J&J's Eye Disease Gene Therapy Bota-Vec Fails Phase 3 Trial

Johnson & Johnson's gene therapy, bota-vec, designed to treat X-linked retinitis pigmentosa (XLRP), a genetic condition leading to vision impairment, has not succeeded in achieving its primary target in a critical Phase 3 trial known as the LUMEOS study. This update was recently made on the Johnson & Johnson healthcare provider website. Although the one-year results of the LUMEOS study did not meet the primary endpoint, the outcomes were described as "directionally supportive."

The primary goal of the LUMEOS study was to evaluate the improvement in patients' ability to navigate a vision-guided mobility assessment maze. Despite the primary endpoint not being met, Johnson & Johnson is analyzing the comprehensive data, including significant improvements in several secondary endpoints, to consider strategic options going forward, according to a company spokesperson.

The setback of this Phase 3 study comes amid a trend where major biopharmaceutical companies are retreating from research and development involving adeno-associated virus (AAV) gene therapies. Recently, Vertex joined this shift, halting its research in this area. Previously, other major companies such as Pfizer, Takeda, Roche, and Biogen had scaled back their involvement in AAV gene therapy research or clinical initiatives.

Johnson & Johnson had acquired bota-vec from the smaller biotech company MeiraGTx in 2023, paying $65 million upfront. This acquisition followed earlier licensing deals for bota-vec and two other gene therapies targeting eye diseases. The bota-vec therapy works by delivering functional copies of the RPGR gene through viral vectors known as adeno-associated viruses. Mutations in this gene are responsible for X-linked retinitis pigmentosa, which primarily affects males.

The LUMEOS Phase 3 trial included 95 participants divided into three groups: those receiving a low dose of the gene therapy, a high dose, and a deferred treatment group acting as a control, scheduled to receive the therapy following the measurement of the primary endpoint.

In 2021, Biogen experienced a similar challenge when its gene therapy for XLRP did not succeed in a mid-stage trial. Meanwhile, Beacon Therapeutics is conducting research on a gene therapy for this rare eye condition and secured $170 million in funding last year to support its efforts.

Johnson & Johnson, along with other industry players, continues to assess the landscape of gene therapy for XLRP, focusing on the potential clinical relevance of their findings and determining the next steps for their gene therapy initiatives.