2. 2
Aerie â Building a Major Ophthalmic
Pharmaceutical Company
â˘âŻ $4.5B US/EU/JP Market with significant unmet needs - growing to more
than $8B by 2023
â˘âŻ Multiple MOAs, once daily, high efficacy and safety
â˘âŻ Late-stage/potential blockbuster revenue opportunity
Large Market
Opportunity
â˘âŻ Inhibits ROCK and NET, targets diseased tissue
â˘âŻ Consistent IOP lowering, lowers Episcleral Venous Pressure
â˘âŻ Expect P3 efficacy data mid-2015, NDA filing mid-2016
Rhopressaâ˘
Triple Action
â˘âŻ Fixed combination of Rhopressa⢠and latanoprost
â˘âŻ P2b achieved all clinical endpoints, P3 start mid-2015
â˘âŻ Potentially most efficacious IOP-lowering therapy
Roclatanâ˘
Quadruple Action
â˘âŻ Full patent protection through at least 2030
â˘âŻ Plan to commercialize products ourselves in North America (and
potentially Europe) while partnering in Japan
All Products Fully
Owned by Aerie
3. 3
Accomplishments Since OIS in November 2013
â˘âŻ Completed IPO â raised $68M
â˘âŻ Reported successful Roclatan⢠Phase 2b results in June 2014
â˘âŻ Initiated Rhopressa⢠Phase 3 efficacy and safety studies in 3Q 2014
â˘âŻ Added key senior executives in 2014 for Commercial, R&D and
Business Development
â˘âŻ Recently announced additional $125M financing
â˘âŻ Initiated efforts to grow pipeline via internal R&D, in-license
and/or acquisition
4. 4
Rhopressa⢠Registration Trial Overview
â˘âŻ Primary efficacy endpoint: IOP at all time points through Day 90
â˘âŻ Non-inferiority design vs. timolol
wďˇâŻ 95% CI within 1.5 mmHg at all time points, within 1.0 mmHg at a majority of time
points
â˘âŻ Entry IOP: Over 20 mmHg and less than 27 mmHg
wďˇâŻ FDA agreed with Aerie proposal for entry IOPs with no expected impact on label
wďˇâŻ Rhopressa⢠non-inferior to latanoprost at entry IOPs of 22-26 mmHg in Phase 2b
â˘âŻ FDA discussions on Phase 3 design now complete
wďˇâŻ Combined trials to include approximately 1,300 total patients
wďˇâŻ 100 patients of 12 month safety data needed for NDA filing
5. 5
Rhopressa⢠Registration Trial Design
* PGAs have been shown to be less effective when dosed BID
âRocket 1â
90-Day Efficacy
Registration Trial
â˘âŻ Rhopressa⢠0.02% QD ~200 patients
â˘âŻ Timolol BID ~200 patients
âRocket 2â
One Year Safety
(3 Mo. Interim Efficacy)
Registration Trial
â˘âŻ Rhopressa⢠0.02% QD ~230 patients
â˘âŻ Rhopressa⢠0.02% BID* ~230 patients
â˘âŻ Timolol BID ~230 patients
âRocket 3â
One Year Safety
Registration Trial
Canada
â˘âŻ Rhopressa⢠0.02% QD ~90 patients
â˘âŻ Rhopressa⢠0.02% BID ~90 patients
â˘âŻ Timolol BID ~60 patients
6. 6
Roclatan⢠Phase 2b Clinical Trial Design
Phase 2b Protocol
Roclatan⢠0.01%
vs.
Roclatan⢠0.02%
vs.
Rhopressa⢠0.02%
vs.
Latanoprost
All Dosed QD PM
~300 Patients
28 Days
â˘âŻ Primary efficacy endpoint:
Mean diurnal IOP on Day 29
â˘âŻ Two concentrations of Roclatan⢠vs.
Rhopressa⢠0.02% and latanoprost
â˘âŻ Trial design follows FDA requirement for
fixed-dose combination
wďˇâŻ Statistically significant difference at
measured time points
wďˇâŻ Higher combo efficacy vs. components of
at least 1â3 mmHg, as previously
accepted by FDA for product approval
7. 7
Mean IOP at Each Time Point â Primary Efficacy Measure
0.02% Roclatan⢠Achieved Statistical Superiority Over
Individual Components at All Time Points (p<0.001)
Roclatan⢠Phase 2b, Intent to Treat
14
15
16
17
18
19
20
21
22
23
24
25
26
27
28
29
Pre- 8AM 8AM 10AM 4PM 8AM 10AM 4PM 8AM 10AM 4PM 8AM 10AM 4PM 8AM
Study Qual 1 Baseline Day 8 Day 15 Day 29 Day 30
mmHg+/-SEM
0.02% Rhopressa⢠(n=78) 0.005% Latanoprost (n=73) 0.02% Roclatan⢠(n=72)
9. 9
Roclatan⢠Phase 2b Responder Analysis:
Goal is to Achieve Lowest IOP Possible
10%
21% 25%
40%
8%
18%
29%
47%
38%
46%
57%
69%
0%
20%
40%
60%
80%
100%
⤠15 mmHg ⤠16 mmHg ⤠17 mmHg ⤠18 mmHg
%ofSubjects
Reduction
0.02% Rhopressa⢠(n=78) 0.005% Latanoprost (n=73) 0.02% Roclatan⢠(n=72)
Day 29 â % of Subjects with IOP Reduced to < 18 mmHg
10. 10
Asymptomatic, Transient, Self Limiting
Most Commonly Observed AE in Roclatanâ˘
Phase 2b was Conjunctival Hyperemia
40%
0%
20%
40%
60%
80%
100%
Roclatan
80% of Hyperemia Was Rated as Mild by Biomicroscopy