Xechem International - PowerPoint PPT Presentation

1 / 27
About This Presentation
Title:

Xechem International

Description:

Title: Xechem International Author: wpursley Last modified by: ems Created Date: 1/13/2004 11:16:52 PM Document presentation format: On-screen Show – PowerPoint PPT presentation

Number of Views:102
Avg rating:3.0/5.0
Slides: 28
Provided by: wpu5
Category:

less

Transcript and Presenter's Notes

Title: Xechem International


1
(No Transcript)
2
CepTor Corporation
  • Focusing on Targeted Therapies for Neuromuscular
    and Neurodegenerative Orphan Diseases

3
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.

4
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

5
Technology
  • Receptor mediated drug targeting to cells
  • Therapeutic protease inhibition to prevent tissue
    degradation
  • Available for new and existing therapeutic
    compounds

6
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

7
Technology
  • 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

8
Technology(Disease Etiology and Therapeutic
Target Validation)
9
Pipeline
10
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
11
Worldwide Market Potential
Billions
12
Research
  • 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

15
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

16
MYODUR Results in MDX Model for DMD
Propriety unpublished data
17
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
Jul06
18
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

19
NEURODUR Results in EAE Model for MS
Propriety unpublished data
20
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

21
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

22
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

23
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 gt95

24
Orphan 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

25
Orphan Drug PlatformBusiness Model
  • High Commercial Value
  • Market Exclusivity
  • Regulatory advantages
  • Target marketing
  • Distribution and reimbursement infrastructure
  • amortization
  • Low COGS

26
Two-Year Business Plan
  • Enter phase III with MYODUR
  • Have 3 products in the clinic
  • Close 2 corporate partnerships
  • Conduct appropriate financings

27
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
Write a Comment
User Comments (0)
About PowerShow.com