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Introduction Chris Buyse, CFO 30 October 2012
Today s Agenda 03.00 PM Welcome Agenda House keeping Chris Buyse, CFO 03.05 PM Leveraging JETREA to grow ThromboGenicsas an integrated biopharmaceutical company Dr Patrik De Haes, CEO 03.25 PM JETREA - shifting the treatment paradigm for symptomatic VMA US perspective Dr. Peter Kaiser 03.45 PM JETREA - shifting the treatment paradigm for symptomatic VMA EU perspective Dr. Tim Jackson 04.05 PM JETREA -commercial launch strategy and plan Ram Palanki, Global Head, Marketing & Sales 04.25 PM JETREA global Market Access: payer value platform Dr Paul De Nijs, Global Head, Market Access 04.45 PM JETREA Financial returns Chris Buyse, CFO 05.00 PM Questions & Answers (opening conference call) 3
Commercializing JETREA - Transforming ThromboGenics Dr Patrik De Haes, CEO 30 October 2012
ThromboGenics Journey 2001 2006 2012 Focus R&D in cardiovascular &oncology R&D in cardiovascular, oncology & ophthalmology JETREA FDA approval US launch -January 2013 EMEA approval pending Pipeline Broad/vascular Vascular, Ophthalmology, Oncology Ophthalmology Cash position - 45 M 186 M (30.06.12) Market cap - 100M 1.30 B Employees 20 40 > 135 5
Key Strategic Decisions Transition from early-stage R&D to late-stage clinical development Transition from a vascular to an ophthalmology company Strengthen financial resources Commercialization in USA via own organization Strategic partnership with Alcon for EU and ROW 1, Gaston Geenslaan, Leuven, Belgium 6
Investment Highlights 2012 Alcon agreement for Europe and Rest-of-World: 375 mio+ royalties Private placement 77.8 mio US : JETREA (ocriplasmin) granted Priority Review Positive recommendation from FDA Advisory Committee NEJM publication on JETREA Phase III results US FDA Approves JETREA for treatment of Symptomatic VMA Upcoming milestones : US :launchof JETREA in January 2013 EU : CHMP opinion in Jan 2013 Anticipated EC approval in March 2013 7
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What is JETREA? JETREA Truncated form of human plasmin Produced by recombinant DNA technology First Pharmacological Treatment for symptomatic Vitreomacular Adhesion (VMA) Liquefaction of the vitreous Unique dual mode of action : Liquefaction of vitreous Separation of vitreous from retina Separation of the vitreous 9
Symptomatic VMA is a Progressive Disease Normal OCT VMA causing Traction VMA causing Macular Hole Normal Vision Distortion of Vision Central Blindness 10
JETREA US approval crucial to our success Key inflexion point in ThromboGenics development FDA approved label Symptomatic VMA : broad label Finalizing US launch plans for JETREA (January 2013) US organization readiness: recruitment, training, processes Partnership with Alcon working well. On track to launch JETREA in the EU in 2013 11
Vision & Strategy Continue to grow ThromboGenicsas a profitable integrated biopharmaceutical company developing and commercializing novel ophthalmic medicines 12
Delivering our strategy Successfully commercialize JETREA in the US via our own commercial organization Support Alcon s successful commercialization of JETREA outside the US Further develop our JETREA franchise via new formulations and indications costs shared equally with Alcon Accessing new novel ophthalmic medicines to help expand our ophthalmology franchise sourced via our own research, joint development deals, licensing deals and possibly acquisitions 1, Gaston Geenslaan, Leuven, Belgium 13
Extending the JETREA Franchise New formulations Current formulation has to be stored at -20C New indications Wet age-related Macular Degeneration (wamd) Diabetic Macular Edema(DME)/Diabetic Retinopathy (DR) Retinal Vein Occlusion (RVO) All developments and investments jointly agreed with Alcon 14
