Corporate Presentation

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Corporate Presentation
1
Corporate Presentation
May 2012
NYSE Amex: PBTH
Safe Harbor Statement
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This presentation contains forward-looking statements, including statements regarding the results of
current studies and pre-clinical experiments and the effectiveness of PROLOR’s long-acting protein
programs and are made pursuant to the safe harbour provisions of the Private Securities Litigation Reform
Act of 1995. Investors are cautioned that forward-looking statements involve risks and uncertainties that
may affect PROLOR’s business and prospects, including the risks that PROLOR Biotech may not succeed in
developing any commercial products, and that ongoing studies may not continue to show substantial or
any activity; and other risks and uncertainties that may cause results to differ materially from those set
forth in the forward-looking statements. In addition to the risks described above, investors should
consider the economic, competitive, governmental, technological and other factors discussed in PROLOR
Biotech’s filings with the Securities and Exchange Commission.
PROLOR: Leader in BioBetter Drugs
Developing biobetter long acting proteins and peptides
Reversible Pegylation
Technology
(Peptides and small molecules)





CTP Technology
(Recombinant Proteins)
Dramatically reduce injection frequency
Reduce Drug load
Reduce potentially 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
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PROLOR’s Pipeline of Market Leaders
Indication
Growth
Hormone
Disorders
Product
Market
Size
hGH –CTP
(Adults)
$3B
Preclin.
Ph I
Ph II
1/week injection
hGH –CTP
(Children)
1/week injection
Factor VIIa- CTP
$1.3B
2/week injection
Factor IX - CTP
$0.7B
1/week injection
$2B
1/week injection
Hemophilia
Diabetes Type GLP1/Glucagon
II & Obesity
dual receptor
agonist
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Ph III
CTP: Clinically Validated Technology

Merck ’s long-acting FSH-CTP (Elonva®) received EU
marketing authorization in 2010


Single FSH-CTP injection replaces 7 daily FSH injections in
fertility treatment
Two licensees of CTP technology: PROLOR & Merck
Merck holds license for 4 fertility-focused proteins
 PROLOR holds license for all other human therapeutics
of natural or non-natural sequence

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Phase
II designII Study Design
MOD-4023
Phase
Stage I ( 4 weeks treatment)
Stage II ( 4 months treatment)
MOD-4023.
Daily hGH
MOD-4023 Treatment-
Daily hGH
4 months study
Data analysis
Dose finding Stage :
3 Doses
Dose Confirmatory Stage
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hGH-CTP Phase II Positive Data
-- Analysis & Conclusions-
A single weekly injection of hGH-CTP can replace 7 daily
injections of commercially available GH

Identified potential kick-start dosing for Phase III trial

Very Good safety & tolerability profile.

Majority of the patients were maintained within the normal range
of IGF-1, using only 50-65% of their cumulative daily GH dosing”

Estimated COG 50-75% of commercially available GH
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hGH-CTP Phase II & III Planning

Pediatric GHD Phase II trial- has initiated and on track




Q2-Q3 filing for adult Phase III trial- approval on track




52-56 patients,
12 months efficacy & safety
Key outcome: Height velocity
120-150 patients
6 months efficacy & 12 months safety
Key outcome: Improvement in truncal fat
Received U.S. orphan drug status for children & adults

Expect to be first long-acting GH to market
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Current Market Size: $3 Billion, indicated
for children & adults – orphan designation
hGH-CTP
Major Players: Lilly, Roche (Genentech), Pfizer,
Novo Nordisk, Merck-Serono
Marketed Products: all requiring
single daily injections
hGH-CTP Value Proposition: Single weekly
injection replacing 7 consecutive daily injections,
using same pen/needle device (31G needle)
Clinical Stage: Successfully completed Phase II
(adults), Phase III (adults) expected initiation later in
2012; Phase II (pediatrics) initiated early 2012
Competitive Positioning: Expected to be first-to-market longacting hGH; Superb clinical, safety and immunogenicity profile,
non-viscous drug product, granted orphan designation in the U.S.
CMC Status – High-yielding, high-quality
GMP process and drug product with
commercial CMO (Rentschler, Germany)
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2nd drug delivery technology platform for
peptides & small molecules –
Reversible PEGylation
MOD-6030: A Long Lasting Oxyntomodulin
Dual GLP-1 / Glucagon Receptor Agonist
 GLP-1 and the Glucagon receptors
agonist
 Induce appetite suppressant
mechanism
 Studies conducted in humans to learn
the effect of Oxyntomodulin on body
weight and appetite control, show
favorable results.
OXM directly stimulates insulin secretion
from murine islets and induces glucose
tolerance in animal model.
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MOD-6030—Long-Acting GLP-1/Glucagon Dual
Receptor Agonist versus Native Oxyntomodulin:
Previous 3rd party oxyntomodulin studies:

