![]() Leading Innovation in Glaucoma The Next Generation Company Overview February 26, 2014 Exhibit 99.1 |
![]() 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 AR-13324 product candidate relate to the results in our Phase 2 clinical trials. Our product candidate PG324 has only been tested in preclinical animal models. 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. 2 Important Information 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 our prospectus filed with the SEC on October 28, 2013 and 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 Aerie – Next Generation in Glaucoma Therapies AR-13324 Novel Dual Action All Products Fully Owned by Aerie PG324 Breakthrough Triple Action • Fixed combination of AR-13324 and latanoprost • Potential for maximal IOP lowering • Expect P2b results mid-2014, P3 readiness mid-2015 • Inhibits ROCK and NET, targets diseased tissue • Consistent IOP lowering, may lower EVP • Expect P3 efficacy data mid-2015, NDA filing mid-2016 Large Market Opportunity • $4.5B US/EU/JP Market with significant unmet needs • Multiple MOAs, once daily, high efficacy and safety • Late-stage/potential blockbuster revenue opportunity • Full patent protection through at least 2030 • Will retain US rights and plan to partner in JP/EU |
![]() 4 Currently Prescribed Glaucoma Therapies Once Daily 2-3 Times Daily Non-PGA Market PGA: Prostaglandin Analogue; BB: Beta Blocker; AA: Alpha Agonist; CAI: Carbonic Anhydrase Inhibitor US Glaucoma Market IMS TRx data, FY 2012 PGA Market Bimatoprost, 11% Travoprost, 12% Latanoprost, 27% BB, 15% BB Fixed Combo, 14% AA, 14% CAI, 6% |
![]() Largest Rx market in ophthalmology - US 28.5M Rx; annual US sales $1.9B ($4.5B US/EU/JP) 2.2M glaucoma patients in the US expected to grow to over 3M by 2020 ~50% of patients use multiple medications to control disease No drugs with new MOA launched in 20 years None of the existing drugs target the diseased tissue, or Trabecular Meshwork (primary drain), or lower EVP None of the existing drugs have shown consistent efficacy across a broad range of pressures Significant Glaucoma Market Opportunities 1 National Eye Institute 1 5 |
![]() Inflow (fluid production) AA, BB, CAI PGAs decrease increase increase Aerie’s Novel Products Address All IOP Control Mechanisms and May Lower EVP Outflow Secondary Drain (Uveoscleral) Outflow Primary Drain (Trabecular Meshwork) 6 Triple Action PG324 Dual Action AR-13324 |
![]() 7 The Competitive Landscape Clinical Efficacy Dosing/ Day Tolerability Peak Product Sales* (US/EU) Targeting Diseased Tissue PGA High 1x Hyperemia Iris Color Change Other Cosmetic $1.7B No Beta Blocker Moderate 2x Cardiopulmonary Contraindications >$500M No CAI Low 2 - 3x Sulfonamide Contraindication Bitter Taste >$500M No Alpha Agonist Low 2 - 3x Allergy Drowsiness >$400M No AR-13324 High; Consistent 1x Hyperemia Yes PG324 Potentially Highest 1x TBD Yes * Peak sales for best-in-class franchise |
![]() 8 New PGAs - not usable as add-on to current PGAs Glaucoma Competitors in Pipeline AR-13324 is the only new MOA drug dosed once-daily AR -13324 (Aerie) ROCK/NET inhibitor (qd) Phase 2b K-115 (Kowa) ROCK inhibitor (bid) Phase 3 AMA0076 (Amakem) ROCK inhibitor (bid) Phase 2a INO-8875 (Inotek) Adenosine-A1 agonist (bid) Phase 2 OPA-6566 (Acucela) Adenosine-A2a receptor Phase 1/2 SYL040012 (Sylentis) beta Phase 2 New MOAs RNAi blocker (bid) (Japan) New PGAs 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 (bid) |
![]() 9 Once-daily, consistent IOP lowering 1 drug - 2 MOAs, targets diseased tissue, and may lower EVP Well tolerated; no systemic drug-related AEs Efficacy expected to be > current drugs for majority of patients Once-daily Combination of 2 drugs - 3 MOAs, and may lower EVP Well tolerated; no systemic drug-related AEs Efficacy expected to be superior to all current drugs AR-13324 Dual-Action PG324 Triple-Action Future drug of choice for 80% of patients - IOP <26mmHg Future drug of choice for patients requiring maximal IOP lowering Aerie Franchise: Highly Differentiated Product Profiles |
