After its bet on a rare eye disease gene therapy ended in a phase 3 fail last year, Johnson & Johnson is now returning the asset to sender as MeiraGTx buys back full rights to botaretigene sparoparvovec (bota-vec) for just $25 million upfront.
As part of the deal, J&J could also see a one-time milestone payment connected to bota-vec’s potential approval and commercial performance, according to an April 16 release, as well as future sales royalties. MeiraGTx said it will seek out a speedy filing in the U.S. and E.U., with a launch planned for 2027 should it gain approval.
The odds on any approval however appear long. J&J fully acquired the rights to bota-vec in a 2023 Christmastime deal worth as much as $415 million, after the therapy was co-developed by the pharma’s Janssen unit. The therapy failed to improve vision-guided mobility in patients with X-linked retinitis pigmentosa in a key phase 3 trial back in May last year. That flop has led to J&J shedding the asset.
Outside of the clinic, bota-vec’s road through the FDA may also be rocky, as other gene therapies have struggled to gain traction under outgoing top biologics regulator Vinay Prasad, M.D. With no successor to Prasad yet named, it is unclear how the agency will handle approvals moving forward.
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Bota-vec is designed to restore a functional copy of the retinitis pigmentosa GTPase regulator gene to the retina. Without this gene, patients, who are mostly men, gradually lose sight as they age until often becoming legally blind around 40.
MeiraGTx remains optimistic about bota-vec’s odds in front of regulators despite the phase 3 fail, the company said in the release, due to feedback from physicians who participated in the trial.
“MeiraGTx has heard from numerous investigators about the clinically meaningful benefit that bota-vec has afforded a significant number of patients who participated in the study,” the biotech said, “with unprecedented improvements demonstrated in each of the 3 domains of vision.”
Investors felt less optimistic as shares in MeiraGTx fell 16% by mid-morning trading Thursday.
New York-based MeiraGTx’s ultimate goal is to become a commercial company, according to the release, with aspirations for both bota-vec and AAV-hAQP1, a gene therapy for radiation-induced dry mouth, to hit the market in the next two years.
