Opthea Limited · ASX:OPT

Opthea — Investor Presentation (March 2016)

23 slides from Opthea's investor presentation, March 2016

  1. Slide 1: Developing OPT-302: A novel therapy for wet AMD

    Opthea Limited (ASX:OPT, OTCQX:CKDXY) Developing OPT-302: A novel therapy for wet AMD March 2016 Megan Baldwin PhD, CEO & Managing Director; megan.baldwin@opthea.com

  2. Slide 2: Disclaimer

    Disclaimer Investment in Opthea Limited (‘Opthea’) is subject to investment risk, including possible loss of income and capital invested. Neither Opthea nor any other member company of the Opthea Group guarantees any particular rate of return or performance, nor do they guarantee the repayment of ca

  3. Slide 3: Opthea Limited

    Opthea Limited  Clinical stage biotechnology company OPT-302 Wet AMD Program: Milestones  Developing a novel therapy for wet AMD  Wet AMD is the leading cause of blindness in the Western IND Approval for OPT-302 world in older adults June 2015   Technology is based on targeting signals that con

  4. Slide 4: Financial Position (Unaudited)

    Financial Position (Unaudited) Key Financial Details ASX: OPT Substantial Shareholders % Holding Ticker Symbol ASX:OPT Biotechnology Value 18% Fund (BVF)* Share Price (as at Mar 1 2016) ~A$0.385 Baker Bros (NY, USA) 9% Total Ordinary Shares on Issue 150,190,303 Packer & Co. 8.5% Options on Issue 49,

  5. Slide 5: Board of Directors and Executive Management

    Board of Directors and Executive Management Megan Baldwin, PhD, MAICD CEO and Managing Director Appointed CEO Feb ’14, joined company in 2008. Over 19 years experience in research and drug development of therapies targeting angiogenesis. Previously held roles at Genentech (now Roche) in US, in R&D a

  6. Slide 6: The disease process of 'wet' (neovascular) AMD

    The disease process of ‘wet’ (neovascular) AMD Normal Retina ‘Wet’ AMD Choroid 6 Figure: The Angiogenesis Foundation

  7. Slide 7: Monitoring Wet AMD & Treatment Efficacy

    Monitoring Wet AMD & Treatment Efficacy  Trial endpoints are well defined & accepted by FDA/EMA  Non-invasive & routine  Highly quantitative  Trial endpoints are relatively short (Ph 2 typically 6 months, Ph 3 typically 12 months) Visual Acuity Anatomical Changes by Imaging (OCT,FA) Eye Chart (R

  8. Slide 8: Lead Program: OPT-302 for Wet AMD

    Lead Program: OPT-302 for Wet AMD  Lead molecule: – OPT-302 (soluble VEGFR-3, VEGF-C/-D ‘Trap’)  Mechanism: – Blocks VEGF-C and VEGF-D:  Inhibits blood vessel growth  Inhibits vessel leak  Strategy: – To investigate activity as a monotherapy – To develop OPT-302 for use in combination with exis

  9. Slide 9: Resistance to anti-VEGF-A monotherapy

    Resistance to anti-VEGF-A monotherapy  Long-term single-agent therapy with VEGF-A inhibitors is associated with sub-optimal EyleaTM LucentisTM response  Sub-optimal improvements in VA (<15- letter gain)  Persistent fluid on OCT  Resistance to VEGF-A monotherapy may be related to other VEGF famil

  10. Slide 10: Large and growing market opportunity

    Large and growing market opportunity  Wet AMD is the leading cause of blindness in the western world  Increasing prevalence due to ageing population 1/7 ~1.8m  Prevalence expected to double by 2020  Approved therapies for wet AMD target VEGF-A, but not VEGF-C or VEGF-D Our approach is novel and

  11. Slide 11: An unmet medical need despite availability of VEGF-A inhibitors

    An unmet medical need despite availability of VEGF-A inhibitors Despite receiving a VEGF-A inhibitor (Lucentis®, Eylea® or Avastin®): >50% do not achieve significant vision gain 2/3 will continue to have fluid at the back of the eye 25% will have further vision loss at 12 mos 11

  12. Slide 12: Our goal: To improve vision of wet AMD patients

    Our goal: To improve vision of wet AMD patients  To increase the number of patients who experience a significant gain in vision  To increase the magnitude of the vision gain  To prolong response to therapy and prevent visual decline  Potential to reduce dosing frequency 12

