Transcript Presentation
Company Presentation
Company Presentation
Molecules for Better
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
Small Molecules for Better Sight
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Overview
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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: Indication Dry Eye Syndrome Glaucoma Uveitis* *Preparatory work for a Phase 2 Pre-clinical Phase 1 Phase 2 Phase 3
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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
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CF101
F o r t h e Tr e a t m e n t o f O p h t h a l m i c D i s e a s e s
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CF101 – Profile
Chemical Profile
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Highly Selective A3AR Agonist Nucleoside derivative Molecular weight - 510.29 Water insoluble Orally bioavailable Half life time – 8-9 hours MOA Anti-Inflammatory Effect
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Broad anti-inflammatory effect demonstrated in pre-clinical pharmacology studies : Rheumatoid Arthritis Osteoarthritis Inflammatory Bowel Disease Uveitis Pulmonary Inflammation Sepsis Excellent Safety Profile
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~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
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CF101 for Ophthalmic Diseases – Rational
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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
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CF101 – Ophthalmic Market Opportunity
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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
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Dry Eye Syndrome
R e s u l t s o f f o r m e r P h a s e 2 s t u d y
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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
90 CF101, n=39 Placebo, n=37 80 70 60 P=0.006
CF101 vs Placebo 50 40 0 2 4 6 8 10 Treatment time (week) End of Dosing 12 14
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 Small Molecules for Better Sight
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 - % change CF101 Placebo 0 n=35 n=36 -1 -2 Basline vs Week 12 NS -3 -4 -5 -6 -7 Basline vs Week 12 P=0.048
IOP (intra ocular pressure) change at week 12 from baseline in the CF101-treated group (left column) significant decreased. the treatment of Glaucoma Avni et al. Ophthalmology 2010; 117:1287-93 Small Molecules for Better Sight
Uveitis
P h a r m a c o l o g y S t u d i e s
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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
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Development Status
Indication Dry Eye Syndrome Glaucoma Uveitis Status Ongoing Phase 3
237 patient study Enrolment completed Data release - 2013-Q4
Ongoing Phase 2
132 patient study Interim Analysis after first 44 patients Preparatory work for a
Phase 2 study
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Contact Information
OphthaliX Inc.
10 Bareket Street Kiryat Matalon Petah-Tikva 49170 Israel [email protected]
+972-3-9241114
For additional information, please contact: Phil Carlson / Diane Imas KCSA Strategic Communications [email protected] / [email protected]
212-896-1233 / 212-896-1242
Small Molecules for Better Sight