J&J gene therapy treatment fails primary endpoints

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

Botaretigene sparoparvovec (bota-vec) is 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. Johnson & Johnson (J&J) purchased the full rights to bota-vec in a 2023 deal with MeiraGTx.²

The LUMEOS phase 3 clinical trial enrolled 95 patients, in which 58 of those patients received either a low or high single dose of bota-vec. XLRP is a severe and rare form of retinitis pigmentosa, a progressive eye disease. Most of the enrolled patients were male, as XLRP most often affects the male population, manifesting in childhood.

The primary endpoint of improvement in the ability of patients 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 endpoints, as we evaluate strategic options and next steps,” said a J&J spokesperson.¹

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

Multiple secondary endpoint improvements were tied to bota-vec, though the results have a p-value lower than 0.05.

Twenty-two out of 55 patients treated with bota-vec showed improvement on 2 or more endpoints, contrary 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