|Aerie CEO Vicente Anido Jr.|
After persuading the FDA to lower the bar on its second Phase III study for its glaucoma drug Rhopressa, Aerie Pharmaceuticals ($AERI) has delivered the efficacy data it asserts regulators will sanction in an upcoming new drug application. And CEO Vicente Anido Jr. is promising $1 billion-plus in blockbuster sales revenue, though not everyone shares his unbridled enthusiasm for this drug.
In a dramatic turnaround for Irvine, CA-based Aerie, which was forced to concede that its first Phase III had failed to hit the primary endpoint last April, the FDA provided written approval in June to change the primary endpoint for their second late-stage study--Rocket 2--"to include patients with baseline intraocular pressures ranging from above 20 mmHg (millimeters of mercury) to below 25 mmHg." The former range for the primary endpoint of above 20 mmHg to below 27 mmHg was moved to a secondary endpoint range for Rocket 2.
That proved the key ingredient for success today, with once- and twice-daily dosing proving noninferior compared to twice-daily timolol, a beta blocker, which is the second most frequently used class of meds for glaucoma.
But the results were a bit messy overall.
Had the FDA maintained the old primary endpoint, Aerie would have missed it again. Twice-daily dosing also spurred an increase in adverse events, though, with eye redness proving a routine bane for patients. The high patient dropout rate of 40% was quickly flagged by analysts on Twitter and won't be ignored by payers if Aerie remains on track to a 2017 market launch.
Shares of the company soared 72% in after-market trading. And Anido, who rarely misses a chance to tout the company's prospects, says that an NDA is on track for filing in mid-2016 that will make the biotech a major contender in the ophthalmology market.
"We are very impressed by these Rhopressa Phase III results from the Rocket 2 study," Anido said in a statement that went out after the market closed. "This product has demonstrated great promise with its novel mechanisms of action, including its ability to target the diseased tissue responsible for elevated IOP in glaucoma. The clear success demonstrated in this clinical trial, combined with the preclinical research to date on the disease-modification potential of Rhopressa, represent key building blocks in driving Aerie toward becoming a major ophthalmic pharmaceutical company."
- here's the release