Key take away We are on track fast growing profitable ophthalmology company Key focus areas : JETREA significant U.S. commercial opportunity EMA approval will open up other significant markets Alcon to commercialize JETREA in Europe and Rest-of-World Continued shareholder support > Approval > Shareholder return Full profits from US sales Attractive royalty on net sales generated ex-us Continue to invest in R&D and in-licensing opportunities in ophthalmology Commitment to create incremental shareholder value 15
JETREA - shifting the treatment paradigm for symptomatic VMA Peter Kaiser, MD Professor of Ophthalmology Cleveland Clinic, Lerner College of Medicine Cole Eye Institute
Normal Vitreoretinal Architecture
Posterior Vitreous Detachment (PVD) Early Liquefaction Extensive Liquefaction Separation at the Vitreoretinal Interface
Vitreomacular Separation and Adhesion Separation Adhesion
Patients With Unresolved VMA Can Experience a Spectrum of Symptoms Metamorphopsia Decreased Visual Acuity Central Vision Defect
Clinical Course of Symptomatic VMA VMA Symptomatic Progression Spontaneous Resolution
Current Management of Symptomatic VMA Watchful waiting Vitrectomy
Symptomatic VMA: Watchful Waiting
Pars Plana Vitrectomy Using Three Ports
Symptomatic VMA: Vitrectomy
Improvement After Vitrectomy Baseline 20/80 6 months post-vitrectomy 20/40 12 months post-vitrectomy 20/25
Vitrectomy Considerations Risks Retinal tear and detachment Endophthalmitis Hemorrhage Small risk of complete visual loss Post-operative treatment burden Pain and inflammation Multiple post-operative follow-up visits Lost work time Cataract requiring 2 nd operation to remove 7 14 day face-down positioning in some patients
Face-Down Position
Patient / Physician Dilemma Only available treatment is surgery Watchful waiting often employed first Condition remains untreated Continues to progress in an unpredictable manner Complications can occur Surgical outcomes may be worsened by irreversible damage Surgery indicated by individual patient need Need for visual acuity Rate of progression Presence of macular hole Overall benefit / risk
Rationale for Pharmacologic Treatment Option Resolve the underlying condition Avoid risks and burdens of surgery Intervene earlier Reduce progression and damage Improved outcomes Reduce discomfort
JETREA : Truncated Form of Human Plasmin Plasmin JETREA
JETREA Induced Vitreoretinal Separation
Resolution of VMA 20/60 20/100 20/30 20/30
Clinical Program
Study Design TG-MV-006 (US) Study Design TG-MV-007 (US + EU) JETREA 125 µg R 2:1 IVT Day 7 Day 14 Day 28 Month 3 Month 6 Placebo Primary Endpoint
Key Inclusion Criteria Symptomatic VMA Presence of vitreomacular adhesion on OCT Central vitreal adhesion within 6 mm OCT field surrounded by elevation of the posterior vitreous cortex Symptoms considered by investigator as due to VMA such as Metamorphopsia Decreased visual acuity Other visual complaints BCVA 20/25 in study eye
Symptomatic VMA Patient Population Vitreomacular Traction Vitreomacular Traction with Macular Hole
Efficacy
Non-Surgical VMA Resolution at Day 28 Primary Endpoint Placebo JETREA 30 25 P=0.003 27.9 P<0.001 25.3 P<0.001 26.5 Patients, % 20 15 10 5 13.1 6.2 10.1 0 N= TG-MV-006 TG-MV-007 Combined 107 219 81 245 188 464
Proportion of Patients with Non-Surgical VMA Resolution Placebo (N=188) JETREA (N=464) 30 25 Patients, % 20 15 10 5 0
Secondary Endpoints
Total PVD at Day 28 Secondary Efficacy Endpoint Placebo JETREA 20 P=0.014 16.4 P<0.001 P<0.001 15 13.4 Patients, % 10 6.5 10.6 5 3.7 0 N= 0.0 TG-MV-006 TG-MV-007 Combined 107 219 81 245 188 464
Non-Surgical Macular Hole Closure at Day 28 Placebo JETREA 50 40 P=0.002 43.9 P<0.028 36.7 P<0.001 40.6 Patients, % 30 20 10 12.5 6.7 10.6 0 N= TG-MV-006 TG-MV-007 Combined 32 57 15 49 47 106
