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8-K - 8-K - AERIE PHARMACEUTICALS INC | d265889d8k.htm |
Company Overview Investor Presentation September 2017 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 trial
results for Rhopressa TM
(netarsudil ophthalmic solution) 0.02% relate to the results in its first Phase 3 registration trials named Rocket 1 and Rocket 2, or
Rocket 4 which will be used primarily for European regulatory filing purposes, and for
Roclatan TM
(netarsudil/latanoprost
ophthalmic solution) 0.02%/0.005% relate to the results in its Phase 3 registration
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, 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 Companys 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. Any top line 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. 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 Managements 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.
For Investor Use |
3 Aerie Late Stage IOPLowering Products (IP 2030+) Pipeline Activities Rhopressa Potential for disease modification, 24 hour IOP lowering AR-13154 Pre-clinical molecule for diseases of the retina Drug Delivery - Front and back of the eye Rhopressa (netarsudil ophthalmic solution) 0.02%
Aerie-owned new chemical entity targeting the diseased tissue PDUFA goal set for February 28, 2018; entering launch mode Roclatan (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005%
Fixed combination of Rhopressa and latanoprost Two P3s achieved primary efficacy endpoints NDA preparation underway Aerie Building a Major Ophthalmic Pharmaceutical Company Data on file Product candidates have not been approved by the FDA For Investor Use |
4 Rhopressa TM (netarsudil ophthalmic solution) 0.02% Roclatan TM (netarsudil/latanoprost ophthalmic solution) 0.02%/0.05% Positioning as First Line Therapy: Benefits of Rhopressa TM while also targeting the secondary drain Achieved statistical superiority to market-leading latanoprost in P3 trials
Potential to become the most efficacious IOP-lowering medication for glaucoma and ocular hypertension, if approved Positioning as Adjunctive Therapy: Once-daily dosing directed at site of pathology, the trabecular meshwork Consistent IOP lowering over 12 months and across a broad range of baseline IOPs, as demonstrated in clinical trials Lowering of episcleral venous pressure, among other demonstrated attributes Aeries Lead Glaucoma Therapies Data on file Product candidates have not been approved by the FDA For Investor Use |
5 2016 US Glaucoma Market PGA: Prostaglandin Analogue; BB: Beta Blocker; AA: Alpha Agonist; CAI: Carbonic Anhydrase Inhibitor
Source: IMS Data
16% 13% 10% 8% 36% 9% 8% 2 4 Times Daily Once Daily For Investor Use 36M TRx approximates 60M units per year for 1.7 bottles/Rx PGA Market Non-PGA Market US Glaucoma Market 2016 $2.8B; 36M TRx, Market Share in TRx Latanoprost Travoprost Bimatoprost Other CAI AA Fixed Combo BB Tafluprost |
6 **Rhopressa achieved non-inferiority to timolol at primary endpoint
range
of > 20 mmHg to < 25 mmHg
*Rhopressa achieved non-inferiority to timolol at pre-specified secondary
endpoint range of > 20 mmHg to < 24 mmHg; also non-inferior at < 25
mmHg Rhopressa Trials Included in NDA
Submission (PDUFA date February 28, 2018)
Rhopressa 0.02% QD
202 patients timolol BID 209 patients (Total enrollment: 411 patients) Rhopressa 0.02% QD 251 patients Rhopressa 0.02% BID 254 patients timolol BID 251 patients (Total enrollment: 756 patients) 1 Post-hoc analysis ClinicalTrials.gov Identifier: NCT02207491, NCT02207621 Product candidates have not been approved by the FDA For Investor Use Rocket 1 3 Mo. Safety and Efficacy* Registration Trial U.S. Rocket 2 12 Mo. Safety, 3 Mo. Interim Efficacy** Registration Trial U.S. 1 |
7 Additional Rhopressa Phase 3 Trial Data Included in NDA: Rocket 4 Under Review; Mercury 1 Supportive ClinicalTrials.gov Identifier: NCT02246764, NCT02558374 Product candidates have not been approved by the FDA Rhopressa 0.02% QD 351 patients timolol BID 357 patients *Rhopressa TM achieved non-inferiority to timolol at primary endpoint range of > 20 mmHg to < 25 mmHg; also non-inferior up to < 30 mmHg; consistent safety results
*Rhopressa TM achieved non-inferiority to timolol at primary endpoint range of > 20 mmHg to < 25 mmHg; also non-inferior up to < 30 mmHg; consistent safety results
Roclatan 0.02%/0.005% QD
238 patients Rhopressa TM 0.02% QD 244 patients
