Ophthotech Corp. (NASDAQ:OPHT) gained $0.29 (10%) to $3.09 on Thursday on news that it acquired exclusive, worldwide rights to develop and commercialize a preclinical ophthalmic gene therapy from the University of Florida Research Foundation and the University of Pennsylvania.
The deal gives the biotech an adeno-associated virus (AAV) gene therapy to treat rhodopsin (RHO; OPN2)-mediated autosomal dominant retinitis pigmentosa. The AAV is designed to knock down mutant RHO expression and deliver a functional replacement gene.
Ophthotech will pay the universities a $500,000 upfront fee for the therapy and an annual license maintenance fee in the low double-digit thousands, plus up to $23.5 million for clinical, marketing approval and reimbursement milestones and up to $70 million for commercial sales milestones. The universities will also be eligible for royalties in the low single-digit percentages of net sales.
Ophthotech will work with several researchers at both universities, and conduct preclinical and natural history studies with the University of Pennsylvania. Ophthotech anticipates beginning a Phase I/II trial of the AAV therapy in early 2020.
The deal marks the biotech's second gene therapy collaboration since February when it announced plans to seek out such partnerships with companies and academic institutions to expand its pipeline.
The first collaboration with University of Massachusetts Medical School gave Ophthotech access to "minigene" technology designed to deliver a shortened form of a gene via a standard-sized AAV vector. The partners' research will focus on two degenerative retinal diseases: Leber congenital amaurosis type 10 due to centrosomal protein 290kDa (CEP290) mutations; and autosomal recessive Stargardt disease due to ATP-binding cassette sub-family A member 4 (ABCA4; ABCR) mutations. Ophthotech has an option to exclusive rights to patents or patent applications from the research.
Ophthotech does not have any other gene therapies in its pipeline.
It is developing its sole clinical stage asset, Zimura avacincaptad pegol, for several ophthalmic indications. It expects Phase IIb top-line data in 2H19 and 2020 for the synthetic aptamer that inhibitors complement 5 (C5) to treat dry age-related macular degeneration (AMD) and autosomal recessive Stargardt disease, respectively.