Still signing up patients for its phase III trial with Fovista, the antiplatelet-derived growth factor agent that would be used with anti-VEGF therapy in wet age-related macular degeneration (AMD), Ophthotech Corp. pulled down a $200 million up-front ex-U.S. deal with Novartis AG that could be worth more than $1 billion, not including royalties, which are also part of the deal.

The deal keeps Fovista "anti-VEGF" agnostic, so physicians have a choice of which therapy they might use with it, said David Guyer, CEO of New York-based Ophthotech, who spoke to BioWorld Today ahead of a conference call with investors on Monday.

"Part of choice also is that some physicians may want a co-formulation, and market conditions change over time, so we like the ability to have it both ways," said Guyer. Included in the agreement is the possibility of mixing Fovista with an anti-VEGF from Novartis, of Basel, Switzerland, which will also develop a Fovista pre-filled syringe.

"They certainly have great expertise with that technology," Guyer said. In March, Novartis disclosed that it had come up with one for the wet AMD anti-VEGF therapy Lucentis (ranibizumab, Roche AG).

Along with Lucentis, another compound for which Fovista was designed to add extra punch is Eylea (aflibercept, Regeneron Pharmaceuticals Inc.). Used off-label for wet AMD is Avastin (bevacizumab, Roche AG). Anti-VEGFs keep abnormal blood vessels from growing behind the eye, which can cause eventual blindness, and Fovista is designed to make the same blood vessels shrink away. (See BioWorld Insight, July 22, 2013.)

Under terms of the deal, Ophthotech gets $200 million up front, plus another $130 million in enrollment-based milestone payments related to the phase III trial with Fovista, which Guyer described as "purely about recruitment." The study is "on target for top-line data in 2016. Since the endpoint for the trial is 12 months, you can subtract a year, and that's when recruitment is expected to be finished," he said, and the remaining milestone money would be paid.

Also included in the Novartis deal are contingent, ex-U.S. marketing approval-based milestone payments totaling as much as $300 million, and sales milestones outside the U.S. that could add up to another $400 million.

Ophthotech said earlier this month that the phase III Fovista program had opened trial sites for the company's third experiment, testing the drug in combination with Avastin and Eylea. The work builds on the strength of a phase IIb trial that tested Fovista 0.3 mg in combination with Lucentis 0.5 mg, Fovista 1.5 mg with Lucentis 0.5 mg, or sham with Lucentis 0.5 mg. Patients in the 1.5 mg Fovista/0.5 mg Lucentis group gained a mean of 10.6 letters of vision on the ETDRS standardized chart after 24 weeks of treatment, compared to 6.5 letters for patients receiving Lucentis monotherapy, with no significant safety issues in either treatment group.

Wet AMD therapies sold for $5 billion last year, despite a 60 percent penetrance by off-label Avastin, which means the market is much larger still, and destined to grow as the population ages, Guyer said. Ophthotech went public in September 2013 and more than doubled the hoped-for amount, selling 7.6 million shares at $22 each to garner $167 million. (See BioWorld Today, Sept. 26, 2013.)

Ophthotech's shares (NASDAQ:OPHT) were up 22 percent in after-hours trading, or $7.15, selling for $38.61.

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