Aerie Initiates Fourth Phase III Clinical Trial of Rhopressa

First Patient Dosed in 90-Day Efficacy Trial Named Rocket 4

IRVINE , Calif.--(BUSINESS WIRE)--Aerie Pharmaceuticals, Inc. (NASDAQ:AERI), a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class glaucoma therapies, today announced that on September 23, dosing commenced of the first patients enrolled in Rocket 4, the Company’s fourth Phase 3 clinical trial of Rhopressa^TM, a novel once-daily, triple-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 700 patients in this two-arm 90-day efficacy study, comparing once-daily Rhopressa^TM for non-inferiority to twice daily timolol. The range for the primary endpoint mirrors Aerie’s successful Rocket 2 trial, and includes patients with baseline IOPs ranging from above 20 mmHg (millimeters of mercury) to below 25 mmHg. The 90-day efficacy results of this study are expected to be reported in approximately one year. Rocket 4 also includes a secondary endpoint range of baseline IOPs from above 20 mmHg to below 27 mmHg, and also a secondary endpoint timeframe of six months. Rocket 4 is designed to provide six-month safety data for a minimum of 300 patients to support expected future filings for regulatory approval in Europe. To ensure a proper cohort of patients in the study for primary and secondary endpoint purposes, Rocket 4 includes enrollment of patients with baseline IOPs up to 30 mmHg.

“Based on our prior discussions with the FDA, and the successful Rocket 2 results we announced last week, we remain on track to file our Rhopressa^TM NDA in mid-2016. The Rocket 4 clinical trial design includes the same clinical endpoint ranges used for our successful Rocket 2 clinical trial, from above 20 mmHg to below 25 mmHg, and is designed to provide additional clinical data for Rhopressa^TM and adequate six-month safety data to prepare us for our future expected European regulatory filings,” said Vicente Anido, Jr., Ph.D., Chairman and Chief Executive Officer at Aerie.

Dr. Anido continued, “In addition to looking forward to the results of Rocket 4 in about a year, we continue to learn more about this drug as we further analyze the successful Rocket 2 clinical trial results in addition to new preclinical research findings, the results of which we expect to share on November 13, 2015, at our event at the AAO (American Academy of Ophthalmology) meeting in Las Vegas, Nevada.”

Triple-Action Rhopressa^TM

Rhopressa^TM 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 Rhopressa^TM also lowers episcleral venous pressure, which contributes approximately half of IOP in healthy subjects. Further, Rhopressa^TM provides an additional mechanism that reduces fluid production in the eye and therefore lowers IOP. Biochemically, Rhopressa^TM is known to inhibit both Rho Kinase (ROCK) and norepinephrine transporter (NET). Recent preclinical studies have shown that Rhopressa^TM 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 Rhopressa^TM.

There are four Phase 3 registration trials for Rhopressa^TM. “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,” has just commenced. Based on the successful results of Rocket 2, Aerie expects to submit a New Drug Application filing for Rhopressa^TM in mid-2016.

About Aerie Pharmaceuticals, Inc.

Aerie is a 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. Aerie recently reported the successful results of its second Phase 3 registration trial in the United States named Rocket 2, where the primary efficacy endpoint was to demonstrate non-inferiority of IOP lowering for Rhopressa^TM compared to timolol. Rocket 3, a 12-month safety-only study, is currently in progress, and Aerie has now commenced a fourth Phase 3 trial for Rhopressa^TM, named Rocket 4. Aerie also completed in 2014 a Phase 2b clinical trial in which Roclatan^TM met the primary efficacy endpoint, demonstrating the statistical superiority of Roclatan^TM to each of its components, and recently commenced the first Phase 3 registration trial for Roclatan^TM, named Mercury 1. Aerie also recently announced research collaborations with GrayBug, Inc. and Ramot at Tel Aviv University as it further builds its pipeline for future growth.

Forward-Looking Statements

This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “exploring,” “pursuing” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the success, timing and cost of our ongoing and anticipated preclinical studies and clinical trials for our current product candidates, including statements regarding the timing of initiation and completion of the studies and trials; our expectations regarding the clinical effectiveness of our product candidates and results of our clinical trials; the timing of and our ability to obtain and maintain U.S. Food and Drug Administration or other regulatory authority approval of, or other action with respect to, our product candidates; our expectations regarding the commercialization of our product candidates; our expectations related to the use of proceeds from our initial public offering and the issuance and sale of our senior secured convertible notes; our estimates regarding anticipated capital requirements and our needs for additional financing; the potential advantages of our product candidates; our plans to pursue development of our product candidates for additional indications and other therapeutic opportunities; our plans to explore possible uses of our existing proprietary compounds beyond glaucoma; and our ability to protect our proprietary technology and enforce our intellectual property rights. By their nature, forward-looking statements involve risks and uncertainties because they relate to events, competitive dynamics and industry change, and depend on regulatory approvals and economic circumstances that may or may not occur in the future or may occur on longer or shorter timelines than anticipated. We discuss many of these risks in greater detail under the heading “Risk Factors” in the quarterly and annual reports that we file with the Securities and Exchange Commission (SEC). In particular, the preclinical research discussed in this press release is preliminary and the outcome of such preclinical studies may not be predictive of the outcome of later clinical trials. Any future clinical trial results may not demonstrate safety and efficacy sufficient to obtain regulatory approval related to the preclinical research findings discussed in this press release. Forward-looking statements are not guarantees of future performance and our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

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