Small Molecules for Better Sight

Small Molecules for Better SightSmall Molecules for Better Sight

March 2013

Company Presentation

March 2013

Company Presentation

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


Overview • Company

OphthaliX (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:

Dry Eye Syndrome

Glaucoma

Uveitis*

Indication Pre-clinical Phase 1 Phase 2 Phase 3

*Preparatory work for a Phase 2


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


CF101Fo r t h e Tre at m e nt o f O p ht h a l m i c D i s e a s e s


CF101 – Profile

• Highly Selective A3AR Agonist

• Nucleoside derivative • Molecular weight -

510.29 • Water insoluble• Orally bioavailable• Half life time – 8-9

hours

Chemical Profile Anti-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


CF101 for Ophthalmic Diseases – Rational

• Dry Eye/Uveitis - A3AR agonists have a broad anti-inflammatory effectDemonstrated 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


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


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


Phase 2 Dry Eye Syndrome: Successfully Concluded

40

50

60

70

80

90

0 2 4 6 8 10 12 14

Treatment time (week)

Succ

ess

rate

(% p

atie

nts

impr

ovem

ent o

f >25

%) CF101, n=39

Placebo, n=37

P=0.006CF101

vsPlacebo

Fluorescein staining - Improvement Over Time

Follo

w-u

p

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


CF101 for Glaucoma Treatment

IOP - % change

-7

-6

-5

-4

-3

-2

-1

0CF101 Placebo

IOP

(% c

hang

e)

Basline vs Week 12P=0.048

n=35 n=36

Basline vs Week 12NS

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


Uveitis P h a r m a co l o g y S t u d i e s


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


Development Status

Indication Status

Dry Eye Syndrome Ongoing Phase 3 237 patient studyEnrolment completedData release - 2013-Q4

Glaucoma Ongoing Phase 2 132 patient studyInterim Analysis after first 44 patients

Uveitis Preparatory work for a Phase 2 study


OphthaliX Inc. 10 Bareket Street Kiryat MatalonPetah-Tikva49170 [email protected]+972-3-9241114

For additional information, please contact:

Phil Carlson / Diane ImasKCSA Strategic [email protected] / [email protected] / 212-896-1242

Contact Information

Small Molecules for Better Sight

Documents

Transcript of Small Molecules for Better Sight