According to the company, 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. The company plans to discuss findings with regulators in Q3.
This week, ProQR Therapeutics N.V. provided an update on its sepofarsen program, following a comprehensive post-hoc analysis of the data from the Phase 2/3 Illuminate trial in people with CEP290-mediated Leber congenital amaurosis 10, or LCA10, a severe inherited retinal disease.
Following the top-line announcement in February that Illuminate, ProQR’s pivotal Phase 2/3 trial of sepofarsen in CEP290-mediated LCA10, did not meet the primary endpoint of Best Corrected Visual Acuity (BCVA) at Month 12 compared to a sham procedure control group, comprehensive post-hoc analyses of the trial were undertaken which revealed:
The company noted that the post-hoc analyses showed that the efficacy signal seen with sepofarsen when comparing active treatment and sham eyes to their corresponding contralateral eyes across BCVA, FST, and other endpoints, including PROs, was more consistent with the results seen in earlier findings, where the contralateral eye was used as the control.
Based on these results, ProQR will focus on the following core activities related to sepofarsen: