MeiraGTx Enters Strategic Collaboration With Janssen

MeiraGTx Holdings plc recently announced it has entered into a broad strategic collaboration with Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop and commercialize gene therapies for the treatment of inherited retinal diseases (IRDs).

Under the agreement, the two companies will collaborate in the clinical development of MeiraGTx’s leading IRD pipeline, including product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3 and X-linked retinitis pigmentosa (XLRP). In addition, MeiraGTx and Janssen are entering into a research collaboration covering MeiraGTx’s pipeline of pre-clinical programs for IRDs. The two companies are also entering into a research collaboration to further develop AAV manufacturing technology as well as clinical and commercial manufacturing supply agreements for the clinical and research programs.

“We are very excited to collaborate with Janssen, a leader in the world of innovation and the creation of new medicines. 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. “This important collaboration supports our leading position in gene therapy development and manufacturing and reaffirms our commitment to advancing a broad portfolio of breakthrough gene therapies that may improve the lives of patients suffering from IRDs worldwide.”

Under the terms of the agreement, the two companies will collaborate in the clinical development of MeiraGTx’s leading IRD pipeline, including product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3 and X-linked retinitis pigmentosa (XLRP). MeiraGTx will receive $100 million in cash as an upfront payment and is eligible to receive additional payments for achieving development and sales milestones related to its CNGB3, CNGA3 and XLRP programs of up to $340 million, and untiered royalties of 20 percent 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 and Janssen are also entering into a research collaboration to develop a pipeline of IRD gene therapy candidates; a significant portion of the costs of the research collaboration will be paid by Janssen. Janssen has the right to opt-in to programs coming out of this research collaboration at the time of clearance of an Investigational New Drug (IND) application by the United States Food and Drug Administration (FDA). Janssen will fund all clinical development and commercialization costs following opt-in. MeiraGTx will receive an opt-in payment, development milestones and a high teens untiered royalty on annual net sales of commercialized products coming out of this collaboration.

The two companies are also entering into a research collaboration to further develop AAV manufacturing technology and will share the costs of this research collaboration.

This is MeiraGTx’s second collaboration with Janssen, having entered into a research collaboration in October 2018 to develop regulatable gene therapy treatments using MeiraGTx’s proprietary riboswitch technology.

IRDs are a group of rare eye conditions caused by an inherited gene mutation that are often characterized by progressive retinal degeneration which leads to severe vision impairment, loss or blindness. MeiraGTx currently has three ongoing clinical programs in IRDs, with a fourth program expected to enter clinical development in 1H2019, as well as a deep pipeline of pre-clinical IRD programs for a variety of gene mutations.

The transaction is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 in the United States, and is expected to close in the first calendar quarter of 2019.

MeiraGTx is a vertically integrated, clinical stage gene therapy company with five 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, as well as a potentially transformative gene regulation technology. 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: inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Though initially focusing on the eye, salivary gland and central nervous system, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases. For more information, please visit www.meiragtx.com.