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OIS@AAO
October 13, 2016
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 3 registration 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, including the updated guidance presented herein, 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 topline Mercury 1
data presented herein is preliminary and based solely on information available to us as of the date of this document and
additional information about the results may be disclosed at any time, including at our Investor Day on October 5, 2016. In
addition, 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 Condition and Results of
Operations.” Such forward-looking statements only speak as of the date they are made. We undertake no obligation to
publicly update or revise any forward-looking statements, whether because of new information, future events or otherwise,
except as otherwise required by law.
3
Aerie Late Stage IOP–Lowering Products
Pre-Clinical Research
• Rhopressa™
• Potential for disease modification and neuroprotection
• AR-13154
• Significant lesion size reduction in wet AMD
• Drug Delivery - Front and back of the eye
• Rhopressa™ (netarsudil ophthalmic solution) 0.02%
• Inhibits ROCK, NET, lowers EVP, targets diseased tissue
• NDA filed Q3 2016, entering launch mode
• Roclatan™ (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005%
• Fixed combination of Rhopressa™ and latanoprost
• Two P3’s in process; first P3 achieved primary efficacy endpoint
Aerie – Building a Major Ophthalmic
Pharmaceutical Company
Data on file
4
Rhopressa™ Trials for Q3 2016 NDA Submission
“Rocket 1”
90-Day Efficacy
Registration Trial
U.S.
 Rhopressa™ 0.02% QD ~200 patients
 timolol BID ~200 patients
(Total enrollment: 411 patients)
“Rocket 2”
One Year Safety
(3 Mo. Interim
Efficacy)
Registration Trial
U.S.
 Rhopressa™ 0.02% QD ~230 patients
 Rhopressa™ 0.02% BID ~230 patients
 timolol BID ~230 patients
(Total enrollment: 756 patients)
ClinicalTrials.gov Identifier: NCT02207491, NCT02207621
5
Rocket 2 and Rocket 1 Performance
Maximum Baseline IOP <25 mmHg
Rocket 2
Rocket 1
Data on file
6
Rocket 2: 12 Month 8am IOP Efficacy
Safety Population, Completed Patients (<27 mmHg)
 Difference in 8am mean IOP from
Day 90 (Month 3) to Month 12:
RhopressaTM QD:
 Full population: +0.1 mmHg
 Subset analyses: -0.3 to +0.3 mmHg
Timolol BID:
 Full population: +0.5 mmHg
 Subset analyses: +0.1 to +1.4 mmHg
Day 90 Month 12
AR-13324 0.02% q.d. (n=118)
BL W2 W6 M3 M6 M9 M12
Data on file: AR-13324 – CS302
7
Roclatan™ Registration Trial Design
“Mercury 1”*
One Year Safety
(3 Mo. Interim
Efficacy)
Registration Trial
U.S.
