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Aerie Craters As Glaucoma Drug Comes up Short in Phase 3

Xconomy New York — 

Aerie Pharmaceuticals revealed this afternoon the first results from a group of late-stage trials for its glaucoma drug, and unfortunately for the Bedminster, NJ-based company, they don’t look pretty.

The drug, known as Rhopressa, failed the primary goal of Aerie’s (NASDAQ: AERI) study. It didn’t do a better job of lowering intra-ocular pressure than timolol, a glaucoma drug that’s been around for decades. Worse, the drug looks to have durability issues. Aerie reported that about 36 of the 182 patients on its drug in the trial showed “signs of loss of efficacy” as treatment continued. That’s a big problem for a drug meant to treat a chronic condition like glaucoma, where patients take drops daily for the rest of their lives to protect their vision.

As has been the case in previous studies, the main side effect associated with Rhopressa was redness; about 35 percent of patients on the drug experienced it, though 80 percent of those cases were reported as mild.

For Aerie’s part, the company said that Rhopressa wasn’t inferior to timolol in patients with eye pressure levels lower than 26 millimeters of mercury, or mmHg (the typical method of measurement for eye pressure). It also said that the drug was at least on par with, and often better than timolol in patients with pressure levels below 24mmHg. Additionally, a majority of the patients who saw the drug’s effects wane over time had pressure levels over 26mmHg, and 80 percent of glaucoma patients have measurements lower than 26 mmHg.

“We are obviously disappointed that we missed the primary endpoint…we expected Rhopressa to demonstrate better performance based on the results we saw in the previous Phase 2b studies,” said CEO and chairman Vicente Anido Jr., in a statement. “However, if we had set the high end of the baseline range just one mmHg less, we would have demonstrated non-inferiority compared to timolol at all nine measured time points and numerical superiority at the majority of time points. We believe Rhopressa shows great promise at [intra ocular pressures] where the majority of patients are represented.”

Investors aren’t buying the explanation. In after-hours trading, Aerie’s shares have plummeted more than 70 percent, to $10.60 apiece from $35.39 per share at Thursday’s close.

Glaucoma is essentially a plumbing problem for the eye. Its drainage systems get clogged with fluid, which in turn puts pressure on and damages the optic nerve. If the pressure isn’t relieved, patients can lose their vision. The concept behind Rhopressa is to impact the eye’s drainage systems from multiple angles, including its main drain, a mesh-like filter known as the trabecular meshwork—a target none of the standards of care, prostaglandin analogues (PGAs) like latanoprost (Xalatan), directly impact. (Timolol, meanwhile, is what’s known as a beta blocker, a class of drugs that are typically used as adjunctive therapies for people who don’t respond to PGAs).

Aerie has been hoping Rhopressa might replace or work in tandem with PGAs—drugs that, while very effective, irritate the eyes and can change the color of peoples’ pupils, among other issues. But today’s results are a setback, which opens the door for other competitors with different approaches, among them Lexington, MA-based Inotek Pharmaceuticals (NASDAQ: ITEK). Shares of Inotek climbed 10 percent in post-market trading. (For more on Aerie, its competitors, and the state of glaucoma care, check out this story from March.)

Aerie is running two other Phase 3 studies for Rhopressa; they’ll both produce data by the end of the year. Anido Jr., however, mentioned in a statement that Aerie may need to run an additional late-stage trial depending on the results of the remaining studies.