OPKO Health 8K to Acquire Two Phase 3 Products | OPK 9 Jan 13

Corporate Presentation

January 2013

Exhibit 99.2

Cytochroma 2011

Forward-Looking Statements

Certain statements and information included in this presentation are “forward-looking

statements” under the Private Securities Litigation Reform Act of 1995 (PSLRA), including

expectations regarding (1) the market opportunity and growth potential of the CKD patient

population, (2) how to reach Cytochroma’s target physician audience, (3) new potential

indications for the CTAP101 Capsules, (4) the commercial opportunity for the CTAP101

Capsules, (5) the Phase 3 and NDA timelines for CTAP 101 through 2015, (6) the pipeline

for clinical programs for CKD patients and early stage pipeline products, and (7) the U.S.

market opportunity for Cytochroma’s product candidates generally. Many factors could

cause actual activities or results to differ materially from the activities and results

anticipated in these forward-looking statements. These factors include that the various

conditions to the closing of the transaction between Opko and Cytochroma may not be met,

as well as risks inherent in funding, developing and obtaining regulatory approvals of new,

commercially-viable and competitive products and treatments. In addition, these forward-

looking statements may also be adversely affected by scientific developments relating to

chronic kidney disease, general market factors, competitive product development, product

availability, federal and state regulations and legislation, the regulatory process for new

products and indications, manufacturing issues that may arise, patent positions and

litigation, among other factors.

Cytochroma 2011

A clinical stage specialty pharmaceutical

company focused on Chronic Kidney Disease

Cytochroma 2011

To Improve People’s Lives by Treating and Preventing

Clinical Consequences of Vitamin D Insufficiency and Secondary

Hyperparathyroidism

Company Overview

Mission:

Cytochroma 2011

To Improve People’s Lives by Treating and Preventing

Clinical Consequences of Vitamin D Insufficiency and Secondary

Hyperparathyroidism

Company Overview

Mission:

Cytochroma 2011

Product

Indication

Research

Preclinical

Phase I

Phase II

Phase III

Territorial

Rights

CTAP101

Capsules

Low 25D and SHPT*

(CKD Stage 3-4 Patients)

Worldwide

Fermagate

Tablets

Hyperphosphatemia

(CKD Stage 5 Patients)

Worldwide

CTA018

Injection

Moderate to Severe SHPT*

(CKD Stage 5 Patients)

Worldwide

CTAP201

Injection

Mild to Moderate SHPT*

(CKD Stage 5 Patients)

Worldwide

Phosphate Transport

Inhibitors

Hyperphosphatemia

Worldwide

CYP24 Inhibitors

CKD and pre-CKD

Worldwide

SHPT = Secondary Hyperparathyroidism

Cytochroma Product Pipeline

A clinical-stage specialty pharmaceutical company focused on CKD

Cytochroma 2011

Vitamin D: Central To SHPT Etiology & Treatment

Vitamin D

prohormone

Liver Enzyme

Vitamin D

hormone

Kidney Enzyme

Parathyroid

hormone

Target Tissues

(Parathyroid gland)

Vitamin D Hormone Replacement Therapies

(Paricalcitol, Doxercalciferol, Calcitriol)

Hyperphosphatemia

Hypocalcemia

Ca-Free Binder Therapies

CalcimimeticTherapies

Ca-Based Binder Therapies

Vitamin D Repletion

Therapies

CYP24 Inhibitor

Therapies

Inactivated

Vitamin D

CYP24 Enzyme

CTAP101 Capsules

Light blue boxes indicate

Cytochroma’s areas of focus

Fermagate Tablets

Cytochroma 2011

Market Opportunity: Chronic Kidney Disease (U.S.)

*National Kidney Foundation 2002

**US Renal Data Service 2009 Annual Data Report

Sources: Levin, A et al., Kidney International 2007; 71: pp.31-38.

Gonzalez, E et al. Am J Nephrol 2004;24:503-510.

LaClair, R et al. Am J Kidney Dis 2005;45:1026-1033.