2-Line Gain in BCVA Without Vitrectomy at Month 6 Placebo JETREA Patients, % 30 25 20 15 10 5 0 N= P=0.002 P=0.035 P<0.001 25.6 22 23.7 11.2 11.1 11.2 TG-MV-006 TG-MV-007 Combined 107 219 81 245 188 464
Change in VFQ-25 Scores from BL to Month 6 Placebo (N=188) Mean Change in VFQ-25 Score 10 JETREA (N=464) 8 6 4 2 0-2 -4 CC-45
JETREA Efficacy Conclusions Higher rate of VMA resolution Higher rate of total PVD induction All other secondary endpoints favor ocriplasmin Full-thickness macular hole closure in 40% of patients 2 and 3-line improvements in visual acuity Lower rate of vitrectomy Improvement in VFQ-25
Safety
Exposure to Intravitreal JETREA Total (any dose) 976 7 Completed Studies 741 Ongoing Studies (as of May 31, 2012) 235 125 µg Dose 582 Phase 3 465 Phase 2 276
Study Eye Ocular AEs: Day 0 7 Placebo (N=187) JETREA (N=465) 15 10 Patients, % 5 0
Study Eye Ocular AEs: Day 8 Month 6 Placebo (N=187) JETREA (N=465) 15 10 Patients, % 5 0
Patients with 2-Lines Vision Decrease: Day 0 7 Placebo N=187 JETREA N=465 3 (1.6%) Day 7 36 (7.7%) 2 (1.1%) Month 6 6 (1.3%)
JETREA Safety Conclusions Most AEs mild to moderate and transient The majority of acute VA decreases resolved Clinical review showed the most frequent cause of VA loss at Month 6 Disease progression Complications of vitrectomy Generally well-tolerated
Clinical Perspective
svma Cole Eye Institute
Baseline 20/30 Cole Eye Institute
3 months later... 20/30 Cole Eye Institute
4 months later... 20/40 Cole Eye Institute
5 months later... 20/50 Cole Eye Institute
svma Progression 20/20 20/40 20/25 20/60 Cole Eye Institute
VMT Cole Eye Institute
Vitreomacular traction syndrome Cole Eye Institute
Vitreomacular traction syndrome Cole Eye Institute
Vitreomacular traction syndrome 20/50 Cole Eye Institute
Macular Holes Cole Eye Institute
Stage 2 Macular Hole UHR-OCT Courtesy of Jay Duker, MD Cole Eye Institute
Stage 2 Macular Hole QuickTime and a decompressor are needed to see this picture. Cole Eye Institute
Stage 2 Macular Hole Cole Eye Institute
AMD Cole Eye Institute
s/p Lucentis x 4 (minimally classic CNV) 20/80
s/p Lucentis x 5 (minimally classic CNV) 20/80
s/p Lucentis x 6 (minimally classic CNV) 20/80
DME Cole Eye Institute
Baseline 20/60 Cole Eye Institute
s/p Avastin x 4 20/30 Cole Eye Institute
DME Cole Eye Institute
Baseline 20/40 Cole Eye Institute
s/p Avastin x 3 20/30 Cole Eye Institute
JETREA - For symptomatic VMA Mr. Tim Jackson Consultant King s College Hospital, London, UK
JETREA : Overview Current Treatment Paradigm Future Treatment Paradigm *VRI Disorder *VRI Disorder Watch & Wait JETREA Vitrectomy Vitrectomy *Vitreoretinal Interface
Watchful Waiting Watchful waiting in VMT 11% spontaneously resolved with stabilization of VA If no resolution, 92% had visual loss 64% lost 2-lines of visual acuity Watchful waiting in Macular Hole 8.6% spontaneous closure after 6 years, no VA improvement Gradual decline to mean VA of 20/200 Longer duration produces lower surgical success 1. Hikichi, et al. Am J Ophthalmol. 119:1995. 2. Chew, et al. Arch Ophthalmol. 177:1999.
Vitrectomy Vitrectomy in VMT 1-3 Successful traction relief in almost all patients VA improved in 35-89% of patients VA worsening in 4-23% Vitrectomy in Macular Hole 4-6 Anatomic closure in 44-94% of patients VA improved in 19-80% of patients (+/- hole closure), seen at 3 months post-op, continuing to 1-2 years 2-lines worsening in 22% of patients 1. Sonmez, et al. Retina. 28:2008. 2. Witkin, et al. Ophth Surgery, Lasers, Imaging. 41:2010. 3. Haller, et al. Ophth. 117: 2010. 4. Christensen, et al. BJO. 93, 2009. 5. Lois, et al. IOVS 52: 2011. 6. Freeman. et al. Arch Ophth. 115, 1997.