![]() 10 AR-13324 AR-13324 NET RKI Trabecular Meshwork 1. ROCK inhibition relaxes TM, increases outflow 2. NET inhibition reduces fluid production 3. ROCK inhibition may lower Episcleral Venous Pressure (EVP) Episcleral Veins Schlemm’s Canal Mechanisms of Action: NET RKI Cornea Ciliary Processes Uveoscleral Outflow |
![]() 11 AR-13324 Demonstrated Strong IOP Lowering in Phase 2b 13 glaucoma NCEs advanced from Phase 2 to Phase 3 since 1970s - All approved Once-daily PM dosing of 0.02% AR-13324 is highly effective - IOP -5.7 and -6.2 mmHg on D28 and D14 - Consistent efficacy through D28 AR-13324 efficacy results within ~1 mmHg of latanoprost Favorable tolerability profile No systemic side effects AR-13324 Efficacy at 8 AM Diurnal Average IOP - Entry IOP 22-36 mmHg (n=221) |
![]() AR-13324 and latanoprost clinically and statistically equivalent in patients with moderately elevated IOPs of 22 - 26 mmHg Latanoprost loses ~1 mmHg efficacy AR-13324 maintains consistent efficacy Latanoprost does not target the diseased tissue – AR-13324 does Latanoprost 0.02% AR-13324 Latanoprost 0.02% AR-13324 Phase 2b baseline IOP entry requirements: 24, 22, 22 mmHg (8am, 10am, 4pm) Full Patient Population Moderately Elevated IOP 12 Differentiated AR-13324 Efficacy Profile Informs Phase 3 Study Design Baseline 22 – 36 mmHg (n=221) Baseline 22 – 26 mmHg (n=106) |
![]() 13 New MOA May Represent Breakthrough Recently reported study confirms AR-13324 lowered EVP by 35% in a preclinical in vivo model 2 1 Aerie Phase 1 PK Study Press Release dated January 9, 2014 2 Aerie Press Release dated February 25, 2014 AR-13324 lowered average IOP by over 30% - from 16 to 11 mmHg 1 Currently available drugs are typically less effective at lower IOPs Consistent efficacy of AR-13324 across baseline IOPs may be explained by lowering of EVP High efficacy of AR-13324 in normotensive volunteers with average IOP of 16 mmHg also supports lowering of EVP No currently marketed products are known to lower EVP, which contributes up to half of IOP in normotensive subjects |
![]() 10,444 individuals were screened for the prevalence of Primary Open-Angle Glaucoma (POAG) 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 14 Baseline IOP (mmHg) Percentage of POAG Patients Identified Cumulative Percentage < 15 13% 13% 16-18 24% 37% 19-21 22% 59% 22-24 19% 78% 25-29 10% 88% 30-34 9% 97% > 35 3% 100% ~80% of Glaucoma IOPs Are < 26 mmHg at Time of Diagnosis |
![]() Latanoprost and timolol lose 0.5 mmHg efficacy for every 1 mmHg drop in baseline IOP Timolol less effective than latanoprost at all baselines AR-13324 equivalent/ non-inferior to latanoprost at baselines 22 – 26 mmHg Timolol is the standard comparator for glaucoma Phase 3 trials Pooled data from three latanoprost registration studies. Hedman and Alm; European Journal Ophthalmology;2000 0 -2 -4 -6 -8 -10 -12 -14 -16 Timolol (n = 369) Latanoprost (n = 460) 16 18 20 22 24 26 28 30 32 34 36 38 Untreated diurnal IOP (mmHg) Latanoprost and Timolol Show Reduced Efficacy at Lower Baseline IOPs 15 |
![]() AR-13324 Registration Trial Design Primary efficacy endpoint: IOP at all time points through Day 90 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 Planned entry IOP: Minimum 21 mmHg, maximum 26 mmHg - FDA has agreed to Aerie proposal for entry IOPs with no expected impact on label - AR-13324 non-inferior to latanoprost at entry IOPs of 22-26 mmHg in Phase 2b Phase 3 Protocol AR-13324 0.02% dosed QD PM vs. Timolol dosed BID ~1200 patients 90 days efficacy 100+ patients for 1 year safety 16 |
