AbbVie Expands Ophthalmology Pipeline with Regenxbio Gene Therapy Deal

AbbVie has significantly bolstered its ophthalmology pipeline through a strategic licensing agreement with Regenxbio. The deal, which involves an upfront payment of $370 million, grants AbbVie access to RGX314, a promising gene therapy for wet age-related macular degeneration (AMD) and other eye diseases.

RGX314 represents a novel approach by delivering a gene that codes for an antibody against VEGF, a key player in the progression of wet AMD. This method targets the same pathway as current leading treatments like Bayer's Eylea and Novartis/Roche's Lucentis but aims to do so with a single treatment rather than ongoing injections.

Currently, RGX-314 is undergoing a pivotal phase 2/3 trial to evaluate its safety and efficacy as a one-off therapy administered under the retina, compared to the standard regimen of Lucentis injections. Additionally, Regenxbio is conducting two phase 2 trials exploring the administration of RGX-314 into the suprachoroidal space, a less invasive procedure that can be performed in a doctor's office.

AbbVie's commitment extends beyond the initial payment, with up to $1.4 billion in potential milestones tied to the project's success. Both companies will share the costs of further studies, underscoring their shared belief in the therapy's potential.

The ultimate goal of RGX-314 is to provide a durable treatment option that could eliminate the need for frequent eye injections, offering a significant quality of life improvement for patients with wet AMD and diabetic retinopathy. If successful, RGX-314 could follow in the footsteps of Luxturna, the first FDA-approved gene therapy for an inherited retinal disease, albeit targeting conditions with a broader patient base.

Despite the challenges faced by Luxturna in meeting sales expectations, AbbVie and Regenxbio are optimistic about RGX-314's prospects, given its application to more prevalent diseases. The therapy utilizes an adeno-associated virus (AAV) as a delivery vector, a method that has recently come under scrutiny due to safety concerns in other gene therapy trials. However, recent FDA advisory discussions have not resulted in additional restrictions, maintaining a positive outlook for gene therapy development.

Under the terms of the agreement, Regenxbio will share in the profits from RGX-314's net sales in the U.S. and receive royalties from sales elsewhere, retaining certain commercial rights in the U.S. The deal is anticipated to be finalized by the end of 2021, marking a significant step forward in the treatment of eye diseases through gene therapy.