OPKO HEALTH, INC. - FORM 8-K - EX-99.1

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8-K - 8-K - OPKO HEALTH, INC.	d851650d8k.htm

Annual J.P. Morgan Healthcare Conference

January 2015

Exhibit 99.1

Cautionary Statement

This presentation contains "forward-looking statements,” as that term is defined under the Private Securities Litigation Reform Act of 1995

(PSLRA), which statements may be identified by words such as “expects,” “plans,” “projects,” “will,” “may,” “anticipates,” “believes,”

“should,” “intends,” “estimates,” "potential" and other words of similar meaning, including statements regarding our estimated revenues

and financial projections, our ability to achieve high levels of growth, the potential for our products under development, the potential of the

4Kscore™ to reduce prostate biopsies by 30-58% and predict the risk of aggressive prostate cancer, our ability to develop, test and launch

new products, the expected timing of the clinical studies and regulatory submissions relating to our products under development, the

outcome of our clinical trials and validation studies and that such outcomes will support commercialization, the expected market penetration

and size of the market for our products under development, including without limitation, Rolapitant™, Rayaldee™ (CTAP-101), hGH-CTP™,

the 4Kscore, and our point-of-care diagnostic products for Total-PSA, testosterone, and Vitamin D, the potential benefits of our products

under development, including whether the 4Kscore will improve selection of candidates for prostate biopsy, predict the risk of distant

metastases, and result in $2 to 4 billion in healthcare savings, whether MOD-6031 will provide superior long-term therapy for obesity and

Type II diabetes patients, our ability to successfully commercialize our product candidates such as Rolapitant, the 4Kscore, Rayaldee (CTAP-

101) and hGH-CTP, as well as products for other markets such as urology, women’s health, cardiology, oncology, iPTH, and infectious

disease, whether we will be able to develop Rayaldee (CTAP-101) for additional indications and whether Rayaldee (CTAP-101) will take

significant market share in Stage 3 and 4 CKD patients with SHPT, whether Rayaldee (CTAP-101) will raise serum total 25-hydroxyvitamin D

(25D) more effectively than any over-the-counter (OTC) or prescription (Rx) product currently marketed without the risk of hypercalcemia,

whether we can reach more than half of the CKD population with a small sales force, expectations regarding patent coverage, the expected

timing for commencing, completing and obtaining results for our clinical trials, the timing for release of trial data and seeking and obtaining

FDA and European regulatory approvals, and the timing of commercial launch of our product candidates, as well as other non-historical

statements. These forward-looking statements are only predictions and reflect our views as of the date they were made, and we undertake

no obligation to update such statements. Such statements are subject to many risks and uncertainties that could cause our activities or actual

results to differ materially from the activities and results anticipated in forward-looking statements, including risks inherent in funding,

developing and obtaining regulatory approvals of new, commercially-viable and competitive products and treatments, general market

factors, competitive product development, product availability, federal and state regulations and legislation, and integration issues arising

from the transactions, delays associated with development of novel technologies, unexpected difficulties and delays in validating and testing

product candidates, the regulatory process for new products and indications, manufacturing issues that may arise, the cost of funding

lengthy research programs, the need for and availability of additional capital, the possibility of infringing a third party’s patents or other

intellectual property rights, the uncertainty of obtaining patents covering our products and processes and in successfully enforcing them

against third parties, and the possibility of litigation, among other factors, including all of the risks identified under the heading Risk Factors

in our Annual Report on Form 10-K and other filings with the Securities and Exchange Commission.

OPKO Health

Focus on Large Market Potential Products

Diagnostics

–

Claros

1 immunoassay system for rapid, lab quality in-office testing

–

4Kscore™

blood test for aggressive prostate cancer risk

Pharmaceuticals

–

New Vitamin D products for SHPT

–

Platform technologies to make peptides and proteins long-acting

for growth hormone deficiency, hemophilia, obesity

–

Calcium-free, magnesium-based phosphate binder

–

Approved third generation hepatitis B vaccine

Opportunistic

Investments

–

Innovative technologies

Antibodies

RNAi

Anti-virals

Cardiovascular devices

International

Markets

–

Established businesses in:

Mexico

Chile

Spain

Brazil

Israel

Uruguay

4Kscore Test to Identify Risk for Aggressive

Prostate Cancer

•

Two types of prostate cancer:

–

Low grade, non-lethal

–

Aggressive, lethal

•

4Kscore: ONLY

blood test to predict risk of aggressive form

•

Backed by 10 years of clinical research at Memorial Sloan-

Kettering Cancer Center

•

Validated by OPKO in a prospective, blinded study of 1,012

men

•

Global market for pre-biopsy test (not screening): $800M

•

Use of 4Kscore would reduce 30-58% of unnecessary prostate

biopsies

•

Healthcare savings of $2-4 billion in the US

4Kscore Commercial Update

•

4Kscore US clinical trial manuscript in press (Eur. Urol.)

•

4Kscore CPT code application accepted by AMA CPT

Editorial Board (MAAA Category 3)

•

4Kscore was discussed at NCCN Guidelines meeting

Nov. 2014 for potential inclusion in 2015 guidelines

•

Over 465 US urologists have used the 4Kscore test in

routine practice

•

Launched by OPKO in Europe September 15, 2014;

samples tested at University of Barcelona –

barnaclinic+

•

Mexico launch in January 2015; samples will be tested at

OPKO Lab in Nashville, TN

Claros

1: Rapid Testing in the Physician Office

mins

Finger stick blood

sample

1-2

mins

Claros 1 Platform Addresses Large Dx Markets

•

Testosterone

–

15 million US tests: $525 M

–

510(k) filing in 2Q2015

•

PSA

–

30 million US tests: $750 M

–

Intended use to focus on screening (early detection) claim

–

PMA filing in 1Q2016

•

Vitamin D

–

70 million US tests: $3.5 B

–

On track to support launch of Rayaldee 1Q2016

•

Lyme Disease

–

Claros 1 selected as platform for NIH funded development

–

OPKO has exclusive option to diagnostic test ($120M US Market)

OPKO Pharmaceuticals —

Advanced, Deep Pipeline

Product

Indication

Preclinical

Phase 1

Phase 2

Phase 3

Milestone

Market Size

Rayaldee™

(CTAP101)

SHPT

(CKD Stage 3-4 Patients)

Phase 3 results

reported

3Q 2014

$12.0 BN

hGH-CTP

hGH deficiency

$3.5 BN

Alpharen™

(Fermagate)

Hyperphosphatemia

(CKD Stage 5 Patients)

$1.2 BN

Rolapitant

CINV

NDA accepted

November, 2014

$1.5 BN

Sci-B-Vac™

Hepatitis B

(CKD Stage 5 Patients)

$0.2BN

Lunacalcipol™

(CTA018)

Moderate to severe

SHPT

(CKD Stage 5 Patients) &

Psoriasis

$1.5 BN

CTAP201

Mild to moderate SHPT

(CKD Stage 5 Patients)

$1.1 BN

Factor VIIa-CTP

Hemophilia

$1.7 BN

AntagoNAT

Platform

Cancer, CV, metabolic

and orphan disease

$1.0 BN

Oxyntomodulin

Diabetes, Obesity

$15 BN

Collaboration with Pfizer

Outlicensed to TESARO

Rayaldee (CTAP101) –

A Late-Stage Investigational Drug

Product Overview

•

Oral formulation of 25D

addresses significant unmet

need

•

Achieves reliable increases in

serum 25D and reductions in

plasma PTH in Stage 3-4 CKD

•

Lower risk of side effects

compared to active 1,25D

products

•

Phase 1 cancer trail begun

Clinical Status

Intellectual Property

* 25-Hydroxyvitamin D3

** 1,25-Dihydroxyvitamin D

•

Clinical development guided

by prominent Scientific

Advisory Board

•

Positive Phase 3 data

•

NDA submission target:

1Q2015

•

Rayaldee US patents issued,

protected through 2028

•

Additional global patents

allowed or pending

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

*US Renal Data Service 2013 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.

