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| 8-K - FORM 8-K - AERIE PHARMACEUTICALS INC | d433226d8k.htm |
 Company Overview Investor Presentation August 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™ (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™ (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 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. 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 “Management’s 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 IOP–Lowering 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 P3’s 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™ (netarsudil ophthalmic solution) 0.02%
Roclatan™ (netarsudil/latanoprost ophthalmic solution) 0.02%/0.05% Positioning as First Line Therapy: • Benefits of Rhopressa™ 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 Aerie’s Lead Glaucoma Therapies Data on file Product candidates have not been approved by the FDA For Investor Use |
 5 2016 US Glaucoma Market US Glaucoma Market – 2016 $2.8B; 36M TRx, Market Share in TRx PGA: Prostaglandin Analogue; BB: Beta Blocker; AA: Alpha Agonist; CAI: Carbonic Anhydrase Inhibitor
Source: IMS Data
Non-PGA Market PGA Market 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 Bimatoprost Travoprost Latanoprost Tafluprost BB Fixed Combo AA CAI Other |
 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) “Rocket 1” 3 Mo. Safety and Efficacy* Registration Trial U.S. Rhopressa™ 0.02% QD 202 patients timolol BID 209 patients (Total enrollment: 411 patients) “Rocket 2” 12 Mo. Safety, 3 Mo. Interim Efficacy** Registration Trial U.S. 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 TM 1 |
 7 Rhopressa Phase 3 Trial Data Filed as Supportive to NDA Filing ClinicalTrials.gov Identifier: NCT02246764, NCT02558374 Product candidates have not been approved by the FDA “Rocket 4” 6 Mo. Safety, 3 Mo. Interim Efficacy* Trial Rhopressa™ 0.02% QD 351 patients timolol BID 357 patients *Rhopressa™ 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
“Mercury 1” 12 Mo. Safety, 3 Mo. Interim Efficacy** Registration Trial Roclatan™ 0.02%/0.005% QD 238 patients Rhopressa™ 0.02% QD 244 patients
latanoprost QD 236 patients **Rhopressa™ achieved non-inferiority to latanoprost
at
range of > 20 mmHg to < 25 mmHg
For Investor Use TM |
 8 Rocket 2: Safety/Tolerability Overview of Rhopressa 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 TM |
 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 Rx’s 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 American Society of Cataract Refractive Surgeons (ASCRS) May 2017 World Glaucoma Congress Helsinki June 2017 Association of Research in Vision and Ophthalmology (ARVO) May 2017 American Glaucoma Society (AGS) March 2017 Building Aerie’s 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 |
 11 **Roclatan™ achieved statistical superiority to components at primary endpoint range of > 20 mmHg to < 36 mmHg, similar to Mercury 1 *Roclatan™ achieved statistical superiority to components at primary endpoint range of > 20 mmHg to < 36 mmHg Roclatan U.S. Registration Trial Results “Mercury 1” 12 Mo. Safety, 3 Mo. Interim Efficacy* Registration Trial Roclatan™ 0.02%/0.005% QD 238 patients Rhopressa™ 0.02% QD 244 patients latanoprost
QD 236 patients “Mercury 2” 3 Mo. Safety and Efficacy** Registration Trial Same structure as Mercury 1, except 3 months Roclatan™ 0.02%/0.005% QD 245 patients Rhopressa™ 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
TM |
 12 12 Roclatan Maintained Superior Efficacy Over Individual Components for 12 Months Mean IOP at Each Time Point (ITT) • Roclatan™ statistically superior to latanoprost and Rhopressa™ at all
time points • Roclatan™ IOP-lowering 1-3 mmHg greater than monotherapy through Month 12 ++ Data on File Based on Mercury 1 Topline 12-month Product candidates have not approved by the FDA For Investor Use TM |
 13 13 Roclatan Maintained Superior Efficacy Over Individual Components for 12 Months Mean Diurnal IOP at Each Visit (ITT) p<0.0001 at All Visits vs. Latanoprost and Rhopressa ++ Data on File Based on Mercury 1 Topline 12-month Product candidates have not approved by the FDA For Investor Use TM ™ |
 14 Roclatan Phase 3 Month 12 Responder Analysis: Goal is to Achieve Lowest IOP Possible At Month 12: % of Patients with IOP Reduced to 18 mmHg or Lower *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 TM 16% 26% 37% 49%49% 57% 12% 22% 35% 66% 27% 43% 60% 72% 82% 0% 20% 40% 60% 80% 100% < 14 mmHg < 15 mmHg < 16 mmHg < 17 mmHg < 18 mmHg Rhopressa™ (n=148) Latanoprost (n=203) Roclatan™ (n=158) *** ** IOP on Treatment |
 15 Mercury 1: Safety/Tolerability Overview of Roclatan 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™ 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 TM |
 16 Roclatan Next Steps • Roclatan NDA filing expected 1H 2018 - Pre-NDA meeting expected 2H 2017 - Final stability data on critical path • Aerie Ireland plant and 2 contract manufacturers are expected to support Roclatan™ U.S. commercial activities • Mercury 3 expected to commence in Europe 3Q 2017; regulatory submission in Europe expected in 2H 2019 Product candidates have not been approved by FDA For Investor Use TM ™ |
 17 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
® ) expected to start 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 in U.S. on Japanese / Japanese Americans • Phase 3 trials expected to be conducted in Japan commencing 2H 2018 For Investor Use |
 18 Advancing the Pipeline Product candidates have not been approved by FDA • Rhopressa™ • Potential for disease modification • 24-hour IOP lowering • AR-13154 (and related compounds) • Significant lesion size reduction in wet AMD models • Drug Delivery • Front of the eye implants for glaucoma • Back of the eye implants for retinal diseases For Investor Use |
 19 Netarsudil* Blocks TGF-beta-Induced Expression of Fibrosis Proteins in Cultured Human TM Cells TGF-beta Levels are Elevated in the Aqueous Humor of
Patients with Glaucoma • 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 *Active ingredient of Rhopressa™ Data on File Control TGF
2 (8ng/ml) TGF 2 (8 ng/ml) + Netarsudil (500nM) |
 20 Netarsudil* Causes Expansion of TM Tissue, Opening Spaces for Increased Outflow Control + Netarsudil TM: Trabecular Meshwork SC: Schlemm’s 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 |
 21 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) Netarsudil (n=8) Baseline (n=8) ** p<0.01 *** p<0.001 |
 22 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 ROCK PKC X 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 |
 23 Topical AR-13154(S) Provides Additive Efficacy to Eylea® in Proliferative Diabetic Retinopathy Model Oxygen-induced retinopathy model of PDR (mouse) 0.06% AR-13154(S) delivered topically from P12 to P17 Eylea ® delivered IP 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 -37% -34% -57% ** *** *** *** 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 |
 24 • Bioerodible implant technology using polyester amide (PEA) polymers • 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 DSM Collaboration – Implant Delivery Technology For Investor Use |
 25 Evaluating Aerie’s 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 Aerie molecules inhibit both ROCK1 and ROCK2 ROCK |
 26 Summary • Key Clinical Priorities • Rhopressa™: PDUFA date set for February 28,
2018
• Roclatan™: Mercury 3 expected to commence 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 Aerie’s 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 |
 Rhopressa and Roclatan 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 27 2017 2018 TM TM |