RARE Daily

MeiraGTx Enters Asset Purchase Agreement with Janssen Related to its XLRP Gene Therapy for Up to $415 Million

December 21, 2023

Rare Daily Staff

MeiraGTx entered an asset purchase agreement with Janssen Pharmaceuticals, a Johnson & Johnson company, for the remaining interests in the gene therapy bota-vec for the treatment of XLRP, as well as a commercial supply agreement and a and a technology transfer agreement for bota-vec manufacturing.

Under the terms of their asset purchase agreement, MeiraGTx will receive $130 million in and near-term milestone payments, including $65 million at signing, an additional $50 million anticipated in the first quarter of 2024, with the remaining $15 million in milestone payments expected later in 2024. The Company will receive up to an additional $285 million in cash payments upon first commercial sales of bota-vec in the U.S. and EU and for manufacturing technology transfer. J&J will be responsible for any royalty or milestone amounts that become payable on bota-vec to UCL Business (University College London). MeiraGTx has also entered into a commercial supply agreement with J&J for bota-vec manufacturing.

“The initial $130 million upfront and near-term milestone payments, combined with the $30 million investment we received from Sanofi in October, increases our cash runway to mid-2026, without including the additional $285 million in potential payments associated with this transaction,” said Alexandria Forbes, president and CEO of MeiraGTx.

X-linked retinitis pigmentosa (XLRP) is a severe form of retinitis pigmentosa, a group of rare, inherited retinal diseases that cause serious vision impairment and blindness. Retinitis pigmentosa affects about 1 in 3,500 people, with XLRP accounting for approximately 15 percent of all retinitis pigmentosa cases.

XLRP is most commonly caused by a defect in the RPGR gene, which encodes a protein called X-linked retinitis pigmentosa GTPase regulator (RPGR) that plays a vital role in the development of the cells that make up the retina, a thin layer of tissue found on the back wall of the eye. Absence of this functional protein results in poorly functioning rod and cone photoreceptors, parts of the eye which are responsible for vision at low and high light levels, as well as seeing color.

Botaretigene sparoparvovec (bota-vec) is designed to treat the most common form of XLRP caused by mutations in the eye-specific form of the RPGR gene called RPGR open reading frame 15 (RPGR ORF15). Both rods and cones photoreceptors require RPGR ORF15 to function.

In 2019 MeiraGTx and Janssen entered a collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases that included Janssen receiving worldwide exclusive rights to commercialize product candidates for XLRP among others.

“This puts us in a strong financial position and allows us to increase our focus on our two late-stage clinical programs in Xerostomia and Parkinson’s disease and our leading end-to-end manufacturing capabilities, with potential strategic activities around each of these wholly-owned assets,” said Forbes.

Photo: Alexandria Forbes, president and CEO of MeiraGTx

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