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®&
Divestment of sepofarsen and ultevursen completed – Théa to continue development of sepofarsen and ultevursen for patients with LCA10 and Usher syndrome
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).
ProQR has announced the completion of an in-depth strategic review designed to deliver on our commitment to advance RNA therapies for diseases with high unmet need. We are also sharing additional analyses of the sepofarsen Illuminate trial.
The EFF Spring Virtual Summit hosted by ProQR on April 15 is a virtual meeting featuring the latest clinical data for USH2A mediated Usher syndrome and retinitis pigmentosa and a live panel discussion.
ProQR shared additional analyses from the Phase 2/3 Illuminate trial of sepofarsen for LCA10 and provided an update on the company strategy. Management to host a conference call today at 8:00am EDT (14:00 CET).
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
ProQR Therapeutics today announced positive results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations.
