For people dealing with the creeping blindness of glaucoma, the most effective treatments are eye drops called prostaglandin analogues, or PGAs. They’re not a cure, but taken chronically, they’re the best way to slow the progression of the disease, which is why they’ve been the standard of care for glaucoma for more than two decades.
With data released this afternoon, however, Aerie Pharmaceuticals might have something better.
Bedminster, NJ, and Irvine, CA-based Aerie (NASDAQ: AERI) said its drug known as Roclatan has met its main goal in a 718-patient Phase 3 trial, the first of three late-stage studies. The drug—a combination of the most prescribed PGA, latanoprost (Xalatan), and Aerie’s experimental netarsudil (Rhopressa)—did a better job lowering the eye pressure of glaucoma patients than either latanoprost alone or netarsudil alone, for each of nine different time points measured over a three-month span. Patients were given a drop of either Roclatan, netarsudil, or latanoprost, once a day over the course of the trial.
Shares of Aerie surged more than 62 percent, to $34.35 apiece, in post-market trading on Wednesday.
Glaucoma occurs when the eye’s drainage systems become clogged, which in turn puts pressure on and damages the optic nerve. The goal for a glaucoma drug is to lower that pressure and protect people from losing their vision. About 2.7 million people in the U.S. live with glaucoma, a figure expected to climb to 4.3 million by 2030, according to the National Eye Institute.
“It’s great to have a medication that is essentially a [PGA]-plus that can achieve a few extra points on top of our first-line drug,” says Manishi Desai, a glaucoma specialist at Boston University School of Medicine, adding that the drug might be particularly useful for patients who don’t respond to their current medications. Desai isn’t involved with Aerie.
Aerie said that Roclatan lowered patients’ eye pressure levels by between 1 to 3 millimeters of mercury, or mmHg (the typical measurement for eye pressure), more than either latanoprost or netarsudil, throughout the study.
The company also said Roclatan lowered patients’ eye pressure by an average of 1.3 to 2.5 mmHg more than latanoprost, and by an average of 1.8 to 3.0 mmHg more than netarsudil. The drug’s effects didn’t wane over the 90 days people were tested.
But is the drug tolerable? Eye redness, or hyperemia, is a notorious side effects of PGAs. Netarsudil has also been shown to cause redness. Roclatan is a combination of the two, so it’s no surprise that the problem is even worse with the drug. About 53 percent of patients on Roclatan reported redness, compared to 41 percent of those on netarsudil and 33 percent of patients on latanoprost. Aerie says that 80 percent of those cases were mild, and the problem didn’t get worse with more treatment.
Other side effects reported included itchy eyes, an increase in tear production, brown or grey deposits in the cornea (what’s known as corneal verticillata), and leaky blood vessels in the eye. Aerie said there were no serious side effects, and chief medical officer Richard Lewis said in a statement that the safety data the drug has generated “points to a safe and tolerable product.”
“The results are what I may have expected—high efficacy with significant hyperemia,” says Richard Madonna, the chairman of clinical education at SUNY College of Optometry in New York. Madonna also isn’t involved with Aerie.
Although the cases of redness were reported as mild, Madonna wants to see how things play out over time, and whether the condition causes people to stop taking the drug. Thirty seven patients (15.5 percent) on Roclatan and 43 (17.6 percent) of those on netarsudil dropped out before three months of treatment, compared to 13 (5.5 percent) on latanoprost. 25 of those that dropped out on Roclatan and 23 patients on netarsudil stopped treatment because of side effects, compared to none on PGAs. Desai says the drop-outs are “a little concerning” given that most of those that left the trial did so because of side effects.
“Will the efficacy outweigh the lack of tolerability?” Madonna said.
The answer could come as Aerie reports more data. Patients in the current trial, known as Mercury 1, will stay on Roclatan for 12 months for safety evaluation. Aerie has two other Phase 3 trials underway. If Mercury 2, which will evaluate the drug’s effectiveness over 90 days, is successful, Aerie could file for FDA approval by the end of 2017. A third study, Mercury 3, is underway in Europe.
PGAs have been the standard of care for two decades, and beta blockers like timolol—today used as an adjunctive therapy for people who don’t respond to PGAs—were introduced in the late 1970s. Aerie hopes Roclatan can replace PGAs—many of which are generic—and provide another option for those who don’t respond to them. Aerie filed for FDA approval of netarsudil earlier this month. (Check out this story for more on Aerie, netarsudil, Roclatan, and the field of glaucoma treatments as a whole.)