Aerie Pharmaceuticals took a beating from investors earlier this year when its glaucoma drug failed the first of a few Phase 3 trials. But today the company has bounced back, thanks to a helping hand from the FDA.
Bedminster, NJ and Irvine, CA-based Aerie (NASDAQ: AERI) said late Wednesday that its glaucoma drug, known as Rhopressa, hit its primary goal of what’s called “Rocket 2,” the second of a group of late stage studies. The goal was to be at least as good as timolol—a decades-old glaucoma drug—at lowering the pressure in the eyes of people who are starting out with pressure levels between 20 and 25 millimeters of mercury, or mmHg (the typical method of measurement for eye pressure). Rhopressa did not meet its secondary goal—successfully treating patients whose pressure started in a range between 20 and 27 mmHg.
Aerie expects to file an application with the FDA in the middle of next year.
Aerie is succeeding here, in part, because of the FDA’s help. Rhopressa didn’t just fail its first Phase 3 trial, Rocket 1; it was alarmingly losing its effectiveness over time in some patients. That’s not what you want to see from an eye drop for a chronic condition like glaucoma, where patients take drops for the rest of their lives to protect their vision.
Aerie, however, claimed that the drug would have passed that trial if the patient baseline was between 20 and 26 mmHg, and that the waning effect was primarily seen in patients with eye pressures over 26 mmHg.
The company and CEO Vicente Anido Jr. (pictured above) also said at the time that Rhopressa was at least as effective as timolol for patients with eye pressures lower than 26 mmHg. It also said Rhopressa was at least on par with and potentially better than timolol in patients with pressures below 24 mmHg. (Specifically, Aerie said the drug was superior to timolol at seven of nine tracked timepoints over the course of treatment.)
The takeaway is that Rhopressa is better at lowering eye pressure in patients starting out with lower pressure readings. A majority of glaucoma patients have eye pressures below 26 mmHg, according to Aerie.
After the trial failed in April, Aerie made its case to the FDA, and the agency agreed in June to change the primary goal for its coming study. That put Aerie’s assertions to the test. Today, Aerie claims its assertion has paid off with positive top-line results—among them, importantly, a note that the drug lowered patients’ eye pressure over the course of the 90-day treatment period, and didn’t wane.
Investors are cheering. Aerie’s shares were up more than 70 percent to about $31 apiece in post-market trading late Wednesday. That price is approaching the company’s share price prior to the trial flop in April.
Still, Aerie has questions to answer. One thing Aerie must continue to watch going forward, for instance, is just how big a deal the hyperemia, or redness is that its drug appears to cause. In today’s slideshow presentation, the company said that 35 percent of patients on the once-daily dose and 50 percent of those on the twice-a-day dose reported redness. Aerie asserted that a majority of those cases were mild, but Aerie’s slides showed that 9 percent of people on the once-daily dose and 17 percent of those on the twice-daily dose dropped out of the study early specifically because of redness. (It should be noted that standard-of-care prostaglandin analogues, or PGAs, also cause redness.)
What’s more, 40 percent of the folks on the twice-daily dose overall discontinued treatment early. Some other less frequent side effects seen in the study, according to Aerie’s slides, were blurry vision, corneal deposits, and subconjunctival hemorrhages (bursted blood vessels in the eye). And of course, Aerie still doesn’t know just how long Rhopressa will remain effective over time—something to be measured in further testing.
There hasn’t been much in the way of new treatments for glaucoma in many years. PGAs have been the standard of care for two decades, and so-called beta blockers like timolol—used as an adjunctive therapy for people who don’t respond to PGAs—were introduced in the late 1970s. Aerie hopes Rhopressa can at least supplant timolol, and either work with, or in some cases replace PGAs, which are effective but can irritate and change the color of peoples’ eyes, among other issues. (For more on Aerie, Rhopressa, and the field of glaucoma treatments as a whole, check out this story from March.)
Aerie is holding a conference call this afternoon to discuss the data.