J&J Gene Therapy Treatment For Vision Loss Fails to Meet Primary Endpoints

Johnson & Johnson acquired the entire rights to a gene treatment for a rare condition some years ago; the pharmaceutical giant may now be going through buyer’s remorse. The therapy did not enhance vision-guided mobility in individuals with X-linked retinitis pigmentosa (XLRP) during a phase 3 study, as reported on May 2.

The LUMEOS trial included 95 participants, 58 of whom were administered either a low or high single injection of botaretigene sparoparvovec (bota-vec), an experimental gene therapy utilizing an adeno-associated virus to deliver a functioning variant of the retinitis pigmentosa GTPase regulator (RPGR) gene to the retina.

XLRP is an unusual and aggressive variant of retinitis pigmentosa, an advanced eye condition that results in the degeneration of photoreceptors, which eventually results in blindness. The illness typically appears in childhood and mostly impacts males, with the LUMEOS study recruiting just a limited number of women affected by the condition.

In the collective evaluation of all participants who received bota-vec, the trial’s main aim of enhancing patients’ capacity to visually go through a virtual maze was not achieved; nonetheless, J&J indicated that the results were directionally favorable.

“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 stated.

All patients administered Bota-vec encountered at least a solitary treatment-emergent negative effect, with 86% of them classified as mild or severe, as per the statement. 53% of individuals had at least one negative outcome related to Bota-vec.

Bota-vec was associated with improvements in many secondary endpoints of the experiment, notably patient-reported eyesight and ratings on a letter chart vision assessment. Although the findings exhibited a p-value less than .05, the values are intended only for descriptive reasons and were not presented as statistically significant by the firm.

According to Johnson & Johnson, out of the 55 individuals who were treated, 22 of them exhibited improvement on multiple endpoints, while one patient in the control group did not demonstrate any improvement. As of the 25th of April, the firm continued to conduct a phase 3 follow-up research for patients participating in the original late-stage experiment.

Initially, the Janssen division of Johnson & Johnson worked along with the genetic medicines biotech company MeiraGTx to create Bota-vec technology. In a deal that could potentially be worth $415 million, Johnson & Johnson purchased the complete rights to the program at the end of December 2023.