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CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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Page 1: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases
Page 2: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

CepTor Corporation

Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

Page 3: 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.

Page 4: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 5: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

Technology

• Receptor – mediated drug targeting to cells

• Therapeutic protease inhibition to prevent tissue degradation

• Available for new and existing therapeutic compounds

Page 6: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 7: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 8: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

Technology(Disease Etiology and Therapeutic Target Validation)

Page 9: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

Pipeline

Product Indication

C-301 Epilepsy

MYODUR

C-202

C-203

Muscular Dystrophy

ALS

CIDP

NEURODUR Multiple Sclerosis

Research Pre-Clin IND

Page 10: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 11: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 12: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 13: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 14: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 15: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 16: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

MYODUR Results in MDX Model for DMD

Propriety unpublished data

Page 17: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 18: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 19: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

NEURODUR Results in EAE Model for MS

Propriety unpublished data

Page 20: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 21: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 22: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 23: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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%

Page 24: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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

Page 25: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

Orphan Drug PlatformBusiness Model

• High Commercial Value • Market Exclusivity • Regulatory advantages• Target marketing• Distribution and reimbursement infrastructure amortization • Low COGS

Page 26: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

Two-Year Business Plan

• Enter phase III with MYODUR

• Have 3 products in the clinic

• Close 2 corporate partnerships

• Conduct appropriate financings

Page 27: CepTor Corporation Focusing on Targeted Therapies for Neuromuscular and Neurodegenerative Orphan Diseases

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