MeiraGTx announced on Apr. 16 that it is reacquiring the rights to its eye disease gene therapy, bota-vec, from Johnson & Johnson for $25 million upfront. The company said it plans to seek regulatory approval for the treatment despite a failed Phase 3 trial last year.
The move comes as MeiraGTx aims to address X-linked retinitis pigmentosa, an inherited eye disease caused by mutations in the RPGR gene that leads to early childhood blindness, particularly in males. The company believes data from the LUMEOS trial are sufficient to support global regulatory applications and hopes this will provide a new treatment option for patients with significant unmet medical needs.
Under the terms of the agreement, Johnson & Johnson will receive a milestone payment if bota-vec is approved in the United States and royalties starting in mid-2029. The companies originally partnered in 2019 through a deal valued at up to $440 million, with J&J leading clinical development since Phase 1.
Although bota-vec did not meet its primary endpoint of improving visual-guided mobility in last year's LUMEOS trial, MeiraGTx maintains that there were improvements in vision and patient-reported outcomes. Rachel Huckfeldt, director of Inherited Retinal Disorders Clinical Trials at Mass Eye and Ear and one of the study’s principal investigators, said "the study showed improvements in visual acuity as seen on a standard letter reading test" and noted patients also reported daily life improvements.
MeiraGTx said experts and patient advocates support seeking regulatory approval based on these findings. "Investigators around the world are enthusiastically supporting filing for regulatory approval of bota-vec in order to allow access to treatment for the patients they are seeing in their clinic today who are waiting for this potentially life-changing therapy," MeiraGTx said.
The company expects to file for approvals with U.S. and European regulators by 2027. CEO Alexandria Forbes said: "We are extremely pleased to have reacquired bota-vec for the treatment of XLRP... This is a unique opportunity... with data supporting a meaningful benefit in patients with no alternative treatment." If approved, MeiraGTx would become a commercial-stage biotech firm.
