Aerie Pharmaceuticals Initiates First Phase 3 Registration Trial of RoclatanTM

Aerie Pharmaceuticals, Inc.

, a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class glaucoma therapies, today announced that on September 18, dosing commenced of the first patients enrolled in Mercury 1, the Company's first Phase 3 registration trial of RoclatanTM, a novel once-daily, quadruple-action eye drop being tested for its ability to lower intraocular pressure (IOP) in patients with glaucoma or ocular hypertension. Aerie anticipates total enrollment of approximately 690 patients in this three-arm one-year safety study with a 90-day efficacy readout. RoclatanTM, which is a fixed-dose combination of Aerie's product candidate RhopressaTM and latanoprost, the market leading prostaglandin analogue (PGA), demonstrated in its successful Phase 2b clinical trial in 2014 that it has the potential to be the most efficacious therapy for patients with glaucoma and ocular hypertension. The clinical endpoint of Mercury 1 is similar to the efficacy endpoint in the Company's previous Phase 2b clinical trial and compares RoclatanTM for superiority over each of its two components, with all three arms dosed once daily. The range for the primary endpoint evaluates patients with maximum baseline IOPs ranging from above 20 to below 36 mmHg (millimeters of mercury). The 90-day efficacy results of this study are expected to be reported in approximately one year. "Having just reported the successful Phase 3 efficacy results for our single-agent product candidate RhopressaTM, we believe RoclatanTM, which combines RhopressaTM with the market leading PGA in a fixed-dose combination, has significant IOP-lowering potential. Should RoclatanTM perform as well in Mercury 1 as it did in the RoclatanTM Phase 2b trial, we will have a very promising product candidate, with the potential to be the most efficacious therapy for patients with glaucoma and ocular hypertension," said Vicente Anido, Jr., Ph.D., Chairman and Chief Executive Officer at Aerie. Dr. Anido continued, "We expect to initiate two additional RoclatanTM Phase 3 trials in 2016. Mercury 2 will be a second registration trial in the United States structured similarly to Mercury 1 and designed to measure efficacy over 90 days. In addition, Mercury 3 will be a third registration trial in Europe designed to assist with European approval, and is expected to be a non-inferiority trial comparing RoclatanTM to a widely prescribed fixed-dose combination product marketed in Europe." Quadruple-Action Roclatan™ Roclatan™ is a once-daily eye drop that combines our triple-action Rhopressa™ with latanoprost, a PGA that is the most widely prescribed glaucoma product. If approved, we believe that Roclatan™ would be the first glaucoma product to lower IOP through all known mechanisms: (i) increasing fluid outflow through the trabecular meshwork (TM), the eye's primary drain, (ii) increasing fluid outflow through the uveoscleral pathway, the eye's secondary drain, (iii) reducing fluid production in the eye, and (iv) reducing episcleral venous pressure (EVP). A successful 28-day Phase 2b clinical trial for Roclatan™ was completed in June 2014. RoclatanTM achieved its primary efficacy endpoint on day 29 and demonstrated statistical superiority over the product's individual components at all time points. We believe that Roclatan™, if approved, would be the only glaucoma product that covers the full spectrum of known IOP-lowering mechanisms, giving it the potential to provide a greater IOP-lowering effect than any currently approved glaucoma product. The first Phase 3 registration trial for RoclatanTM, named Mercury 1, commenced in September of 2015 and is the subject of this press release. It is expected that there will be two additional Phase 3 trials for RoclatanTM, both commencing in 2016. Triple-Action RhopressaTM RhopressaTM is a novel triple-action eye drop that we believe, if approved, would become the only once-daily product available that specifically targets the trabecular meshwork, the eye's primary fluid drain and the diseased tissue responsible for elevated IOP in glaucoma. Preclinical results have demonstrated that RhopressaTM also lowers episcleral venous pressure, which contributes approximately half of IOP in healthy subjects. Further, RhopressaTM provides an additional mechanism that reduces fluid production in the eye and therefore lowers IOP. Biochemically, RhopressaTM is known to inhibit both Rho Kinase (ROCK) and norepinephrine transporter (NET). Recent preclinical studies have shown that RhopressaTM may have disease-modifying properties, including an anti-fibrotic effect on the trabecular meshwork and the potential to increase perfusion of the trabecular meshwork. Preclinical research is also currently underway to evaluate the potential neuroprotective benefits of RhopressaTM. There are four Phase 3 registration trials for RhopressaTM. "Rocket 2," the successful results of which were recently reported, is a 12-month safety trial with a 90-day efficacy readout. Safety data for the 12-month period of the Rocket 2 trial is expected late 2015 or early 2016. "Rocket 1," the results of which were initially reported in April 2015, was a 90-day efficacy trial that did not achieve its primary endpoint, but did achieve its pre-specified secondary endpoint. "Rocket 3" is a 12-month safety-only study in Canada which is currently in progress. A fourth Phase 3 trial, named "Rocket 4," is expected to commence in late September 2015. Based on the successful results of Rocket 2, Aerie expects to submit a New Drug Application filing for RhopressaTM in mid-2016.