AbbVie bets $370M on pivotal eye disease gene therapy, puts up $1.4B in biobucks 

AbbVie is betting $370 million on gene therapy. Having dipped its toe into the sector through other deals, AbbVie is now immersing itself through a deal with Regenxbio for eye disease gene therapy RGX-314.

Regenxbio has worked through a 2019 clinical hold, which led it to sue the FDA, to establish itself as a front-runner in the race to develop a gene therapy for use in wet age-related macular degeneration (AMD) and other eye diseases. The prospect, RGX-314, encodes for an antibody fragment designed to inhibit the VEGF growth factor that is implicated in eye diseases including AMD.  

Anti-VEGF antibodies such as Regeneron’s Eylea and Roche’s Lucentis are already used to treat wet AMD. AbbVie is betting that there is a market for a one-time treatment that frees patients from the need to receive repeat doses of anti-VEGF antibodies.

Here’s how the deal breaks down. AbbVie is paying Regenxbio $370 million and committing to up to $1.4 billion in milestones. In return, AbbVie will gain an even split of the profits in the U.S. and all the revenues, minus tiered royalties, in the rest of the world. Regenxbio will wrap up ongoing trials of the gene therapy before collaborating with AbbVie on future work, including a second pivotal study.

RELATED: Regenxbio sues FDA over gene therapy clinical hold

Other tasks are split based on geography, with AbbVie taking the lead on the production of RGX-314 for commercial supply outside of the U.S. and clinical development and commercialization globally. Regenxbio will make RGX-314 for clinical development and U.S. commercial supply.

Regenxbio is testing the subretinal delivery of RGX-314 to wet AMD patients in a pivotal trial. Other midphase clinical trials are testing delivery of the gene therapy into another part of the eye, known as the suprachoroidal space. The route of administration could make it easier for physicians to give RGX-314 in their offices, rather than in surgical settings.  

Other companies are targeting the same indications. Adverum Biotechnologies has a VEGF therapy in the clinic, but a dose-limiting toxicity recently prompted it to drop plans to advance the candidate in diabetic macular edema (DME). Work in wet AMD is continuing, but analyst expectations were lowered by the setback in DME.

Shares in Regenxbio jumped 20% in premarket trading, rising to close to $40 a pop.

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