J&J's XLRP Gene Therapy Bota-Vec Falls Short of Primary End Points

This article originally appeared on our sister site, Ophthalmology Times®.

Data from the LUMEOS phase 3 clinical trial demonstrated a rare disease gene therapy referred to as botaretigene sparoparvovec (bota-vec) has failed to improve the vision-guided mobility of patients with X-linked retinitis pigmentosa (XLRP).¹

Johnson & Johnson (J&J) purchased the full rights to bota-vec, an investigational gene therapy utilizing an adeno-associated virus to move a functional copy of the retinitis pigmentosa GTPase regulator (RPGR) gene to the retina, in a 2023 deal with MeiraGTx.^1,2

A total of 95 patients were enrolled in LUMEOS and 58 of these patients received bota-vec in a single low or high dose. A progressive eye disease, XLRP constitutes a severe and rare form of retinitis pigmentosa. Because XLRP most commonly appears in men, with manifestation occurring in childhood, the majority of the patients enrolled were male.

LUMEOS' primary end point, improvement in the ability to visually navigate a virtual maze, was not met.

“We’re working to understand the totality of the data, inclusive of the clinical relevance of improvement shown on the majority of secondary end points, as we evaluate strategic options and next steps,” a J&J spokesperson said.¹

At least one treatment-emergent adverse event was experienced by all enrolled patients, with 86% of these events considered mild or moderate in severity.¹ Furthermore, 53% of patients experienced at least 1 adverse event associated with bota-vec.¹

Multiple secondary end point improvements were tied to bota-vec, though the results have a p-value lower than .05.

Twenty-two out of 55 patients treated with bota-vec showed improvement on 2 or more end points, as opposed to 0 patients in the control group. J&J was still running a phase 3 follow-up study as of April 25, for patients in the initial late-stage.

REFERENCE
1. Incorvaia D. J&J gene therapy fails to improve visual navigation in late-stage rare eye disease trial. Fierce Biotech. Published May 5, 2025. Accessed May 5, 2025. https://www.fiercebiotech.com/biotech/jj-gene-therapy-fails-improve-visual-navigation-late-stage-rare-eye-disease-trial
2. Armstrong A. MeiraGTx gifts remaining gene therapy rights to J&J for up to $415M. Fierce Biotech. Published December 21, 2023. Accessed May 5, 2025. https://www.fiercebiotech.com/biotech/meiragtx-gifts-remaining-gene-therapy-rights-jj-415m