AbbVie and REGENXBIO Announce $1.75 Billion Collaboration to Advance Ophthalmic Gene Therapy RGX-314

On September 13, 2021, AbbVie and REGENXBIO announced a strategic collaboration agreement to co-develop and commercialize RGX-314. RGX-314 is a potential one-time gene therapy for the treatment of wet age-related macular degeneration (AMD), diabetic retinopathy (DR), and other chronic retinal diseases.

Wet age-related macular degeneration (AMD) is characterized by vision loss due to the formation of new, leaky blood vessels in the retina. RGX-314 delivers a transgene to cells via an AAV8 vector. The transgene encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). Dysregulation of the VEGF pathway promotes the growth of new leaky blood vessels and leads to fluid accumulation in the retina. Intravitreal anti-VEGF injections currently represent the standard of care for wet AMD. However, these therapies require lifelong, repeated intraocular injections to maintain efficacy. Due to the treatment burden, patients typically experience progressive vision loss and reduced treatment frequency over time.

Diabetic retinopathy (DR) is the leading cause of vision loss in adults aged 24 to 75 years worldwide. As DR progresses, a significant proportion of patients develop vision-threatening complications, including diabetic macular edema (DME) and neovascularization, which can lead to blindness. Current treatment options for patients with DR include anti-VEGF therapy, retinal laser therapy, or surgery.

▲RGX-314 Overview (Image source: REGENXBIO Inc. official website)

REGENXBIO is advancing the development of RGX-314 through two distinct ocular administration routes. The therapy is currently being evaluated in a pivotal trial utilizing subretinal delivery for patients with wet age-related macular degeneration (AMD), as well as in two Phase 2 clinical trials utilizing suprachoroidal delivery for patients with wet AMD and diabetic retinopathy (DR). Under the terms of the collaboration agreement, REGENXBIO will be responsible for completing the ongoing RGX-314 trials, and both companies will collaborate to share the costs of additional RGX-314 trials, including the planned second pivotal trial evaluating subretinal delivery for wet AMD and future studies. AbbVie will lead the global clinical development and commercialization of RGX-314.

In addition, AbbVie will pay REGENXBIO an upfront payment of $370 million, and REGENXBIO is eligible to receive up to $1.38 billion in additional development, regulatory, and commercial milestone payments.

References:

[1] AbbVie and REGENXBIO Announce Eye Care Collaboration. Retrieved September 13, 2021, from https://www.prnewswire.com/news-releases/abbvie-and-regenxbio-announce-eye-care-collaboration-301374836.html

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