U.K. biotech Exonate won a £4.9 million (approximately $6.1 million) Seeding Drug Discovery Award from the Wellcome Trust to support preclinical development of an eye drop-formulated vascular endothelial growth factor (VEGF)-targeting therapy for wet age-related macular degeneration (wet AMD). The award comes less than 2 months after the Cambridge-headquartered firm closed its third, £1.5 million ($1.87 million) funding round with new and existing investors.

Exonate was spun out of the University of Nottingham, U.K., in 2013. The firm is developing small-molecule inhibitors of the serine/threonine-protein kinase SRPK1, a regulator of messenger RNA (mRNA) splicing that promotes the production of pathogenic forms of VEGF in angiogenesis-related diseases. Working out of laboratories at the University of Nottingham, Exonate is developing a pipeline of SPHINX molecule SPRK1 inhibitors in collaboration with a team at the University of New South Wales.

Exonate says studies in wet AMD and cancer have shown that SPRK1 inhibition reduces pathologic VEGF formation, angiogenesis, and disease-progression, but without inhibiting production of protective forms of VEGF. The firm aims to develop cost-effective, topical formulations that specifically block pathologic forms of VEGF in wet AMD, and so cause fewer off-target effects, side effects, and toxicity than current injected forms of anti-VEGF therapy. The aim is to expand the technology to other therapeutic areas, potentially including cancer.

“This award represents a strong endorsement of the approach taken by the company to discover and develop novel small molecules with a more targeted mode of action,” stated Catherine Beech, M.B., Ch.B., Exonate CEO. “The funding will enable us to accelerate our current program to develop safer, more cost-effective drugs that can be easily administered as eye drops, improving adherence and benefiting patients. Exonate's early data is very promising and we have a clear aspiration to successfully deliver medicines in areas of unmet need.”

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