J&J will hand over the rights to bota-vec for $25 million upfront, clearing MeiraGTx to seek regulatory approvals in the U.S. and EU in 2027.
MeiraGTx is buying back the rights to an eye disease gene therapy from Johnson & Johnson, pledging to package the existing data and make a run to regulators despite a failed Phase 3 trial last year.
The genetic medicines biotech insists there was plenty of good to be found in the Phase 3 LUMEOS trial of bota-vec that could support global regulatory applications, according to a Thursday release. The gene therapy has been under development for X-linked retinitis pigmentosa, an inherited eye disease caused by mutations in the RPGR gene that causes blindness in early childhood, particularly in males.
J&J will hand over the rights to bota-vec for $25 million upfront, with a one-time milestone payment due upon U.S. approval and certain sales performance. The Big Pharma will also be eligible for royalties starting in mid-2029.
The partners began working together in 2019, with J&J offering $100 million upfront and up to $340 million in various milestones and other payments. The entire deal was worth up to $440 million.
MeiraGTx has been collaborating on the clinical development of bota-vec since Phase 1, with J&J taking the lead.
But the gene therapy took a stumble last year, when it failed to improve visual-guided mobility of patients in the LUMEOS trial, according to a report from Fierce Biotech at the time.
Nevertheless, MeiraGTX believes the trial provided approvable data. The biotech called on experts and patient advocates to support its case that LUMEOS showed improvements in vision and other patient reported outcomes.
Rachel Huckfeldt, director of Inherited Retinal Disorders Clinical Trials at Mass Eye and Ear and one of the trial’s principal investigators, said in a statement on Thursday that the study showed improvements in visual acuity as seen on a standard letter reading test. Patients also reported improvements in their daily lives.
Huckfeldt and other experts also highlighted the significant unmet need for patients with X-linked retinitis pigmentosa.
“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 in the release.
The company plans to move quickly now that bota-vec is back in the fold, with regulatory filings in the U.S. and EU to come in 2027.
“We are extremely pleased to have reacquired bota-vec for the treatment of XLRP,” said MeiraGTx CEO Alexandria Forbes. “This is a unique opportunity to gain an asset at this stage in development with data supporting a meaningful benefit in patients with no alternative treatment, many of whom are waiting for this life changing therapy and hoping for expeditious approval.”
If bota-vec is approved, the milestone would mark MeiraGTx’s transition to a commercial-stage biotech. The company is also developing AAV-hAQP1 in radiation-induced xerostomia, with a pivotal readout expected in the second quarter of 2027. That would mean two potential product launches for the small biotech to come over the next two years.
