ProQR Therapeutics has announced that the company’s pivotal Illuminate trial of sepofarsen for the treatment of Leber congenital amaurosis 10 (LCA10) did not meet its primary endpoint of Best Corrected Visual Acuity (BCVA) measured at 12 months. The drug was found to be generally well tolerated, although investigators observed signs of retinal thinning and cataracts.
LCA is considered by some retinal experts to be a severe form of retinitis pigmentosa. Sepofarsen, an ASO which uses ProQR’s proprietary Axiomer RNA editing technology, aims to restore vision loss in people with LCA due to a specific mutation in the CEP290 gene. Sepofarsen works by binding to the mutated CEP290 RNA to enable correct splicing. The cells in the retina can then produce the CEP290 protein again.
ProQR’s Phase 2/3 study assessed 36 participants aged eight years or older at 14 study sites in 9 countries. Participants were randomized to three equal groups (1:1:1) of the target registration dose sepofarsen (160 μg/80 μg loading dose/maintenance doses), a low dose of sepofarsen for masking (80 μg/40 μg loading dose/maintenance doses), or sham procedure.
Sepofarsen failed to correct vision for either dosing group and participants had no statistical difference in the sensitivity of their field of vision or mobility compared to the control group.
The results shocked both the company and investors as the drug had achieved highly positive results in both preclinical and Phase 1/2 testing.
ProQR executives said that the results didn’t diminish their expectations for follow-on candidates in their inherited retinal disease pipeline although some analysts viewed the matter differently.
ProQR is based in the Netherlands but is also listed on the NASDAQ (PRQR). The share price fell 70% on the bad news.
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