Need for Non-Surgical Therapy Only available treatment is vitrectomy Watchful waiting not optimal in symptomatic patients Condition remains untreated Continues to progress in an unpredictable manner Complications can occur Surgical outcomes may be worsened by irreversible damage
JETREA Single injection Minimally invasive procedure Rapid onset of action Ease of administration
JETREA Benefits Resolve VMA High rate of macular hole closure Improvement in quality of life Greater vision improvement Decreased need for surgery Non-responders can still receive vitrectomy
Clinical Perspective
VMA Resolution at Day 28 JETREA Placebo
Case Study: Resolution & Progression 20/100 20/20
Case Study: Resolution & Progression 20/100 20/20 20/30 20/125
JETREA Response Profile All JETREA responders have VMA resolution by day 28 30 VMA Resolution & FTMH Closure (%) 25 20 15 10 5 0 70% 80% 100% 0 7 14 21 28 Time, Post Injection
Predictable Response Profile With a Single Injection All JETREA responders have VMA resolution by day 28 Average time between initial visit and scheduling of vitrectomy > 28 days Vitrectomy remained a viable option for nonresponders in MIVI-TRUST 93.1% MH closure rate in phase III nonresponders (92.3% in placebo arm)
Patient cases
Symptomatic VMA 92 Images Courtesy of Tufts University Eye Center
Symptomatic VMA: VA 20/30 93 Images Courtesy of Tufts University Eye Center
Symptomatic VMA in ERM: VA 20/40 94 Images Courtesy of Tufts University Eye Center
s/p ranubizumab x 6 (occult CNV) 20/60 Cole Eye Institute
Geographic Atrophy with VMA:VA 20/200 96 Images Courtesy Pravin U Dugel, MD
Conclusions Limited treatment options available Watchful waiting Vitrectomy JETREA Benefit in one in four patients as evaluated by CRC Low risks in non-responders Vitrectomy outcomes preserved in non-responders Favorable benefit/risk for JETREA to treat symptomatic VMA, including when associated with macular hole
JETREA : Current Treatment Paradigm Future Treatment Paradigm *VRI Disorder *VRI Disorder Watch & Wait JETREA Vitrectomy Vitrectomy *Vitreoretinal Interface
JETREA (Ocriplasmin) - Commercial Launch Strategy & Plan Ram Palanki ThromboGenics, Global Head Marketing & Sales 30 October 2012
JETREA FDA Approved Prescribing Information 100
ASC3 VMA Is a Core Pathology in Many Retinal Disorders VMA on OCT Vitreomacular Traction Macular Hole Macular Pucker /ERM Retinal Tears/Detachment Diabetic Macular Edema Diabetic Retinopathy Age-Related Macular Degeneration (AMD) Retinal Vein Occlusion 101
Slide 101 ASC3 insert label slide before Alexandra Schietekatte, 29-Oct-12
Developments in OCT have led to improved diagnosis of VMA in Retina Clinics Past Present 1 The Future * Evolution of OCT as a diagnostic has helped identify VMA as distinct clinical entity 1 Image courtesy of Dr. DafnaGoldenberg, Tel Aviv Medical Center *Image courtesy of Heidelberg Engineering 102
OCTs Routinely Performed in Ophthalmology Clinics Optical Coherence Tomography (OCT) Performed routinely in clinics Nearly every ophthalmologist clinic in US has access to OCT* Spectral domain: makes diagnosing VMA easy, fast, precise & accurate ~10.5 1 million OCT procedures performed in 2011 *David Huang, MD, Ph.D, OCT News, March 2009 103 1 Centers for Medicare and Medicaid Services. Physician/Supplier Procedure Summary (PSPS) File. 2008-2011
US Target customer and Market Opportunity US Retinal Specialists 2100 US Market Opportunity* 2100 retina specialists treat 270,000 eligible patients** *Eligibility based on Jan 2011 TG primary Quant research ( 55 US retinal physicians) and CMS Claims Data (SAF); CMS Claims Data (PSPS). ** This number doesn t include penetration rates 104
Medicare Volume of IntravitrealInjections Has Steadily Increased Since 2008 2,500,000 2,000,000 1,500,000 1,000,000 No. of Units Billed of CPT 67028 1 1,718,730 1,369,959 1,088,946 2,103,596 500,000 0 2008 2009 2010 2011 1 Centers for Medicare and Medicaid Services. Physician/Supplier 105 Procedure Summary (PSPS) File. 2008-2011.
JETREA Promotional Launch Strategy
JETREA Launch Strategic Imperatives Rapidly build awareness of JETREA s FDA Approval Build JETREA patient base between FDA Approval and January 2013 Product Launch Increase breadth and depth of JETREA usage in 2013 and beyond
JETREA Strategic Imperative Strategic Imperative Operating Strategy Rapidly build awareness of JETREA s FDA Approval JETREA.com launched OCT 17 Personal letters to US KOLs and all Investigators sent OCT 18 FDA Approval E-blast to 10,000+ retina specialists and ophthalmologists sent OCT 18-19 Dear Dr. Smith, I am pleased to announce the US Food and Drug Administration approval of JETREA (ocriplasmin) 2.5 mg/ml Intravitreal Injection.
