Johnson & Johnson’s Janssen Pharmaceutical will partner with MeiraGTX Holdings to develop, manufacture, and commercialize its clinical-stage inherited retinal disease portfolio, the companies said today, through a global collaboration and license agreement that could generate more than $440 million for MeiraGTx.

The collaboration—the second between the companies launched in the past three months—includes MeiraGTx’s leading product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa (XLRP).

MeiraGTx has agreed to give Janssen the exclusive option to license new treatments for other inherited retinal diseases from a pipeline of products addressing novel gene targets that the companies plan to develop through a research collaboration with Janssen that includes the companies exploring new targets for other inherited retinal diseases.

“Janssen is excited to expand our portfolio with the addition of innovative assets that have the potential to improve sight or prevent the progression to blindness in inherited retinal diseases which currently have no treatment options,” James List, MD, PhD, global therapeutic area head, cardiovascular & metabolism, Janssen Research & Development, said in a statement.

Added Mathai Mammen, MD, PhD, global head, Janssen Research & Development, “Through this collaboration we look forward to deepening our expertise in gene therapy and leveraging our breadth of research and development expertise to help bring forward new treatment options for people living with inherited retinal diseases.”

Janssen agreed to pay MeiraGTx $100 million in upfront cash, and up to $340 million in payments tied to achieving development and sales milestones related to its CNGB3, CNGA3, and XLRP programs, plus untiered royalties of 20% of annual net sales for the CNGB3, CNGA3, and XLRP programs.

Janssen has agreed to fund all clinical development and commercialization costs for these programs, MeiraGTx said—and a “significant” portion of the costs of the research collaboration.

Both of the company’s two achromatopsia candidates are in Phase I/II trials—AAV-CNGB3 (NCT03001310) and AAV-CNGA3 (NCT03758404)—as is the company’s XLRP candidate AAV-RPGR (NCT03252847).

Research, manufacturing partnerships

Under the research partnership, Janssen retains the right to opt-in to programs upon clearance of an IND application by the FDA. Janssen agreed to fund all clinical development and commercialization costs following opt-in, as well as pay MeiraGTx an opt-in payment, development milestones, and untiered royalty in the high teens on annual net sales of commercialized products coming out of this collaboration.

Janssen and MeiraGTx are also partnering on research to further developing adeno-associated virus (AAV) manufacturing technology, with both companies agreeing to split their costs. Plans also call for the companies to sign clinical and commercial manufacturing supply agreements for the clinical and research programs.

“By combining Janssen’s extensive clinical, regulatory, and commercial expertise and global reach with MeiraGTx’s deep experience in gene therapy development and manufacturing, we aim to accelerate the development of our pipeline of potential IRD gene therapies to address the needs of patients globally,” said Alexandria Forbes, PhD, president and chief executive officer of MeiraGTx.

In October, MeiraGTx agreed to use its proprietary riboswitch technology to engineer regulatable gene therapy constructs encoding proprietary gene sequences from Janssen, in the first collaboration between the companies.

That first collaboration, whose value was not disclosed, centered around MeiraGTx’s gene regulation platform, which incorporates on/off switch for gene expression into the gene therapy vector which can then be activated using a small molecule. Through the platform, gene therapies can be switched on and off according to patients’ need and the dosing requirements of their therapy. According to MeiraGTx, temporal control overlaying spatial regulation of gene expression has the potential to increase the utility and flexibility of gene therapy.

MeiraGTx was formed and began operations in 2015, through acquisitions that included the gene therapy portfolio of Kadmon, the biopharma founded by Waksal’s brother, Sam Waksal, PhD, following guilty pleas that led to a prison sentence in an insider trading case that involved his former company, ImClone Systems, and drew in Martha Stewart.

“Any event in life changes one. That was an event that probably changed me in many ways. I think I am a better person,” he told CNBC in 2015.

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