Johnson & Johnson bought the full rights to a rare disease gene therapy a few years ago, but now the Big Pharma may be experiencing buyer’s remorse. The treatment failed to improve the vision-guided mobility of patients with the eye disease X-linked retinitis pigmentosa (XLRP) in a phase 3 trial, according to results shared May 2.
The LUMEOS trial enrolled 95 patients, 58 of whom received either a low or high single dose of botaretigene sparoparvovec (bota-vec), an investigational gene therapy that uses an adeno-associated virus to shuttle a functional copy of the retinitis pigmentosa GTPase regulator (RPGR) gene to the retina.
XLRP is a severe and rare form of retinitis pigmentosa, a progressive eye disease that causes photoreceptors to break down over time, eventually leading to blindness. The disease often manifests in childhood and mainly affects boys, with the LUMEOS trial only enrolling a handful of women with the disease.
In a pooled analysis of all who received bota-vec, the trial’s primary endpoint of improvement in the ability of patients to visually navigate through a virtual maze was not met, though J&J said the result was “directionally supportive."
"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," a J&J spokesperson told Fierce Biotech.
All patients given bota-vec experienced at least one treatment-emergent adverse event, with 86% of the events considered mild or moderate in severity, according to the announcement. Fifty-three percent of patients had at least one adverse event associated with bota-vec.
Bota-vec was tied to improvements in several of the trial’s secondary endpoints, including patient-reported vision and scores on a letter chart visual acuity test. While the results had a p-value smaller than .05, the values are for descriptive purposes only and were not touted as statistically significant by J&J.
All in all, 22 out of 55 treated patients showed improvement on two or more endpoints while no patient in the control group did, according to J&J. As of April 25, the company was still running a phase 3 follow-up study for patients in the initial late-stage trial.
Bota-vec was originally developed by J&J’s Janssen unit in collaboration with genetic medicines biotech MeiraGTx. In late December 2023, J&J bought the full rights to the program in a deal potentially worth $415 million.
At the time of publication, MeiraGTx had not responded to a request for comment from Fierce Biotech.