latanoprost QD 236 patients **Rhopressa TM achieved non-inferiority to latanoprost
at
range of > 20 mmHg to < 25 mmHg
**Rhopressa TM achieved non-inferiority to latanoprost
at
range of > 20 mmHg to < 25 mmHg
For Investor Use Rocket 4 6 Mo. Safety, 3 Mo. Interim Efficacy* Trial Mercury 1 12 Mo. Safety, 3 Mo. Interim Efficacy** Registration Trial |
8 Rocket 2: Safety/Tolerability Overview of Rhopressa TM QD (Interim 12-Month) Product candidates have not been approved by the FDA There were no drug-related systemic or serious adverse events The most common adverse event was conjunctival hyperemia with ~50% incidence*, the majority mild and sporadic Other ocular AEs AEs occurring in ~5-23% of patients included: conjunctival hemorrhage, cornea verticillata, blurry vision and reduced visual acuity For Investor Use |
9 9 Rhopressa Commercialization Prep Hired Chief Commercial Officer and VPs of Sales, Market Access, Commercial Operations, and Medical Affairs in late 2016 / early 2017, and Chief Compliance Officer in 2015 Expect to hire U.S. salesforce of 100 reps immediately after approval; fully trained by end of
2Q18
- Target top 12,000 prescribers; 80% of Rxs Commenced initial scientific market access meetings with
top Medicare Part D / Commercial
Payors
Developing launch plan Product candidates have not been approved by FDA For Investor Use |
10 Building Aeries Presence in the Medical Community 5 Posters, including Enhancing Efficacy by Continuous Delivery of AR-13154 in an Animal Model of Proliferative Diabetic Retinopathy, Carbajal et al. 4 Presentations, including 24 Hour IOP Lowering of Netarsudil, Peace et al. 3 Presentations, including 3-Month Interim Results of Mercury 1, Asrani et al., and Aqueous Humor Dynamics of Netarsudil Ophthalmic Solution 0.02% in Healthy Volunteers, Sit et al. Aerie Medical Affairs Booth highlighted, in addition to presentation Data on File American Glaucoma Society (AGS) March 2017 American Society of Cataract Refractive Surgeons (ASCRS) May 2017 Association of Research in Vision and Ophthalmology (ARVO) May 2017 World Glaucoma Congress Helsinki June 2017 |
11 **Roclatan TM achieved statistical superiority to components at primary endpoint range of > 20 mmHg to < 36 mmHg, similar to Mercury 1 *Roclatan TM achieved statistical superiority to components at primary endpoint range of > 20 mmHg to < 36 mmHg Roclatan TM U.S. Registration Trial Results Roclatan TM 0.02%/0.005% QD 238 patients Rhopressa TM 0.02% QD 244 patients latanoprost
QD 236 patients Same structure as Mercury 1, except 3 months Roclatan TM 0.02%/0.005% QD 245 patients Rhopressa TM 0.02% QD 255 patients latanoprost QD 250 patients ClinicalTrials.gov Identifier: NCT02558400, NCT02674854 Product candidates have not been approved by
the FDA For Investor Use
Mercury 1 12 Mo. Safety, 3 Mo. Interim Efficacy* Registration Trial Mercury 2 3 Mo. Safety and Efficacy** Registration Trial |
12 Roclatan Phase 3 Month 12 Responder Analysis: Goal is to Achieve Lowest IOP Possible *p<0.05, **p<0.01, ***p<0.0001 ++ Data on File Based on Mercury 1 Topline 12-month Product candidates have not approved by the FDA For Investor Use At Month 12: % of Patients with IOP Reduced to 18 mmHg or Lower 16% 26% 37% 49% 57% 12% 22% 35% 49% 66% 27% 43% 60% 72% 82% 0% 20% 40% 60% 80% 100% 14 mmHg 15 mmHg 16 mmHg 17 mmHg 18 mmHg IOP on Treatment Rhopressa (n=148) Latanoprost (n=203) Roclatan (n=158) |
13 Mercury 1: Safety/Tolerability Overview of Roclatan TM QD Topline 12 Month There were no drug-related systemic or serious adverse events The most common adverse event was conjunctival hyperemia with ~60% incidence, scored as mild on biomicroscopy for ~70% of these patients and sporadic Other ocular AEs AEs occurring in ~5-18% of subjects receiving Roclatan TM included: cornea verticillata, conjunctival hemorrhage, eye pruritus, lacrimation increased, visual acuity reduced, blepharitis and punctate keratitis. ++ Data on File Based on Mercury 1 Topline 12-month Product candidates have not approved by the FDA For Investor Use |
14 Roclatan Next Steps Roclatan NDA filing expected 1H 2018 Aerie Ireland plant and 2 contract manufacturers are expected to support Roclatan U.S. commercial activities Mercury 3 commenced in Europe 3Q 2017 - Trial conducted in U.K., France, Germany, Italy, Spain and Belgium
- Regulatory submission in Europe expected in 2H 2019 Product candidates have not been approved by FDA For Investor Use |
