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Janssen Acquires Remaining Rights to Eye Disease Gene Therapy From MeiraGTx

NEW YORK – Johnson & Johnson subsidiary Janssen Pharmaceuticals is purchasing the remaining interests in botaretigene sparoparvovec (bota-vec), an investigational gene therapy for a form of retinitis pigmentosa, from MeiraGTx in a deal worth up to $415 million, the companies said Thursday.

Janssen and MeiraGTx inked a collaboration and license agreement in 2019 to develop, manufacture, and commercialize gene therapies for inherited retinal diseases, and bota-vec is one of the treatments they've been developing as a treatment for patients with X-linked retinitis pigmentosa (XLRP) caused by certain mutations in the RPGR gene. The gene therapy is designed to deliver a functional copy of RPGR and aims to counteract loss of retinal cells and preserve, and potentially restore, patients' vision. 

Last year, MeiraGTx received a $30 million milestone payment from Janssen when it began dosing XLRP patients with bota-vec in the Phase III Lumeos trial.

Janssen has now decided to pay MeiraGTx $130 million upfront to retain the remaining rights to develop, manufacture, and commercialize bota-vec, including $65 million at signing, another $50 million in Q1 2024, and $15 million in milestone payments later in 2024. As part of the latest deal, MeiraGTx also entered into a commercial supply agreement and a technology transfer agreement for bota-vec manufacturing. As such, MeiraGTx is also eligible for an additional $285 million after rights to manufacturing technology are transferred to Janssen and upon the first commercial sale of bota-vec in the US and EU.

"We are very happy to announce the execution of the agreements related to bota-vec for the treatment of XLRP, which provide us with significant near-term cash," MeiraGTx President and CEO Alexandria Forbes said in a statement.

With the $130 million upfront and near-term payments from Janssen, plus a $30 million investment it received from Sanofi in October, MeiraGTx said the company's cash runway has extended into mid-2026. New York and London-headquartered MeiraGTx plans to use the funds to develop its late-stage clinical programs in xerostomia and Parkinson's disease as well as its gene regulation platform, which the firm is using to prioritize development of targets in metabolic disease, immunology, and oncology.