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

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Presentation on theme: "Small Molecules for Better Sight March 2013 Company Presentation March 2013 Company Presentation."— Presentation transcript:

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

2 Small Molecules for Better Sight 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 Forward Looking Statements 2

3 Small Molecules for Better Sight Overview Company OphthaliX (OTC: OPLI) is a clinical-stage company focused on the development of ophthalmic drugs Technology Targeted therapy (A3 adenosine receptor) Drug CF101 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: Dry Eye Syndrome Glaucoma Uveitis* Indication Pre-clinicalPhase 1Phase 2 Phase 3 *Preparatory work for a Phase 2

4 Small Molecules for Better Sight 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 Small Molecules for Better Sight CF101 For the Treatment of Ophthalmic Diseases

6 Small Molecules for Better Sight CF101 – Profile Highly Selective A3AR Agonist Nucleoside derivative Molecular weight - 510.29 Water insoluble Orally bioavailable Half life time – 8-9 hours Chemical ProfileAnti-Inflammatory Effect MOA Broad anti-inflammatory effect demonstrated in pre-clinical pharmacology studies : Rheumatoid Arthritis Osteoarthritis Inflammatory Bowel Disease Uveitis Pulmonary Inflammation Sepsis Excellent Safety Profile ~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 Small Molecules for Better Sight 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 Small Molecules for Better Sight CF101 – Ophthalmic Market Opportunity – 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) 1 - GlobalData

9 Small Molecules for Better Sight Dry Eye Syndrome Results of former Phase 2 study

10 Small Molecules for Better Sight Phase 2 Dry Eye Syndrome: Successfully Concluded 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 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

11 Small Molecules for Better Sight CF101 for Glaucoma Treatment 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 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

12 Small Molecules for Better Sight Uveitis Pharmacology Studies

13 Small Molecules for Better Sight CF101 for the Treatment of Uveitis: Pharmacology Studies 13 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 Posterior Uveitis Anti-inflammatory effect of CF101

14 Small Molecules for Better Sight Development Status IndicationStatus Dry Eye SyndromeOngoing Phase 3 237 patient study Enrolment completed Data release - 2013-Q4 GlaucomaOngoing Phase 2 132 patient study Interim Analysis after first 44 patients UveitisPreparatory work for a Phase 2 study

15 Small Molecules for Better Sight 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 Contact Information


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