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Company Presentation
MarchMarch
2013 2013
Small Molecules for Better Sight
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 Sight2
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:
Indication
Pre-clinical
Phase 1
Phase 2
Phase 3
Dry Eye Syndrome
Glaucoma
Uveitis*
*Preparatory work for a Phase 2
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
Small Molecules for Better Sight
CF101
F o r t h e Tr e a t m e n t o f
Ophthalmic Diseases
Small Molecules for Better Sight
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
Anti-Inflammatory Effect
Broad anti-inflammatory effect demonstrated in
pre-clinical pharmacology studies :
•
•
•
•
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Rheumatoid Arthritis
Osteoarthritis
Inflammatory Bowel Disease
Uveitis
Pulmonary Inflammation
Sepsis
Excellent Safety Profile
MOA
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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
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 neuroprotective effects on retinal ganglion cells
Prevention of retinal ganglion cells loss both in vitro and in vivo
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
Small Molecules for Better Sight
Dry Eye Syndrome
Results of former Phase 2
study
Small Molecules for Better Sight
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
CF101, n=39
Placebo, n=37
80
P=0.006
CF101
vs
Placebo
70
60
Follow-up
(% patients improvement of >25%)
Success rate
90
50
End of Dosing
40
0
2
4
6
8
10
12
14
Treatment time (week)
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
IOP (% change)
-1
-2
Basline vs Week 12
NS
-3
-4
-5
-6
-7
Basline vs Week 12
P=0.048
IOP ocular
(intrapressure)
ocular pressure)
at week
12 from
in thegroup
CF101-treated
(left column)
IOP (intra
change at change
week 12 from
baseline
in thebaseline
CF101-treated
(left column)group
was statistically
significant
decreased. significant decreased. These data led to the conclusion that CF101 may be utilized for
was statistically
the treatment of Glaucoma
Avni et al. Ophthalmology 2010; 117:1287-93
Small Molecules for Better Sight
Uveitis
Pharmacology Studies
Small Molecules for Better Sight
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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Small Molecules for Better Sight
Development Status
Indication
Status
Dry Eye Syndrome
Ongoing Phase 3
237 patient study
Enrolment completed
Data release - 2013-Q4
Glaucoma
Ongoing Phase 2
132 patient study
Interim Analysis after first 44 patients
Uveitis
Preparatory work for a
Phase 2 study
Small Molecules for Better Sight
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