|Aerie CEO Vicente Anido|
Last spring, as Aerie Pharmaceuticals ($AERI) execs rushed to man the ramparts as its share price tanked following the failure of its glaucoma drug Rhopressa, the company insisted that a slight change to its late-stage program would have been enough to get them over the goal line. So this morning, in a head-snapping turnaround, shares jumped 50% as the FDA proved amenable to making some changes that might let another Phase III trial succeed.
The biotech announced this morning that the FDA had provided written approval to change the primary endpoint for its second Phase III 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 will now represent a secondary endpoint range for Rocket 2."
Also, the FDA has proven agreeable to using a statistical methodology that will demonstrate that the trial is powered to reach those endpoints without having to add new patients to the mix.
One of the reasons why Aerie found itself in the crosshairs of investors in April is that CEO Vicente Anido had been confident in boasting to Reuters that the company was on track to gaining a blockbuster approval that should have set the stage for billion-dollar sales for its glaucoma drug. Failing at the first Phase III wasn't in the script. But with the trial changes, Anido is back to predicting a success.
"If Rocket 2 results resemble those of Rocket 1, we believe we may have a much greater opportunity for success in meeting the clinical endpoint of non-inferiority to timolol," noted Anido in a statement. "We are also very appreciative of the thoughtful guidance provided by the FDA, and believe their feedback will prove very useful as our programs progress. Looking ahead, we expect to commence our next Phase III registration trial for Rhopressa, named Rocket 4, in the third quarter of 2015. Rocket 4 is expected to be established with a primary endpoint range of above 20 mmHg to below 25 mmHg."
Investors seemed agreeable, bidding up the shares in a reverse of the spring-time rout. Aerie, though, may face some future problems trying to explain that first setback to payers, provided they go on to an approval.
Aerie had already set a low bar for itself for the first Rocket, going after evidence of noninferiority of its once-daily therapy with twice-daily timolol, a beta blocker, in lowering intraocular pressure. There were signs of a drop in efficacy during the trial and a third of the patients in the Rhopressa arm suffered from red eye--a condition that was widely viewed as a potential stumbling block for the biotech.
- here's the release