JETREA Strategic Imperative Strategic Imperative Build JETREA patient base between FDA Approval and January 2013 Product Launch Operating Strategy Educate physicians about JETREA and symp. VMA at major congresses (AAO, OSN NY, Retina 2013) Communicate JETREA January 2013 availability Continued presence in Trade Media Ensure availability of sales collateral and brand campaign for January 2013
JETREA Strategic Imperative Strategic Imperative Increase breadth and depth of JETREA usage in 2013 Operating Strategy Develop and maintain KOL advocacy through promotional speakers bureau program Monitor and adapt key promotional messages Continue education of physicians and ancillary staff about JETREA and symptomatic VMA
Sales & Reimbursement Field Teams 111
Sales & Reimbursement Field Organization ThromboGenics Quintiles National Director Sales & Reimbursement Quintiles Project Lead Quintiles Manager East National Training Manager Manager Commercial Operations Quintiles Manager West Area Manager Northeast Area Manager Southeast Area Manager Midwest Area Manager West 7 RAM 4 RBM 7 RAM 4 RBM 7 RAM 4 RBM 7 RAM 4 RBM RAM Retina Account Manager RBM Reimbursement Business Manager 112
Retina Account Manager Deployment Criteria 28 RAMs 2800 Retina-Focused MDs Stratified according to: - Volume of patients seen Key procedure codes Key diagnostic codes Regional differences Retina Specialist Distribution 113
Reimbursement Business Manager Deployment Criteria 16 RBMs National Plans +200 Regional Affiliates Large Plan Affiliates ~1700 Small Population Plans Stratified according to: Volume of Business Size and influence of accounts Back office infrastructure Aligned for optimal coverage with RAM territories 114
Retina Account Manager Territories Sized for Optimal coverage at Launch and Beyond
Field Team Recruiting Activity on Schedule 44 Field Positions 28 Retina Account Managers 16 Reimbursement Business Managers 3,260 Applicants 2,169 Phone Screens 160 DDI Score Tested 150 F2F Interviews 60 TG Senior Management Final Interviews Offer Letters 10/18 11/1 On Boarding Starts 11/1 11/12 116
Field Readiness Timeline Months Before Launch 13 12 11 10 9 8 7 6 5 4 3 2 1 Jan 12 Feb12 Mar 12 Apr 12 May 12 Jun 12 Jul 12 Aug 12 Sep 12 Oct 12 Nov 12 Dec 12 Jan 13 National Sales Director Quintiles Project Leader Work Order Development Q Project Kickoff Area Managers Trainer Quintiles Field Managers Ad Com RBM Manager (16) RBM Commercial Ops (28) RAM Home Study Modules S A L E S Interviewing and Selecting RAM & RBM Candidates Area Mtgs Training Materials Development Territory Mapping & Analytics Area Manager Recruiting & Hiring Launch Meeting Develop SFA & CRM Solutions Develop Bonus & Incentive Plans Disease Awareness, Reimbursement & Patient Pipeline Development 117
JETREA Reimbursement Support Services
Types of Medical Billing Codes ICD-9 Diagnosis Codes Communicate patient condition CPT Codes Report the type of service or procedure provided HCPCS Codes Supplement the CPT system by reporting the medical product used Medical Coding 119
Review of ICD-9 Codes ICD-9 diagnosis codes are used on claims to justify medical necessity for the items and services listed on the claims as provided to the patient by a HCP ICD-9 codes have a high degree of specificity; without a diagnosis code, claims must be coded with a not otherwise specified code, which can make it difficult for payers to establish medical necessity for other drugs or services indicated on the claim form. The diagnosis code for symptomatic VMA is 379.27 Vitreomacular adhesion, Vitreomacular traction. Excludes: traction detachment with vitreoretinal organization (361.81) 120
HCPCS codes for drugs Jan 2013 Apr 2013 Jun 2013 Oct 2013 Jan 2014 J- codes Created to describe drugs that cannot be self administered Used in physician office settings Q- codes C- codes Temporary HCPCS codes, created outside the annual cycle of new J-codes Temporary codes for drugs eligible for Medicare transitional pass-through payments Used in physician office settings Used in hospital outpatient department setting & ASC 121
JETREA Reimbursement Support Platform: Our Commitment to Providers & Patients 100% Commitment ensuring patient access Seamless and hassle-free reimbursement support experience Exceeds industry standards of reimbursement support Reimbursement Support Service Platform for JETREA 122