“Mercury 2”**
90-Day Efficacy
Registration Trial
U.S. and Canada
“Mercury 3”
6 Mo. Efficacy and
Safety
Registration Trial
Europe
 Roclatan™ QD ~230 patients
 Rhopressa™ 0.02% QD ~230 patients
 latanoprost QD ~230 patients
 Roclatan™ QD ~230 patients
 Comparator (TBD) ~230 patients
* ClinicalTrials.gov Identifier: NCT02558400
** ClinicalTrials.gov Identifier: NCT02674854
 Roclatan™ QD ~230 patients
 Rhopressa™ 0.02% QD ~230 patients
 latanoprost QD ~230 patients
8
Mercury 1: Study Design And Analysis
• Trial design follows FDA requirement for fixed dose combination
– Superiority of combination over each individual component
– Statistically significant difference at each measured time
point
– Higher combo efficacy vs. components at ~1-3 mmHg, as
previously accepted by FDA for product approval
(SimbrinzaÂŽ)
• The primary statistical modeling of the primary efficacy analysis
was agreed with the FDA using the intent to treat (ITT)
population with imputation for any missing data
Data on file
9
RoclatanTM Achieved Statistical Superiority Over
Individual Components at All Time Points
Mean IOP at Each Time Point (ITT)
***p<0.0001 vs Latanoprost and RhopressaTM
Data on file and PG324 - CS301 Top Line Data
10
Mercury 1: RoclatanTM Phase 3 Responder
Analysis
Day 90: % of Patients with IOP Reduced to 18 mmHg or Lower
***
***
***
14%
23%
32%
42%
54%
15%
25%
39%
54%
69%
33%
44%
61%
71%
82%
0%
20%
40%
60%
80%
100%
≤ 14 mmHg ≤ 15 mmHg ≤ 16 mmHg ≤ 17 mmHg ≤ 18 mmHg
%ofPatients
IOP on Treatment
Rhopressa™ (n=198) Latanoprost (n=223) Roclatan™ (n=200)
###
###
***p<0.0001 vs Latanoprost and RhopressaTM
###p<0.0001 vs RhopressaTM, p<0.05 vs Latanoprost
Data on file and PG324 - CS301 Top Line Data
11
Mercury 1: Roclatan™ Summary
• Demonstrated superiority over both latanoprost and Rhopressa™ for the
primary efficacy analysis at all 9 time points (p<0.0001)
• IOP-lowering effect was greater (1-3 mmHg) than monotherapy with either
latanoprost or Rhopressa™ throughout the duration of the study
• There were no drug-related serious adverse events and was no evidence of
treatment-related systemic effects
• The main adverse event was conjunctival hyperemia, ~50% of patients and
was scored as mild for ~80% of these patients
Next Steps:
• Mercury 2: Topline 3-month expected H1 2017
• Mercury 3 (Europe): Efficacy study, comparing to a leading combo product
marketed in Europe, expected to commence in H1 2017
• RoclatanTM NDA filing expected near year-end 2017
Data on file and PG324 - CS301 Top Line Data
12
Mercury 1: Rhopressa™ Arm Performance
Summary
• Demonstrated non-inferiority to latanoprost in patients
with baseline IOP < 25 mmHg
• Efficacy relative to latanoprost improves as baseline IOP
declines
• RhopressaTM maintained consistent IOP lowering across
all baseline IOPs including > 25 mmHg
• Stable efficacy from week 2 to month 3
• Adverse event profile consistent with previous studies
Data on file and PG324 - CS301 Top Line Data
13
Aerie Summary
• Key Clinical Priorities
• RhopressaTM: NDA filing submitted Q3 2016
Rocket 4 in process (not required for NDA filing)
• RoclatanTM: Mercury 1 and Mercury 2 completion
Mercury 3 commencement 1H 2017 (EU)
• Research Initiatives
• Rhopressa™ disease modification, neuroprotection, sustained release
• AR-13154 potential in wet AMD, etc.
• Evaluating Aerie’s 3,000+ proprietary molecules
• Business Development and Expansion Opportunities
• Drug delivery opportunities for front and back of the eye
• EU/JP clinical path and commercialization strategy
• Ireland Manufacturing Facility
• Fully Financed
14
2016 2017
RhopressaTM and RoclatanTM Key Milestones
Q3-2017: Roclatan™
P3 Mercury 1
Topline safety (12 mos)
Near YE 2017:
Roclatan™ NDA filing
expected
1H-2017: Roclatan™
P3 Mercury 3 (EU)
to be initiated
Q1-2016: Rhopressa™
Rocket 2 Topline safety (12 mos)
Q3-2016: Rhopressa™
NDA filed
Q4-2016: Rhopressa™
Rocket 4 Topline efficacy (3 mos)
Q3-2016: Roclatan™
P3 Mercury 1
Topline efficacy (3 mos)
Q2-2017: Roclatan™
P3 Mercury 2
Topline efficacy (3 mos)
Q1-2016: Roclatan™
P3 Mercury 2
initiated
15
15

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Public Device & Biopharma Ophthalmology Company Showcase - Aerie Pharmaceuticals

  • 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 3 registration 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, including the updated guidance presented herein, 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 topline Mercury 1 data presented herein is preliminary and based solely on information available to us as of the date of this document and additional information about the results may be disclosed at any time, including at our Investor Day on October 5, 2016. In addition, 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 Condition and Results of Operations.” Such forward-looking statements only speak as of the date they are made. We undertake no obligation to publicly update or revise any forward-looking statements, whether because of new information, future events or otherwise, except as otherwise required by law.
