Aerie Pharmaceuticals, Inc.
Developing new therapies for sight-threatening diseases of the eye.
We are a publicly traded (NASDAQ: AERI), clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class therapies for the treatment of patients with glaucoma and other diseases of the eye.
Glaucoma is one of the largest segments in the global ophthalmic market. In 2016, branded and generic glaucoma product sales exceeded $5.0 billion in the United States, Europe and Japan in aggregate, according to IMS. Glaucoma is a progressive disease in which elevated levels of intraocular pressure, or IOP, are associated with damage to the optic nerve, resulting in irreversible vision loss and potentially blindness. Additionally, glaucoma is a highly individualized disease and there is no direct correlation of the level of IOP at diagnosis with damage to the optic nerve.
According to the National Eye Institute, it is estimated that over 2.7 million people in the United States suffer from glaucoma, a number that is expected to reach 4.3 million by 2030. Furthermore, The Eye Diseases Prevalence Research Group has estimated that only half of the nation’s glaucoma sufferers know that they have the disease. Prescription volume for glaucoma products in the United States alone was 36 million in 2016 and is expected to grow, driven in large part by the aging population.
If approved, we believe our innovative product candidates could be effective across the majority of glaucoma patients. They are once-daily, well-tolerated eye drops that are designed to provide eye-care professionals with the first novel intraocular pressure-lowering mechanisms of action, or MOAs, to treat patients with glaucoma or ocular hypertension in over 20 years.
Our lead product candidate, Rhopressa™1 2 3 (netarsudil ophthalmic solution) 0.02%, successfully completed its second Phase 3 registration trial in September 2015. This trial, named Rocket 2, achieved its primary endpoint of non-inferiority of Rhopressa™ ophthalmic solution to comparator timolol across a range of baseline IOPs from above 20 millimeters of mercury (mmHg) to below 25 mmHg. In addition to successfully achieving non-inferiority to timolol at this endpoint range, the topline 12-month safety data from Rocket 2 confirmed a positive safety profile for the drug and demonstrated a consistent IOP lowering effect throughout the 12-month period at the specified timepoint.
Rhopressa™ ophthalmic solution also achieved non-inferiority to timolol in its first Phase 3 trial, named Rocket 1, at the pre-specified secondary endpoint range of baseline IOPs from above 20 mmHg to below 24 mmHg, while not achieving non-inferiority to timolol at the primary endpoint range of below 27 mmHg due to performance at the top millimeter of the range. We resubmitted the NDA for Rhopressa™ ophthalmic solution on February 28, 2017 using the Rocket 2 results as pivotal and Rocket 1 as supportive. In May 2017, we received notification from the FDA that the FDA has completed its initial 60-day review of the Rhopressa™ NDA and determined that the application is sufficiently complete to permit a substantive review. The Prescription Drug User Fee Act (PDUFA) goal date for the completion of the FDA’s review of the Rhopressa™ NDA is set for February 28, 2018.
With FDA approval in sight, why Aerie's keen on global strategy too
[Orginally published on November 21, 2017 by Jennifer Henderson, Triangle Business Journal]
Aerie – which is developing a pipeline of treatments for glaucoma and other eye diseases and is beefing up its research and development team locally – is also well on its way pursuing regulatory approval in both Europe and Japan moving forward.
“The company’s success to date, both clinically and financially, is what is allowing it to pursue all of these broad markets,” says Adnan Butt, a managing director at Guggenheim Securities.
Of the company pursuing approval of its pipeline in both the U.S. – with FDA approval expected in February or sooner – as well as in Europe and Japan to follow, Butt says “The progression is logical.”
However, the strategy is unique, he says, in the fact that it’s “a broader strategy than we typically expect” of a small- to mid-sized biotechnology company.
Aerie has a unique lead drug and pipeline – there is little competition in the space – and it’s also had success in clinical trials, Butt says. Additionally, the company has “the ability to grow organically” when it comes to it’s pipeline.
The company stated Thursday that it’s started dosing for a Phase 2 clinical trial aimed at progressing toward Japanese regulatory approval for its lead eye drop product Rhopressa for the treatment of glaucoma. While the Phase 2 trial will be conducted in the U.S., subsequent Phase 3 trials will need to be completed in Japan.
“Our global expansion strategy is now in full execution mode for both Japan and Europe, with the previously announced initiation of Mercury 3 in Europe,” said Vicente Anido, Jr., chairman and CEO at Aerie, in a statement.
“The Japanese glaucoma market is one of the largest in the world at approximately $1 billion annually, and we believe there continues to be substantial unmet need in this market,” he added.At the beginning of the year, Aerie said that it would shell out $25 million in construction and equipment costs for a new manufacturing plant in Ireland.
Going forward, the plant provides Aerie both “multiple sources of supply” and “control,” says Butt. It also plays into the company’s global access to the European and Asian markets, he confirms.