Comprehensive Provider and Patient Reimbursement Support Platform Reimbursement Support Benefit Verification Prior Authorization Denials and Appeals Billing and Coding Information Claims Tacking Patient Assistance Web Based Application Portals Alternate Coverage Research and Triage Foundation Co-pay Assistance Product Replacement Credit/Debit Card Triage & Referral Management Spillage Spoilage Mishandling 123
Development Milestones for Launch Readiness NOV 12 DEC 12 JAN 13 11/2 Physician Ad Board Content map for website 11/9 Final Reimbursement Branding Concept 11/15 11/21 Training Messaging and content development 12/10 Training 12/24 WEBSITE READINESS 1/9 Training 1/2013 LAUNCH To 11/21 Website development and planning To 12/7 Website beta testing To 12/24 Website implementation activities To 1/11 Website optimization 124
Strategic partnership with Alcon: Maximizing the global JETREA opportunity
EU Target Customer and Market Opportunity France, 250 EU Big 5 + Belgium Belgium, 55 UK, 225 EU Big 5 Market Opportunity Germany, 600 Spain, 350 Italy, 500 2000 retina specialists treat 230,000 eligible patients VMA/VMT 151,372 MH, 79,355 Maximizing the JETREA opportunity in EU and ROW with our partner Alcon Launch in over 40 countries *US clinical prevalence data extrapolated to EU-5 country population. Eligibility based on Jan 2011 TG primary Quant research (42 EU re nal physicians). ** This number doesn t include penetration rates 126
Global Strategic Leadership of ThromboGenics Guarantee global positioning and global branding for Jetrea Develop One global brand concept Align Key message across the globe Leverage US strategy to ensure global alignment and maximize efficiency Optimizing communication between US and OUS to reduce redundancy in efforts and leverage successful tactics in other parts of the world 127
Strategic input in commercialization plans outside the US Strategic guidance in the development of the commercialization plans of Alcon s local affiliates UK, Germany and France Belgium, Italy, Spain, Canada, Australia Status Approved Under Review 128
JETREA - Global Market Access : Payer Value Platform Dr Paul De Nijs ThromboGenics, Global Head Market Access 30 October 2012
Introduction JETREA : building Market Access Foundation for successful Market Access Strategy Raise internal awareness for MA requirements Focus on optimising the label Determine the true value of JETREA for providers & payers Establish optimal launch price for US and EU Execution of Market Access plan Data generation Communication platform Infrastructure for MA implementation: Germany -UK -France Health Economist Multi-disciplinary Task Force for Health Technology Assessment 130
Early Value Assessment Systematic Approach Objective: Optimal Pricing & Reimbursement strategy for JETREA in US/EU5 INSIGHT & UNDERSTANDING: GAP ANALYSIS Pricing, reimbursement and funding landscape before/after market entry OPTIMAL P&R STRATEGY: Key value drivers Evidence required Price-volume relationship Reimbursement thresholds & restrictions Profit-optimal US target price EU price corridor & optimal EU launch sequence Value Harvesting Value Demonstration Value Identification Systematic early value assessment foundation for value identification and demonstration 131
Early Value Assessment US-EU payer/physician/kol research 15 100 (out of 2500) 11 50 (out of 225) / 6 50 (out of 600) / 3 3 / 5 No primary research, results based on SKP experience & expert judgement 5 25 (out of 500) 3 Payer / QUAL. Spec./ QUANT. KOL/ QUAL. SOLID METHODOLOGY Clear Objectives Representative Sample Relevant Comparators State-of-the-art price finding methods SKP Market Research Oct 10- Feb 11 132
Early Value Assessment Initial Value Perception Physicians / KOLs Rarely answered Frequently answered Overall Alignment with Payers + 1. Possibility to avoid/replace surgery 2. Less invasive compared to VE 3. Safety/tolerability 4. Efficacy in general 5. Improvement in VA 6. Efficacy: Non-surg. symptomatic VMA resol. 7. Addit. treatment option for symptomatic VMA 39% 38% 28% 28% 25% 25% 15% - 1. Efficacy: Insufficient symptomatic VMA resolution 2. Safety/tolerability in general 3. Efficacy: Low effect with ERM 4. Side effects: Subretinal fluids 19% 15% 24% 45% Mirror payers perceptions: avoiding / replacing surgery is biggest perceived advantage, but efficacy needs clarification 133
Fact based Market Access Approach VALUE HARVESTING: What the research showed us Unmet need Value demonstration Determination of Jetrea key attributes for physicians and payers & data generation Budget Impact / Cost Effectiveness Key Value Messages Clinical effect Value Response Kit supporting value communication & value negotiation
