Eye Disease Gene Therapy Misses Endpoint: J&J

Johnson & Johnson says its gene therapy botaretigene sparoparvovec missed the primary endpoint in the Phase 3 LUMEOS study that is evaluating it for treating X-linked retinitis pigmentosa (XLRP). The company describes XLRP as a genetic eye disease, which primarily affects males, caused by a defect in the RPGR gene on the X chromosome that leads to progressive vision loss.

The primary outcome was the change from baseline to Week 52 in vision-guided mobility assessment (VMA) as measured by the ability to navigate through a VMA maze, according to the company. “At Week 52, the primary endpoint (VMA) was not statistically significant but was directionally supportive,” the company says.

Secondary endpoints assessed changes after treatment administration in retinal function, functional vision, visual function. J&J says the study did demonstrate improvement in all three visual domains.