Our experience and expertise with back of the eye diseases enables us to concentrate on developing and commercializing therapies for both dry and wet Age-Related Macular Degeneration (AMD) in an accelerated manner.

Ophthotech is focused on developing and commercializing therapies for age-related macular degeneration (AMD), an expanding, multi-billion dollar therapeutic area. The company’s lead product candidate, an anti-PDGF agent (E10030), is the first therapy to demonstrate both disease modification and significant visual gain in the majority of patients with wet AMD when co-administered with an anti-VEGF agent. Wet AMD remains a major unmet need as most patients fail to gain significant vision with monotherapy using an anti-VEGF agent, the current standard of care. A highly powered 445-patient Phase 2b trial of E10030 is fully enrolled, with results expected in the first half of 2012.

About Ophthotech

Ophthotech Corp. is a privately held biopharmaceutical company based in Princeton, NJ and New York, NY focused on developing and commercializing therapies for dry and wet AMD. Ophthotech is developing a pipeline of compounds with strong scientific foundations for the treatment of AMD, with the goal of bringing them to market in an accelerated manner. For more information, please visit www.ophthotech.com.
 

Investors
Tom Biancardi
Ophthotech Corp.
VP, Finance and Operations
609-945-6050
tom.biancardi@ophthotech.com

Media
Jennifer Devine
SmithSolve LLC
On behalf of Ophthotech Corp.
973-442-1555 ext. 102
jennifer.devine@smithsolve.com
 

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Princeton, NJ – April 26, 2010 – Ophthotech Corp. (“Ophthotech”), a privately held biopharmaceutical company focused on developing ophthalmic therapies for back-of-the-eye diseases, today announced that the first patient has been enrolled in its Phase II randomized, controlled clinical trial of E10030, an anti-PDGF aptamer, in combination with Lucentis® for the treatment of wet age-related macular degeneration (AMD).

The Phase II trial will assess the efficacy and safety of the combination treatment regimen with approximately 444 patients enrolled at sites in the US, Europe and Latin America. In a Phase I clinical study, 59 percent of patients treated with E10030 and Lucentis, an anti-VEGF agent, gained significant vision (3-line gain or better) 12 weeks after the start of therapy. Notably, there was a mean decrease of 86% in the area of choroidal neovascularization (CNV) at 12 weeks. In comparison, current standard of care utilizing monotherapy anti-VEGF treatment does not induce significant neovascular regression and results in 3-line or better visual gain in approximately one third of patients.

"With current standard of care, the majority of wet AMD patients do not achieve significant visual gain nor neovascular regression,” said Samir Patel, MD, president and CEO of Ophthotech. “Combination therapy with E10030 demonstrates a significant advancement in the treatment of wet AMD. The magnitude of neovascular regression noted in this Phase 1 study appears to be associated with a significant visual gain in the majority of these patients after only 12 weeks, a result not seen with anti-VEGF treatment alone."

Wet AMD is characterized by the abnormal growth of blood vessels (neovascularization) beneath the retina, which leak blood and fluid and can cause permanent damage to cells in the center of the retina (the macula). This form of AMD is the most severe form of the disease, and often leads to permanent vision loss.

E10030 is an aptamer targeting PDGF-B, a key molecule involved in the recruitment and maturation of pericytes. Pericytes in neovascular tissue have been shown to be protective and play a major role in anti-VEGF treatment resistance. E10030 strips the pericytes from the neovascular tissue rendering it highly sensitive to an anti-VEGF attack.

About anti-PDGF E10030

E10030 is an aptamer-based compound directed against PDGF-B. Pharmacology studies indicate that E10030 binds to PDGF-B with high specificity and affinity and inhibits the functions of PDGF-B both in vitro and in vivo. In preclinical studies involving models of ocular neovascularization, concurrent inhibition of PDGF-B and VEGF-A signaling was superior to inhibition of the VEGF-A pathway alone and demonstrated the potential to regress neovascularization.

E10030 is one of three compounds that Ophthotech is developing to treat wet and dry AMD. Additional molecular entities include an aptamer targeting the complement factor C5 and volociximab, an anti-angiogenic monoclonal antibody targeting the α5β1 integrin, both currently in Phase I studies.

About AMD

Age-related macular degeneration (AMD) is the leading cause of blindness for people over the age of 50 in the United States and Europe. The role of abnormal neovascularization, or angiogenesis, in the pathogenesis of neovascular AMD has been well established. There are two forms of the disease, namely "dry" and "wet" AMD. The “wet” form is characterized by the growth of new blood vessels into the central region of the retina. These new vessels cause severe visual loss due to retinal damage caused by subsequent leakage and scar formation. Anti-VEGF therapies and photodynamic therapies have been approved for “wet” AMD. “Dry” AMD accounts for up to 90 percent of all cases of AMD. There is no approved therapy for “dry” AMD, which afflicts 8 million patients in the United States and an additional 8 million in Europe. Visual loss in “dry” AMD is typically not as severe as “wet” AMD, however, over time, “dry” AMD can progress to the wet form of the disease.

