 Aerie Pharmaceuticals, Inc. Company Overview November 18-19, 2014 Building a Major Ophthalmic Pharmaceutical Company Exhibit 99.1 |
 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 data results for our Rhopressa ™ and Roclatan ™ product candidates relate to the results in our Phase 2 clinical trials. 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, including beliefs, expectations, estimates, projections and statements relating to our business plans, prospects and objectives, and the assumptions upon which those statements are based. Words such as “may,” “will,” “should,” “would,” “could,” “believe,” “expects,” “anticipates,” “plans,” “intends,” “estimates,” “targets,” “projects” 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. 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 Operation.” 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 Rhopressa TM Triple Action All Products Fully Owned by Aerie Roclatan TM Quadruple Action • Fixed combination of Rhopressa TM and latanoprost • P2b achieved all clinical endpoints, P3 start mid-2015 • Potentially most efficacious IOP-lowering therapy • Inhibits ROCK and NET, targets diseased tissue • Consistent IOP lowering, lowers Episcleral Venous Pressure • Expect P3 efficacy data mid-2015, NDA filing mid-2016 Large Market Opportunity • $4.5B US/EU/JP Market with significant unmet needs - growing to more than $8B by 2023 • Multiple MOAs, once daily, high efficacy and safety • Late-stage/potential blockbuster revenue opportunity • Full patent protection through at least 2030 • Plan to commercialize products ourselves in North America (and potentially Europe) while partnering in Japan • Grow pipeline via R&D efforts, in-license and/or acquisition Aerie – Building a Major Ophthalmic Pharmaceutical Company |
 4 • Largest Rx market in ophthalmology: $4.5B US/EU/JP* • US Glaucoma patients: 2.7M growing to 4.3M by 2030 1 • Patients on more than one drug to control disease: 50% No commonly prescribed drugs: • target the diseased tissue • relax the main fluid drain and lower EVP • show consistent efficacy across a broad range of pressures * IMS 2013 1 National Eye Institute Glaucoma Market Unmet Needs Expanding Market |
 5 FY 2013 U.S. Glaucoma Market = $2.0B; 31.5M TRx Market Share in TRx Current Product Dashboard PGA: Prostaglandin Analogue; BB: Beta Blocker; AA: Alpha Agonist; CAI: Carbonic Anhydrase Inhibitor Once Daily 2-3 Times Daily Bimatoprost Travoprost Latanoprost BB BB Fixed Combo AA CAI 10% 10% 31% 15% 14% 10% 8% Non-PGA Market PGA Market |
 6 New PGAs: not usable as add-on to current PGAs Rhopressa TM and Roclatan TM : advanced triple and quadruple MOAs Glaucoma Competitors in Pipeline * Approved in Japan 9/29/2014 New MOAs Rhopressa TM (Aerie AR-13324 ) ROCK/NET inhibitor (qd) Phase 3 Roclatan TM (Aerie PG324 ) ROCK/NET inhibitor + PGA (qd) Phase 3 K-115 (Kowa ) ROCK inhibitor (bid) Approved in Japan* Adjunctive Therapy AMA0076 (Amakem) ROCK inhibitor (bid) Phase 2a INO-8875 (Inotek) Adenosine -A1 agonist (bid) Phase 2 OPA-6566 (Acucela ) Adenosine -A2a agonist (bid) Phase 1/2 SYL040012 (Sylentis) RNAi beta blocker (qd) Phase 2 BOL -303259 (B+L) NO donating latanoprost (qd) Phase 3 DE-117 (Santen) EP2 agonist (qd) Phase 2a ONO-9054 (Ono) FP/EP3 agonist (qd) Phase 1 New PGAs |
