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Aerie Pharmaceuticals Reports Topline Result from Rhopressa® Mechanism of Action Study

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Clinical confirmation of the effect of Rhopressa® on
trabecular meshwork outflow facility in patients with open-angle
glaucoma or ocular hypertension

Aerie Pharmaceuticals, Inc. (NASDAQ:AERI), an ophthalmic pharmaceutical
company focused on the discovery, development and commercialization of
first-in-class therapies for the treatment of patients with open-angle
glaucoma, retina diseases and other diseases of the eye, today
announced topline efficacy results from a double-masked, randomized,
placebo-controlled study (AR-13324-CS206; NCT03233308) designed to
evaluate the effect of Rhopressa® (netarsudil ophthalmic
solution) 0.02% on aqueous humor dynamics in patients with open-angle
glaucoma or ocular hypertension. While several pre-clinical models and a
previous study in healthy volunteers examined the effect of Rhopressa®
on trabecular meshwork outflow facility, this study is the first
performed on glaucoma patients to confirm that it lowers intraocular
pressure (IOP) primarily through this mechanism.

The study enrolled patients who were diagnosed with open-angle glaucoma
or ocular hypertension in both eyes, with unmedicated baseline IOP
between 20 and 30 mmHg (millimeters of mercury). The primary endpoint of
the study was the mean change from baseline in the mean diurnal outflow
facility, measured using previously published methods.1

In the study, Rhopressa® produced a statistically significant
increase in trabecular outflow facility of approximately 35 percent over
baseline. A complete analysis of the study data will be presented at a
future scientific congress.

"Elevated IOP in open-angle glaucoma is due to dysfunction of the
trabecular outflow pathway. This study confirms that in patients with
elevated IOP, Rhopressa® lowers IOP by increasing trabecular
outflow facility," said Casey C. Kopczynski, Ph.D., Chief Scientific
Officer, Aerie Pharmaceuticals. "We believe this will give physicians
additional confidence that Rhopressa® targets the tissue
responsible for elevated IOP in their patients, and it will help them
understand their options for combining this novel trabecular outflow
drug with other therapies that lower IOP through different mechanisms."

This topline finding is consistent with the published results of a study
of similar design in healthy volunteers (AR-13324-CS102; NCT02406287) in
which Rhopressa® was also shown to exert a statistically
significant effect on trabecular outflow facility.1

About Rhopressa®

Rhopressa® (netarsudil ophthalmic solution) 0.02%, is a novel
once-daily eye drop approved for the reduction of elevated intraocular
pressure (IOP) in patients with open-angle glaucoma and ocular
hypertension. A Rho kinase (ROCK) inhibitor, Rhopressa® is
specifically designed to increase outflow of aqueous humor (the fluid
inside the eye) through the trabecular meshwork, the main fluid drain of
the eye.

In clinical trials of Rhopressa®, the most common adverse
reactions were conjunctival hyperemia, cornea verticillata, instillation
site pain, and conjunctival hemorrhage. More information about Rhopressa®,
including the full product label, is available at www.rhopressa.com.

About Aerie Pharmaceuticals, Inc.

Aerie is an ophthalmic pharmaceutical company focused on the discovery,
development and commercialization of first-in-class therapies for the
treatment of patients with open-angle glaucoma, retina diseases and
other diseases of the eye. Aerie's first product, Rhopressa® (netarsudil
ophthalmic solution) 0.02%, a once-daily eyedrop approved by the U.S.
Food and Drug Administration (FDA) for the reduction of elevated
intraocular pressure (IOP) in patients with open-angle glaucoma or
ocular hypertension, was launched in the United States in April 2018. In
clinical trials of Rhopressa®, the most common adverse
reactions were conjunctival hyperemia, cornea verticillata, instillation
site pain, and conjunctival hemorrhage. More information about Rhopressa®,
including the full product label, is available at www.rhopressa.com.
Aerie's advanced-stage product candidate,
Roclatan™ (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005%, a
fixed-dose combination of Rhopressa® and the
widely-prescribed PGA (prostaglandin analog) latanoprost, achieved its
3-month primary efficacy endpoint in two Phase 3 registration trials,
Mercury 1 and Mercury 2, and also showed safety and efficacy throughout
12 months in Mercury 1. Aerie submitted the Roclatan™ New Drug
Application (NDA) in May 2018 and, in July 2018, the FDA set the PDUFA
(Prescription Drug User Fee Act) goal date for the completion of the
FDA's review of the Roclatan™ NDA for March 14, 2019. Aerie continues to
focus on global expansion and the development of additional product
candidates and technologies in ophthalmology, including for wet
age-related macular degeneration and diabetic macular edema. More
information is available at www.aeriepharma.com.

