label extension study of sepofarsen, including preliminary second eye treatment data
Top-line data now expected to be announced in Q1 2022Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
… Positive Top-Line Results from the Phase 1/2 Study of Sepofarsen in LCA10 Patients Reported rapid, significant and … Illuminate, were more likely to respond to treatment with sepofarsen. Table: Change from baseline in treated eye …
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators in Q3
LEIDEN, Netherlands & CAMBRIDGE, Mass., June 08, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced a virtual presentation of data fro
ProQR will focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas. This comes following feedback from the European Medicines Agency (EMA) related to ProQR’s sepofarsen for Leber Congenital Amaurosis type 10 (LCA10).
