Aerie Pharmaceuticals Inc (NASDAQ:AERI) just put out its second quarter financials, and alongside the release the company gave us some insight into its pipeline. Specifically, and of note, it expects to file a new drug application (NDA) with the FDA for its lead glaucoma candidate Rhopressa sometime before the end of September. If the FDA accepts the NDA quickly, we could be looking at a mid to late 2017 approval for the drug, and a chance for Aerie to start generating revenues and working towards a neutral cash flow. The NDA filing will no doubt serve up some immediate bullish momentum on its announcement, so ahead of this, here’s a look at the drug in question, and the science on which the application is based.
So, as we’ve said, it’s called Rhopressa, and it’s a triple action drug designed to reduce intra ocular pressure (IOP). It’s called triple action because it has three distinct MOAs working towards a common goal – reduced IOP. The first is the inhibition of what’s called ROCK, which is a type of kinase associated with the prevention of fluid drainage out of the inner eye. Inhibiting this kinase increases drainage, and reduces pressure in the process. The second is the reduction of blood. pressure in the veins that surround the eye. The third, through the inhibition of another kinase called NET, it reduces the production of fluid (which complements mechanism one).
So that’s the drug, what about the data?
The submission is based on a phase III registration trial that kicked off back in the summer of 2014. The trial was a bit up and down, and the company took a hit when interim safety data suggested that Rhopressa had a pretty rough safety profile; one that would make it inferior to a competitor drug. However, when an extension post topline looked at long term safety, the drug wasn’t nearly as bad from a toxicity perspective as everyone first thought, and the company picked up its strength.
Looking at efficacy, Rhopressa performed pretty well in the trial, meeting its primary efficacy endpoint by demonstrating non-inferiority compared to twice-daily timolol (which is the competitor mentioned in the above paragraph). It also hit on a few secondaries, and the dosing maintained efficacy across a 90-day period, which is exactly what the company was looking for.
What are the chances of approval? Well, it’s always tough to tell how these things are going to turn out – we’ve seen a number of drugs pick up CRLs already this year – but from the data we’ve got, and without taking into consideration the manufacturing element of the NDA (because we don’t have access to that yet), it looks like it’s got a pretty good chance of getting an agency green light.
What’s the market like?
Rhopressa is targeting an adjuvant indication at first, so it will have a restricted target market. Having said this, analysts suggest that the market will be somewhere in the region of 50% of glaucoma patients, which is not too bad, especially since there is the opportunity to expand into first line going forward. It’s estimated that more than 3 million US individuals have glaucoma in the US alone, but circa 50% of these don’t realize they have it. Of course, this 50% won’t be in the prescribed portion, so Aerie will be looking at targeting 50% of the 50% of the 3 million that know they have it and actively seek treatment. In other words, somewhere in the region of 750,000 individuals. We don’t yet have a price point, but looking at the market tolerance for cost, analysts have put a peak sales estimate on the drug of a little over $600 million.
So there we go. The takeaway on this one is that the NDA filing for a drug that has bounced back and forth between biotech analysts for close to half a decade is finally nearing NDA submission, and things are looking good for Aerie and it’s shareholders. T
o reiterate the timeframes, September submission, November/December acceptance, late Q3 2017 PDUFA. That’s if all goes smoothly.