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", "opportunities" 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: our
expectations regarding the commercial launch and sales of Rhopressa®
and Roclatan™ and any future product candidates, if approved; our
commercialization, marketing, manufacturing and supply management
capabilities and strategies; third-party payer coverage and
reimbursement of Rhopressa® and Roclatan™ and any future
product candidates, if approved; the glaucoma patient market size and
the rate and degree of market adoption of Rhopressa® and
Roclatan™ and any future product candidates, if approved, by eye-care
professionals and patients; the timing cost or other aspects of the
commercial launch of Rhopressa® and Roclatan™ and any future
product candidates, if approved; the success, timing and cost of our
ongoing and anticipated preclinical studies and clinical trials for
Rhopressa®, with respect to regulatory approval outside the
United States, and Roclatan™ and any future product candidates,
including statements regarding the timing of initiation and completion
of the studies and trials; our expectations regarding the effectiveness
of Rhopressa®, Roclatan™ and any future product candidates
and results of our clinical trials and any potential preclinical
studies; the timing of and our ability to request, obtain and maintain
FDA or other regulatory authority approval of, or other action with
respect to, as applicable, Rhopressa®, Roclatan™ and any
future product candidates in the United States, Canada, Europe, Japan
and elsewhere, including the expected timing of, and regulatory and/or
other review of, filings for, as applicable, Rhopressa®,
Roclatan™ and any future product candidates; the potential advantages of
Rhopressa®, Roclatan™ and any future product candidates; our
plans to pursue development of additional product candidates and
technologies in ophthalmology, including development of Rhopressa®
and Roclatan™ for additional indications, our preclinical retina
programs and other therapeutic opportunities; our plans to explore
possible uses of our existing proprietary compounds beyond glaucoma and
ophthalmology; our ability to protect our proprietary technology and
enforce our intellectual property rights; and our expectations regarding
collaborations, licensing, acquisitions and strategic operations,
including our ability to in-license or acquire additional ophthalmic
products, product candidates or technologies. By their nature,
forward-looking statements involve risks and uncertainties because they
relate to events, competitive dynamics, industry change and other
factors beyond our control, and depend on regulatory approvals and
economic and other environmental 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, FDA
approval of Rhopressa® does not constitute FDA approval of
Roclatan™, and there can be no assurance that we will receive FDA
approval for Roclatan™ or for any future product candidates. FDA
approval of Rhopressa® also does not constitute regulatory
approval of Rhopressa® in jurisdictions outside the United
States, and there can be no assurance that Rhopressa® will
obtain regulatory approval in other jurisdictions. Our receipt of a
Prescription Drug User Fee Act (PDUFA) goal date notification for
Roclatan™ does not constitute FDA approval of the Roclatan™ New Drug
Application (NDA), and there can be no assurance that the FDA will
complete its review by the PDUFA goal date of March 14, 2019, that the
FDA will not require changes or additional data that must be made or
received before it will approve the NDA, if ever, or that the FDA will
approve the NDA. In addition, the research discussed in this press
release is preliminary and the outcome of such studies may not be
predictive of the outcome of later studies. 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.

1 Kazemi A, McLaren JW, Kopczynski CC, Heah TG, Novack GD,
Sit AJ. The effects of netarsudil ophthalmic solution on aqueous humor
dynamics in a randomized study in humans. J Ocul Pharmacol Ther. 2018
Feb 22. doi: 10.1089/jop.2017.0138.

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