Andrew Rae, President & CEO / TSX-V: ICO

Transcription

1 ico Therapeutics Andrew Rae, President & CEO / TSX-V: ICO

2 Forward Looking Statements Certain of the statements contained in this presentation are forward-looking statements which involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements of the Company, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. 2

3 About ico Therapeutics Targeting ocular diseases Lead program: Pipeline: Partnerships: Ph 2 for Diabetic Macular Edema (DME) - Human Monoclonal Antibody Targeting eotaxin-1 - Oral Amphotericin B Delivery System JDRF, ISIS, AstraZeneca MedImmune & Immune Pharmaceuticals Efficient use of capital Experienced management & board 3

4 Validated Therapeutic Arena Pfizer Lpath >$500 M (wamd) Santen Macusight $50 M upfront + undisclosed milestones ( wamd & DME) Novartis Genentech Undisclosed (wamd & DME) Bayer Regeneron >$320 M (wamd & DME) Sanofi-Aventis Fovea 370 M (DME & Other Ophthalmic Therapies) Alcon ThromboGenics 375 (VMA & Other Ophthalmic Therapies) Alcon ESBATech $589 M (Ophthalmic Platform) Novartis Alcon $12.9 B (Ocular Drug and Device Pipeline) Allergan Molecular Partners $1.4 B (wamd) DME = diabetic macular edema wamd = wet age-related macular degeneration 4

5 ico 007 Novel 2 nd generation antisense candidate for the treatment of Diabetic Macular Edema Partner: Isis Pharmaceuticals

6 Diabetic Macular Edema (DME) Leading cause of blindness in working age adults Abnormal Retina Proliferation of new, permeable blood vessels allows blood to leak into retinal area, causing swelling and deformation Current treatments: Steroids Laser Anti-VEGF Normal Retina 6

7 ico-007: MOA - VEGF Plus Growth factors Modulate signal ico-007 inhibits the production of c-raf, thereby preventing the signaling of growth factors, which in turn prevents the production of new and permeable blood vessels Retina 7

8 ico-007 & C-Raf Kinase Recent results have shown that the pathways activated by Ras/Raf play a crucial role in diabetes-associated complications including retinopathy. 8

9 Phase 1: Patient Examples Single Injection 110ug dose (sample patient) 350ug dose (sample patient) 700ug dose (sample patient) 1000ug dose (sample patient) Bas eline 528 microns 391 microns 867 microns 423 microns 6 months 379 microns 228 microns 124 microns 187 microns Delta: 149 microns Delta: 163 microns Delta: 743 microns Delta: 236 microns The number of patients in study is low, statistical significance cannot be inferred 9

10 ideal Physician-sponsored US Phase 2 Randomized, multi-center Up to 208 patients 4 Study Groups Monotherapy dose 1 (350 ug) Monotherapy dose 2 (700 ug) Combination (350 ug) with Ranibizumab Combination (350 ug) Laser Photocoagulation 174 patients treated (April 26) Primary endpoint Change in visual acuity at 8 months Secondary endpoints 12 month follow up Visual acuity & reduction of retinal thickness Duration of effect Safety of repeated injections Pharmacokinetic assessments (PK) 10

11 Competitive Landscape Category Product Device Anti-VEGF Steroids Laser Avastin Macugen Eylea Lucentis (approved) Iluvien, Ozurdex Triamcinolone, etc. Current Product Profile Issues Treatment burden Side effects Cost Biological targets 11

12 ico-007 Product Differentiation Novel c-raf kinase-targeted antisense specific for DME Targets more than just VEGF: Existing competitors target VEGF only, while DME is caused by multiple growth factors Longer half life = fewer injections: Competitors require monthly injections costly, time consuming and a logistical burden on both patients and physicians Easy to manufacture No known side effects comparable to select competitors 12

13 Market Opportunity U.S. Diabetes Patients 21 million 40 million 10% Penetration % of diabetes patients report visual impairment (5.3 million Diabetic Retinopathy patients today) (1.6 million DME patients today) $ $10,000 per annual treatment (only clinically significant cases) *Lucentis = $24,000 annually M 13

14 ico-007 Next Steps Received Health Canada Clearance for Phase 2 Completed fill finish manufacturing to produce clinical supply US FDA Clearance for Phase 2 US Phase 2 trial initiated Clinical Update Primary endpoint from Ph 2 ideal study at 8 months Ongoing regional partnership discussions 14

