Aerie Pharmaceuticals, Inc. Company Overview May 12, 2014 Leading Innovation in Glaucoma The Next Generation Exhibit 99.2 |
2 Important Information 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™ product candidate relate to the results in our Phase 2 clinical trials. Our product candidate Roclatan™ is currently in 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 Aerie – Next Generation in Glaucoma Therapies Rhopressa TM Triple Action All Products Fully Owned by Aerie Roclatan TM Quadruple Action • Fixed combination of Rhopressa TM and latanoprost • Potential for maximal IOP lowering • Expect P2b results late June or early July 2014, P3 readiness mid-2015 • Inhibits ROCK and NET, targets diseased tissue • Consistent IOP lowering, lowers 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 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 Glaucoma Market Glaucoma Market 1 National Eye Institute show consistent efficacy across a broad range of pressures Expanding Market Unmet Needs |
5 increase Decreases Fluid Inflow/Production (Ciliary Processes) Aerie Covers the IOP-lowering Spectrum Aerie Covers the IOP-lowering Spectrum Increases Fluid Outflow: Secondary Drain (Uveoscleral Pathway) Increases Fluid Outflow: Primary Drain (Trabecular Meshwork); Lowers EVP (Episcleral Venous Pressure) Rhopressa™ Rhopressa™ Roclatan™ Roclatan™ AA, BB, CAI AA, BB, CAI PGAs PGAs |
Once Daily 2-3 Times Daily Non-PGA Market PGA: Prostaglandin Analogue; BB: Beta Blocker; AA: Alpha Agonist; CAI: Carbonic Anhydrase Inhibitor PGA Market FY 2013 US Glaucoma Market = $2.0B; 31.5M TRx Market Share in TRx Current Product Dashboard Bimatoprost, 11% Travoprost, 10% Latanoprost, 32% BB, 15% BB Fixed Combo, 14% AA, 10% CAI, 8% 6 |
7 Aerie Competitive Advantage Aerie Competitive Advantage Clinical Efficacy Clinical Efficacy Doses Doses Per Per Day Day Tolerability Tolerability Peak Product Peak Product Sales* Sales* Targeting Targeting Diseased Diseased Tissue Tissue Rhopressa TM High; Consistent 1 Hyperemia Yes Roclatan TM Potentially Highest 1 TBD Yes PGA High 1 Hyperemia Iris Color Change Other Cosmetic $1.7B No Beta Blocker Moderate 2 Cardiopulmonary Contraindications >$500M No CAI Low 2 - 3 Sulfonamide Contraindication Bitter Taste >$500M No Alpha Agonist Low 2 - 3 Allergy Drowsiness >$400M No * Peak worldwide sales for best-in-class franchise. |
8 New MOAs AR-13324 (Aerie) ROCK/NET inhibitor (qd) Phase 2b K-115 (Kowa) ROCK inhibitor (bid) Phase 3 (Japan) 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 (bid) Phase 2 New PGAs - not usable as add-on to current PGAs Glaucoma Competitors in Pipeline Rhopressa TM is the only new MOA product dosed once-daily 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 |
9 Future drug of choice for the 80% of patients with IOP of 26 mmHg or less Future product of choice for patients requiring maximal IOP lowering Triple-Action Rhopressa TM Quadruple-Action Roclatan TM Aerie Market Positioning 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 superior to all currently marketed drugs For patients with IOPs above 26 mmHg Also for patients at any IOP with significant disease progression |
10 Triple-Action Rhopressa TM TM Cornea Uveoscleral Outflow Rhopressa Trabecular Meshwork 1. ROCK inhibition relaxes TM, increases outflow 2. NET inhibition reduces fluid production 3. ROCK inhibition lowers EVP Episcleral Veins Schlemm’s Canal Mechanisms of Action: RKI NET Ciliary Processes RKI NET RKI TM |
11 13 glaucoma NCEs advanced from Phase 2 to Phase 3 since 1970s - All approved Once-daily PM dosing of 0.02% Rhopressa TM is highly effective - IOP -5.7 and -6.2 mmHg on D28 and D14 - Consistent efficacy through D28 Rhopressa TM efficacy results within ~1 mmHg of latanoprost Favorable tolerability profile No systemic side effects Rhopressa TM Efficacy 8 AM Diurnal Average IOP - Entry IOP 22-36 mmHg (n=221) Rhopressa 0.01% (n=74) Rhopressa 0.02% (n=71) Latanoprost (n=76) Rhopressa TM : Strong IOP Lowering in Phase 2b TM Rhopressa 0.02% q.d. TM TM |
Rhopressa TM and latanoprost clinically and statistically equivalent in patients with moderately elevated IOPs of 22 - 26 mmHg Latanoprost loses ~1 mmHg efficacy Rhopressa TM maintains consistent efficacy Latanoprost does not target the diseased tissue – Rhopressa TM does Phase 2b baseline IOP entry requirements: 24, 22, 22 mmHg (8am, 10am, 4pm) Differentiated Rhopressa TM Efficacy Profile Latanoprost Baseline 22 – 26 mmHg (n=106) Latanoprost 0.02% Rhopressa™ Baseline 22 – 36 mmHg (n=221) Full Patient Population Moderately Elevated IOP 0.02% Rhopressa™ 12 |