About Ophthotech

Ophthotech Corp. is a privately held biopharmaceutical company based in Princeton, NJ and New York, NY focused on developing and commercializing therapies for back-of-the-eye diseases. Ophthotech plans to develop a pipeline of compounds with strong scientific foundations for the treatment of AMD and bring them to market in an accelerated manner. For more information, please visit http://www.ophthotech.com.

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NEW YORK, December 15, 2009 — Ophthotech Corp., a biopharmaceutical company focused on developing novel ophthalmic therapies for both wet and dry age-related macular degeneration (AMD), announced today that it has closed a $30 million Series B financing round.

The financing round was led by Clarus Ventures, which will be represented on Ophthotech's board of directors by Nicholas Galakatos, Ph.D., managing director of Clarus. Clarus is joined in the current round by existing investors SV Life Sciences, Novo A/S and HBM BioVentures. Proceeds from the financing will be used to fund a Phase 2 combination trial of Ophthotech's lead product candidate for treatment of wet AMD, E10030, and development activities for the company's other targeted therapies for wet and dry AMD.

"In its Phase 1 combination therapy trial, E10030, which targets PDGF, has demonstrated unprecedented visual gain in patients with wet AMD. This remarkable visual outcome was accompanied for the first time by robust neovascular regression, reflecting the disruption of underlying pathology. E10030 has the potential of becoming a new standard for wet AMD treatment," said Samir Patel, M.D., president and chief executive officer of Ophthotech. "We welcome Nick Galakatos and Clarus Ventures to Ophthotech at an important time, as we look to move E10030 into a larger, randomized Phase 2 wet AMD study. Their deep experience in drug development and business strategy will be invaluable as we move all of our potentially breakthrough programs forward."

Galakatos added, "Ophthotech has an innovative pipeline of best-in-class products for the treatment of AMD. Our investment decision was driven by the quality of the pipeline and the experience of the management team, who successfully developed Macugen® into the first marketed anti-VEGF therapy for the eye. We are excited to be working with this team again."

E10030, an aptamer targeting PDGF, has demonstrated potency when combined with anti-VEGF agents such as Lucentis. In this Phase 1 combination study, 59 percent of patients treated with a combination of E10030 and Lucentis gained significant vision (3-line gain) 12 weeks after therapy. All patients demonstrated neovascular regression, a first in wet AMD therapy, with no evidence of drug-related adverse events.

In addition to E10030, Ophthotech is pursuing multiple therapeutic targets to develop next-generation treatments for wet AMD and potentially the first approved treatment for dry AMD.

About AMD

AMD is the leading cause of blindness for people over the age of 50 in the United States and Europe. The role of abnormal neovascularization, or angiogenesis, in the pathogenesis of neovascular AMD has been well established. There are two forms of the disease, "dry" and "wet" AMD. The wet form is characterized by the growth of new blood vessels into the central region of the retina. These new vessels cause severe visual loss due to retinal damage caused by subsequent leakage and scar formation. Anti-VEGF therapies and photodynamic therapies have been approved for wet AMD. Dry AMD accounts for up to 90 percent of all cases of AMD. There is no approved therapy for dry AMD, which afflicts 8 million patients in the United States and an additional 8 million in Europe. Visual loss in dry AMD is typically not as severe as wet AMD, however, over time, dry AMD can progress to the wet form of the disease.

About Ophthotech

Ophthotech Corp. is a privately held biopharmaceutical company focused on developing and commercializing therapies for back-of-the-eye diseases. Ophthotech plans to develop a pipeline of compounds with strong scientific foundations for the treatment of AMD and bring them to market in an accelerated manner. In August of 2007, Ophthotech announced a Series A venture financing and two separate in-licensing deals with Archemix Corp. and (OSI) Eyetech. A third in-license from Biogen Idec and PDL BioPharma was announced in January of 2008. Ophthotech's venture investors include SV Life Sciences, HBM BioVentures and Novo A/S. For more information, please visit http://www.ophthotech.com.

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Fort Lauderdale, Fla, May 4, 2009 – Ophthotech Corp. today announced positive results of a phase 1 clinical study evaluating E10030, its novel anti-platelet derived growth factor (anti-PDGF) in conjunction with an anti-vascular endothelial growth factor (anti-VEGF), to treat wet age-related macular degeneration (wet AMD). Anti-PDGF therapy resulted in enhanced visual outcome and was associated with significant neovascular regression. The results were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2009 Annual Meeting in Fort Lauderdale.

59% percent of patients treated with anti-PDGF and anti-VEGF gained significant vision (3-line gain) at week 12 after therapy. 100% of treated patients demonstrated neovascular regression. E10030 was well tolerated with no evidence of drug-related adverse events. Current standard of care treatment utilizing monotherapy anti-VEGF results in 3-line visual gain in approximately one third of patients and without significant neovascular regression.