  • 3. 3 Aerie Late Stage IOP–Lowering Products Pre-Clinical Research • Rhopressa™ • Potential for disease modification and neuroprotection • AR-13154 • Significant lesion size reduction in wet AMD • Drug Delivery - Front and back of the eye • Rhopressa™ (netarsudil ophthalmic solution) 0.02% • Inhibits ROCK, NET, lowers EVP, targets diseased tissue • NDA filed Q3 2016, entering launch mode • Roclatan™ (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005% • Fixed combination of Rhopressa™ and latanoprost • Two P3’s in process; first P3 achieved primary efficacy endpoint Aerie – Building a Major Ophthalmic Pharmaceutical Company Data on file
  • 4. 4 Rhopressa™ Trials for Q3 2016 NDA Submission “Rocket 1” 90-Day Efficacy Registration Trial U.S.  Rhopressa™ 0.02% QD ~200 patients  timolol BID ~200 patients (Total enrollment: 411 patients) “Rocket 2” One Year Safety (3 Mo. Interim Efficacy) Registration Trial U.S.  Rhopressa™ 0.02% QD ~230 patients  Rhopressa™ 0.02% BID ~230 patients  timolol BID ~230 patients (Total enrollment: 756 patients) ClinicalTrials.gov Identifier: NCT02207491, NCT02207621
  • 5. 5 Rocket 2 and Rocket 1 Performance Maximum Baseline IOP <25 mmHg Rocket 2 Rocket 1 Data on file
  • 6. 6 Rocket 2: 12 Month 8am IOP Efficacy Safety Population, Completed Patients (<27 mmHg)  Difference in 8am mean IOP from Day 90 (Month 3) to Month 12: RhopressaTM QD:  Full population: +0.1 mmHg  Subset analyses: -0.3 to +0.3 mmHg Timolol BID:  Full population: +0.5 mmHg  Subset analyses: +0.1 to +1.4 mmHg Day 90 Month 12 AR-13324 0.02% q.d. (n=118) BL W2 W6 M3 M6 M9 M12 Data on file: AR-13324 – CS302
  • 7. 7 Roclatan™ Registration Trial Design “Mercury 1”* One Year Safety (3 Mo. Interim Efficacy) Registration Trial U.S. “Mercury 2”** 90-Day Efficacy Registration Trial U.S. and Canada “Mercury 3” 6 Mo. Efficacy and Safety Registration Trial Europe  Roclatan™ QD ~230 patients  Rhopressa™ 0.02% QD ~230 patients  latanoprost QD ~230 patients  Roclatan™ QD ~230 patients  Comparator (TBD) ~230 patients * ClinicalTrials.gov Identifier: NCT02558400 ** ClinicalTrials.gov Identifier: NCT02674854  Roclatan™ QD ~230 patients  Rhopressa™ 0.02% QD ~230 patients  latanoprost QD ~230 patients
  • 8. 8 Mercury 1: Study Design And Analysis • Trial design follows FDA requirement for fixed dose combination – Superiority of combination over each individual component – Statistically significant difference at each measured time point – Higher combo efficacy vs. components at ~1-3 mmHg, as previously accepted by FDA for product approval (SimbrinzaÂŽ) • The primary statistical modeling of the primary efficacy analysis was agreed with the FDA using the intent to treat (ITT) population with imputation for any missing data Data on file
  • 9. 9 RoclatanTM Achieved Statistical Superiority Over Individual Components at All Time Points Mean IOP at Each Time Point (ITT) ***p<0.0001 vs Latanoprost and RhopressaTM Data on file and PG324 - CS301 Top Line Data