 7 2023 U.S. Glaucoma Market and Market Share Projections* Projected Market Share in TRx PGA: Prostaglandin Analogue; BB: Beta Blocker; AA: Alpha Agonist; CAI: Carbonic Anhydrase Inhibitor Data Source: IMS 2013 FY TRX. Internal projection *Projected market shares compiled by an independent market research company U.S. Glaucoma Market = $4.9B; 41.7 M TRx TRx Growth 2013-2023 = 2.3% Roclatan™ + Rhopressa™ May Capture Over 40% Market Share Based on 200 U.S. Physician Responses to Survey PGA’s BB BB Fixed Combo AA CAI Rhopressa™ Roclatan™ 31% 18% 25% 6% 9% 5% 5% |
 8 increase Decreases Fluid Inflow/Production (Ciliary Processes) Increases Fluid Outflow: Secondary Drain (Uveoscleral Pathway) Increases Fluid Outflow: Primary Drain- Lowers EVP - Rhopressa™ Roclatan™ AA, BB, CAI PGAs Aerie Products Cover the IOP-lowering Spectrum Trabecular Meshwork(TM); (Episcleral Venous Pressure) |
 Aerie Product Market Positioning* * Confirmed by Market Research Future drug of choice for the 80% of patients with IOP of 26 mmHg or less Triple-Action Rhopressa™ Future product of choice for patients requiring maximal IOP lowering Quadruple-Action Roclatan™ 9 Also for PGA users as add-on therapy Also for PGA non-responders and those with tolerability concerns Also for patients with low-tension glaucoma Efficacy potentially greater than all currently marketed drugs For patients with IOPs above 26 mmHg Also for patients at any IOP with significant disease progression |
 10 Rhopressa ™ NET RKI Trabecular Meshwork Triple-Action Rhopressa™ Episcleral Veins Schlemm’s Canal Ciliary Processes Cornea Uveoscleral Outflow ROCK inhibition relaxes TM, increases outflow NET inhibition reduces fluid production ROCK inhibition lowers Episcleral Venous Pressure (EVP) IOP-Lowering Mechanisms NET RKI |
 11 Rhopressa™: Powerful Compound for Physiological Lowering of IOP – Phase 2b Results Once-daily PM dosing of 0.02% Rhopressa™ is highly effective IOP -5.7 and -6.2 mmHg on D28 and D14 Rhopressa™ efficacy results within ~1 mmHg of latanoprost Favorable tolerability profile with no systemic side effects Mean Diurnal IOP – Entry IOP 22-36 mmHg (n=221) 25.6 19.5 20.0 25.5 18.4 18.7 13 18 23 28 Baseline Day 14 Day 28 Rhopressa™ 0.02% (n=71) Latanoprost (n=76) |
 12 Rhopressa ™ Differentiated Efficacy Profile Phase 2b baseline IOP entry requirements: 24, 22, 22 mmHg (8 AM, 10 AM, 4 PM) Rhopressa™ and latanoprost clinically and statistically equivalent in patients with moderately elevated IOPs of 22-26 mmHg Latanoprost loses ~1 mmHg efficacy @ baselines of 22-26 mmHg Rhopressa™ maintains consistent efficacy Rhopressa™; 1 st product to treat the diseased tissue (trabecular meshwork) with a once-daily (QD) dose Baseline: 22 – 36 mmHg (n=221) Baseline: 22 – 26 mmHg (n=106) -5.7 -6.8 -8 -6 -4 -2 0 0.02% Rhopressa™ Latanoprost -5.8 -5.9 -8 -6 -4 -2 0 0.02% Rhopressa™ Latanoprost |
 13 Sustained Effect of Rhopressa™ vs. Latanoprost * 36 hours post dose 8 AM Mean IOP (mmHg) by Treatment Group 0.02% Rhopressa ™ Roclatan™ Phase 2b 0.02% Rhopressa ™ Rhopressa™ Phase 2b 0.005% latanoprost Rhopressa™ Phase 2b (n = 78) (n = 71) (n = 76) Baseline 26.6 27.2 26.8 Day 8 20.0 21.1 20.0 Day 29 20.3 21.2 19.2 Day 30* 21.0 22.2 22.4 Rhopressa™ Duration is Superior to Latanoprost 36 Hours After Last Dose |