  13. Slide 13: OPT-302 has comparable single-agent and additive activity with Eylea in mouse AMD

    OPT-302 has comparable single-agent and additive activity with Eylea® in mouse AMD Combined inhibition of VEGF-A (Eylea®), VEGF-C and VEGF-D (OPT-302) is more effective than inhibition of VEGF-A alone Control OPT-302 70% Wet AMD lesion area 78% 91% EYLEA™ OPT-302 + EYLEA® * * Pairwise comparison: OP

  14. Slide 14: Ophthotech and Opthea: Distinct approaches for wet AMD combination therapy

    Ophthotech and Opthea: Distinct approaches for wet AMD combination therapy Activity in in mouse wet AMD model p<0.02 p<0.05 Control Fovista® Macugen® Fovista ® + Macugen® FOVISTA™ OPT-302 Preclinical: Activity as Monotherapy? NO YES Preclinical: Activity in Combination? YES YES Potential Use in DME?

  15. Slide 15: OPT-302 Phase 1/2A: Protocol OPT-302-1001

    OPT-302 Phase 1/2A: Protocol: OPT-302-1001 Dose-escalation & dose-expansion of repeated IVT injections Phase 1: Dose-escalation Phase 2A: Dose-expansion (Open-label) (Randomised) OPT-302 (2.0 mg) OPT-302 (2.0 mg) Monotherapy* Monotherapy* Long term follow-up at Week 24 IVT Q4W x 3 IVT Q4W x 3, ~n=15

  16. Slide 16: Phase 1/2A Trial Endpoints

    Phase 1/2A Trial Endpoints Primary Endpoint of Phase 1/2A trial:  Safety Secondary Endpoints:  Preliminary measures of clinical activity  Vision (Eye-Chart)  Size of lesion  Fluid Visual Acuity Anatomical Changes by Imaging (OCT,FA) Eye Chart (Retinal fluid, thickness, area) 16

  17. Slide 17: Clinical Advisory Board & Investigators; Near-term Clinical Milestones

    Clinical Advisory Board & Investigators Near-term Clinical Milestones OPT-302 Wet AMD Program:  Clinical Advisory Board of internationally recognised and Milestones experienced key opinion leaders from Australia and US IND Approval for OPT-302  Extensive experience in development of novel and FDA

  18. Slide 18: OPT-302: Intellectual Property

    OPT-302: Intellectual Property Summary covering sVEGFR-3 IP for Eye Disease COMPOSITION OF MATTER TERM Covering sVEGFR-3 (inc. OPT-302) • Granted Patents: Europe, Japan, Canada, Australia 2022 • Granted Patent: USA 2026 Covering OPT-302 ~2034 • Recently filed new specific composition of matter PCT i

  19. Slide 19: Listed Ophthalmology Companies in Phase 1-3

    Listed Ophthalmology Companies in Phase 1-3 $2.3BN PDGF-B aptamer (Phase 3) Wet AMD Market Cap (US $m) Listed Ophthalmology ROCK inhib., small mol. $593M Companies Ph1-3 Glaucoma (Phase 3) Gene therapy $357M Opthea Eye diseases (Phase 1/2) Ohr Ocular Ther. $226M a-VEGF-A gene therapy (Phase 1/2) Ava

  20. Slide 20: Opthea: Corporate Highlights

    Opthea: Corporate Highlights  Completed $17.4M fundraising (Nov ‘14) to advance wet AMD program  Supported by institutional healthcare investors from US, EU and Australia  Fully funded through 2017 and Phase 1/2A and Phase 2B clinical studies in wet AMD patients  Changed company name (Circadian

  21. Slide 21: OPT-302: Program Highlights

    OPT-302: Program Highlights  OPT-302 is a fully owned asset with broad development potential in a range of eye diseases  Structurally similar to multi-billion dollar marketed product (Eylea), but with differentiated mechanism of action  Targets validated pathway involved in wet AMD progression 

  22. Slide 22: OPT-302 Investor Snapshot

    OPT-302 Investor Snapshot OPT-302: OPT-302 + a-VEGF-A achieves more Asset - Soluble form VEGFR-3 complete blockade of VEGF pathway - A ‘trap’ similar to Eylea™ with distinct MOA Strategy - Targets a mechanism of resistance - Trial to investigate monotherapy and Inhibits VEGF-C & VEGF-D combination s

  23. Slide 23: Megan Baldwin, PhD CEO & Managing Director

    Megan Baldwin , PhD CEO & Managing Director Opthea Limited (ASX:OPT, OTCQX:CKDXY) Megan.baldwin@opthea.com +61 (0) 447 788 674

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