Ophthotech in-licenses pair of aptamers

In a pair of in-licensing deals with Archemix Corp. and OSI Pharmaceuticals, Ophthotech Corp. recently acquired rights to aptamer compounds for the treatment of macular degeneration. Ophthotech is a new company formed by former executive of Eyetech, which OSI Pharmaceuticals acquired in late 2005.

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PRINCETON, N.J.—Ina pair of in-licensing deals with Archemix Corp. and OSI Pharmaceuticals,Ophthotech Corp. recently acquired rights to aptamer compounds for thetreatment of macular degeneration. Ophthotech is a new company formed by formerexecutive of Eyetech, which OSI Pharmaceuticals acquired in late 2005.

With Archemix Corp.—a well-known aptamer pioneer—the companysigned an exclusive, worldwide licensing agreement for all ophthalmic uses ofArchemix's proprietary aptamers targeting the C5 component of the complementcascade in both the wet and dry forms of age-related macular degeneration(AMD). Archemix will focus its on-going aptamer program on cardiovascular andhematological diseases and cancer. Terms of the agreements between the twoprivate companies were not disclosed.

"Of principal importance," says Dr. Samir Patel, presidentand CEO of Ophthotech, "is that these developments indicate that angiogenesisand the dry form of AMD have come a long way over the past five years. Webelieve the in-licensing strongly addresses dry AMD which is the form thataccounts for up to 90 percent of all cases of the disease and for which no majortherapy exists today." Dr. Patel notes that the disease affects approximatelyeight million people in the United Statesand a like number in Europe.

The second agreement is with OSI Pharmaceuticals which,subsequent to merging with Eyetech, Inc., decided to divest itsanti-platelet-derived growth factor (PDGF) aptamer program. At roughly the sametime, Dr. Patel and others, who now form the Ophthotech management team, leftEyetech to establish the new company.

Under the terms of the agreement, OSI will transfer toOphthotech all rights in the PDGF aptamer program, including rights to itspreclinical compound E10030, in exchange for an up-front cash payment, anequity interest in Ophthotech and potential future milestones and royalties.Again, terms of the agreement were not disclosed.

In preclinical studies, E10030 has shown the potential toregress neovascularization when used in combination with a vascular endothelialgrowth factor (VEGF) inhibitor. Anti-VEGF agents alone have shown the abilityto slow or halt, but not to regress the damage done by AMD. "We are excited todevelop this Phase-I-ready anti-PDGF aptamer for AMD," Dr. Patel says. "Studiessuggest that this compound, in combination with anti-VEGF agents, may causeregression of abnormal blood vessels in AMD. This is not observed withanti-VEGF therapy alone."

Dr. Patel adds, "As we know, based on improved understandingof the disease process, inflammation plays an important role in AMD as well asin coronary heart disease and elsewhere. In wet AMD, for example, histologicalexamination reveals tissue packed with inflammation cells. In addition, wet AMDpatients have inflammation biomarkers suggesting a complement-mediatedmechanism. "We believe that the anti-C5 aptamer blockade represents a potentialbreakthrough therapy for dry and wet AMD."

With the $36 million of financing raised in the initialround of funding, Ophthotech expects to have sufficient resources to executeits strategy. Participants in the private round include SV Life Sciences, HBMBioVentures and Novo A/S.

Former members of the Eyetech management team who havejoined Dr. Patel are Henry Simon (now with lead investor SV Life Sciences andformer Chairman of the Board of Eyetech), and Dr. David R. Guyer, (former CEOand co-founder of Eyetech, who will also serve as Ophthotech's Chairman of theBoard).

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