A1261863904wNZUF

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Revolutionizing iron load disorders treatment

• Team
• Daniela Couto
• Ana Carina Silva
• Ana Fernandes
• Cláudia Vistas

PIs Pedro Oliveira Marco Marques IBB-Institute
for Biotechnology and Bioengineering,
IST Tutor Professor Miguel Prazeres
3rd July 2008
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Market Analysis - Clinical Need
Iron load disorders
gt 0.5 billion people
More than 0.5 billion people require removal of
iron from their bodies
Transplant or Death
Iron accumulation
Organ damage
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How is Iron Removed?
Bacteria
Iron Capture molecules
Core IP
Iron
Iron Capture has a library with more than 300
producers!
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Key Features
Facilitates FDA trials
New opportunity for IP
Higher efficiency
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Market Funneling
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Market Analysis - Market Size
The iron removal drug Exjade launched in 2005
reached the billion dollar mark in 2006. Frost
Sullivan, 2006
Iron Capture technology
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Market Analysis - Competition
Drugs Iron Capture Desferal (Novartis) Ferriprox (Apotex Inc.) Exjade (Novartis)
Oral route of administration ? ? ? ?
Long effective period ? ? ? ?
High iron removal efficiency ? ? ? ?
Minimize side effects ? ? ? ?
Price ? ? ? ?
An ideal iron capture molecule would be taken
orally, three times a week and without so many
side effects like the alternatives available on
the market Dr. Rita Fleming, Hematologist
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Market Analysis - SWOT Analysis
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Value chain for Iron Capture business strategy
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Product development process for Iron Capture
business strategy
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Project scheduling
()
Today
Actual progress
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Conclusions and Further Work
Iron Capture needs 800,000 to proceed with RD
and firm during the next 36 months.
The expected return after IP licensing several
orders of magnitude higher
Iron Capture offers a unique value proposition
over existing products in the market place a
novel technology that will produce iron capture
molecules with higher ability to remove iron,
able to confer highest therapeutic efficacy.

• Future Work
• Optimization of iron capture molecule production
• Protect the technology by filing a patent
• Pilot a business plan
• Licensing negotiations

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• It is not the strongest of the species that
  survives, nor the most intelligent
• but the one most responsive to change.
• 
  
  Charles Darwin

PIs Pedro Oliveira Marco Marques Tutor Professo
r Miguel Prazeres

• Team
• Daniela Couto
• Ana Carina Silva
• Ana Fernandes
• Cláudia Vistas

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Chemical vs. Biological Synthesis

• In this research project DSM, Delft University of
  Technology and the University of Groningen
  cooperatively investigate how synthetic process
  steps in the current production of antibiotics
  may be replaced by an entirely biological
  production process where a single
  micro-organismperforms all the required
  synthetic steps. Such integration leads to
  obvious advantages of less waste, less production
  of carbondioxide and higher overall yields.

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Market Analysis - Clinical Need
Iron overload Iron overload
Hereditary hemochromatosis Secondary overload - Iron-loading anemia and/or transfusion Dietary iron overload Chronic liver diseases Hepatitis C and B Miscellaneous causes of iron overload Atherosclerosis and vascular injury Restenosis Cancer Papillomavirus
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Market Analysis - Competition and IP
Pharmaceutical Drugs/ Characteristics Advantages Disadvantages
Deferoxamine (Desferal) Route of Administration SQ, IV Half-life 20 minutes Primary routes of iron excretion Urine/Stool Dose range 20-60 mg/kg/d Price 8.8 /g - Long-term experience - Effective in maintaining normal or near-normal iron stores Reversal of cardiac disease May be combined with deferiprone - Requires parenteral infusion - Ear, eye, bone toxicity - Poor compliance
Deferiprone (Ferriprox) Route of administration PO Half-life 2 to 3 hours Primary route of iron excretion Urine Dose range 50-100 mg/kg/d Price 5.5 /g - Orally active - Safety profile well established - Enhanced removal of cardiac iron - May be combined with deferoxamine - May not achieve negative iron balance in all patients - Risk of agranulocytosis and need for weekly blood counts
Deferasirox (Exjade) Route of administration PO Half-life 8 to 16 hours Primary route of iron excretion Stool Dose range 20-30 mg/kg/d Price 60 /g - Orally active Once-daily administration Demonstrated equivalency to deferoxamine at higher doses - Trials in several hematologic disorders - Limited long-term data - Need to monitor renal function - May not achieve negative iron balance in all patients
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Solution Requirements

• In our technology, we aim to produce a higher
  chelator able to confer the following properties
  when incorporated into a drug
• oral route of administration
• plasma half-life enough to give constant
  protection from labile plasma iron
• high therapeutic index
• high molar chelating efficiency
• none important side effects or only in
  iron-depleted subjects
• high ability to chelate intracellular cardiac and
  other tissue iron.

