– MeiraGTx enters into an Asset Purchase Agreement related to botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) for a total of up to $415 million

– MeiraGTx to receive $130 million in upfront and near-term milestone payments as part of the agreement

– MeiraGTx to receive up to an additional $285 million upon first commercial sales of bota-vec in U.S. and EU and manufacturing technology transfer

LONDON, UK and NEW YORK, NY, USA I December 21, 2023 I MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced an asset purchase agreement with Janssen Pharmaceuticals, Inc. (J&J), a Johnson & Johnson company, for the remaining interests in bota-vec for the treatment of XLRP, as well as a commercial supply agreement and a technology transfer agreement for bota-vec manufacturing.

“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 as well as cash upon potential approval and commercialization of this important gene therapy, and additional revenue from the commercial manufacture of bota-vec,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “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.”

Dr. Forbes continued, “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. In addition, we continue to expedite the development of our Riboswitch gene regulation platform prioritizing targets in metabolic disease, immunology and oncology, which we believe have the potential to significantly alter outcomes in these broad disease areas and include several areas of interest from Sanofi following their investment earlier this quarter.”

Agreements related to bota-vec:

  • MeiraGTx to monetize potential future royalties and milestones related to bota-vec with J&J acquiring remaining rights for development, manufacturing, and commercialization of bota-vec.
  • MeiraGTx to receive $130 million in upfront 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 plc (University College London).
  • MeiraGTx has also entered into a commercial supply agreement with J&J for bota-vec manufacturing.

Evercore Group L.L.C. is serving as financial advisor and Morgan Lewis & Bockius LLP is serving as legal advisor to MeiraGTx.

For more information related to our clinical trials, please visit www.clinicaltrials.gov

About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, and a transformative gene regulation platform technology that allows precise, dose-responsive control of gene expression by oral small molecules with dynamic range that can exceed 5000-fold. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring, and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: ocular diseases, including both inherited retinal diseases as well as large degenerative ocular diseases, neurodegenerative diseases, and severe forms of xerostomia. Though initially focusing on the eye, central nervous system, and salivary gland, MeiraGTx plans to expand its focus to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

For more information, please visit www.meiragtx.com