Johnson & Johnson Acquires Phase 3 Ophthalmic Gene Therapy for X-Linked Retinitis Pigmentosa in Deal Worth Over $415 Million

▎Edited by the WuXi AppTec content team

MeiraGTx announced today that it has reached an agreement with Johnson & Johnson,Johnson & Johnson to Acquire Development Rights for Gene Therapy Botaretigene Sparoparvovec (Bota-Vec, Formerly Known as AAV-RPGR) for the Treatment of X-Linked Retinitis Pigmentosa (XLRP)MeiraGTx can receive up to $415 million in upfront and milestone payments.

Bota-vec is a gene therapy based on adeno-associated virus vector (AAV), designed to treat eye-specific

Open Reading Frame 15 (ORF15) of the RPGR Gene

RPGR ORF15) variants cause XLRP.

The protein encoded by RPGR is essential for the function of rod and cone photoreceptor cells. The Phase 1/2 clinical trial of Bota-vec has been completed in adult and pediatric patients, and the Phase 3 Lumeos clinical trial completed patient enrollment in 2023. Existing trial results show that bota-vec treatment has acceptable safety, with efficacy assessments in proof-of-concept studies indicating improvements in patients' retinal sensitivity, visual acuity, and functional vision.

Bota-Vec has been granted Fast Track designation and Orphan Drug designation by the FDA, as well as PRIME designation and Orphan Drug designation from the European Medicines Agency.

Under this agreement, MeiraGTx will receive an upfront payment and near-term milestone payments totaling $130 million. Additionally, MeiraGTx is also eligible to receive up to $285 million in milestone payments following the first commercial sales of bota-vec in the United States and the European Union, as well as the successful transfer of manufacturing technology.

[1] MeiraGTx Announces Asset Purchase Agreement Involving its AAV-RPGR Collaboration for up to $415 million. Retrieved December 21, 2023, from https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-announces-asset-purchase-agreement-involving-its-aav

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