15 Expanding Aerie Franchise to Europe and Japan Europe (16 Glaucoma Market: 128M monthly units, >2X U.S.)
Expect to file EU MAA for Rhopressa in 2018 Current clinical plan expected to satisfy EU regulatory requirements (including Rocket 4 for Rhopressa and Mercury 3 for Roclatan) Mercury 3: 6-month safety and 90-day efficacy registration trial for Europe,
comparing Roclatan for non-inferiority to a fixed-dose combo in Europe
(Ganfort
® ) started 3Q 2017. Approximately 250 patients per arm. Commenced construction of Ireland Plant to support worldwide commercial supply Japan (16 Glaucoma Market: 52M monthly units)
Preparing to advance clinical development on our own Phases 1 and 2 to be conducted on Japanese / Japanese Americans Phase 3 trials expected to be conducted in Japan commencing 2H 2018 For Investor Use |
16 Advancing the Pipeline Product candidates have not been approved by FDA Rhopressa Potential for disease modification in glaucoma 24-hour IOP lowering AR-13154 (and related compounds) Potential opportunity for Aerie retina program Drug Delivery Back of the eye implants for retinal diseases Front of the eye implants for glaucoma For Investor Use |
17 Netarsudil* Blocks TGF-beta-Induced Expression of Fibrosis Proteins in Cultured Human TM Cells TGF 2: Transforming growth factor 2; SMA: Smooth muscle actin; FSP1: Fibroblast-specific protein 1 Lin, C-W et. al., J. Ocul Pharmacol Ther (2017) in press For Investor Use Data on File Product candidates have not been approved by FDA TGF-beta Levels are Elevated in the Aqueous Humor of
Patients with Glaucoma
*Active ingredient of
Rhopressa |
18 Netarsudil* Causes Expansion of TM Tissue, Opening Spaces for Increased Outflow Control + Netarsudil TM: Trabecular Meshwork SC: Schlemms Canal Control = buffered saline solution Increasing Trabecular Outflow, Reducing Fibrosis Could Stop Degeneration of Outflow Tissues in Glaucoma *Active ingredient of Rhopressa Source: Ren R et al. Invest Ophthalmol Vis Sci. 2016; 57(14):6197-6209. For Investor Use Product candidates have not been approved by FDA |
19 Rhopressa 24-hour IOP Pilot Study Demonstrates Effective Nocturnal Efficacy Netarsudil (active ingredient of Rhopressa) equally effective during nocturnal and diurnal periods Current glaucoma medications either have no efficacy at night (beta blockers, alpha agonists) or reduced efficacy at night (PGAs, CAIs) 1 - 6 AR-13324-CS204 1. Liu JH, et al. Am J Ophthalmol. 2004; 138:389-395. 2. Gulati V, et al. Arch Ophthalmol. 2012; 130:677-684. 3. Liu JH,
et al. Ophthalmology. 2009; 116:449- 454. 4. Liu JH, et al.
