ceptor corporation focusing on targeted therapies for neuromuscular and neurodegenerative orphan...
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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
• 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
• Receptor – mediated drug targeting to cells
• Therapeutic protease inhibition to prevent tissue degradation
• Available for new and existing therapeutic compounds
Technology
• 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 defectAutoimmuneTrauma Membrane ElevatedEtiology unknown Permeability Ca2+
Ischemia etc.
Cellular Dysfunction, Widespread degradation CalpainInstability, cell death of multiple substrates activation
(receptors, kinases, Calpain
Cytoskeleton, etc.) inhibitor
Tissue
preservation
Technology(Disease Etiology and Therapeutic Target Validation)
Pipeline
Product Indication
C-301 Epilepsy
MYODUR
C-202
C-203
Muscular Dystrophy
ALS
CIDP
NEURODUR Multiple Sclerosis
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
0 1 2 3 4 5 6
C-203 (CIDP)
C-202 (ALS)
C-301 (Epilepsy)
NEURODUR (Multiple Sclerosis)
MYODUR (Muscular Dystrophy)
$ Billions
Research
Neuromuscular Neurodegenerative
- Cardiomyopathys - Amyotrophic lateral sclerosis (ALS)* - Cancer cachexia* - Ototoxicity- AIDS wasting - Retinal degeneration- Denervation injury - Spinal cord injury
- Alzheimer’s* - Huntington’s* - Chronic inflammatory
demyelinatingpolyneuropathy (CIDP)*
*Internal Development
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
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
Jul06
Toxicology and Safety Toxicology and Safety Pharmacology StudiesPharmacology Studies
IND SubmissionIND Submission
Phase I-II DMD Clinical StudyPhase I-II DMD Clinical StudyAssay DevelopmentAssay Development
Pre-Clinical Pre-Clinical StudiesStudies
Jan05 Apr05 Jul05 Oct05 Jan06 Apr06 Oct06 Jan07
US and EU ODA FilingUS and EU ODA Filing
Non-GMP ManufacturingNon-GMP ManufacturingGMP ManufacturingGMP Manufacturing
Pre-formulation, Characterization, Solubility, Stability, and FormulationPre-formulation, Characterization, Solubility, Stability, and Formulation
Multiple 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
NEURODUR Results in EAE Model for MS
Propriety unpublished data
Epilepsy (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
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 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
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 Gaucher disease$300K/year/patient
REPLAGAL Fabry disease$170K/year/patient
Factor VIII Hemophilia$70K/year/patient
hGH GH deficiency$20K/year/patient
Orphan Drug PlatformBusiness Model
• High Commercial Value • Market Exclusivity • Regulatory advantages• Target marketing• Distribution and reimbursement infrastructure amortization • Low COGS
Two-Year Business Plan
• Enter phase III with MYODUR
• Have 3 products in the clinic
• Close 2 corporate partnerships
• Conduct appropriate financings
Investor 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