Key Value Messages 1 Eye functioning deteriorates with age 2 Current treatment paradigm negatively impacts patient outcome 3 JETREA benefits lead to paradigm shift in treatment of symptomatic VMA BI / CE Unmet need Key Value Messages Clinical effect 4 5 6 JETREA leads to improved visual acuity & Quality of Life (QoL) and to reduced need for vitrectomy Early treatment leads to improved patient outcomes Increased response rate in special patient populations 7 JETREA treatment leads to predictable budget impact 8 JETREA treatment is cost-effective in most patient populations
First-in-class: Significant impact on Pricing and Reimbursement First-in-class Innovation Impact on P&R JETREA is first-in-class - First pharmacological treatment for treatment of symptomatic VMA JETREA has a unique MoA - Pharmacological detachment of the vitreous body from the retina - Single intra-ocular injection + Paradigm shift in therapy - No need for watchful waiting - Patients can be treated earlier - Improved outcome High burden of disease for patients of symptomatic VMA First-in-class can impact: - Level of access - Reimbursement and price level - Speed of reimbursement decision Vitrectomy: nonpharmacological (surgical) treatment, leads to: - Delay of treatment - Negative impact on patient outcome 136
Jetrea Market Access USA Implementation Plan 137
Medicare is the Primary Payer for U.S. VMA Patients Insurance Coverage for U.S. VMA Patients Intravitreal Drug Coverage in the U.S. Medicaid Uninsured 8% 4% Specialty pharmacy 59% 29% Private insurance Medical benefit Buy and bill Medicare Both Medicare patients would be covered under Medicare Part B % of VMA patients Pharmacy benefit Most private plans cover IVT injected drugs under medical benefits with the options for physicians to acquire them through specialty pharmacy or buy and bill Source: Simon-Kucher & Partners research 2010, quantitative research with n=100 physicians,,n=3 KOLs and n=15 payers 138
Launch Timing Impacts Coding Filings and Coding Impacts Payer Adoption ICD-9 Diagnosis Code Appl Submitted for VMA/VMT (Jan 2011) Approval and Use of New ICD-9 Diagnosis Code (Oct 2011) ICD-9 Diagnosis Coding HCPCS J Coding for MD Office HCPCS C Coding for HOPD/ASC Payer Advocacy and Coverage FDA Approval Oct 2012 2011 2012 2013 Throughout 2011 and Q1-Q3 2012: Payer education and advocacy for coverage (both disease/indication and product) -Payer Advisory Boards -Dossier Development -Health Economics -Local Coverage Policies -KOL Referral Network -CAC Education and Advocacy (Dec 2012 Sub; Apr 2013 Effective) Transitional Pass Through Status and C Code for HOPD/ASC Reimbursement (Jan 2013 Sub; Jan 2014 Effective) HCPCS J Code for MD Office Reimbursement 139 2014
Payer to HCP Sphere NAM NAM HI NAM NAM 140
US Private Payer: ~ 30% Insurance Coverage for U.S. VMA Patients Intravitreal Drug Coverage in the U.S. Medicaid Uninsured 8% 4% Specialty pharmacy 59% 29% Private insurance Medical benefit Buy and bill Medicare Both Medicare patients would be covered under Medicare Part B % of VMA patients Pharmacy benefit Most private plans cover IVT injected drugs under medical benefits with the options for physicians to acquire them through specialty pharmacy or buy and bill Source: Simon-Kucher & Partners research 2010, quantitative research with n=100 physicians,,n=3 KOLs and n=15 payers 141
Metrics for Success Payer Coverage and Time to Payment 95% of public and commercial lives have covering JETREA 1 year post launch 90% of Claims paid in 60 Days or less after J-Code implementation Launch Distribution Efficient Launch/Sales Volume (ongoing sales ins/outs) Customer Satisfaction (ATU) on Access Option Buy/Bill and SP AR Days managements Patient Assistance % of Patients approved for PAP after referral to programs Post launch awareness (measure 6 months and 12 months post) of PAP programs 142
Metrics for Success Payer Coverage and Time to Payment 95% of public and commercial lives have covering Jetrea 1 year post launch 90% of Claims paid in 60 Days or less after J-Code implementation Launch Distribution Efficient Launch/Sales Volume (ongoing sales ins/outs) Customer Satisfaction (ATU) on Access Option Buy/Bill and SP AR Days managements Patient Assistance % of Patients approved for PAP after referral to programs Post launch awareness (measure 6 months and 12 months post) of PAP programs 143
