Ophthotech Nabs $175M To Fund Late-Stage Trial For Eye Drug

5/29/13Follow @benthefidler

Ophthotech was founded by former Eyetech Pharmaceuticals executives to change the standard of care for the “wet” form of age-related macular degeneration: the leading cause of blindness among adults in the western world.

With a new $175 million round of financing the New York company will get its shot to prove its new drug candidate works in a large-scale clinical trial.

New York-based Ophthotech announced today that is has raised the new round of cash from Novo A/S and its current investors, which include SV Life Sciences, Novo Ventures (the VC arm of Novo Nordisk), HBM Healthcare Investments, and Clarus Ventures.

The financing comes in a couple parts. One is a $50 million Series C round of preferred equity financing, which is being supplied by both Novo, and Ophthotech’s other investors. The remaining $125 million is being committed by Novo Nordisk as part of a product royalty sale agreement. Essentially, Ophthotech is providing Novo with an undisclosed percentage royalty on future sales of its drug candidate E10030 (which it hopes to market under the name Fostiva). In return, Novo has agreed to pay $125 million in three equal installments. The first part of the Series C and the royalty financing deal has already closed, which means that Ophthotech should have about $58.3 million of new cash for its coffers today, and that it stands to collect roughly another $116.7 million from Novo and its other investors if its drug can hit all of its milestones.

Ophthotech will use the cash to bankroll a large late-stage study of E10030, a treatment for the wet form of AMD, a condition in which blood vessels leak behind the eye, leading to distorted vision and potentially blindness. Ophthotech hasn’t revealed the design of the study yet, but it will include 1,900 patients across 200 testing sites, and begin before the end of September.

The market for age-related macular degeneration has exploded over the past several years, as the standard of care has changed from laser therapies that didn’t actually improve patients’ vision—rather, it stopped their vision from getting worse while destroying the very retinal tissue clinicians were trying to save—to injectable pharmaceuticals that do. Roche/Genentech’s ranibizumab (Lucentis) and bevacizumab (Avastin), and Regeneron Pharmaceuticals’ afilbercept (Eylea) have all become hit drugs for treating the disease. All of them, however, fight AMD by inhibiting the vascular endothelial growth factor (VEGF) that researchers say causes the condition to worsen. Ophthotech’s E10030, instead, blocks platelet-derived growth factor (PDGF), a different protein that has also been implicated in the wet form of AMD.

This is important because the essential problem in the wet form of AMD is abnormal blood vessel growth in the macula, a part of the retina. While blocking VEGF on its own only stops those abnormal blood vessels from continuing to grow, scientists believe adding PDGF to the equation may cause them to recede. Ophthotech’s plan is to combine the two treatments into one regimen, giving patients one injection of an anti-VEGF such as ranibizumab, and another of E10030. While the idea of getting two injections in the eye in one sitting certainly isn’t a fun idea for potential patients, Ophthotech’s selling point is that combining the two simply works better than solely using any anti-VEGF on its own.

“Giving combination therapy via two injections does not increase the treatment burden for the patient or physician,” says Ophthotech CEO David Guyer. “The patient visits are unchanged.”

Ophthotech isn’t the only company to target PDGF—a startup in Waltham, MA called Kala Pharmaceuticals is developing a drug, for example, that blocks both VEGF and PDGF—but Ophthotech is the farthest along in development. The company in 2012 completed a mid-stage study of 449 patients over 24 weeks. In the study, patients randomly received either injections of 0.3 mg of E10030 and 0.5 mg of ranibizumab; 1.5 mg of E10030 and 0.5 mg of ranibizumab; or 0.5 mg of ranibizumab and a placebo injection. Ophthotech reported in October that the group taking the largest dose of E10030 with Roche’s drug had better results than those taking ranibizumab alone: in the former group, patients read an average of 10.6 letters on a standardized visual acuity chart more than they had at the beginning of the study. By comparison, patients taking ranibizumab alone could read an average of 6.5 more letters than they could before—a 62 percent benefit. Ophthotech reported that it was a statistically significant improvement, meaning it was highly unlikely that the results were due to chance, and that the drug was well tolerated by patients.

Guyer says that the next study will test patients who haven’t yet been treated for wet AMD, meaning the company hopes to establish the combination regimen with any anti-VEGF as a first-line therapy for the disorder.

“We are anti-VEGF agnostic,” Guyer says. “We believe that our drug will be shown to work with any of the anti-VEGF’s.”

He added that the primary goal for the study—which is also the standard FDA regulatory endpoint for approval of such drugs—will be the same. The only difference will be that the late-stage trial will test patients over the course of a full year instead of 24 weeks.

Guyer is the co-founder and former CEO of Eyetech Pharmaceuticals, which crafted the first VEGF blocker, pegaptanib (Macugen), to be approved by the FDA for wet AMD. Eyetech had already developed what is now known as E10030 when it was acquired by OSI Pharmaceuticals for $935 million in 2005—the year before ranibizumab was approved by the FDA and stole that drug’s thunder. Guyer, who subsequently left the company to work as a partner at SV Life Sciences, licensed the newer drug from OSI in 2007 and formed Ophthotech around it with Eyetech’s other co-founder, Samir Patel (Patel is now Ophthotech’s president).

“It’s a drug we’ve known for a very long time and we’re fortunate enough to do the development on,” he says.

Guyer wouldn’t reveal how much money Ophthotech has raised to date, though he did say that Novo Ventures has been a part of the investment group since the company’s inception. Guyer added that though he believes a small company like Ophthotech can successfully sell E10030 in the U.S., the company would likely look to find a partner to help sell the drug internationally.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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