AbbVie Strikes Gene Therapy Pact with REGENXBIO to Tackle Eye Diseases

On September 13th, AbbVie announced a partnership worth up to $1.8 billion with Maryland-based drugmaker REGENXBIO Inc to develop and commercialize RGX-314, a potential one-time gene therapy for treating wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR), and other chronic retinal diseases.

This is AbbVie’s second gene therapy deal this year after it forked out $90 million for a multi-year, strategic collaboration with Thousand Oaks, CA-based Capsida Biotherapeutics in April.

Under the terms of the deal, AbbVie will pay REGENXBIO an upfront payment of $370 million. In addition, REGENXBIO is also eligible to receive up to $1.38 billion in additional development, regulatory and commercial milestones. Since the news, REGENXBIO’s shares which closed at around $33 when trading closed last Friday, jumped to $40 per share. Its share price currently stands at $45.68.

“We are committed to finding solutions for patients living with difficult-to-treat retinal diseases and to helping preserve and protect our patients from visual impairment and devastating vision loss,” said Tom Hudson, MD, Senior Vice President, R&D, and CSO of AbbVie. “In collaboration with REGENXBIO, we aim to make a remarkable impact for the millions of patients suffering from vision loss associated with retinal diseases.”

One Time Gene Therapy for Eye Diseases

Wet AMD is a chronic disorder that causes visual distortions, reduced central vision, decreased intensity of colors, and a blind spot in the field of vision. It develops when new and abnormal blood vessels grow uncontrolled under the macula, causing swelling, bleeding, and/or fibrosis. VEGF signaling is the preferred target for this disease as it is known to cause endothelial cell proliferation and increase vascular permeability.

Approximately 20 million people worldwide live with the condition, and it is set to become more prevalent as the global population ages. This calls for a need for a personalized treatment that achieves optimal visual outcomes with minimal clinic visits and intravitreal injections.

At present, Roche’s Lucentis, Regeneron’s Eylea, and Novartis’ Beovu are all available for treating wet AMD, but they require multiple dosing and monthly monitoring of patients. If approved, REGENXBIO hopes that its one-time gene therapy could hold an edge over these treatments.

RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway.

“AbbVie is a strong, complementary partner for REGENXBIO. We expect to leverage AbbVie’s global developmental and commercial infrastructure within eye care with our expertise in AAV gene therapy clinical development and deep in-house knowledge of manufacturing and production to continue the development of RGX-314,” said Kenneth T. Mills, President and CEO of REGENXBIO.

As per the deal, REGENXBIO will complete the ongoing trials of RGX-314, but both companies will share costs on additional drug trials, including the planned second pivotal trial evaluating subretinal delivery for the treatment of wet AMD and future trials.

Author

Rajaneesh K. Gopinath