AbbVie and REGENXBIO have formed a strategic partnership to develop and commercialize a potential gene therapy for age-related macular degeneration (AMD), diabetic retinopathy (DR) and other chronic retinal diseases.
As part of the agreement, REGENXBIO will receive a significant upfront payment of $370 million and up to $1.38 billion in development, regulatory and commercial stages. If the applicant is approved, the companies will equally share profits from net sales in the United States and AbbVie will pay tiered royalties on net sales in the rest of the world.
Shares of REGENXBIO rose more than 20% on the news.
RGX-314 consists of the NAV vector AAV8, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF), part of the pathway by which new leaky blood vessels grow and contribute to the accumulation of fluid in the retina.
The potential therapy has two potential routes of delivery to the eye, the more standard subretinal delivery procedure and delivery into the suprachoroidal space via the SCS microinjector, which can be performed under local anesthesia.
RGX-314 is currently being tested in a pivotal trial for wet AMD using subretinal delivery and two separate Phase II clinical trials using office-based suprachoroidal delivery for patients with wet AMD and of RD.
AbbVie was a “strong and complementary partner” for REGENXBIO, said its president and CEO, Kenneth T Mills. “We plan to leverage AbbVie’s global eye care development and commercial infrastructure with our AAV gene therapy clinical development expertise and deep in-house manufacturing and production knowledge to further develop RGX-314,” he said.
Under the agreement, REGENXBIO will be responsible for the ongoing trials of RGX-314, then the companies
collaborate and share the costs of additional testing. Later, AbbVie will lead the clinical development and commercialization of RGX-314 globally while REGENXBIO will partner in the commercialization of the therapy in the United States. On the manufacturing side, REGENXBIO will manage supplies for clinical development and commercial supply in the United States, and AbbVie will lead commercial supply outside of the United States.
Wet AMD – a major cause of vision loss affecting up to two million people in the United States, Europe and Japan – can be treated with current anti-VEGF therapies, but these require repeated treatment every lifelong and patients may experience decreased vision over time.
DR is the leading cause of vision loss in adults aged 24-75 worldwide, with current treatment options such as “watchful waiting”, anti-VEGF treatment, retinal laser or surgical.