![]() 17 AR - 13324 Summary Potential drug of choice for 80% of patients – those with IOPs < 26mmHg Once daily, high efficacy, consistent IOP lowering, good tolerability and safety profile through Phase 2 The only drug to target the diseased tissue, with added MOAs of lowering fluid production and potentially lowering EVP Next Steps: Phase 3 expected to commence in mid-2014 Phase 3 90-day efficacy and short-term safety results expected mid-2015 NDA filing expected by mid-2016 |
![]() AR-13324 FIXED COMBINATION WITH LATANOPROST AR-13324 MOAs: NET RKI 18 TM Outflow Ciliary Processes Uveoscleral Outflow Latanoprost Cornea 1. ROCK inhibition restores TM outflow 2. NET inhibition reduces fluid production 3. ROCK inhibition may lower Episcleral Venous Pressure 4. PGA receptor activation increases uveoscleral outflow Triple-Action PG324 NET RKI |
![]() First product to lower IOP through all three known mechanisms, and also potentially lower EVP - Once-daily: 1 drop, 2 drugs, 3 MOAs High efficacy in predictive primate model Human proof of concept established in prior ROCKi/PGA combination trials - Demonstrated significant IOP lowering beyond PGA alone Dose Dose Dose PG324 vs. Latanoprost, Primates (n=6/group) Latanoprost 0.02% PG324 19 Day 2 - No IOP measured PG324: Triple-Action Fixed Combination |
![]() Primary efficacy endpoint: Mean diurnal IOP on Day 28 Two concentrations of PG324 vs. AR-13324 0.02% and latanoprost Trial design similar to registration trial for fixed-dose combination - 1-3 mmHg superiority vs. components previously accepted by FDA 20 PG324 Phase 2b Clinical Trial Design Phase 2b Protocol PG324 0.01% vs. PG324 0.02% vs. AR-13324 0.02% vs. Latanoprost All dosed QD PM ~300 patients 28 days |
![]() Potential for use by patients requiring maximal IOP lowering and/or patients with advanced disease state Potential to be the most efficacious IOP-lowering drug in glaucoma with at least three and potentially four MOAs Expected to be the first PGA fixed combination product in the US, and dosed as a single drop once daily Next Steps: Phase 2b first patient dosed early 1Q 2014 Phase 2b results expected mid-2014 Phase 3 preparatory work commences second half of 2014 Phase 3 readiness expected mid 2015 PG324 Summary 21 |
![]() H1 2014 H2 2014 H2 2015 H1 2015 H1 2016 22 Key Milestones Financed by IPO Proceeds Early-2014 : PG324 Start Phase 2b clinical trial Mid-2014 : AR-13324 Start Phase 3 registration trials Mid-2015 AR-13324 Efficacy results from Phase 3 expected Mid-2016 AR-13324 NDA filing expected Mid-2014: PG324 Results from Phase 2b expected Mid-2015 : PG324 Phase 3-prep : : |
![]() Financial Summary AR-13324 Phase 3 registration trials Direct clinical - $25.0M Non-clinical - $12.4M PG324 Phase 2b clinical trial Direct clinical - $2.5M Non-clinical - $1.0M Phase 3 enabling activities Non-clinical - $6.3M G&A and working capital - $20.8M Use of IPO Proceeds: $68M through Mid-2016 Additional PG324 Needs: $40M through Mid-2017 PG324 Phase 3 registration trials Direct clinical - $27.5M 12 Months G&A and working capital - $12.5 23 Targeted Mid-2017: Expected NDA Approval for AR - 13324 Expected NDA Filing for PG324 |
![]() Market Cap (dollars in millions) 24 Stock & Market Cap Performance 10/31/13 - $243.7 12/31/13 - $418.2 2/21/14 - $490.6 |
![]() AR-13324 Novel Dual Action All Products Fully Owned by Aerie PG324 Breakthrough Triple Action • Fixed combination of AR-13324 and latanoprost • Potential for maximal IOP lowering • Expect P2b results mid-2014, P3 readiness mid-2015 • Inhibits ROCK and NET, targets diseased tissue • Consistent IOP lowering, may lower EVP • Expect P3 efficacy data mid-2015, NDA filing mid-2016 Large Market Opportunity • $4.5B US/EU/JP Market with significant unmet needs • Multiple MOA’s, once daily, high efficacy and safety • Late-stage/potential blockbuster revenue opportunity • Full patent protection through at least 2030 • Will retain US rights and plan to partner in JP/EU 25 Aerie – Next Generation in Glaucoma Therapies |