Metrics for Success Payer Coverage and Time to Payment 95% of public and commercial lives have covering Jetrea 1 year post launch 90% of Claims paid in 60 Days or less after J-Code implementation Launch Distribution Efficient Launch/Sales Volume (ongoing sales ins/outs) Customer Satisfaction (ATU) on Access Option Buy/Bill and SP AR Days managements Patient Assistance 85% of Patients approved for PAP after referral to programs Post launch awareness (measure 6 mo and 12 mo post) of PAP programs 144
JETREA Market Access EU Implementation Plan 145
Project Milestones & Timelines Market Access: UK -Germany -France November 2012 NICE UK STA Feb 2013 STA Submission June 2013 ACD September 2013 FAD UK October 2013 Final Guidance GERMANY Early Benefit Assessment Submission April 2013 P&R submission FRANCE 146
Launch Readiness Market Access USA ThromboGenics: Leverage of Global Market Access strategy and materials Implementation plan adapted to payer environment: Medicare Medicaid Private Payers Patient Assistance Programs EU/RoW partnership ThromboGenics - Alcon: Leverageof Market Access teams in EU HTA applications jointly prepared Market Access Value Platform created 147
JETREA - Financial Update Chris Buyse, CFO 30 October 2012
A Track Record of Creating Value 40 38 36 34 32 30 28 26 24 22 20 18 16 14 12 10 8 6 4 2 0 IPO 35mn Private Placement May 2007 23.9mn Private Placement Nov 2009 42mn Private Placement Dec 2010 56mn Private Placement Mar 2012 77.8mn Jul-06 Oct-06 Jan-07 Apr-07 Jul-07 Oct-07 Jan-08 Apr-08 Jul-08 Oct-08 Jan-09 Apr-09 Jul-09 Oct-09 Jan-10 Apr-10 Jul-10 Oct-10 Jan-11 Apr-11 Jul-11 Oct-11 Jan-12 Apr-12 Jul-12 Oct-12 Source: Thomson Datastream. Average liquidity over the last 3 months: approximately 170,000 shares per day Average daily transaction value around 5.5 million 149
Strong Shareholder Base October 2012 150
ThromboGenics Financial Position Well funded to support upcoming launch of JETREA in the US Finished H1 2012 with cash of approximately 186 million Will be profitable in 2012 due to 75 million upfront payment from Alcon EU launch of JETREA will generate a further 90 million in milestone payments from Alcon anticipated in 2013 ThromboGenics will sustain its profitability through the commercialization of JETREA Thrombogenics corporate tax burden will benefit from a combination of the application of the Belgianpatent income deduction regime and the existing deduction carry forwards of ThromboGenics NV 151
Realizing Value from JETREA s US Market Success ThromboGenics will account for all of the US sales of JETREA COSTS - Producing the final JETREA product FujiFilm and Patheon - The costs of our US organization - Supply chain costs needed to distribute JETREA - Other costs REVENUES 152
Realizing Value from JETREA s EU/ROW Market Success -Further 90 million in milestones from Alcon around the launch of JETREA in Europe Potential to receive a further 210 million in sales milestones Attractive royalties which give us an important share of the economics of Alcon s sales of JETREA JETREA product supply income COSTS We will need to bear the costs of: Supplying the final JETREA product to Alcon Our strategic marketing, market access and medical affairs teams operating across the top 5 EU markets and Belgium Other costs REVENUES 153
JETREA shareholder value creation Potential global market for JETREA should allow ThromboGenics to generate significant returns for shareholders Returns will be based on two highly attractive revenue streams: US sales via our own commercial organization Royalties and sales milestones from Alcon our partner for JETREA outside the US Sales and marketing costs are expected to grow more slowly than revenues post the launch phase Sales-related costs such as IP/licensing fees will remain below 5% of sales ThromboGenics is highly leveraged to the success of JETREA 154
Key take aways ThromboGenics next key milestone is JETREA s US launch in January 2013 High awareness of JETREA amongst key targets in the US retina community Confident there is a significant market for JETREA in US We are on track to have our first-class, focused US commercial organization in place to support the launch of JETREA Alcon preparing to launch JETREA in 2013 in the EU, if approved Market access strategies being implemented for both the US and Europe Well financed to support commercialization of JETREA Expect JETREA to generate significant shareholder returns 155
Commercializing JETREA Transforming ThromboGenics Questions & Answers 30 October 2012
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Commercializing JETREA Transforming ThromboGenics Questions & Answers 30 October 2012
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