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Market Analysis - Competition and IP

• Keystone Biomedical Inc. is the holder of several
  patents regarding the chemical synthesis of
  siderophores
• Geraci et al, US Patent 5.952.492, 1999
• Geraci et al., US Patent 6.335.443 B1, 2002
• Gaudioso Weglarz, US Patent 6.063.919, 2002).

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Iron Capture

• Innovative and highly qualified team
• Iron Capture has envisioned a novel technology
  that will produce chelators with higher ability
  to chelate iron, able to confer highest
  therapeutic efficacy.
• We have an extended library with more than 300
  potential siderophore producers.
• We will work for revolutionizing the chelating
  agents market.

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Market Analysis - Three dimensions for examining
opportunities
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Manufacturing process of Iron Capture products
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Financial Projections
    1st year 2nd year 2nd year 3rd year
Instrumentation Fermentors 100000
Instrumentation HPLC 25000
Instrumentation Orbital 5000
Instrumentation LCMS 75000
Consumables Consumables 20000 20000 20000 20000
Facilities rent Facilities rent 18000 18000 18000 18000
Human resources 4 technicians 56000 56000 56000 56000
Human resources CEO and CFO 56000 56000 56000 56000
Patent   100000 100000 100000 100000
Toxicology tests   30000 30000
Total (per year) Total (per year) 355000 215000 215000 215000
TOTAL 800,000 800,000 800,000 800,000 800,000
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Alliance or Acquisition?
Novartis Alliances Novartis Alliances
Idenix Pharmaceuticals QLT
skyePharma Myogen
Biosite MorphoSys
Speedel Cellzome
Merck Xenogen
Genentech/Tanox Vernalis
Schering AG Biotec
Dr. Reddys Syngene
Novartis Business Units
Pharmaceutical RD StrategiesInnovation and
Productivity Rationale Novartis Alliances
(World), 2005.
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Market Funneling
Iron Chelating Agents
Water Treatment
Pharmaceutical
Agronomy
Ceramic Industry
Iron Detection Methods
Anti- corrosion
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Application Field Comments
Water Treatment in the treatment of drinkable water the contamination with iron ions is not considered a relevant issue since the maximum value of iron concentration would never be achieved because the water is captured from a water spring the contamination of these waters is only possible to occur after leaving the water treatment station domain, through the pipelines that conduct water throughout all the city due to ion release arising from degradation or corrosion alternatives are welcome given the limitations of traditional chemical processes for the waters decontamination process this technology can be useful to decontaminate iron-based lixiviation waters produced during mineral extractions
Agronomy according with the CEE legislation 2003/2003 there is not a recommendation to perform iron analysis to liquid or solid fertilizers. The analysis to the soils also does not require irons elements measurements
Geology is not applicable directly into the minerals extraction, although could be used perhaps for removing contaminating iron ions from ceramics, which have high economic impact in this industry
Anti-corrosion could be used on the identification of the presence of steel corrosion, rust, for example, the presence of iron ions could induce a change of color
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Application Field Comments
Pharmaceutical Industry hemochromatosis is an iron overload disease characterized by peaks of iron overload that are not possible to predict or to enclose in proper levels the only treatment available for hemochromatosis is the use of deferroxamine as chelating agents the deferroxamine is a treatment that cannot be administrated to children with ages inferior to 3 years old and these chelating agents are only capable of immobilize 20 mg of iron ions per day the existent therapies used are inadequate and rise some ethical problems once phlebotomy requires opening a vein for letting blood Chemical agents are known to have to some hypersensitivity reactions and some other side effects Desferal is an efficient drug, but because it requires parenteral infusion, patients often refuse this treatment an ideal iron-chelator would be taken orally, three times a week and without so many side effects like the alternatives available on the market