  • 10. 10 Mercury 1: RoclatanTM Phase 3 Responder Analysis Day 90: % of Patients with IOP Reduced to 18 mmHg or Lower *** *** *** 14% 23% 32% 42% 54% 15% 25% 39% 54% 69% 33% 44% 61% 71% 82% 0% 20% 40% 60% 80% 100% ≤ 14 mmHg ≤ 15 mmHg ≤ 16 mmHg ≤ 17 mmHg ≤ 18 mmHg %ofPatients IOP on Treatment Rhopressa™ (n=198) Latanoprost (n=223) Roclatan™ (n=200) ### ### ***p<0.0001 vs Latanoprost and RhopressaTM ###p<0.0001 vs RhopressaTM, p<0.05 vs Latanoprost Data on file and PG324 - CS301 Top Line Data
  • 11. 11 Mercury 1: Roclatan™ Summary • Demonstrated superiority over both latanoprost and Rhopressa™ for the primary efficacy analysis at all 9 time points (p<0.0001) • IOP-lowering effect was greater (1-3 mmHg) than monotherapy with either latanoprost or Rhopressa™ throughout the duration of the study • There were no drug-related serious adverse events and was no evidence of treatment-related systemic effects • The main adverse event was conjunctival hyperemia, ~50% of patients and was scored as mild for ~80% of these patients Next Steps: • Mercury 2: Topline 3-month expected H1 2017 • Mercury 3 (Europe): Efficacy study, comparing to a leading combo product marketed in Europe, expected to commence in H1 2017 • RoclatanTM NDA filing expected near year-end 2017 Data on file and PG324 - CS301 Top Line Data
  • 12. 12 Mercury 1: Rhopressa™ Arm Performance Summary • Demonstrated non-inferiority to latanoprost in patients with baseline IOP < 25 mmHg • Efficacy relative to latanoprost improves as baseline IOP declines • RhopressaTM maintained consistent IOP lowering across all baseline IOPs including > 25 mmHg • Stable efficacy from week 2 to month 3 • Adverse event profile consistent with previous studies Data on file and PG324 - CS301 Top Line Data
  • 13. 13 Aerie Summary • Key Clinical Priorities • RhopressaTM: NDA filing submitted Q3 2016 Rocket 4 in process (not required for NDA filing) • RoclatanTM: Mercury 1 and Mercury 2 completion Mercury 3 commencement 1H 2017 (EU) • Research Initiatives • Rhopressa™ disease modification, neuroprotection, sustained release • AR-13154 potential in wet AMD, etc. • Evaluating Aerie’s 3,000+ proprietary molecules • Business Development and Expansion Opportunities • Drug delivery opportunities for front and back of the eye • EU/JP clinical path and commercialization strategy • Ireland Manufacturing Facility • Fully Financed
  • 14. 14 2016 2017 RhopressaTM and RoclatanTM Key Milestones Q3-2017: Roclatan™ P3 Mercury 1 Topline safety (12 mos) Near YE 2017: Roclatan™ NDA filing expected 1H-2017: Roclatan™ P3 Mercury 3 (EU) to be initiated Q1-2016: Rhopressa™ Rocket 2 Topline safety (12 mos) Q3-2016: Rhopressa™ NDA filed Q4-2016: Rhopressa™ Rocket 4 Topline efficacy (3 mos) Q3-2016: Roclatan™ P3 Mercury 1 Topline efficacy (3 mos) Q2-2017: Roclatan™ P3 Mercury 2 Topline efficacy (3 mos) Q1-2016: Roclatan™ P3 Mercury 2 initiated
  • 15. 15 15