 14 Rhopressa™ EVP-Lowering Breakthrough Phase 2b data provided first sign of EVP-lowering: Phase 1 study in low baseline IOP subjects: Preclinical in vivo study: Note: Timolol and latanoprost reported to have no effect or to increase EVP in animal models Consistent Efficacy Across Baseline IOPs Lowered Average IOP by Over 30% From 16 Down to 11 mmHg Lowered EVP by 35% |
 15 Baseline IOP* ~80% of U.S. Glaucoma Patients Have IOPs that are 26 mmHg at Time of Diagnosis The Baltimore Eye Survey * Sommer A, Tielsch JM, Katz J et al. Relationship between intraocular pressure and primary open angle glaucoma among white and black Americans: The Baltimore eye survey. Arch Ophthalmol 1991;109:1090-1095 ** IWASE et al Tajimi study group. Japan Glaucoma Society. Ophthalmology, 2004 Sep, 111 (9): 1641-8. 21 mmHg (Normal Tension Glaucoma) >21 - 26 mmHg >26 - <35 mmHg 10,444 Individuals Were Screened for the Prevalence of Primary Open-Angle Glaucoma (POAG) and the IOP at Time of Diagnosis 92% of Japanese Patients with POAG, IOPs Were 21 mmHg** 60% 20% 20% |
 16 Latanoprost and Timolol Show Reduced Efficacy at Lower Baseline IOPs Pooled data from three latanoprost registration studies. Hedman and Alm; European Journal Ophthalmology;2000 Latanoprost and timolol lose efficacy as baseline IOPs decline Timolol at least 1 mmHg less effective than latanoprost across all published baselines Rhopressa™ equivalent/ non-inferior to latanoprost at baselines 22–26 mmHg Timolol is the standard comparator for glaucoma Phase 3 trials -16 -14 -12 -10 -8 -6 -4 -2 0 16 18 20 22 24 26 28 30 32 34 36 38 Untreated Diurnal IOP (mmHg) Timolol (n=369) Latanoprost (n=460) |
 17 Rhopressa™ Registration Trial Overview Primary efficacy endpoint: IOP at nine time points through Day 90 Phase 3 entry IOP is >20 mmHg and <27 mmHg Non-inferiority design vs. timolol 95% CI within 1.5 mmHg at all time points, within 1.0 mmHg at a majority of time points Combined trials to include approximately 1,300 total patients 100 patients with 12 months of safety data needed for NDA filing Should meet efficacy requirements for EMA filing 300 patients with 6 months safety data needed for EMA filing and 100 with 12 months |
 18 Rhopressa™ Registration Trial Design * PGAs have been shown to be less effective when dosed BID “Rocket 1” 90-Day Efficacy Registration Trial Rhopressa™ 0.02% QD ~200 patients Timolol BID ~200 patients “Rocket 2” One Year Safety (3 Mo. Interim Efficacy) Registration Trial “Rocket 3” One Year Safety Registration Trial Canada Rhopressa™ 0.02% QD ~230 patients Rhopressa™ 0.02% BID * ~230 patients Timolol BID ~230 patients Rhopressa™ 0.02% QD ~90 patients Rhopressa™ 0.02% BID ~90 patients Timolol BID ~60 patients |
 19 ROCK inhibition relaxes TM, increases outflow NET inhibition reduces fluid production ROCK inhibition lowers EVP PGA receptor activation increases uveoscleral outflow Rhopressa ™ Quadruple-Action Roclatan™ Fixed Combination of Rhopressa™ with Latanoprost IOP-Lowering Mechanisms Ciliary Processes Latanoprost Cornea Uveoscleral Outflow NET RKI NET RKI Trabecular Meshwork Episcleral Veins Schlemm’s Canal |
