ARVO 2025 News: J&J’s XLRP Gene Therapy Didn’t Meet Primary Endpoint in Phase 3 Clinical Trial

Johnson & Johnson reported that botaretigene sparoparvovec (bota-vec), its gene therapy for people with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR, did not meet the primary endpoint in the Phase 3 LUMEOS clinical trial. The primary endpoint in LUMEOS was the effect of bilateral treatment on patients’ performance in a vision-guided mobility assessment (VMA), i.e., performance in navigation of a maze under reduced lighting.

Though bota-vec did not meet its primary endpoint in LUMEOS, some patients did show a response to the treatment with improvement in the VMA. A responder was someone who navigated the VMA under reduced lighting that was two lux levels lower than VMA lighting prior to treatment. Some patients also had improvements in low-luminance visual acuity (LLVA) which is their ability to read an eye chart in reduced light, and static perimetry which measures retinal sensitivity at different locations (loci) in the retina.

All patients treated in LUMEOS had at least on treatment emergent events, most of which were mild or moderate.

In the LUMEOS trial, 95 patients were randomized to receive a bilateral high or low dose of the treatment or assigned to a control group which is eligible for deferred treatment. The company reported results at 52 weeks after treatment for 55 treated patients and 30 controls.

Johnson & Johnson has not announced its next steps for bota-vec.

Results from LUMEOS were presented by Michael Clark, MD, Johnson & Johnson, at the 10^th Retinal Therapy Innovation Summit hosted by the Foundation and Oregon Health & Science University on May 2, 2025, in Salt Lake City, Utah. The Summit was held before the 2025 Association for Research in Vision and Ophthalmology (ARVO) meeting.

XLRP is an inherited retinal disease that usually affects males. Women are usually unaffected carriers but can also be affected.

A small number of women were enrolled in LUMEOS.