MeiraGTx Enters Gene Therapy Collaboration with Janssen for IRDs Worth up to $440M

Rare Daily Staff

Janssen Pharmaceutical is entering into a broad strategic collaboration with MeirGTx to develop and commercialize gene therapy programs to treat inherited retinal diseases and will pay a $100 million upfront fee and up to $340 million in milestones.

Inherited retinal diseases are a group of rare eye conditions caused by an inherited gene mutation that are often characterized by progressive retinal degeneration that leads to severe vision impairment, loss, or blindness. MeiraGTx currently has three ongoing clinical programs in inherited retinal disease with a fourth program expected to enter clinical development in the first half of 2019, as well as a pipeline of preclinical inherited retinal disease programs for a variety of gene mutations.

Janssen will receive worldwide exclusive rights to commercialize product candidates for the inherited genetic visual disorders achromatopsia caused by mutations in either CNGB3 or CNGA3, X-linked retinitis pigmentosa, and options to additional inherited retinal disease programs. Janssen has agreed to fund all clinical development and commercialization costs for these programs.

In addition, MeiraGTx and Janssen are entering into a research collaboration covering MeiraGTx’s pipeline of pre-clinical programs for inherited retinal disease. The two companies are also entering into a research collaboration to further develop viral vector manufacturing technology, as well as 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, president and CEO of MeiraGTx.

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 application by the United States Food and Drug Administration to begin human clinical trials. 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.

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

February 4, 2019
Photo: Alexandria Forbes, president and CEO of MeiraGTx