Staff Writer

Optherion Inc. raised $37 million in a Series A round to advance preclinical research with a new approach to diagnosing and treating age-related macular degeneration (AMD).

The New Haven, Conn.-based company is pursuing the genetic link between AMD and mutations in the genes for Complement Factors H and B, as well as variations in Chromosome 10. According to Optherion's President and CEO Colin Foster, the initial focus is on genetic mutations in Complement Factor H, which occur in about 50 percent of AMD patients, resulting in an inactive form of Complement Factor H protein.

Optherion proposes to create a genetic test to identify patients with Complement Factor H mutations and treat them with a recombinant form of Complement Factor H protein. Foster said the company has several options regarding delivery of the protein, but it initially will focus on intravenous infusion. That systemic delivery has "wonderful benefits," he added, because patients with the mutation often are vulnerable to other diseases including cardiovascular disease, rheumatoid arthritis, asthma and stroke, all of which the replacement protein might have a beneficial effect on.

The recombinant Complement Factor H protein program is about two years from the clinic, Foster said. The company initially is targeting the program to dry AMD but also is studying it in wet AMD and Membranoproliferative Glomerulonephritis Type II (MPGN-II), a renal disease often caused by the same genetic variations underlying AMD. Other programs to diagnose and treat mutations in Complement Factor B and Chromosome 10 also are under way.

For each of the programs, Foster sees companion diagnostics as a key way to ensure "we give the right patient the right therapeutic."

Optherion was founded in 2005 by Gregory Hageman, a professor at the University of Iowa and co-discoverer of the AMD genetic links; ophthalmologist Sandy Gordon; and serial company builder David Scheer of Scheer & Co. Foster joined shortly afterward, leaving his post as CEO of Bayer Pharmaceuticals in the U.S. and region head for Bayer Pharmaceuticals in North America.

Quaker BioVentures and Domain Associates LLC led the Series A round, which is expected to last about two years. Additional investors included Johnson & Johnson Development Corp., Purdue Pharmaceutical Products LP, Pappas Ventures, Biogen Idec New Ventures and GE Healthcare Financial Services.

Foster said the reception from venture capitalists during the Series A fundraising was "very enthusiastic," despite the rapidly increasing amount of competition in the AMD space.

AMD affects more than 50 million people worldwide. About 10 percent of them have wet AMD, the more serious form of the disease, while the majority have dry AMD, which eventually can progress to wet AMD.

The market for wet AMD is dominated by the vascular endothelial growth factor (VEGF) inhibitor Lucentis (ranibizumab, Genentech Inc.), which generated $420 million in the first half of the year. The success of Lucentis and the off-label use of its less expensive cousin Avastin (bevacizumab), a VEGF inhibitor for cancer, have curbed competitors such as Macugen (pegaptanib sodium, OSI Pharmaceutical Inc.) and Visudyne (verteporfin, QLT Inc. and Novartis AG).

Yet plenty of companies see room in the wet AMD market for a little competition. Regeneron Pharmaceuticals Inc. is conducting a Phase III trial of its VEGF Trap-Eye, which offers less frequent injections than Lucentis, while Opko Health Inc. is in Phase III with the anti-VEGF siRNA drug bevasiranib, which may prove complementary to Lucentis.

Others developing wet AMD drugs include Allergan Inc., Pfizer Inc., TargeGen Inc. and Ophthotech Corp., just to name a few. (See BioWorld Today, July 12, 2007, and Aug. 6, 2007.)

There are no drugs approved specifically for dry AMD, but its large market has attracted significant interest. In Phase II trials are Sirion Therapeutics Inc.'s ST-602, an oral fenretinide that reduces lipofuscin buildup in dry AMD, and Othera Pharmaceuticals Inc.'s eyedrop OT-551.

Earlier in development are Ophthotech's Complement C5 targeted aptamer and Pfizer's TNX-234, as well as planned studies with TargeGen's wet AMD drug in patients at risk of progressing from the dry to the wet form of the disease.

Foster said another advantage of Optherion's systemic administration is that the protein "could be used alongside" other therapies approved for AMD.