The common wisdom in venture capital circles is that investment money flows fastest to the most mature products in the pipeline. Risk is out. Short-term gains--or what passes as short-term in the time-hungry drug discovery world--and clearly defined exit strategies are in.

And then along comes a company like Optherion, with its lead therapy squarely in the preclinical phase, about two years from the clinic. The company is staking its hopes on some intriguing academic work, but the timeline needed to advance it is pushed too far into the future to provide any clearly defined development path. Partnerships? Buyouts? An IPO at some stage? It’s all a possibility.

None of that, though, frightened away an A-list group of investors, which includes Domain Associates, Pappas Ventures, J&J Development Corp. and Biogen Idec’s very busy investment arm. Together, they chipped in $37 million for Optherion’s first round.

Optherion CEO Colin Foster says it’s all about the science, the small team of researchers and executives devoted to the program and the prospects of a highly targeted therapy that marries a diagnostic and therapy in fighting a disease that counts tens of millions of patients worldwide.

“The company is rooted in science from the University of Iowa and Yale,” says Foster, the former North American chief for Bayer’s pharmaceutical ops. Scientists at those institutions laid down the IP groundwork for the company linking variations in Complement Factor H (CFH) and Complement Factor B (CFB)--both regulators of the alternative complement pathway--to AMD, the leading cause of blindness in the elderly. The principal investigator at Iowa, Dr. Gregory Hageman, is the company’s chief scientific officer.

“We seek to provide patients with the correct form of Factor H and mute the over-expression of the alternative complementary pathway,” says Foster. And this is one therapy that will be developed with a companion diagnostic test to identify which patients are likely to benefit from it.
Think personalized medicine, here.

It’s a huge target population, and Foster says Optherion has a scientific rifle that can hit it. In one construct, says Foster, a combination of genetic variations along with key risk factors like obesity and smoking increased a person’s risk of AMD hundreds of times over the norm.
“We want to be sure we’re giving the therapy to a patient who is going to benefit from the link between genetics and disease,” says Foster. “There aren’t that many other examples where you have so few genetic variations associated with such a pervasive disease. And by bringing diagnostics and therapeutics together, I think that’s a huge opportunity to make a difference.”

Coincidentally, the same genetic variations are at play in an orphan indication called Membranoproliferative Glomerulonephritis Type II, which is also known as Dense Deposit Disease. It’s an end-stage renal disease that usually surfaces when victims are between 5 and 25 and the company plans to pursue a parallel program for that indication as well.

Optherion’s $37 million round can get the company to the end of the preclinical stage, says Foster. At that point a new venture round would likely be in order, but Foster isn’t going much farther than that in projecting how the company is likely to develop. There are simply too many variables at work to make a crystal ball work that far out.
Foster can see the money being used to beef up Optherion’s New Haven-based workforce from 14 to around 30 or 35, with most of the new additions coming in to support the science and development of its lead therapy.

At that point, its scientists will know a lot more about the therapy they’re working with, and its chance of making it in the clinic.