The market for age-related macular degeneration (AMD) treatments is expected to balloon over the next few years as the population gets older, and plenty of people think there’s room for some billion dollar drugs there. After all, the disease is one of the main causes of blindness in older adults. That fact helped New Haven-based Optherion score $37 million in series A financing from the likes of Quaker BioVentures, Domain Associates, Johnson & Johnson Development Corporation, Purdue Pharmaceutical Products, Pappas Ventures, Biogen Idec New Ventures, and GE Healthcare Financial Services.
The AMD market is already starting to get crowded with new and emerging treatments for the “wet” form of this disease (in which abnormal blood vessels grow and leak behind the retina), including Genentech’s Lucentis. But Optherion’s first products will aim at the “dry” form. There are no approved drugs yet for dry AMD, which is much more common and usually precedes the wet type. That could mean an even bigger opportunity. And Optherion isn’t ignoring the wet AMD market. “Ultimately, people will probably use cocktails of drugs to treat wet AMD,” says Colin J. Foster, the firm’s president and CEO. “There’s no reason why our drug shouldn’t be used in these as well as for dry AMD.”
The company’s platform rests on research from Yale and the University of Iowa looking at a part of the immune system called the “alternative complement system.” The big news was that certain complement factors (H and B) are very strongly linked to AMD. The genetics and molecular biology here are, for once, relatively straightforward and have been well studied, handing Optherion some prime targets for product development.
Though the company’s first drug is barely more than a glimmer in management’s eyes right now, the plan is to build healthy recombinant complement factor H to replace the bad kind. Optherion is also aiming to develop both diagnostics tests as well as treatments. What’s more, “There’s been an explosion in research showing how the complement system is involved in everything from asthma to heart disease,” says Foster. Along with the AMD projects, Optherion is also working on treatments for a rare but deadly childhood kidney disease.