Post on 28-Dec-2015
Advancing “oral” treatments for
bone & joint diseases especially for
osteoporosis and arthritis
Paul HopperExecutive Chairman
February 2006
2
This presentation contains forward-looking statements that involve
risks and uncertainties. These forward-looking statements are not
guarantees of Bone Medical Limited’s future performance and involve
a number of risks and uncertainties that may cause actual results to
differ materially from the results discussed in these statements.
Factors that might cause the Company’s results to differ materially
from those expressed or implied by such forward-looking statements
include but are not limited to, development and commercialisation of
the Company’s product portfolio; development or acquisition of
additional products; and other risks and uncertainties. Bone Medical
Limited undertakes no duty to update any of these forward-looking
statements to confirm them to actual results.
Safe Harbor Statement
2006 Bone Medical Ltd.CONFIDENTIAL
3
Corporate Overview
2006 Bone Medical Ltd.
• Founded December 2002
• Novel, proprietary oral drug delivery technology for musculoskeletal disease
• Phase I/IIa clinical trial for oral calcitonin- Capsitonin- completed
• Phase I clinical study for oral parathyroid hormone- Perthoxal completed
• Listed Public August 2004
Ticker: (ASX:BNE) (ADR:BMEDY.PK)
Office: Bentley, WA, Australia 6102
Shares Outstanding: 64,291,032 (Ordinary); 9,999,204 (Preferred C)
Market Cap (13-2-06): AU$19.3 million (US$15.2 million)
• Over AU$5.1MM spent to date developing two core compounds
• Approximately AU$1.4 million cash on-hand
CONFIDENTIAL
4
Capsitonin Phase I/IIa clinical trial for “osteoporosis” completed
Capsitonin Pre-IND meeting held with FDA in December 2005
Rapid regulatory process / strategy for Capsitonin- bioequivalence & 505(b)(2)
NDA submission
Perthoxal- oral parathyroid hormone for “osteoporosis” Phase I clinical study
completed
New, unique oral formulation technology with robust IP and long patent life
“Multi-billion” dollar market opportunity(s) for both Capsitonin and Perthoxal
Balanced portfolio between lower-risk drug delivery programs and potential
breakthrough future treatments
Experienced management with experience in science, management and capital
markets
2006 Bone Medical Ltd.
Investment Highlights
CONFIDENTIAL
5
Sources: International Osteoporosis Foundation, CDC, Mayo Clinic, NIAMS, National Center for Chronic Disease Prevention and Health Promotion
• Osteoporosis
Approximately 200 million women worldwide
Incidence is expected to “double” in the next 50 years
Estimated US$48.0 million spent to treat disease in North America and in Europe
• Osteoarthritis
Over 20 million people in the U.S. alone
Most common form of arthritis, approximately 5-10% of population will develop
• Treatments
Calcitonin (sCT): US$600 million
• Injectable and Nasal forms only (no oral form available)
Parathyroid Hormone (PTH): US$450 million
• Injectable form only (no oral from available)
2006 Bone Medical Ltd.CONFIDENTIAL
Market Opportunity
6
• Osteoporosis
Painful and debilitating
• Worldwide cost burden to reach US$131.5 billion by 2050
• Only 1 in 2 vertebral fractures diagnosed by a physician
• Less than 20% of Osteoporosis patients receive timely treatment
• Osteoarthritis (and rheumatoid arthritis)
Painful, physically and emotionally debilitating
• 25% of persons “cannot” perform major daily activities (e.g., lifting, climbing stairs)
2006 Bone Medical Ltd.CONFIDENTIAL
Market Need
“There are no oral formulations available
for calcitonin or parathyroid hormone
limiting the number of treated patients”
“There are no oral formulations available
for calcitonin or parathyroid hormone
limiting the number of treated patients”
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Capsitonin (“oral” sCT)Therapeutic peptide1° Osteoporosis, 2°Osteoarthritis
2006 Bone Medical Ltd.CONFIDENTIAL
Product Pipeline
R&D Pre-clinical Phase I Phase II
Phase III NDA
Perthoxal (“oral” PTH)Therapeutic peptideOsteoporosis
BN007 (collagen tolerance)Oral Immune therapyRheumatoid Arthritis
BN006 (TNF down-regulator)
BN008 (Osteoclast down-reg)
BN005 (Osteoblast up-reg)
Products / Compounds
Novel therapeutic entitiesRheumatoid Arthritis, Osteoarthritis
Studies (trials)designedto support
bioequivalenceand 505(b)(2) FDA
submissions
8 2006 Bone Medical Ltd.
