Janssen Announces $440 Million Collaboration with MeiraGTx to Advance Gene Therapies for Inherited Retinal Diseases

Today, Janssen, a subsidiary of Johnson & Johnson, announced the signing of a global collaboration and license agreement with MeiraGTx. The two companies will jointly develop, manufacture, and commercialize a portfolio of therapies for inherited retinal diseases (IRDs), including candidate products targeting achromatopsia (ACHM) caused by CNGB3 or CNGA3 mutations, as well as X-linked retinitis pigmentosa (XLRP). The companies will also collaborate on research and development to explore new targets for other IRDs and to advance adeno-associated virus (AAV) manufacturing technologies.

Under the terms of this agreement, Janssen will pay MeiraGTx an upfront payment of $100 million to secure exclusive options for preclinical programs targeting inherited retinal diseases (IRDs). Janssen will be responsible for subsequent clinical trial costs, while MeiraGTx is eligible to receive up to $340 million in various milestone payments, as well as royalties on product sales.

A Biotechnology Company Focused on Developing Gene TherapiesPharmaceuticalsMeiraGTx currently focuses its research and development efforts on three primary areas: ophthalmic diseases, salivary gland disorders, and neurodegenerative diseases. MeiraGTx possesses core competencies in the design and optimization of viral vectors, as well as in the manufacturing of gene therapies, and holds potentially transformative gene regulation technologies. The company utilizes adeno-associated virus (AAV) as a gene delivery vector, enabling the optimization of gene therapies for specific diseases through vector customization. Minor variations in the capsid proteins of the vector can modulate the efficiency of gene delivery to different cell types, thereby allowing the selection of specific AAV capsids to most effectively target particular cell types.

Furthermore, MeiraGTx stated that its gene regulation platform is a potentially revolutionary technology that integrates an “on/off” switch for gene expression into gene therapy vectors, which can then be activated by small molecules. In this way, gene therapy can be turned on and off according to patient needs and therapeutic dosing requirements.

Inherited retinal diseases (IRDs) represent an ideal therapeutic area for gene therapy, as most disease-causing genetic mutations have been identified, and the eye is, to some extent, an immune-privileged site. Clinical trials have demonstrated that the delivery of gene therapies using adeno-associated virus (AAV) or lentiviral (LV) vectors in the eye does not result in systemic side effects or elicit significant immune responses. Most investigational therapies aim to introduce functional genes to restore normal expression of proteins encoded by defective genes, such as CNGA/CNGB (for the treatment of achromatopsia) and RPGR (for the treatment of X-linked retinitis pigmentosa [XLRP]).

One Method of Ocular Injection Gene Therapy (Image Source: Reference [2])

ACHM is characterized by significantly reduced visual acuity from birth, extreme photophobia, nystagmus, and achromatopsia. ACHM exhibits genetic heterogeneity, with up to 92% of ACHM patients carrying mutations in the CNGB3 or CNGA3 genes. There are approximately 30,000 ACHM patients nationwide in China. Patients with XLRP may exhibit symptoms during childhood and rapidly progress to blindness between the ages of 20 and 30. To date, there are no effective and approved therapies for the treatment of ACHM and XLRP, and there remains a significant unmet need in these patient populations.

Previously, MeiraGTx’s A002 therapy for achromatopsia (ACHM) caused by CNGB3 gene mutations was granted Rare Pediatric Disease designation and Orphan Drug designation by the U.S. Food and Drug Administration (FDA), as well as PRIME (PRIority MEdicines) designation and Orphan Drug designation by the European Medicines Agency (EMA). Meanwhile, its AAV-RPGR therapy for X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations received Fast Track designation from the FDA.

MeiraGTx’s Product Development Pipeline (Image source: MeiraGTx official website)

Dr. James List, Global Head of Cardiovascular and Metabolic Therapeutics at Janssen Research & Development, stated: “Janssen is pleased to expand our portfolio with innovative therapies that have the potential to improve vision or prevent disease progression leading to blindness in inherited retinal diseases (IRDs) for which there are currently no treatment options. We look forward to collaborating with MeiraGTx to develop these assets and explore potential targets for new IRDs.”

Regarding this new agreement, Dr. Mathai Mammen, Global Head of Janssen Research & Development, stated: “This collaboration continues Janssen’s long-standing tradition of addressing unmet needs of patients worldwide. Through this partnership, we look forward to deepening our expertise in gene therapy and providing new treatment options for patients with inherited retinal diseases (IRDs).”

References:

[1] Janssen Enters Worldwide Collaboration and License Agreement with MeiraGTx to Develop Gene Therapy Programs for Inherited Retinal Diseases. Retrieved Jan. 31, 2019, from https://www.janssen.com/us/sites/www_janssen_com_usa/files/janssen_enters_worldwide_collaboration_and_license_agreement_with_meiragtx_to_develop_gene_therapy_programs_for_inherited_retinal_diseases.pdf

[2] Nature Review: What progress has gene therapy made in the treatment of ophthalmic diseases? Retrieved Jan. 31, 2019, from https://mp.weixin.qq.com/s/lZvSQ7pjUOFscOFoonhfEg

[3] Flash | Gene Therapy Granted PRIME Status, Poised to Become First Treatment for Color Blindness. Retrieved Jan. 31, 2019, from https://mp.weixin.qq.com/s/ca6C1h0x-7W7tIVX1CUFkw

[4] Flash: Gene Therapy Startup Targeting Multiple Diseases Plans $86 Million IPO. Retrieved Jan. 31, 2019, from https://mp.weixin.qq.com/s/L6EQ8aGRchGcfN6M0WmbIA

[5] MeiraGTx Official Website. Retrieved Jan. 31, 2019, from www.meiragtx.com

Source: WuXi AppTec