13 Phase 2b data provided first sign of EVP-lowering: Phase 1 study in low baseline IOP subjects: Preclinical in vivo study: Note: EVP contributes up to half of IOP in normotensive subjects Consistent efficacy across baseline IOPs Lowered average IOP by over 30% from 16 to 11 mmHg Lowered EVP by 35% Rhopressa ™ EVP-Lowering Breakthrough |
14 10,444 individuals were screened for the prevalence of Primary Open-Angle Glaucoma (POAG) ~80% of Glaucoma IOPs Are 26 mmHg at Time of Diagnosis 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 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% |
15 Latanoprost and Timolol Show Reduced Efficacy Latanoprost and Timolol Show Reduced Efficacy at Lower Baseline IOPs at Lower Baseline IOPs 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 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) ™ |
16 Rhopressa TM Registration Trial Overview 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: Over 20 mmHg and less than 27 mmHg - FDA has agreed to Aerie proposal for entry IOPs with no expected impact on label - Rhopressa TM non-inferior to latanoprost at entry IOPs of 22-26 mmHg in Phase 2b • FDA discussions on Phase 3 design now complete - Combined trials to include approximately 1,300 total patients - 100 patients of 12 month safety data needed for NDA filing |
17 Rhopressa TM Registration Trial Design “Rocket 1” 90-Day Efficacy Registration Trial: Rhopressa TM 0.02% QD ~200 patients Timolol BID ~200 patients “Rocket 2” One Year Safety (3 mo. interim efficacy) Registration Trial: Rhopressa TM 0.02% QD Rhopressa TM 0.02% BID ~230 patients Timolol BID ~230 patients 1 PGAs have been shown to be less effective when dosed BID “Rocket 3” One Year Safety-Only Study – Canada: Rhopressa TM 0.02% QD ~90 patients Rhopressa TM 0.02% BID ~90 patients Timolol BID ~60 patients ~230 patients 1 |
Ciliary Processes Cornea Uveoscleral Outflow NET RKI NET RKI Trabecular Meshwork Episcleral Veins Schlemm’s Canal Latanoprost Quadruple-Action Roclatan TM Rhopressa TM MOAs: RKI PGA RHOPRESSA TM FIXED COMBINATION WITH LATANOPROST 1. ROCK inhibition relaxes TM, increases outflow 2. NET inhibition reduces fluid production 3. ROCK inhibition lowers EVP 4. PGA receptor activation increases uveoscleral outflow |
19 Roclatan TM : Quadruple-Action Fixed Combination First product to lower IOP through all three known mechanisms, and also lowers 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 Day 2 - No IOP measured Dose Dose Dose 0.02% Roclatan TM vs. Latanoprost Primates (n=6/group) Latanoprost Roclatan TM |
20 Roclatan TM TM Phase 2b Clinical Trial Design Primary efficacy endpoint: Mean diurnal IOP on Day 28 Two concentrations of Roclatan vs. Rhopressa TM 0.02% and latanoprost Trial design similar to registration trial for fixed-dose combination - 1-3 mmHg superiority vs. components previously accepted by FDA Phase 2b Protocol Roclatan TM 0.01% vs. Roclatan TM 0.02% vs. Rhopressa TM 0.02% vs. Latanoprost All dosed QD PM ~300 patients 28 days TM June or early July 2014 Results expected to be released late |
21 Key Milestones Financed by IPO Proceeds Key Milestones Financed by IPO Proceeds Started Phase 2b clinical trial H1 2014 H1 2014 H2 2014 H2 2014 H2 2015 H2 2015 H1 2015 H1 2015 Start Phase 3 registration trials Late June or Early July 2014: Roclatan TM Results from Phase 2b expected Efficacy results from Phase 3 expected H1 2016 H1 2016 Phase 3 prep NDA filing expected TM Early-2014: Roclatan Early 3Q 2014: Rhopressa Mid-2015: Rhopressa Mid-2016: Rhopressa Mid-2015: Roclatan TM TM TM TM |
22 Rhopressa TM Phase 3 registration trials Direct clinical - $25.0M Non-clinical - $12.4M Roclatan TM 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 Roclatan TM Needs: $40M through Mid-2017 Roclatan TM Phase 3 registration trials Direct clinical - $25.5M Non-clinical - $2.0M 12 Months G&A and working capital - $12.5 Targeted Mid-2017: Expected NDA Approval for Rhopressa TM Expected NDA Filing for Roclatan Financial Summary Financial Summary TM |
23 Market Cap (dollars in millions) 10/31/13 - $243.7 12/31/13 - $418.2 5/9/14 - $348.0 23 Aerie Stock & Market Cap Performance |