Capsitonin - Bone Medical Ltd
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Transcript Capsitonin - Bone Medical Ltd
Advancing “oral” treatments for
bone & joint diseases especially
for osteoporosis and arthritis
Paul Hopper
Executive Chairman
February 2006
Safe Harbor Statement
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.
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2006 Bone Medical Ltd.
Corporate Overview
•
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
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2006 Bone Medical Ltd.
Investment Highlights
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
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2006 Bone Medical Ltd.
Market Opportunity
•
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)
Sources: International Osteoporosis Foundation, CDC, Mayo Clinic, NIAMS, National Center for Chronic Disease Prevention and Health Promotion
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2006 Bone Medical Ltd.
Market Need
•
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)
“There are no oral formulations available
for calcitonin or parathyroid hormone
limiting the number of treated patients”
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2006 Bone Medical Ltd.
Product Pipeline
Products / Compounds
R&D
Pre-clinical
Capsitonin (“oral” sCT)
Phase I
Phase II
Phase III
NDA
Studies (trials)
designed
to support
bioequivalence
and 505(b)(2) FDA
submissions
Therapeutic peptide
1° Osteoporosis, 2°Osteoarthritis
Perthoxal (“oral” PTH)
Therapeutic peptide
Osteoporosis
BN007 (collagen tolerance)
Oral Immune therapy
Rheumatoid Arthritis
BN006 (TNF down-regulator)
BN008 (Osteoclast down-reg)
BN005 (Osteoblast up-reg)
Novel therapeutic entities
Rheumatoid Arthritis, Osteoarthritis
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2006 Bone Medical Ltd.
Lead Compound
“Calcitonin has been used
Safely as a treatment
for osteoporosis
for over 30 years!”
Capsitonin Oral Capsule
(synthetic salmon calcitonin peptide)
Synthetic peptide
of salmon calcitonin
•
Mechanism of Action:
•
Use/Indication:
•
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Osteoporosis, Osteoarthritis
Side Effects:
CONFIDENTIAL
Calcitonin aids in the “formation
of new bone” by interfering with
osteoclast activity.
Mild, some joint ache,
headache
2006 Bone Medical Ltd.
Novel Oral Formulation
New, proprietary oral drug delivery technology
Enteric coating for easy swallowing
• Recombinant peptides (Capsitonin
and Perthoxal) are protected from
gastric degradation
• Released in the upper intestine in an
Capsule Contents:
Drug, stabilizers, solublizers, protease inhibitors,
area with high peptide absorption
various GRAS pharmaceutical excipients
Capsule
contents
• Utilizes existing approved
Improved
transcellular
absorption
●
substances and/or excipients
● ●
●
●
●
● ● ● ●
●
● ● ●
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● ●
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• Opportunity for rapid 505(b)(2) FDA
submission
Gastric (stomach) cellular surface
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2006 Bone Medical Ltd.
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
0.2
350.0
Novartis Study
300.0
0
E56
0
60
120
180
240
300
360
420
480
250.0
S963
200.0
S982
-0.2
-0.4
minutes
100.0
-0.6
5000iu sCT i.j.
-0.8
hours
50.0
Untreated control
0.0
200iu sCT s.c.
0
-1
Decrease in plasma calcium following oral
(tablet) administration of Capsitonin in pigs
compared to subcutaneous (injection)
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S984
150.0
0.5
1
1.5
2
2.5
3
3.5
4
Increase in total plasma Capsitonin following
oral (tablet) administration of to primates
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2006 Bone Medical Ltd.
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
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2006 Bone Medical Ltd.
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
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2006 Bone Medical Ltd.
Lead Compound
“The bone forming
effects of parathyroid
hormone have been
known for 70 years!”
Perthoxal Oral Capsule
(synthetic parathyroid hormone peptide)
Synthetic peptide
of the human hormone
•
Mechanism of Action:
•
Use/Indication:
•
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Osteoporosis
Side Effects:
CONFIDENTIAL
A controlling agent for
maintaining normal calcium
levels in the blood for strong
bone formation of new bone.
Some leg cramps, mild
headache
2006 Bone Medical Ltd.
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:
Calcemia [% change]
10
12.0
500ug PTH orally
50ug PTH s.c. injection
8.0
4.0
8
6
4
2
0
0
0.0
0
1
2
3
4
5
6
2
3
4
5
6
Time [hours]
Increase in plasma calcium following oral
(tablet) administration of PTH in primates
compared to subcutaneous (injection)
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1
Increase in total plasma calcium following
oral (tablet) administration of PTH to primates
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2006 Bone Medical Ltd.
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
CONFIDENTIAL
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
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2006 Bone Medical Ltd.
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
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2006 Bone Medical Ltd.
Competition
•
Calcitonin
Nasal Spray (commercially available)
•
Nasal Spray
Injectable (commercially available)
•
Forcaltonin® (Unigene
Laboratories)
Oral
•
CONFIDENTIAL
Miacalcin® Nasal (Novartis),
Fortical Nasal (Unigene
Laboratories), Calcitonin-Salmon
Nasal Spray (Nastech)
Emisphere Technologies
(w/Novartis), Unigene Laboratories
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2006 Bone Medical Ltd.
Competition
•
Parathyroid Hormone
Injectable (commercially available)
•
Injectable pen device
Forteo (Lilly)
Nasal Spray
•
Nastech Pharmaceuticals
Oral
•
Zelos Therapeutics, Emisphere
Technologies (w/Novartis),
Unigene Laboratories (w/GSK)
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2006 Bone Medical Ltd.
Intellectual Property
•
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.
CONFIDENTIAL
methods of solubilisation.
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2006 Bone Medical Ltd.
Key Management
Board of Directors
Corporate Secretary
Gabriel Chiappini
Executive Chairman
Capitalization, governance, BD
Paul Hopper
Innovate Oncology, Evolve Oncology,
Chief Scientific Officer
R&D, formulation, development
Roger New, Ph.D.
Proxima
VP Clinical & Regulatory
R&D, development, regulatory
Tony Lockett, Ph.D.
Covance, Parke-Davis
VP Commercial Operations
Operations, commercialization, BD, development
Patrick J. Mallon
Mitos Pharmaceuticals, Pfizer, Roche
Chief Financial Officer
Ed Daquino
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2006 Bone Medical Ltd.
Business Strategy
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“Hybrid”
Outsource
clinical
development
distribution
& manufacturing
& promotion
• Minimal infrastructure cost and savings
• Corporate / management focus
Partner Capsitonin
“Hybrid” &
(out-license
2 2 Perthoxaldistribution
and/or co-promotion)
& promotion
•
Rapidity to market and market up-take
•
Minimize infrastructure build & capital needs
Selectively
develop
Selectively
develop
“Hybrid”
and
out-license
distribution
analogs
& acquire
32
compounds
in pipeline
& promotion
oncology
compounds
• Broaden drug pipeline and product offerings
CONFIDENTIAL
• Increase revenues and corporate growth
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2006 Bone Medical Ltd.
Value Proposition
“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
CONFIDENTIAL
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2006 Bone Medical Ltd.
Contact
Paul Hopper
Executive Chairman
Tel: +1 858 200-5636 (San Diego, CA)
[email protected]
Patrick J. Mallon
VP Commercial Operations
Tel: +1 858 205-2501 (San Diego, CA)
[email protected]
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2006 Bone Medical Ltd.
Contact
Leon Ivory
Director
Tel: +61 8 9355 5123 (Western Australia)
[email protected]
Ed Daquino
Chief Financial Officer
Tel: +61 8 9355 5123 (Western Australia)
[email protected]
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2006 Bone Medical Ltd.