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www.fdanews.com/articles/202790-biogens-retinal-disease-candidate-falters-in-phase-23-study

Biogen’s Retinal Disease Candidate Falters in Phase 2/3 Study

May 18, 2021

Biogen’s experimental gene therapy, cotoretigene toliparvovec, failed to show efficacy in a phase 2/3 study of patients suffering from a rare, inherited retinal disease.

The treatment did not demonstrate a significant improvement for male patients with X-linked retinitis pigmentosa after 12 months. The disease is marked by poor night vision followed by a gradual restriction of the field of vision, typically leading to blindness by age 40. The condition currently has no approved treatments.

Although the study did not meet its primary endpoint, the company plans to continue development of the gene therapy and is “encouraged by positive trends in other prespecified clinically relevant endpoints, such as a measure of visual acuity under low light conditions,” said Katherine Dawson, Biogen’s head of therapeutics development.

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