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.
ProQR Therapeutics N.V. today 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:
Aniz Girach, MD, chief medical officer of ProQR Therapeutics, pointed out that the post-hoc analyses show an encouraging efficacy signal when comparing the active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints and are consistent with feedback received from the investigators.
“In prior interactions, EMA preferred the use of the contralateral eye as a control, however the protocol was ultimately harmonized globally to use a parallel sham group based on US regulatory requirements,” Girach said in a statement. “While we were disappointed by the outcome of the primary analysis, we believe that these post-hoc analyses and the observation that approximately a third of the patients benefited across multiple concordant endpoints in this trial, in combination with the high unmet need in LCA10, warrants a discussion with the regulators.”
Data from the Illuminate trial will be presented at the upcoming Retinal Cell and Gene Therapy Innovation Summit on April 29 and the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, May 1-4.