Ophthalmology. 2010; 117:2075-9. 5. Fan S et al. J Glaucoma. 2014; 23:276-81. 6. Liu JH, et al. Am J Ophthalmol. 2016;169:249-257. For Investor Use Pre-dose Post-dose (Day 8/9) Product candidates have not been approved by FDA Netarsudil (n=8) Baseline (n=8) ** ** ** *** ** ** *** *** ** p<0.01 *** p<0.001 |
20 Retina Program: AR-13154 Efficacy Driven Primarily by ROCK, PKC Inhibition Retinal tissue harvested from OIR mouse model AR-13154 effectively converted to active metabolite by esterases Active metabolite keeps ROCK, PKC activity, loses PDGFR, JAK activity ROCK/PKC Action: Vascular Dysfunction and Fibrosis ROCK Action: Inflammation Retinal Esterases AR-13154 Active Inactive Data on file; Carbajal, KS et al., Enhancing Efficacy by Continuous Delivery of AR-13154(S) in an Animal Model of Proliferative Diabetic Retinopathy, ARVO 2017, Poster B0481. ROCK JAK PKC PDGFR 182 Aerie compounds screened against 469 human kinases AR-13154 identified as potent lead
compound For Investor Use
Data on File Product candidates have not been approved by FDA ROCK PKC |
21 Topical AR-13154(S) Provides Additive Efficacy to Eylea® in Proliferative Diabetic Retinopathy Model 0% 20% 40% 60% 80% 100% 120% Vehicle Control (n=55) AR-13154(S) topical (n=28) Eylea 1mg/kg IP (n=26) AR-13154(S) + Eylea (n=18) Total Neovascular Area -37% -34% -57% ** *** *** *** Oxygen-induced retinopathy model of PDR (mouse) 0.06% AR-13154(S) delivered topically from P12 to P17 Confirms AR-13154(S) potential as monotherapy and as adjunct to anti-VEGF therapies; not yet tested in humans Data on file; Carbajal, KS et al., Enhancing Efficacy by Continuous Delivery of AR-13154(S) in an Animal Model of Proliferative Diabetic Retinopathy, ARVO 2017, Poster B0481.
For Investor Use Product candidates have not been approved by FDA For more information on Eylea® please see the product webpage https://www.eylea.us/ |
22 DSM Collaboration Implant Delivery Technology Intravitreal sustained-release, bio-erodible implant technology Potential for treatment of Wet AMD and Diabetic Retinopathy Promising results from ongoing feasibility study Evaluating AR-13154 and related Aerie compounds Linear sustained elution rates over several months Achieved target retinal drug concentrations Executed collaboration/licensing agreement Continue prototype evaluations and IND-enabling activities For Investor Use Data on File Product candidates have not been approved by FDA |
23 Evaluating Aeries 3,000+ Owned Molecules Commencing screening for additional indications beyond ophthalmology ROCK inhibition has potential in: Pulmonary health, including pulmonary fibrosis and bronchial asthma Dermatology indications Cancer Others Relationship tree of human kinases. TK, TKL, STE, CK1, AGC, CAMK, CMGC, Other: Kinase superfamilies
For Investor Use ROCK Aerie molecules inhibit both ROCK1 and ROCK2 |
24 Summary Key Clinical Priorities Rhopressa TM : PDUFA date set for February 28,
2018
Roclatan TM : Mercury 3 commenced 3Q 2017 (EU) U.S. NDA filing expected in 1H 2018 Research Initiatives Rhopressa
disease modification, 24-hour IOP lowering, sustained
release
AR-13154 and related compounds with potential for retinal diseases
Evaluating Aeries 3,000+ proprietary Rho kinase molecules Business Development and Expansion Opportunities Drug delivery opportunities for front and back of the eye (e.g., DSM) EU/JP clinical path and commercialization strategy Ireland Manufacturing Facility Well-Financed: $308M cash and investments at 6/30/17 For Investor Use |
2017 2018 Rhopressa TM and Roclatan TM Key Milestones 1H-2019: Roclatan Potential U.S. Approval and Launch 1H-2018: Roclatan NDA Submission Expected Q1-2017: Rhopressa NDA Resubmitted 1H-2018: Rhopressa Potential U.S. Approval and Launch Q2-2017: Rhopressa Rocket 4 Topline safety (6 mos) Q3-2017: Roclatan P3 Mercury 1 12-month Safety Q2-2017: Roclatan P3 Mercury 2 Topline Efficacy (3 mos) 2H-2018: Rhopressa Potential EU MAA Filing For Investor Use 2H-2018: Rhopressa Potential P3 Commencement in Japan Q3-2017: Roclatan P3 Mercury 3 (EU) Initiation (6 mos) Product candidates have not been approved by the FDA 25 |