Form: 8-K

Current report filing

January 14, 2015

33
rd
Annual J.P. Morgan Healthcare Conference
January 2015
Exhibit 99.1


2
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
3


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
4


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
5


Claros
®
1: Rapid Testing in the Physician Office
10
mins
6
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)
7


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
8


Rayaldee (CTAP101) –
A Late-Stage Investigational Drug
Product Overview
•
Oral formulation of 25D
3
*
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
9


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
10
% of CKD Patients with:
Stage
Kidney Function
CKD Prevalence
Vitamin D
Insufficiency(  25D)
SHPT
(   PTH)
Hyperphosphatemia
(  Phosphorus)
3
Moderate impairment
18.7
Million*
70%
56%
37%
4
Severe impairment
1.4 Million*
80%
60%
50%
Failure
0.5 Million*
90%
90%
70%
5


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
11
Low
serum
25D
and
elevated
plasma
PTH
are
prevalent
in
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
12
Drug
Active
Type
25D
**
Ca
iPTH
Rayaldee
Calcifediol
(25-hydroxyvitamin D
3
)
Rx
Vitamin D
Cholecalciferol/Ergocalciferol
(vitamin D
3
/vitamin D
2
)
OTC
Rx
Rx
Rx
Rx
Drisdol
™*
Ergocalciferol
(vitamin D
2
)
Rocaltrol
™*
Calcitriol
(1  ,25-dihydroxyvitamin D
3
)
Hectorol
™*
Zemplar
™*
Paricalcitol
(19-nor-1  ,25-dihydroxyvitamin D
2
)
Doxercalciferol
(1  -hydroxyvitamin D
2
)
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
13


Natural sequence
No need for linker
hGH-CTP –
The Long Acting hGH
14
+
=
Any Short-Lasting
Protein
CTP
Long-Lasting Protein


Global Collaboration with Pfizer for OPKO’s Long-
Acting Human Growth  Hormone (hGH-CTP)
15
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
16


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
17


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)
18


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
in
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
19


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
20


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
21
*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
on
function
in
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
22
Proprietary Technologies with Significant Upside Potential
(As of September 30, 2014)