J&J’s Janssen, MeiraGTx in $440-Million Gene-Therapy Product and Mfg Pact

By Lillian Torres -

February 8, 2019

Janssen Pharmaceuticals, one of the Janssen Pharmaceutical Companies of Johnson & Johnson, has formed a worldwide collaboration and license agreement, worth up to $440 million, with MeiraGTx, a New York- and London-based clinical stage gene-therapy company, to develop, manufacture, and commercialize MeiraGTx clinical-stage inherited retinal disease portfolio.

The deal includes MeiraGTx’s lead product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa (XLRP). The companies have also formed a research collaboration to explore new targets for other inherited retinal diseases and further develop adeno-associated virus (AAV) manufacturing technology.

Under the agreement, MeiraGTx will grant Janssen an exclusive worldwide license to certain clinical assets in MeiraGTx's inherited retinal disease portfolio. The companies have also formed a research collaboration to develop a pipeline of products addressing novel gene targets, giving Janssen the exclusive option to license new treatments for other inherited retinal diseases.

MeiraGTx currently has three programs in clinical development for ocular indications for inherited retinal diseases. AAV-CNGB3 and AAV-CNGA3, which are gene-therapy candidates designed to restore cone function, are delivered via subretinal injection to the area of the eye where most of the cones in the retina are located. AAV-CNGB3 was granted orphan drug designation (ODD), rare pediatric disease, and fast-track designations by the US Food and Drug Administration (FDA), and orphan medicinal product and PRIME designations by the European Medicines Agency (EMA) for the treatment of achromatopsia caused by mutations in the CNGB3 gene. A Phase I/II clinical trial of AAV-CNGB3 in both adult and pediatric patients is underway.

AAV-RGPR is designed to treat the most common form of XLRP caused by mutations in the RPGR gene. Both rod and cone photoreceptors require RPGR to function. AAV-RPGR has received fast-track designation and ODD from the FDA and orphan medicinal product designation from the EMA. A Phase I/II clinical trial of AAV-RPGR in adult and pediatric patients is underway.

Under the agreement, 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% 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 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.

The new deal 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 US, and is expected to close in the first calendar quarter of 2019.

Source: Janssen Pharmaceuticals and MeiraGTx