Lead Compound
CONFIDENTIAL
• Mechanism of Action: Calcitonin aids in the “formation
of new bone” by interfering with
osteoclast activity.
• Use/Indication: Osteoporosis, Osteoarthritis
• Side Effects: Mild, some joint ache,
headache
Capsitonin Oral Capsule(synthetic salmon calcitonin peptide)
Synthetic peptideof salmon calcitonin
“Calcitonin has been usedSafely as a treatment
for osteoporosisfor over 30 years!”
9
• Recombinant peptides (Capsitonin
and Perthoxal) are protected from
gastric degradation
• Released in the upper intestine in an
area with high peptide absorption
• Utilizes existing approved
substances and/or excipients
• Opportunity for rapid 505(b)(2) FDA
submission
New, proprietary oral drug delivery technology
2006 Bone Medical Ltd.CONFIDENTIAL
Novel Oral Formulation
Enteric coating for easy swallowing
Capsule Contents:
Drug, stabilizers, solublizers, protease inhibitors,
various GRAS pharmaceutical excipients
Gastric (stomach) cellular surface●
●●
●
●●
●●●
●●
● ●●
●●●
●●
●● ●●● ●
●
●
●●●
●
Capsulecontents
Improved transcellular
absorption
10 2006 Bone Medical Ltd.CONFIDENTIAL
Capsitonin Oral Capsule
• Background
Existing injectable and nasal formulations of calcitonin are difficult to administer
affecting patient compliance and limiting its use as a treatment option
• To date, an “oral” formulation of calcitonin has been unable to be developed
• Capsitonin Pre-clinical / Proof-of-concept
-1
-0.8
-0.6
-0.4
-0.2
0
0.2
0 60 120 180 240 300 360 420 480
5000iu sCT i.j.
Untreated control
200iu sCT s.c.
Decrease in plasma calcium following oral (tablet) administration of Capsitonin in pigs
compared to subcutaneous (injection)
minutes
0.0
50.0
100.0
150.0
200.0
250.0
300.0
350.0
0 0.5 1 1.5 2 2.5 3 3.5 4
E56
S963
S982
S984
Increase in total plasma Capsitonin followingoral (tablet) administration of to primates
Novartis Study
hours
11 2006 Bone Medical Ltd.CONFIDENTIAL
Capsitonin - Phase I/IIa
• Study Objective
Phase I/IIa open label safety & tolerability and preliminary
efficacy / activity
• Study Design
12 post-menopausal, female volunteers
Sequential, cross-over design
1. Positive control (Miacalcin Nasal) – active comparator
2. 1250iu Capsitonin
3. 2500iu Capsitonin
Measurement of key biomarkers: serum calcitonin, serum CTX,
and blood calcium
12 2006 Bone Medical Ltd.CONFIDENTIAL
Capsitonin - Phase I/IIa
• Study Results / Outcomes
The study showed that Capsitonin can be delivered orally and exert a
statistically significant biological effect
• Measurement of markers of bone breakdown in the blood showed that
Capsitonin reduced bone destruction
• Calcitonin (in a small number of patients) was measured in blood
• Calcium levels in the blood were also reduced as expected
Safe and well tolerated
13
Lead Compound
CONFIDENTIAL
Perthoxal Oral Capsule(synthetic parathyroid hormone peptide)
Synthetic peptideof the human hormone
• Mechanism of Action: A controlling agent for
maintaining normal calcium levels in the blood for strong bone formation of new bone.
• Use/Indication: Osteoporosis
• Side Effects: Some leg cramps, mild
headache
2006 Bone Medical Ltd.
“The bone formingeffects of parathyroid hormone have been
known for 70 years!”