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

•

Obesity

•

Hypertension

•

Diabetes

Over half of the CKD population can be reached with a small

sales force targeting nephrologists and endocrinologists

% of CKD Patients with:

Stage

Kidney Function

CKD Prevalence

Vitamin D

Insufficiency( 25D)

SHPT

( PTH)

Hyperphosphatemia

( Phosphorus)

Moderate impairment

18.7

Million*

70%

56%

37%

Severe impairment

1.4 Million*

80%

60%

50%

Failure

0.5 Million*

90%

70%

Rayaldee -

Commercial Opportunity

Source: BioTrends Research Group, Inc. December 2013

Untreated

26-44%

Vitamin D

Hormone

14-25%

Nutritional

Vitamin D

39-44%

Untreated

31-47%

Safety

concerns;

exacerbates

vitamin D

insufficiency

Efficacy

Concerns

Stage 3 & 4 CKD Treatment

Low

serum

25D

and

elevated

plasma

PTH

are

prevalent

CKD

Stage

3-4

patients

–

20 million CKD Stage 3-4 patients in the US

–

8 million patients with low serum 25D and high plasma PTH

Rayaldee is expected to take significant market share in Stage 3 and 4 CKD patients

suffering from SHPT – a potential $12 billion revenue opportunity

Comparison of Vitamin D Therapies for Stage 3-4 CKD

*And generics

**25-hydroxyvitamin D

Drug

Active

Type

25D

iPTH

Rayaldee

Calcifediol

(25-hydroxyvitamin D

)

Vitamin D

Cholecalciferol/Ergocalciferol

(vitamin D

/vitamin D

)

OTC

Drisdol

™*

Ergocalciferol

(vitamin D

)

Rocaltrol

™*

Calcitriol

(1 ,25-dihydroxyvitamin D

)

Hectorol

™*

Zemplar

™*

Paricalcitol

(19-nor-1 ,25-dihydroxyvitamin D

)

Doxercalciferol

(1 -hydroxyvitamin D

)

Effect on Blood Levels of:

OPKO Biologics: Leader in BioBetter Drugs

Reversible Pegylation Technology

(Peptides and small molecules)

CTP Technology

(Recombinant Proteins)

Developing biobetter long acting proteins and peptides

•

Dramatically reduce injection frequency

•

Reduce drug load

•

Reduce potential side-effects for most proteins, peptides and

small molecules

•

Maintain drug bio-activity

•

Validated

platform

technologies

–

safe

and

effective

Preclinical & clinical proof-of-concept in multiple compounds

Natural sequence

No need for linker

hGH-CTP –

The Long Acting hGH

Any Short-Lasting

Protein

CTP

Long-Lasting Protein

Global Collaboration with Pfizer for OPKO’s Long-

Acting Human Growth Hormone (hGH-CTP)

Collaboration Terms:

–

$295 M up-front payment

–

$275 M for achievement of regulatory based milestones

–

OPKO responsible for funding development program for the key

indications:

•

Adult and Pediatric Growth Hormone Deficiency (GHD)

•

Pediatric Short for Gestational Age

–

Pfizer responsible for funding:

•

Development programs for additional indications

•

All Post Marketing Studies

•

All Commercialization Activities

–

Initial double digit tiered royalties on sales of Adult GHD

–

Profit sharing commencing upon launch for Pediatric GHD

encompassing combined sales for all indications of OPKO’s hGH-CTP

and Pfizer’s Genotropin

–

Pfizer Genotropin represents about 25% of the world market with

annual revenues exceeding $700 M

Financial

Commercial

Development

•

$3.5 billion market, growing 5% annually

•

Once-a-week injection (current products require daily injections)

•

Small needle size (31 gauge) due to low viscosity

–

Competitive long acting formulations have high viscosity

•

Superb clinical, safety and immunogenicity profile

•

Human growth hormone is used for:

–

Growth hormone deficient children

–

Growth hormone deficient adults

–

Short stature

–

Off label

•

Orphan drug designation in the US & EU for children & adults

hGH-CTP Opportunity

hGH-CTP Clinical Development

Adult Pivotal Phase 3 trial (ongoing)

•

Primary efficacy endpoint: reduction in truncal fat mass

after 6 months vs. placebo

•

Secondary efficacy endpoints include:

–

Reduction in total body fat

–

Increase in lean body mass

•

Single pivotal trial required by FDA for BLA submission

hGH-CTP Clinical Development

Pediatric GHD Phase 2 trial (advanced stage)

