Presentation from OIS@ASCRS 2016
Tom Mitro, President & COO
Video Presentation:
https://www.youtube.com/watch?v=rjLnP6lmRWU&list=PL1dmdBNnPTZJBhQxPOp0vdNg3s3wtN2yw&index=29
2. 2
Important Information
Any discussion of the potential use or expected success of our product candidates is subject to
our product candidates being approved by regulatory authorities. In addition, any discussion of
clinical trial results for RhopressaTM (netarsudil ophthalmic solution) 0.02% relate to the results in
its first Phase 3 registration trials, Rocket 1 and Rocket 2, and for RoclatanTM
(netarsudil/latanoprost ophthalmic solution) 0.02%/0.005% relate to the results in its Phase 2b
clinical trial.
The information in this presentation is current only as of its date and may have changed or may
change in the future. We undertake no obligation to update this information in light of new
information, future events or otherwise. We are not making any representation or warranty that
the information in this presentation is accurate or complete.
Certain statements in this presentation are “forward-looking statements” within the meaning of the
federal securities laws. Words such as “may,” “will,” “should,” “would,” “could,” “believe,” “expects,”
“anticipates,” “plans,” “intends,” “estimates,” “targets,” “projects,” “potential” or similar expressions
are intended to identify these forward-looking statements. These statements are based on the
Company’s current plans and expectations. Known and unknown risks, uncertainties and other
factors could cause actual results to differ materially from those contemplated by the statements.
In evaluating these statements, you should specifically consider various factors that may cause
our actual results to differ materially from any forward-looking statements. In particular, the
preclinical research discussed in this presentation is preliminary and the outcome of such
preclinical studies may not be predictive of the outcome of later 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 presentation. These risks and uncertainties are
described more fully in the quarterly and annual reports that we file with the SEC, particularly in
the sections titled “Risk Factors” and “Management’s Discussion and Analysis of Financial
3. 3
Late Stage, Once-Daily, First-in-Class,
IOP–Lowering Products
• Rhopressa™ (netarsudil ophthalmic solution) 0.02%
• Shown to inhibit ROCK and NET, lower EVP and target diseased tissue
• Summary of two US phase 3 trials:
• Efficacy: non-inferiority to BID timolol at baseline IOP <25 and
stable IOP lowering from month 3 thru month 12
• AE’s: commonly mild, sporadic, non-serious, non-sight threatening,
self-resolving
• NDA filing expected Q3 2016
• Roclatan™ (netarsudil / latanoprost ophthalmic solution) 0.02% /
0.005%
• Fixed combination of Rhopressa™ and latanoprost
• Two P3’s in process; topline efficacy expected Q3 2016
• Both products are patent protected through 2030
Aerie – Building a Major Ophthalmic
Pharmaceutical Company
Source: Aerie Pharmaceuticals RhopressaTM Update public SEC filing, March 17, 2016
4. 4
Early Stage, Promising, Potential Break-Through Areas
• Netarsudil (Rhopressa™ and Roclatan™)
• Potential for disease modification in glaucoma
• AR-13154 (multi-kinase inhibitors for AMD)
• Significant lesion size reduction in wet AMD
Aerie – Preclinical Research Findings
Data on File
5. 5
Netarsudil* May Have Anti-Fibrotic Activity in
Human Trabecular Meshwork
Fibrosismarkers
Control TGFb2 (8ng/ml)
Collagen1AFSP1SMA
TGFb2 (8 ng/ml) +
AR-13324 (500nM)
P. Vasantha Rao, Duke University
• Active ingredient of Rhopressa™ and Roclatan™; TGFb2: Transforming growth factor b2; SMA: Smooth muscle actin; FSP1: Fibroblast-
specific protein 1
• Pattabiraman, Padmanabhan P., et. Al., (2015 March) “Effects of Rho Kinase inhibitor AR-13324 on actin cytoskeleton and TGFb2 and
Netarsudil* Blocks TGF-beta-Induced Expression of Fibrosis
Proteins in Human TM Cells
6. 6
Netarsudil Treated Control Treated
Netarsudil* May Increase Perfusion, Nutrient
Delivery to Human TM Outflow Tissues
Dan Stamer (Duke), Haiyan Gong (Boston University)
Netarsudil’s Ability to Increase Perfusion of Human TM Ex Vivo
Supports Potential to Provide More Nutrients and Antioxidants to the
TM
0.00
10.00
20.00
30.00
40.00
50.00
60.00
Control Experimental
Anterior PEFA** (%)
NetarsudilControl
Left (OS) and right (OD) eyes perfused with fluorescent microbeads
Control = buffered saline solution
*Active ingredient of Rhopressa™ and Roclatan™
** Percent Effective Filtration Area
7. 7
+ Netarsudil
Control
Netarsudil* Causes Expansion of TM Tissue Ex
Vivo, Opening Spaces for Increased Outflow
Dan Stamer (Duke), Haiyan Gong (Boston University)
TM: Trabecular Meshwork
SC: Schlemm’s Canal
Control = buffered saline solution
*Active ingredient of Rhopressa™
# p < 0.01
Increasing Trabecular Outflow, Reducing Fibrosis Could Stop
Degeneration of Outflow Tissues in Glaucoma
FLOWFLOW
8. 8
Laser-induced
choroidal
neovascularization
(CNV) in rats
Compounds delivered
by intravitreal injection
ROCK/JAK2/PDGFRb Inhibitor AR-13154 Numerically More Effective
than Eylea® in Rat Model of AMD
20000
30000
40000
50000
60000
70000
80000
90000
100000
110000
Saline
n=49
0.06 ug/mL
AR-13154
n=28
0.6 ug/mL
AR-13154
n=25
6 ug/mL
AR-13154
n=25
800 ug/mL
Eylea
n=20
um2IsolectinIB4(+SEM)
Total CNV Lesion Area (Day 21)
**
*
* p<0.05 vs. Saline
** p<0.001
®
* Data on file
AR-13154 Selective Multi-Kinase Inhibitors Targeting
Multiple Disease Processes Underlying AMD