ProQR Therapeutics N.V. (NASDAQ: PRQR) Announce Seporfarse Study  in LCA10 Treatment Failed to Meet Primary Endpoint 

ProQR Therapeutics N.V. (NASDAQ: PRQR) has revealed that its viral Phase 2/3 Illuminate study of sepofarsen for CEP290-Mediated Leber congenital amaurosis 10 (LCA10) treatment failed to meet the primary endpoint for Best Corrected Visual Acuity after 12 months.

Sepofarsen failed to meet endpoint 

CEO Daniel A. de Boer said, “Given the results observed in earlier studies of sepofarsen, the Illuminate trial results are unexpected and disappointing, especially for people living with LCA10. ProQR was founded with the goal of developing RNA therapies for patients with high unmet medical needs, and we will continue to advance our robust pipeline of therapies for genetic eye disease. We are deeply grateful to all of the participants, their supporters, and investigators who participated in the Illuminate study.”

LCA10 is a blinding retinal condition that is extremely difficult to treat, and currently, there are no treatments. Attending Ophthalmic Genetics and Retinal Degenerations Physician at the Children’s Hospital of Philadelphia and Head of Ophthalmology at Gent University, Belgium, Dr Bart Leroy said that as ProQR works to enhance medicines for inherited retinal illnesses, they will continue to collaborate to understand the data better.

36 patients above eight years enrolled in the study 

At 14 study locations in 9 countries, 36 patients above eight years with genetically confirmed LCA10 attributable to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290 gene were enrolled in the Illuminate study. Subjects were randomly assigned to one of three groups (1:1:1) the target enrollment dose sepofarsen (160 g/80 g loading dose/maintenance doses), a low dose sepofarsen for masking (80 g/40 g loading dose/maintenance doses), or a hoax protocol, with sepofarsen administered through the use of intravitreal injection and the control protocol simulating an injection with no drug or injection.

Foundation Fighting Blindness CEO Benjamin R. Yerxa, “This was not the outcome we had hoped for and we share in the disappointment many are feeling in the community. We will continue to work alongside ProQR to learn more from the ongoing analyses and as they work to advance RNA therapies to potentially help children, adults, and families who are affected by rare genetic eye diseases.”