“Marked neovascular regression, a first in any study, with an outstanding level of visual gain, is very promising for our patients. My clinical experience with current monotherapy anti-VEGF regimen in wet AMD is consistent with published studies, which show that on average neovascular regression does not occur,” said Dr. Lawrence J. Singerman, Clinical Professor at Case Western Reserve University and a principal investigator in over 50 macular clinical trials.

E10030 is an aptamer targeting PDGF, a key molecule involved in the recruitment and maturation of pericytes. Pericytes in neovascular tissue have been shown to be protective and play a major role in anti-VEGF treatment resistance. E10030 strips the pericytes from the neovascular tissue rendering it highly sensitive to an anti-VEGF attack.

“The objective and robust response of neovascular regression is consistent with the biologic activity of E10030. I look forward to a randomized trial design to confirm the strong proof of concept data of this study,” said Dr. Donald J. D’Amico, Professor and Chairman, Department of Ophthalmology, Weill Cornell Medical College, New York-Presbyterian Hospital.

“It is exciting to see our clinical trial confirm the strong preclinical data from oncology and ophthalmic studies targeting the molecules regulating pericyte and endothelial cell survival. Ophthotech will continue to devote its resources towards an accelerated development of our anti-PDGF compound,” said Dr. Samir C. Patel, President and CEO of Ophthotech Corp.

Wet AMD is characterized by the abnormal growth of blood vessels (neovascularization) beneath the retina, which leak blood and fluid and can cause permanent damage to cells in the center of the retina (the macula). This form of AMD is the most severe form of the disease, and often leads to permanent vision loss.

E10030 is one of three compounds that Ophthotech is developing to treat wet and dry AMD. Additional molecular entities include an anti-C5 aptamer and volociximab, an anti-angiogenic monoclonal antibody targeting the α5β1 integrin, both currently in a Phase I study.

About anti-PDGF E10030

E10030 is an aptamer-based compound directed against PDGF-B. Pharmacology studies indicate that E10030 binds to PDGF-B with high specificity and affinity and inhibits the functions of PDGF-B both in vitro and in vivo. In preclinical studies, E10030 demonstrated the potential to regress neovascularization when used in combination with a VEGF-A inhibitor. In experiments involving models of ocular vascularization, concurrent inhibition of PDGF-B and VEGF-A signaling was superior to inhibition of the VEGF-A pathway alone.

About AMD

Age-related macular degeneration (AMD) is the leading cause of blindness for people over the age of 50 in the United States and Europe. The role of abnormal neovascularization, or angiogenesis, in the pathogenesis of neovascular AMD has been well established. There are two forms of the disease, namely "dry" and "wet" AMD. The “wet” form is characterized by the growth of new blood vessels into the central region of the retina. These new vessels cause severe visual loss due to retinal damage caused by subsequent leakage and scar formation. Anti-VEGF therapies and photodynamic therapies have been approved for “wet” AMD. “Dry” AMD accounts for up to 90 percent of all cases of AMD. There is no approved therapy for “dry” AMD, which afflicts 8 million patients in the United States and an additional 8 million in Europe. Visual loss in “dry” AMD is typically not as severe as “wet” AMD, however, over time, “dry” AMD can progress to the wet form of the disease.

About Ophthotech

Ophthotech Corp. is a privately held biopharmaceutical company based in Princeton, NJ and New York, NY focused on developing and commercializing therapies for back-of-the-eye diseases. Ophthotech plans to develop a pipeline of compounds with strong scientific foundations for the treatment of AMD and bring them to market in an accelerated manner.

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5 Vaughn Drive Suite 106
Princeton, NJ 08540
Phone (609) 945-6050
Fax (609) 452-7435

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Ophthotech Corp.

One Penn Plaza
35th Floor
New York, NY 10119
Phone (212) 845-8200
Fax (212) 845-8250

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SV Life Sciences provides finance to businesses at all stages of development and across the human life sciences sector. These sectors range from biotechnology & pharmaceuticals to medical devices & instruments, to healthcare information technology and services. SV Life Sciences currently advises or manages five funds with capital commitments of approximately $1.6 billion which primarily invest amounts of between $1m and $20m in North America and Europe, but will consider innovative investments in other regions. Our team of 28 professionals has a diverse, complimentary set of skills and experience that allow us to tailor a team to work with almost any life sciences business.

www.svlifesciences.com

The firm is led by Managing Directors Nicholas Galakatos, Dennis Henner, Jeffrey Leiden, Robert Liptak, Nicholas Simon, Michael Steinmetz and Kurt Wheeler. The team includes Partner Emmett Cunningham, Transactional Partner Scott Requadt Principals Finny Kuruvilla and Michele Park, and Senior Advisor Edward Scolnick.

Clarus is presently managing $1.2 billion and is committing $20-$60 million per company. The firm invests globally out of its offices in Cambridge, MA and San Francisco, CA.

www.clarusventures.com