Animals (mice, rats): 2 injections per day




Glycemic control
Food intake reduced
Weight loss observed
Humans: 3 injections per day

Weight Loss of 0.5 kg per week, no special diet or exercise
PROLOR’s studies:
 Can we obtain SAME weight loss & reduction of food intake in
Obesity mice model, with a single weekly injection
?
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MOD-6030 Significant Competitive
Advantages in Weight Reduction
Treatment
Injection
Frequency
Dose per
Injection
Cumulative
Dose in
30 Days
Cumulative
Weight Loss
Day 30 (%)
Cumulative
Food Intake
Reduction (%)
Formulation
Buffer
2x per Day
--
--
0.3% Gain
--
Native OXM
2x per Day
6,000 nm
348,000 nm
16.6% Loss
12% Less
PEG-OXM
1X per
Week
6000nm
30,000nm
1.6% Loss
1% Less
MOD-6030
1x per Week 6,000 nm
30,000 nm
27.6% Loss
30% Less
MOD-6030 provides superior weight loss as compared to native OXM
MOD-6030 enables significant dose reduction ( 12x lower)
Substantial benefit penetrating into the Brain
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MOD-6030 GLP1/Glucagon Dual Receptor
Agonist: High Preclinical Program Valuations

Zealand Pharma – Boheringer Ingelheim deal (2011)
Preclinical oxyntomodulin variant
 Targeted for single daily injection
 $57M in payments in first 2 years, additional $480M in
milestones


Marcadia – Roche deal (2010)
Lead compound: Preclinical GLP-1/Glucagon dual receptor
agonist peptide
 Company acquired
 $287M upfront, $250M in milestones

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Long acting Factor VIIa/IX improve hemophilic
patients’ clinical condition & quality of life
• $2 billion market
• Growing 14% annually
• Requires very frequent IV injections
Anticipated Milestones
Clinical Priorities
Event
Timing
Ongoing
hGH-CTP: Phase II pediatric study -on track
hGH-CTP: Phase III adult study regulatory clearance (FDA+EMA)
Initiate study thereafter in EU, USA
Q2 –Q3 2012
Preclinical Priorities
Event
Timing
MOD-6030: Further diabetes studies & process, analytical &
pharmaceutical development to pre-IND submission level
2012
Factor-VIIa-CTP: process, analytical, and pharmaceutical development to
pre-IND submission level
2012
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Leadership
Phillip Frost, MD.
Chairman
Abraham Havron, Ph.D.
CEO
OPKO
Corporation
Jane Hsiao, Ph.D., MBA
Director
Marian Gorecki, Ph.D.
Director
OPKO
Corporation
Steve Rubin, J.D.
Director
Shai Novik, MBA
President
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Financial Profile

NYSE-Amex and TASE listed (PBTH)

$~9.1 million cash balance March 31 , 2012

Closed underwritten public offering on May 16 2012 of
7.475 million shares, led by Jefferies, Oppenheimer and Ladenburg.

Offering provide an additional net of $35 million of cash.

63 million common shares outstanding post public, 5.5 million options
& 0.5 million warrants .

Cash balance estimated to get the company beyond next two clinical
milestones:


Top line results of Phase II pediatrics
Top line results of Phase III adults.
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Thank You
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Intellectual Property




hGH-CTP: 3 patents granted (US, 2009 & 2011)
CTP Platform patent granted (US, 2011) covering
hormones, high affinity protein ligands, proteins that
induce or regulate an immune response, proteins that
are involved with autocrine and paracrine activities,
mimetics of these therapeutic proteins, others
CTP Interferons patent granted (US, 2011)
Several other patent applications filed (Coagulation
factors, GLP-1/Glucagon dual receptor agonist, others)
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