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 defect Autoimmune Trauma Etiology unknown Ischemia etc.

Membrane Permeability Elevated Ca 2+ Cellular Dysfunction, Instability, cell death Widespread degradation of multiple substrates (receptors, kinases, Cytoskeleton, etc.) Calpain inhibitor Calpain activation Tissue preservation

Technology

(Disease Etiology and Therapeutic Target Validation)

Pipeline

Product MYODUR C-203 Indication Muscular Dystrophy NEURODUR Multiple Sclerosis C-301 C-202 Epilepsy ALS CIDP Research Pre-Clin IND

Product MYODUR NEURODUR C-301 C-202 C-203

Pipeline

Indication Muscular Dystrophy Multiple Sclerosis Epilepsy ALS CIDP Anticipated Near Term Status File Phase I/II IND Receive Orphan Status

JCR License – Pacific Rim

Partner WW Partner WW File for Orphan Designation File for Orphan Designation

Worldwide Market Potential

MYODUR (Muscular Dystrophy) NEURODUR (Multiple Sclerosis) C-301 (Epilepsy) C-202 (ALS) C-203 (CIDP) 0 1 2 3

$ Billions

4 5 6

Research

Neuromuscular Neurodegenerative - Cardiomyopathys - Amyotrophic lateral sclerosis (ALS)* - Cancer cachexia* - Ototoxicity - AIDS wasting - Denervation injury - Retinal degeneration - Spinal cord injury - Alzheimer’s* - Huntington’s* - Chronic inflammatory demyelinating polyneuropathy (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

US and EU ODA Filing IND Submission 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 Apr06 Jul06 Oct06 Jan07 Jan05 Apr05 Jul05 Oct05 Jan06

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 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 Gaucher disease REPLAGAL Fabry disease Factor VIII hGH Hemophilia GH deficiency $300K/year/patient $170K/year/patient $70K/year/patient $20K/year/patient

Orphan Drug Platform Business 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