A 75-100 person specialty salesforce can readily reach Cytochroma’s target

physician audience:

Primary commercialization targets include 4,500-6,000 office-based nephrologists and 1,500-

3,000 endocrinologists

Other commercialization targets include geriatricians and 4,500 dialysis clinics

The CKD patient population is large and growing as a result of

cardiovascular disease, obesity and diabetes

Current U.S. sales are derived predominantly from Stage 5 CKD:

•

Approximately $1.5 billion for SHPT therapies

•

Approximately $0.8 billion for hyperphosphatemia therapies

CKD Stage 3-4 patients represent a significantly greater, and largely untapped market opportunity

% of CKD Patients with:

Stage

Kidney Function

CKD Prevalence

Vitamin D

SHPT

Hyperphosphatemia

(

Phosphorus)

Moderate impairment

7.6 Million*

70%

56%

37%

Severe impairment

0.4 Million*

80%

60%

50%

Failure

0.5 Million**

90%

70%

Insufficiency (

25D)

(

PTH)

Cytochroma 2011

Phase 2b study complete and reported strong

efficacy and safety data

High proportion of patients achieved increase

in 25D levels to target, as well as 30%

reduction in PTH from baseline

Modified-release

(MR) formulation of 25D*

Safe and effective treatment for elevated PTH

(SHPT) associated with low 25D levels in Stages

3-4 CKD

Achieves more reliable increases in serum 25D

and reductions in plasma PTH than nutritional

vitamin D

Lower risk of side effects compared to active

1,25D** products

Preserves protective renal feedback mechanism

Additional potential for new indications in:

•

Geriatric patients with low 25D levels and

elevated PTH

•

Patients with GI or malabsorptive disorders

•

Osteoporosis

•

Organ transplant recipients

CTAP101 Capsules

Product Overview

Clinical Status

Next Steps

End of Phase 2 meeting with FDA in Q1 2012

Phase 3 trials started in H2 2012 under SPA

505(b)(2) NDA filing in H1 2015

Intellectual Property

Formulation and method of use patents pending

CTAP101 US patent issued, protected through 2028

25-Hydroxyvitamin D3

1,25-Dihydroxyvitamin D

Cytochroma 2011

Doxercalciferol Capsules

(Hectorol)

1,25D Hormones:

SHPT only

Nutritional Vitamin D:

SHPT and Low 25D

(Off-label)

* K/DOQI -

Kidney Disease Outcomes Quality Initiative is issued by the National

Kidney

Foundation

and

are

practice

guidelines

for

all

stages

chronic

kidney

disease

and related complications.

Limitations of Existing Therapies

Company

Product

Use

Limitations

Upregulate CYP24, which catabolizes both

administered therapy as well as endogenous 25D

and 1,25D

Limited systemic availability due to sequestering

in fat tissue

Inconsistent effects, dependant on GI functionality

and bile production

Limited efficacy to increase serum 25D levels and

lower plasma PTH to K/DOQI* target levels

Paricalcitol Capsules

(Zemplar)

Calcitriol Capsules

(Rocaltrol /Vectical)

Generic

Manufacturers

Ergocalciferol Capsules

(Drisdol)

Ergocalciferol Drops

(Drisdol)

Bypass protective renal feedback mechanism of

downregulating CYP27B1

Cause aberrations in local and systemic calcium

and phosphorus metabolism, leading to side

effects such as hypercalcemia,

hyperphosphatemia and soft tissue calcification

Cannot correct and may worsen low 25D levels

CTAP101 consistently increases serum 25D and achieves meaningful reductions in plasma

PTH without safety concerns

Cytochroma 2011

The Great Unmet Medical Need

Percent of Nephrologists

Nephrologists report a significant need for a therapy that can raise serum

25D levels and suppress plasma PTH levels

Nearly 60% of nephrologists frequently or always measure 25D levels in CKD Stage 3-4 patients

Nearly 90% of nephrologists use nutritional vitamin D in CKD Stage 3-4 patients

Over 90% of nephrologists agree that nutritional vitamin D has “limited efficacy to raise 25D and lower PTH”

Over 90% of nephrologists agree that 1,25D hormones have “risks of side effects which limit dose and

efficacy”

Over 90% of nephrologists report a moderate-to-high need for a therapy that can safely and effectively raise

25D levels and suppress PTH in CKD Stage 3-4 patients

* As assessed through syndicated market research by BioTrends and proprietary research by Mattson Jack

45%

48%

100%

How much need is

there for a single

therapy that can

safely and effectively

raise 25D levels AND

suppress PTH levels

to target in CKD-ND

patients?

