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Small Molecules for Better Sight

Company Presentation. Company Presentation. March 2013. March 2013. Small Molecules for Better Sight. Forward Looking Statements.

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Small Molecules for Better Sight

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  1. Company Presentation Company Presentation March 2013 March 2013 Small Molecules for Better Sight

  2. Forward Looking Statements Please be advised that the information and projections provided in this presentation may include forward-looking statements with respect to plans, projections or future performance of the Company, the occurrence of which involves certain risks and uncertainties and is not under the control of OphthaliX, including, but not limited to, changes in regulatory environment, OphthaliX‘ success in implementing its research, development, sales, marketing and manufacturing plans, protection and validity of patents and other intellectual property rights, the impact of currency exchange rates and the effect of competition by other companies

  3. Overview • CompanyOphthaliX (OTC: OPLI) is a clinical-stage company focused on the development of ophthalmic drugs • TechnologyTargeted therapy (A3 adenosine receptor) • DrugCF101 is a small molecule orally bioavailable drug which was exclusively licensed from Can-Fite BioPharma (TASE: CFBI; OTC: CANFY) to OphthaliX for the use in the ophthalmic field, and is currently developed for: Phase 3 Indication Pre-clinical Phase 1 Phase 2 Dry Eye Syndrome Glaucoma Uveitis* *Preparatory work for a Phase 2

  4. Platform Technology Target A3 adenosine receptor (A3AR), over-expressed in inflammatory cells Drug Product CF101, an agonist at the A3AR; small orally bioavailable molecule Mechanism of Action Anti-inflammatory and neuro-protective effect towards retinal ganglion cells Therapeutic Index Excellent safety profile and anti-inflammatory effect demonstrated in Phase 2 clinical studies

  5. CF101 For the Treatment of Ophthalmic Diseases

  6. CF101 – Profile Chemical Profile Anti-Inflammatory Effect • Broad anti-inflammatory effect demonstrated in pre-clinical pharmacology studies : • Rheumatoid Arthritis • Osteoarthritis • Inflammatory Bowel Disease • Uveitis • Pulmonary Inflammation • Sepsis • Highly Selective A3AR Agonist • Nucleoside derivative • Molecular weight - 510.29 • Water insoluble • Orally bioavailable • Half life time – 8-9 hours Excellent Safety Profile MOA • ~1,000 patient experience • CF101 is safe and well tolerated • No dose-response relationship is evident between CF101 and adverse events • low incidence (<2%) of serious AEs and similar across all dose groups including placebo

  7. CF101 for Ophthalmic Diseases – Rational • Dry Eye/Uveitis - A3AR agonists have a broad anti-inflammatory effect • Demonstrated in pre-clinical pharmacology studies in Rheumatoid arthritis, Osteoarthritis, Inflammatory Bowel Disease, Uveitis, Pulmonary inflammation and Sepsis • Glaucoma - A3AR agonists induce neuro-protective effects on retinal ganglion cells • Prevention of retinal ganglion cells loss both in vitro and in vivo

  8. CF101 – Ophthalmic Market Opportunity 1 - GlobalData • Dry Eye Syndrome • Dry Eye Syndrome market was valued at $1.9 billion in 2010 and • Forecasted to reach $2.8 billion by 2017 (1) • Glaucoma • Glaucoma market was valued at $3 billion in 2010 (1) • Uveitis • Uveitis market was valued at $0.3 billion in 2010 • Forecasted to reach $1.6 billion by 2017 (1)

  9. Dry Eye Syndrome Results of former Phase 2 study

  10. Phase 2 Dry Eye Syndrome: Successfully Concluded • A Phase 2, Randomized, Double-Masked, Placebo-Controlled, Parallel-Group Study of the Safety and Efficacy of Daily CF101 (1 mg) Administered Orally to Patients with Dry Eye Syndrome Fluorescein staining - Improvement Over Time Follow-up End of Dosing CF101-treated group (blue line) as compared to placebo (pink line) over a 12-weeks dosing period. The difference between the group is clearly apparent and significant (p=0.006). The measurement made at week 14, which is 2 weeks post dosing, shows a clear reduction in effect. Avni et al. Ophthalmology 2010; 117:1287-93

  11. CF101 for Glaucoma Treatment IOP decrease documented in the phase II dry eye study led to the initiation of a phase II study utilizing CF101 for the treatment of Glaucoma IOP (intra ocular pressure) change at week 12 from baseline in the CF101-treated group (left column) was statistically significant decreased. These data led to the conclusion that CF101 may be utilized for the treatment of Glaucoma IOP (intra ocular pressure) change at week 12 from baseline in the CF101-treated group (left column) was statistically significant decreased. Avni et al. Ophthalmology 2010; 117:1287-93

  12. Uveitis Pharmacology Studies

  13. CF101 for the Treatment of Uveitis: Pharmacology Studies Anti-inflammatory effect of CF101 Posterior Uveitis Inhibition of EAU by CF101: fundoscopy. Mice were immunized with a uveitogenic regimen of IRBP and treated with CF101 from Day 7 after immunization. Eyes were examined for disease after pupil dilation under a binocular microscope. Shown are results on Day 20 following immunization. Bar Yehuda et al, Int J Mol Med. 2011; 28(5):727-3 13

  14. Development Status

  15. Contact Information OphthaliX Inc. 10 Bareket Street Kiryat Matalon Petah-Tikva 49170 Israel info@ophthalix.com +972-3-9241114 For additional information, please contact: Phil Carlson / Diane Imas KCSA Strategic Communications pcarlson@kcsa.com / dimas@kcsa.com 212-896-1233 / 212-896-1242

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