Illuminate leaves Proqr in darkness

Proqr had hoped that both doses of its gene therapy sepofarsen might prove effective in its pivotal phase 2/3 Illuminate trial in the rare eye disorder CEP290-mediated Leber’s congenital amaurosis 10 (LCA10). Today it admitted that neither was. The washout was total, with no significant difference between either dose, or between the doses and sham treatment, on the primary or any of the secondary endpoints, and the company’s stock plummeted 74%. On a call today Proqr did not rule out subgroup dredges, but it would be wildly optimistic to hope for anything here. The question for the company is what the failure means for the rest of its pipeline, and while it insisted that there was limited readthrough – and that it has cash to last until 2024 – it will have a nervous wait for readouts of its trials. Topline data from the phase 1/2 trial of QR-504a in Fuchs endothelial corneal dystrophy, as well as results of the phase 1 Aurora study of QR-1123 in retinitis pigmentosa, are expected this year. The remaining LCA10 pipeline is led by Editas with EDIT-101, but this has already proven a bit of a disappointment.

Leber’s congenital amaurosis projects in the clinic
Project Company Mechanism of action Details
Phase 3
Sepofarsen Proqr CEP290 gene therapy Failed pivotal Ph2/3 Illuminate trial Feb 2022
Phase 2
EDIT-101 Editas Crispr Cas9 CEP290 gene therapy Interim data from Ph1/2 Brilliance trial disappointed Sep 2021
SAR439483 Atsena/Sanofi Guanylate cyclase receptor inhibitor Ph1/2 ongoing; primary completion Feb 2022
HORA-RPE65 Coave Retinoid isomerohydrolase gene therapy Only active development is in retinitis pigmentosa
CEP290=centrosomal protein 290. Source: Evaluate Pharma

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