15 ico 008 Human monoclonal antibody treating both the back and front of the eye Partners: AstraZeneca/MedImmune & Immune Pharmaceuticals

16 ico-008 Human monoclonal antibody targeting ti eotaxin-1 Binds with high affinity to CCR3 Good safety & significant clinical history Ph 1 & 2 (n=126) Ocular: Vernal & Atopic Keratoconjunctivitis, Wet Age Related Macular Degeneration Systemic: Inflammatory Bowel Disease, Severe Asthma (License granted to Immune Pharmaceuticals) 16

17 ico 008 Licensing: MedImmune & Immune In-licensing Out-licensing $400, up front payment $500, upfront received $7,000,000 milestone payments $32,000,000 incoming milestones Exclusive rights to entire e body 600,000 Immune shares 200,000 Immune warrants 6.14% ownership with dilution provisions* Royalties on net sales Retained WW rights to all ocular applications i Immune has initiated a Ph 2, double-blind, placebo controlled study with ico-008 in 90 patients with moderate-to-severe ulcerative colitis. Completion of patient enrollment and clinical results are anticipated in * As of the date of the transaction, June 24,

18 Oral Amphotericin B Delivery System Partner: The University of British Columbia

19 Amphotericin B & IV Delivery IT WORKS BUT NOT PRACTICAL IN MANY SITUATIONS Gold standard AmBisome >$400M in sales Premium pricing for safety Variety of Developed World fungal infections Lack of resources in Developing World Inconvenient unmet need: oral formulation 19

20 Proprietary Oral Delivery Platform Lipid capsule Candy Wrapper Proprietary platform Used for insoluble products including vaccines and proteins Expanding the Amp B market: Safety and convenience, HIV Oral treatment for : Goal of WHO & Gates Foundation, Governments & several other global health organizations Non-dilutive funding to date: CPDD/Gates and CIHR, NRC 20

21 Oral Amphotericin B Delivery System Studies HIV/AIDS currently managed using a combination of drugs (HAART) HIV often remains hidden in tissue reservoirs Amphotericin B may be efficient at flushing out latent HIV in reservoirs $1.1 million in non-dilutive grant funding from Canadian government/gates Foundation Next steps: feasibility testing, further pre-clinical studies and clinical trials 21

22 Financials & Corporate Review

23 ico Pipeline 23

24 Upcoming Milestones Milestone Timing Completed ico 007: Ph 2 ideal study initiated ico 008: Licensed systemic rights to Immune Pharma $32+ Million Amp B: Non-dilutive grant funding ico 007: Ph 2 ideal study clinical update Amp B: In vitro testing Q ico 007: Visual acuity at 8 month primary end point in Ph 2 ideal study Q ico 007: Regional partnership discussions Ongoing ico 008: Potential non-dilutive funding of ocular programs Ongoing 24

25 Management and Directors Management Andrew Rae, MBA Co-founder, Director, President & CEO Peter Hnik, MD, MHSc. Chief Medical Officer John Meekison, BA, CIM, P. Log. Co-founder & Chief Financial Officer Non-Executive Directors William Jarosz, JD Chairman of the Board, ico Cartesian Capital Group, LLC Richard Barker, PhD Former Director General of the Association of the British Pharmaceutical Industry, Celgene director Noel Hall Co-founder of Aspreva Douglas Janzen Former President & CEO, Cardiome Strategic Advisory Board Donald Buell, MD ico-009 SAB Chair, Former Senior Medical Director, Astellas USA George Lasezkay, JD Principal, Turning Point Consultants, LLC, Former VP Corp Development, Allergan Extensive public company and life science experience Solid operational and product development expertise Ophthalmic hhl specific expertise 25

26 Financials (as of December 31, 2012) Invested Capital to Date $22.5 million Largest Shareholders Isis Pharmaceuticals (9.2%) Special Situations Funds (~9%) Management (85%) (8.5%) Cash $1.26 million Cash Runway Mid-2013 Share Capital 54.7M (SO) 67.5M (FD) Head Office Vancouver BC, Canada Filings available at 26

27 ico Therapeutics ico Therapeutics TSX-V: ICO