1. Jan 08, 2021

Enrollment complete in Phase 2/3 study of sepofarsen for LCA10

Patient enrollment has been completed in the Phase2/3 Illuminate study of sepofarsen (ProQR Therapeutics) for the treatment of Leber Congenital Amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene, according to a press release

Top-line results are expected in the first half of 2022.

In the study, 36 patients ≥8 years old were randomized to receive sepofarsen at the target registration dose, a low dose, or sham treatment. The primary endpoint is the mean change from baseline in best-corrected visual acuity at month 12, at the registration dose versus sham.

“We are pleased to have completed enrollment of the pivotal Illuminate trial of sepofarsen.  This marks an important milestone for ProQR, as well as for the LCA10 and broader inherited retinal disease community. In surpassing our enrollment target, we were able to accommodate the broad interest to participate in the trial,” said Aniz Girach, MD, Chief Medical Officer of ProQR in the press release. “This speaks to the fact that there are currently no approved treatments for patients with LCA10. If approved, sepofarsen has the potential to be the first therapy to address this high unmet medical need for patients who would otherwise face blindness. We are grateful to those who have supported our efforts in bringing this trial forward, including our investigators, patients, and caregivers. We look forward to sharing the top-line results in the first half of 2022.”

Read the full press release here.