- AbbVie is willing to pay billions of dollars for access to an experimental gene therapy from Regenxbio, a Maryland-based drugmaker, that's being tested as a treatment for several prominent eye diseases.
- Through a collaboration announced Monday, AbbVie will initially trade $370 million in exchange for global rights to develop and commercialize the therapy, known as RGX-314. Regenxbio could later take home another $1.38 billion, provided it achieves certain milestones. The companies have also agreed to equally split any sales of the drug in the U.S., though elsewhere, Regenxbio will be eligible for royalties on net sales.
- RGX-314 is a one-time treatment currently being evaluated in a late-stage study of patients with the "wet" form of age-related macular degeneration, or AMD, a common eye disease that causes vision loss. Results from that trial are expected in 2023. Regenxbio is also sponsoring a pair of mid-stage studies testing its therapy against wet AMD and another eye condition called diabetic retinopathy.
While estimates vary, AMD is thought to affect a large number of people. A study published in 2014, for instance, projected the patient population globally would hit 196 million in 2020 and grow to 288 million by 2040.
Among those patients, the vast majority will have "dry" AMD. Only about one- or two-in-10 will have the more severe, "wet" form of the disease, which is characterized by the formation of unusual blood vessels that leak fluid and damage the eye. The Food and Drug Administration has already approved several treatments for wet AMD, including Roche's Lucentis, Regeneron's Eylea and Novartis' Beovu, all of which work by blocking a protein called VEGF, which encourages the growth of new blood cells.
While Beovu is newer to market, having received FDA approval in late 2019, Lucentis and Eylea consistently reap billions of dollars in annual sales for their makers. Just last year, Regeneron recorded almost $8 billion in global sales of Eylea — which, notably, is also approved for other eye conditions, including diabetic retinopathy.
Regenxbio, meanwhile, has been working to provide patients with another option in RGX-314. The therapy comes as a one-time injection, and is designed to deliver a gene that creates proteins meant to neutralize VEGF. Regenxbio believes this approach holds advantages over currently available treatments, which require monthly or bi-monthly eye injections that patients may view as "repetitive" and "inconvenient."
The therapy now also has the backing of a much larger pharmaceutical company. Per terms of Monday's deal, AbbVie will lead clinical development and global commercialization of RGX-314. The companies have agreed that Regenxbio will assist with commercialization efforts in the U.S., and will be responsible for completing the ongoing trials of its therapy.
AbbVie and Regenxbio also plan to collaborate and share costs on additional trials of RGX-314, including a second pivotal study in wet AMD that would administer the therapy under the retina. The two ongoing, mid-stage studies testing RGX-314 in wet AMD and diabetic retinopathy are trying out a different method, delivering the therapy to a part of the eye called the suprachoroidal space.
While the deal provides support and cash to Regenxbio, for AbbVie, it offers a stronger foothold in eye drug development. Last year, AbbVie completed its $63 billion purchase of Allergan, acquiring in the process an eye care unit headlined by Restasis, a topical drug for tear production. Sales from that unit totaled $1.74 billion during the first six months of this year.
The deal also furthers AbbVie's reach in gene therapy research, an area that's seen increased interest from big pharmaceutical companies. Early this year, AbbVie inked another deal centered on genetic medicines, as it partnered with Caribou Biosciences to improve a certain kind of cell therapy, with the help of CRISPR gene editing technology.
After announcing the deal with AbbVie, Regenxbio's share price rose 24%, to trade at nearly $41 by late Monday morning.