 20 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 Primary efficacy endpoint: Mean diurnal IOP on Day 29 Two concentrations of Roclatan™ vs. Rhopressa™ 0.02% and latanoprost Trial design follows FDA requirement for fixed-dose combination Statistically significant difference at measured time points Higher combo efficacy vs. components of at least 1–3 mmHg, as previously accepted by FDA for product approval |
 21 Roclatan™ Phase 2b Clinical Trial Performance Achieved primary efficacy measure Superiority over each of the components on day 29 Achieved statistical superiority over the individual components at all time points More efficacious than latanoprost by 1.6 – 3.2 mmHg More efficacious than Rhopressa™ by 1.7 – 3.4 mmHg Main adverse event was hyperemia (eye redness): Reported in 40 percent of patients Mild for the large majority of patients No systemic drug-related adverse events |
 22 Mean IOP at Each Time Point 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 0.02% Rhopressa™ (n=78) 0.005% Latanoprost (n=73) 0.02% Roclatan™ (n=72) |
 23 Roclatan™ Phase 2b, ITT Mean IOP (mmHg) * Difference between 0.02% Roclatan™ and latanoprost or Rhopressa™ Roclatan: Produced lowest IOP drop in any trial Was superior to latanoprost by 1.6–3.2 mmHg (p<0.001) Was superior to Rhopressa™ by 1.7–3.4 mmHg (p<0.001) Impressive Rhopressa™ performance Day 8 8 AM 19.6 -2.6 20.0 -3.1 10 AM 18.3 -2.7 18.0 -2.4 4 PM 18.6 -3.1 17.9 -2.3 Day 15 8 AM 19.6 -3.2 19.6 -3.1 10 AM 18.3 -2.4 18.7 -2.8 4 PM 18.3 -2.6 18.4 -2.7 Day 29 8 AM 19.2 -2.4 20.3 -3.4 10 AM 17.7 -1.8 18.6 -2.7 4 PM 18.4 -1.6 18.5 -1.7 Mean Mean Difference* Mean Difference* 17.0 15.6 15.6 16.5 15.8 15.7 16.9 15.9 16.8 0.02% Roclatan ™ (n = 72) 0.005% latanoprost (n = 73) 0.02% Rhopressa ™ (n = 78) |
 24 Day 29 – % of Patients with IOP Reductions of 20% Roclatan™ Phase 2b Responder Analysis: Goal is to Achieve Lowest IOP Possible 9% 17% 24% 45% 66% 11% 28% 46% 65% 81% 0% 20% 40% 60% 80% 100% 40% 35% 30% 25% 20% Reduction 0.02% Rhopressa™ (n=78) 0.005% Latanoprost (n=73) 0.02% Roclatan™ (n=72) 32% 50% 63% 81% 93% |
 25 Roclatan™ Phase 2b Responder Analysis: Goal is to Achieve Lowest IOP Possible Day 29 – % of Subjects with IOP Reduced to 18 mmHg 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 Reduction 0.02% Rhopressa™ (n=78) 0.005% Latanoprost (n=73) 0.02% Roclatan™ (n=72) |
 26 Diurnal IOP in Subset Of High Responders 16 mmHg Roclatan™ Phase 2b High Responders: Consistent IOP Drop by Rhopressa™ and Roclatan™ -9.6 -7.2 -9.9 -9.4 -8.2 -10.6 -9.5 -9.5 -10.2 -12 -11 -10 -9 -8 -7 -6 -5 -4 -3 -2 -1 0 0.02% Rhopressa™ (n=16) 0.005% Latanoprost (n=13) 0.02% Roclatan™ (n=31) Day 8 Day 15 Day 29 |
 27 2014 2015 2016 2017 2018 Key Future Milestones June 2014: Roclatan™ Phase 2b clinical trial completed Mid-2015: Roclatan™ Phase 3 initiated Mid-2016: Roclatan™ Efficacy results from Phase 3 expected Mid-2017: Roclatan™ NDA filing expected 2H 2018: Roclatan™ Launch expected 3Q 2014: Rhopressa™ Phase 3 initiated Mid-2015: Rhopressa™ Efficacy results from Phase 3 expected Mid-2016: Rhopressa™ NDA filing expected 2H 2017: Rhopressa™ Launch expected |
 28 Financing the Future Sufficient Capital to Fund: $190+ Million Raised From IPO (10/13) and Follow on Financing (9/14) FDA approval and launch of Rhopressa™ and Roclatan™ Building of Aerie’s pipeline via internal discovery, licensing and M&A |
 Building a Major Ophthalmic Pharmaceutical Company |