Title: Xechem International
1(No Transcript)
2CepTor Corporation
- Focusing on Targeted Therapies for Neuromuscular
and Neurodegenerative Orphan Diseases
3Disclaimer
- 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.
4Summary
- 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
5Technology
- Receptor mediated drug targeting to cells
- Therapeutic protease inhibition to prevent tissue
degradation - Available for new and existing therapeutic
compounds
6Technology
- 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
7Technology
- Genetic defect
- Autoimmune
- Trauma Membrane Elevated
- Etiology unknown Permeability Ca2
- Ischemia etc.
- Cellular Dysfunction, Widespread degradation
Calpain - Instability, cell death of multiple substrates
activation - (receptors, kinases, Calpain
- Cytoskeleton, etc.) inhibitor
- Tissue
- preservation
8Technology(Disease Etiology and Therapeutic
Target Validation)
9Pipeline
10Pipeline
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
11Worldwide Market Potential
Billions
12Research
- Neuromuscular
Neurodegenerative - - Cardiomyopathys - Amyotrophic lateral
sclerosis (ALS) - - Cancer cachexia - Ototoxicity
- - AIDS wasting - Retinal degeneration
- - Denervation injury - Spinal cord injury
- - Alzheimers
- - Huntingtons
- - Chronic inflammatory
- demyelinating
- polyneuropathy (CIDP)
-
-
-
- Internal Development
13 Management Team
- William Pursley Chairman
and CEO - Norman Barton, M.D., PhD EVP, CMO
- Donald Fallon SVP,
CFO - Leslie deVos VP,
Clinical Operations - Teri Michele, M.D. VP,
Clinical Research - Fran Zbikowski VP, Bus.
Development
14 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
15Duchenne 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
16MYODUR Results in MDX Model for DMD
Propriety unpublished data
17C101 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
Jul06
18Multiple Sclerosis (MS)(NEURODUR)
- Disease background
- - Autoimmune disease
- - Myelin degradation and scaring
- - Brain and spinal cord inflammation
- Incidence 1/700-1000
- Prevalence 400,000 in U.S.
- Worldwide Market Potential 5 billion
- NEURODUR crosses the BBB
19NEURODUR Results in EAE Model for MS
Propriety unpublished data
20Epilepsy (C-301)
- Disease background
- - etiology unknown
- - neurodegenerative electrical
signaling disorder - - uncontrollable convulsions
- 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
21Amyotrophic 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
22Chronic Inflammatory DemyelinatingPolyneuropathy
(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
23Orphan 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 gt95
24Orphan Drug Platform
- Highest value market in the world taken as a
whole - e.g. CEREZYME Gaucher disease 300K/year/pa
tient - REPLAGAL Fabry disease 170K/year/p
atient - Factor VIII Hemophilia 70K/year/patie
nt - hGH GH
deficiency 20K/year/patient
25Orphan Drug PlatformBusiness Model
- High Commercial Value
- Market Exclusivity
- Regulatory advantages
- Target marketing
- Distribution and reimbursement infrastructure
- amortization
- Low COGS
26Two-Year Business Plan
- Enter phase III with MYODUR
- Have 3 products in the clinic
- Close 2 corporate partnerships
- Conduct appropriate financings
27Investor Considerations
- 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