•

4 Cohorts:

–

3 dose levels of once-weekly hGH-CTP

–

Commercially available standard daily rhGH treatment

•

Key outcome: height velocity

•

Positive clinical data presented at ENDO meeting June 2014

–

All doses provided excellent growth response compared to control

group and historical controls

–

Promising safety profile

–

hGH-CTP mean annualized height velocity at 6m ranged from 12.25-

14.37cm, compared to annual height velocity of ~10cm as published by

Bakker (2008) and Ranke (2010) for the same GHD patient population

(peak GH, age)

FVIIa

–CTP:

Long Acting for Treating Hemophilic Patients

•

$1.7 billion market

–

Growing 7% annually

–

Only 25% of patients are treated

•

Current product (NovoSeven®) requires frequent IV doses

–

3-4 times a day during bleeding episodes

–

1-2 times a day for prophylactic treatment

•

Pharmacological

studies

hemophilic

mice

and

dogs

FVIIa-CTP

demonstrated:

–

Reduce injection frequency during on-demand therapy

–

Enable prophylactic treatment while reducing the frequency of

injections to 2-3 times a week

–

Potential for substantial improvement in the quality of life of patients via

subcutaneous administration

•

Phase 2a study in hemophilic patients to be initiated 1Q 2015

•

Orphan drug designation in the US and EU

MOD-6031: Long Acting Oxyntomodulin for Obesity

•

WHO estimates 500 million severely overweight or obese people

•

>$15 billion market -

growing rapidly

•

Oxyntomodulin: Nature’s Appetite Control Mechanism

–

Natural appetite suppressor

–

Secreted by the digestive system following food intake and induces

satiety

–

Increases glucose tolerance

–

Short acting –

requires 3 injections per day

•

MOD-6031 Long Acting Oxyntomodulin-

weekly injection studies in

mutant obese mice and diet induced obese mice demonstrated:

–

Significantly inhibited food intake and reduced body weight by

reducing fat

–

Reduced cholesterol levels

–

Improved glycemic control

•

Phase 1 study to be initiated mid 2015

•

MOD-6031 is expected to provide SAFE long-term therapy for

obesity and diabetes type II patients

Rolapitant –

Potential Near-term Revenue Driver

•

Rolapitant out-licensed to Tesaro in December 2010

–

Payments of up to $121 million

–

Double-digit tiered royalties

•

Differentiated cancer supportive care product with $1.5B US Market

Opportunity

–

Potent neurokinin-1(NK-1) receptor antagonist for chemotherapy-

induced nausea and vomiting (CINV)

–

Opportunity to differentiate on convenience, market access and safety

•

Single dose

•

Long acting

•

NDA accepted November 2014

–

All three Phase 3 trials (MEC* and HEC**) achieved primary endpoint

–

Primary endpoint: complete response (no emesis and no use of rescue

medication)

–

Third Phase 3 trial (HEC) also achieved all secondary endpoints,

including:

•

Complete response in acute (0-24 hrs) and overall (0-120 hrs) phase of CINV

•

Also effective to prevent nausea

*Moderately emetogenic chemotherapy **Highly emetogenic chemotherapy

•

Oral and IV formulations allow full market access

•

Lack of drug-drug interactions

Strategic Investments

•

ARNO Therapeutics, Inc. (OTC: ARNI) (~4% equity interest)

–

Anti-progestins for breast (phase 2), endometrial and prostate cancers

•

Zebra Biologics, Inc. (~19% equity interest)

–

Combinatorial

antibody

libraries

based

function

human

cell

screens

•

OAO Pharmsynthez (MICE: LIFE) (~17% equity interest)

–

Russian developer and marketer of new drugs

•

RXi Pharmaceuticals Corporation (NASDAQ: RXII) (~11% equity interest)

–

sRNA to prevent hypertrophic scars (phase 2)

•

Cocrystal Pharma, Inc. (OTC: COCP) (~15% equity interest)

–

New anti-virals (Hepatitis C, flu, dengue fever)

•

Sevion Therapeutics, Inc. (OTC: SVON) (~ 4% equity interest*)

–

Antibodies against difficult targets (e.g., G protein-coupled receptor, ion

channels)

•

Neovasc, Inc. (NASDAQ: NVCN) (~ 6% equity interest)

–

Cardiology devices

•

ChromaDex, Inc. (OTC: CDXC) (~ 2% equity interest)

–

New nutritional supplement APIs

Proprietary Technologies with Significant Upside Potential

(As of September 30, 2014)

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