Low Need

Moderate Need

High Need

Cytochroma 2011

CTAP101: Significant Commercial Opportunity

BioTrends Research Group, Inc. December 2010

Low serum 25D and high plasma PTH are prevalent in CKD Stage 3-4 patients

•

8.0M CKD Stage 3-4 patients in the U.S.

4.0M patients with low serum 25D and high plasma PTH

~1.0M patients seen by nephrologist

~1.0M patients seen by endocrinologist

Existing

treatments

are

not

effective

have

significant

safety

issues

CTAP101’s efficacy and safety results compare favorably to both nutritional vitamin D and 1,25D hormones in CKD stage 3-4

patients and will drive untreated patients to start on CTAP101 Capsules

Stage 3 CKD Treatment

Stage 4 CKD Treatment

Untreated

44%

1,25D

Hormone

20%

Nutritional

Vitamin D

36%

Nutritional

Vitamin D

38%

Untreated

26%

1,25D

Hormone

36%

Safety concerns

and exacerbates

vitamin D

insufficiency

Efficacy Concerns

CTAP101 Capsules is expected to take significant market share (35% to 50%) in the

CKD

Stage

and

market

–

potential

$1B

revenue

opportunity

Cytochroma 2011

CTAP101: Corrects Vitamin D Insufficiency

Cytochroma 2011

CTAP101: Corrects SHPT (Elevated Plasma iPTH)

Cytochroma 2011

CTAP101 Capsules reliably increase serum 25D and reduce plasma PTH

CTAP101 Capsules are safe in CKD Stage 3-4 patients

CTAP101’s efficacy and safety compare favorably to competing products

No competing products can consistently normalize 25D levels and produce significant reductions

in PTH without safety concerns

The efficacy of OTC vitamin D at raising 25D or lowering PTH is modest; results are mixed and a

relatively low percentage of patients respond

1,25D (calcitriol) doses that significantly reduce PTH (0.5 mcg/day) produce significant increases

in serum calcium and cause hypercalcemia

CTAP101 Capsules: Phase 2b Summary

Repeat dose proof of concept

No clear dosing regimen for OTC vitamin D exists

Lower 1,25D doses (0.25 mcg/day) do not produce consistent, meaningful reductions

in PTH

1,25D analogues can significantly reduce PTH about the same amount as CTAP101

Capsules, but cause elevations in serum and urine calcium which raise safety concerns

and limit their use

Cytochroma 2011

CTAP101: Phase 3 and NDA Timelines

2012

2013

2014

NDA

Filing

CL-3002

Data

CL-3001 trial

SPA

Open Label Extension

2015

CL-3002 trial

NDA

Safety

Update

NDA

Approval

TG Mouse Carcinogenicity Study

Dose ranging

SPA

CL-3001

Data

Cytochroma 2011

CTAP101-CL-3001 & CL-3002 Combined Site Map

= Selected investigator sites for study 3001 (33 Sites)

= Selected investigator sites for study 3002 (30 Sites)

Cytochroma 2011

Corporate Highlights

CTAP101 Capsules is a Highly Differentiated Lead Asset

•

First-in-class modified-release vitamin D prohormone that both corrects vitamin D insufficiency and

controls SHPT* in patients with stage 3 or stage 4 CKD

current

therapy

can

reliably

restore

adequate

serum

25D**

and

suppress

elevated

PTH***

•

Compelling Phase 2b data in hand; Phase 3 program ongoing under SPA.

•

USPTO recently issued a patent covering the product until ‘28

•

NDA filing expected in 1H’15

Broad and Deep Pipeline

•

Two Phase 2 and two Phase 3 clinical programs, all for CKD patients

•

Early stage pipeline includes new inhibitors of phosphate transport in the GI tract, as well as CYP24

Large Market Opportunity

•

Cytochroma’s product candidates address $2.3 billion market in the U.S.

•

Vitamin D insufficiency affects 70-80% of the 8 million CKD Stage 3-4 patients in the U.S.

SHPT = Secondary hyperparathyroidism

25D = 25-hydroxyvitamin D

***

PTH = parathyroid hormone

Corporate Presentation

January 2013

OPKO Health (OPK) 8-KOpko Health to Acquire Two Phase 3 Products