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Watertown-based Eyepoint said Monday that patients administered its experimental treatment for a common cause of age-related blindness failed to maintain vision with less frequent injections compared to a standard treatment — meaning a Phase 3 clinical trial had fallen short of its primary goal.
The study setback imperiled Eyepoint’s plans to seek regulatory approval for the drug, Duravyu, and caused its stock price to plummet. Shares of Bedford-based Ocular Therapeutix, the maker of a competing eye treatment that will be submitted to the Food and Drug Administration later this year, rose in reaction.
Both Eyepoint and Ocular are developing drugs to maintain vision in people with wet age-related macular degeneration, or wet AMD; those drugs can be injected into the eye less frequently than currently approved treatments.
In the Phase 3 study called LUGANO, participants administered two eye injections of Duravyu over one year were able to make out nearly four additional letters on a standard eye chart compared to participants treated with six eye injections of Eylea, made by Regeneron Pharma, over the same one-year treatment period. The difference in visual acuity, however, was not large enough to prove statistical equivalence between Duravyu and Eylea — failing the study’s primary endpoint.
Eyepoint blamed the negative result on nine participants administered Duravyu who had “asymmetric” vision loss of more than 15 letters, or three entire lines on a standard eye chart, for reasons unrelated to wet AMD. Study participants treated with Eylea, collectively, also achieved vision improvement over one year that was better than the company anticipated.
After conducting an ad hoc analysis that excluded those nine patients, Duravyu was statistically equivalent to Eylea, Eyepoint said.
“We believe that Lugano demonstrated clinically meaningful results that reinforce the potential of Duravyu to improve the treatment paradigm in wet AMD,” said Eyepoint CEO Jay Duker on a conference call with analysts and investors.
Eyepoint shares fell 67 percent to $4.87 in Monday trading.
Investors were not buying Eyepoint’s explanation for the Duravyu study failure. Some of the Wall Street analysts who cover Eyepoint downgraded the company, others came to its defense.
To salvage Duravyu, Eyepoint will need to notch a win on a companion Phase 3 study called LUCIA that is nearing completion. Results will be announced this fall.
On the call with analysts Monday, Duker said the company believes the totality of data from two studies — one positive and one negative — will still be strong enough to support an FDA approval. But a successful LUCIA study is now more important than ever, and it’s not entirely clear if Eyepoint’s explanation for the LUGANO results will be borne out. Even if LUCIA is successful, will it be positive enough to overcome the negative results of the LUGANO study?
Ocular, Eyepoint’s main competitor, will be submitting a marketing application to the FDA for its wet AMD treatment, Axpaxli, during the fourth quarter, the company has said. Axpaxli is also intended to be injected twice per year.
Last February, Ocular reported results from its own Phase 3 study that demonstrated Axpaxli’s superiority over Eylea, although results were not as strong as investors had been expecting.
Regeneron’s Eylea (available in low and high doses) and Vabysmo, made by Roche, are the current market-leading treatments for wet AMD, with combined sales topping $15 billion annually. Vabysmo is typically injected into the eye every three or four months. Regeneron’s Eylea is injected every two or three months.
While Ocular and Eyepoint have both argued that patients with wet AMD will prefer less frequent injections, it’s still not clear if retinal physicians will see convenience as a compelling reason to switch, or if insurers will be willing to pay more.