Transcript Xechem International

CepTor Corporation
Focusing on Targeted Therapies for Neuromuscular
and Neurodegenerative Orphan Diseases
Disclaimer
This program contains certain forward-looking statements within the meaning of
Section 27A of the Securities Act of 1933, as amended, and Section 21E of the
Securities Exchange Act of 1934, as amended, which are intended to be covered by
safe harbors created hereby. Such forward-looking statements involve known and
unknown risks, uncertainties, including the ability of the Companies to
successfully develop and commercialize their technologies, and other factors that
may cause the actual results, performance or achievements of the Companies to be
materially different from any future results, performance or achievements of the
Companies expressed or implied by such forward-looking statements.
Summary
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Broad, proprietary platform technology
Experienced management team
All markets minimum $1billion
Low-risk development programs
Efficient orphan drug focus internally
Partnership focused for non-orphans
Technology
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Receptor – mediated drug targeting to cells
Therapeutic protease inhibition to prevent tissue
degradation
Available for new and existing therapeutic compounds
Technology
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Carnitine analogue transport molecule to target skeletal
muscle
Taurine analogue transport molecule to target nerve
tissue, including crossing the blood brain barrier (BBB)
Calpain inhibition to prevent muscle and nerve tissue
degradation
Other therapeutic passenger small molecules
Technology
Genetic defect
Autoimmune
Trauma
Etiology unknown
Ischemia etc.
Cellular Dysfunction,
Instability, cell death
Membrane
Permeability
Widespread degradation
of multiple substrates
(receptors, kinases,
Cytoskeleton, etc.)
Elevated
Ca2+
Calpain
activation
Calpain
inhibitor
Tissue
preservation
Technology
(Disease Etiology and Therapeutic Target Validation)
Pipeline
Product
Indication
MYODUR
Muscular Dystrophy
NEURODUR
Multiple Sclerosis
C-301
Epilepsy
C-202
ALS
C-203
CIDP
Research
Pre-Clin
IND
Pipeline
Product
Indication
Anticipated Near
Term Status
MYODUR
Muscular Dystrophy
File Phase I/II IND
Receive Orphan Status
JCR License – Pacific Rim
NEURODUR
Multiple Sclerosis
Partner WW
C-301
Epilepsy
Partner WW
C-202
ALS
File for Orphan
Designation
C-203
CIDP
File for Orphan
Designation
Worldwide Market Potential
MYODUR (Muscular Dystrophy)
NEURODUR (Multiple Sclerosis)
C-301 (Epilepsy)
C-202 (ALS)
C-203 (CIDP)
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$ Billions
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Research
Neuromuscular
- Cardiomyopathys
- Cancer cachexia*
- AIDS wasting
- Denervation injury
*Internal Development
Neurodegenerative
- Amyotrophic lateral sclerosis (ALS)*
- Ototoxicity
- Retinal degeneration
- Spinal cord injury
- Alzheimer’s*
- Huntington’s*
- Chronic inflammatory
demyelinating
polyneuropathy (CIDP)*
Management Team
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William Pursley
Norman Barton, M.D., PhD
Donald Fallon
Leslie deVos
Teri Michele, M.D.
Fran Zbikowski
Chairman and CEO
EVP, CMO
SVP, CFO
VP, Clinical Operations
VP, Clinical Research
VP, Bus. Development
External Collaborators
Jerry Mandell, M.D. – Former Chairman of Neurology, Ohio State, SAB, phase I/II site
FDA representative
John Griffin, M.D. – Chairman of Neurology, John Hopkins, BOD, phase I/II site
Edwin Kolodny, M.D. – Chairman of Neurology, New York University, SAB,
phase I/II Site
H. Lee Sweeney, PhD – Chairman of Physiology, University of Pennsylvania, SAB,
pre- clinical studies, scientific advisor – Muscular Dystrophy
Association
Frank Sasinowski – Partner, Hyman Phelps and McNamara (regulatory counsel)
Past FDA Chief Counsel, authored orphan drug legislation
Duchenne Muscular Dystrophy (DMD)
(MYODUR)
• Disease background
- x-linked, recessive dystrophin gene defect
- degenerative skeletal muscle disease
- death in late adolescence
• Incidence: 1/3500 male births
• Prevalence: 46,000 in reimbursable markets
• WW Market Potential: $2.8 billion
MYODUR Results in MDX Model for DMD
Propriety unpublished data
C101 Development Timeline Overview
IND Submission
US and EU ODA Filing
Toxicology and Safety
Pharmacology Studies
Pre-Clinical Studies
Assay Development
Phase I-II DMD Clinical Study
Non-GMP Manufacturing
GMP Manufacturing
Pre-formulation, Characterization, Solubility, Stability, and Formulation
Jan05
Apr05
Jul05
Oct05
Jan06
Apr06
Jul06
Oct06
Jan07
Multiple Sclerosis (MS)
(NEURODUR)
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Disease background
- Autoimmune disease
- Myelin degradation and scaring
- Brain and spinal cord inflammation
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Incidence: 1/700-1000
Prevalence: 400,000 in U.S.
Worldwide Market Potential: $5 billion
NEURODUR crosses the BBB
NEURODUR Results in EAE Model for MS
Propriety unpublished data
Epilepsy (C-301)
• Disease background
- etiology unknown
- neurodegenerative electrical signaling disorder
- uncontrollable convulsions
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Incidence: 181,000 new cases each year in U.S.
Prevalence: 2.5 million in U.S.
U.S. Market Potential: $2 billion
Valproic acid most common therapy today
C-301 4X more effective than valproic acid in
mouse model
Amyotrophic Lateral Sclerosis (ALS)
(C-202)
• Disease background
- Etiology unknown
- Motor neuron disease
- Death 3-4 years post diagnosis
• Incidence: 1/5000
• Prevalence: 24,000
• U.S. Market Potential: $1.9 billion
Chronic Inflammatory Demyelinating
Polyneuropathy (CIDP) (C-203)
• Disease background
- autoimmune disease
- myelin and subsequent axonal degeneration
- loss of control of extremities
• Prevalence: 60,000 Worldwide
• Worldwide Market Potential: $2.3B
Orphan Drug Platform
Market (U.S.)
• 6,000 rare diseases
• 24,000,000 patients directly affected
• Since the ODA, 1456 compounds have been granted
orphan drug designation and 269 have been approved
representing an 18% success rate and growing.
• No orphan drug has ever been withdrawn from market
• Orphan drugs are reimbursed at a rate >95%
Orphan Drug Platform
• Highest value market in the world taken as a whole
• e.g. CEREZYME
REPLAGAL
Factor VIII
hGH
Gaucher disease
Fabry disease
Hemophilia
GH deficiency
$300K/year/patient
$170K/year/patient
$70K/year/patient
$20K/year/patient
Orphan Drug Platform
Business Model
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High Commercial Value
Market Exclusivity
Regulatory advantages
Target marketing
Distribution and reimbursement infrastructure
amortization
• Low COGS
Two-Year Business Plan
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Enter phase III with MYODUR
Have 3 products in the clinic
Close 2 corporate partnerships
Conduct appropriate financings
Investor Considerations
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Significant upside near and mid-term
Very experienced management team
Broad-based, proprietary, platform technology
Low-risk development/orphan drug focus
All markets at least $1 billion
Targeting technology available to new and existing
compounds