14 2006 Bone Medical Ltd.CONFIDENTIAL
Perthoxal Oral Capsule
• Background
Existing injectable and nasal formulations of parathyroid hormone (PTH) are
difficult to administer affecting patient compliance and limiting its use as a
treatment option
• To date, an “oral” formulation of PTH has been unable to be developed
• Perthoxal
Pre-clinical / Proof-of-concept:
Time [hours]
0
2
4
6
8
10
0 1 2 3 4 5 6C
alce
mia
[% c
hang
e]0.0
4.0
8.0
12.0
0 1 2 3 4 5 6
500ug PTH orally
50ug PTH s.c. injection
Increase in total plasma calcium followingoral (tablet) administration of PTH to primates
Increase in plasma calcium following oral (tablet) administration of PTH in primates
compared to subcutaneous (injection)
15 2006 Bone Medical Ltd.CONFIDENTIAL
Perthoxal - Phase I
• Study Objective
Phase I open label safety & tolerability study and preliminary pharmacodynamic effects
• Study Design
18 post-menopausal, female volunteers
Sequential, cross-over design (12 of 18 patients only) of two different formulations of Perthoxal
1. Positive control (s.c. injected PTH) – active comparator
2. Formulation (1) 450ug Perthoxal
3. Formulation (2) 400ug Perthoxal
Measurement of key biomarker: serum calcium concentration
16 2006 Bone Medical Ltd.CONFIDENTIAL
Perthoxal - Phase I
• Study Results / Outcomes
The study showed that Perthoxal was able to be delivered safely
and that measurable amounts of calcium were able to be detected in
serum
• One Perthoxal formulation showed levels of measurable calcium
Both Perthoxal formulations were shown to be safe and well
tolerated
17 2006 Bone Medical Ltd.CONFIDENTIAL
Competition
• Calcitonin Nasal Spray (commercially available)
• Miacalcin® Nasal (Novartis), Fortical Nasal (Unigene Laboratories), Calcitonin-Salmon Nasal Spray (Nastech)
Injectable (commercially available)
• Forcaltonin® (Unigene Laboratories)
Oral
• Emisphere Technologies (w/Novartis), Unigene Laboratories
Nasal Spray
18 2006 Bone Medical Ltd.CONFIDENTIAL
Competition
• Parathyroid Hormone
Injectable (commercially available)
• Forteo (Lilly)
Nasal Spray
• Nastech Pharmaceuticals
Oral
• Zelos Therapeutics, Emisphere
Technologies (w/Novartis),
Unigene Laboratories (w/GSK)
Injectable pen device
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• Bone Medical has been granted exclusive worldwide rights
to oral drug delivery technology in the field of
musculoskeletal disease under three patent pending
applications for each product:
1. the use of aromatic alcohols to enhance the uptake of peptides
across the small intestine;
2. pharmaceutical compositions containing certain proportions of
aromatic alcohols; and
3. methods of solubilisation.
2006 Bone Medical Ltd.CONFIDENTIAL
Intellectual Property
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Corporate Secretary
Gabriel Chiappini
2006 Bone Medical Ltd.CONFIDENTIAL
Key Management
Executive Chairman
Paul Hopper
Chief Scientific Officer
Roger New, Ph.D.
VP Clinical & Regulatory
Tony Lockett, Ph.D.
VP Commercial Operations
Patrick J. Mallon
Chief Financial Officer
Ed Daquino
Board of Directors
Operations, commercialization, BD, developmentMitos Pharmaceuticals, Pfizer, Roche
R&D, formulation, developmentProxima
R&D, development, regulatoryCovance, Parke-Davis
Capitalization, governance, BDInnovate Oncology, Evolve Oncology,
21 2006 Bone Medical Ltd.CONFIDENTIAL
Business Strategy
• Minimal infrastructure cost and savings
• Corporate / management focus
• Rapidity to market and market up-take
• Minimize infrastructure build & capital needs
• Broaden drug pipeline and product offerings
• Increase revenues and corporate growth
“Hybrid”
distribution
& promotion22
Partner Capsitonin &
Perthoxal (out-license
and/or co-promotion)
“Hybrid”
distribution
& promotion21
Outsource clinicaldevelopment
& manufacturing
Selectively develop
analogs & acquire
oncology compounds
“Hybrid”
distribution
& promotion23
Selectively developand out-license
compounds in pipeline
22
“Two” products in clinical trials:
Capsitonin - Oral calcitonin for “osteoporosis” and “osteoarthritis”
Perthoxal - Oral parathyroid hormone for “osteoporosis”
Rapid regulatory process: potential bioequivalence and/or 505(b)(2)
FDA submissions
Multi-billion dollar market opportunity(s)
New, unique oral formulation technology with robust IP and long patent life
Balanced portfolio between lower-risk drug delivery programs and potential
breakthrough future treatments
Experienced management
2006 Bone Medical Ltd.
Value Proposition
CONFIDENTIAL
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Paul HopperExecutive Chairman
Tel: +1 858 200-5636 (San Diego, CA)
Hopper@bone-ltd.com
Patrick J. MallonVP Commercial Operations
Tel: +1 858 205-2501 (San Diego, CA)
pmallon@bone-ltd.com
2006 Bone Medical Ltd.CONFIDENTIAL
Contact
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Leon IvoryDirector
Tel: +61 8 9355 5123 (Western Australia)
leonivory@bone-ltd.com
Ed DaquinoChief Financial Officer
Tel: +61 8 9355 5123 (Western Australia)
eddaquino@bone-ltd.com
2006 Bone Medical Ltd.CONFIDENTIAL
Contact