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Medgenics ($MDGN) September 2011 Investor Presentation

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Medgenics (NYSE AMEX: MDGN) is developing and commercializing Biopump, a proprietary tissue-based platform technology for the sustained production and delivery of therapeutic proteins using the patient's own skin biopsy for the treatment of a range of chronic diseases including anemia, hepatitis C and hemophilia. Medgenics believes this approach has multiple benefits compared with current treatments, which include regular and costly injections of therapeutic proteins.

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Medgenics ($MDGN) September 2011 Investor Presentation

  1. 1. Medgenics  Investor   Presentation September  2011   Andrew  L.  Pearlman,  Ph.D.  President  &  CEO  NYSE  Amex:  MDGN  AIM:  MEDU,  MEDG  
  2. 2. Forward-­Looking Statements: This presentation includes certain estimates and other forward-­looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, including statements with respect to anticipated operating and financial performance, clinical results, potential partnerships, licensing opportunities and other statements of expectation. Words such as and variations of these words and similar expressions, are intended to identify these forward-­looking statements. While we believe these statements are accurate, forward-­looking statements are inherently uncertain and we cannot assure you that these expectations will occur and our actual results may be significantly different. These statements by the Company and its management are based on estimates, projections, beliefs and assumptions of management and are not guarantees of future performance. Important factors that could cause actual results to differ from those in the forward-­looking statements include the factors described in the filings with the U.S. Securities and Exchange Commission. The Company disclaims any obligation to update or revise any forward-­looking statement based on the occurrence of future events, the receipt of new information, or otherwise. 2  
  3. 3. Truly Personalized Medicine: Innovative med-­tech and therapeutics company developing sustained protein therapy for chronic diseases. Continuous protein production and delivery from Designed to be better, safer and cheaper, replacing scores of injections, in $130b protein market. Potentially offering major advantages in treating a wide range of chronic diseases starting with anemia, hepatitis C and hemophilia. 3  
  4. 4. Key Considerations:  Publicly Listed: NYSE Amex: MDGN;; LSE AIM: MEDU, MEDG Proprietary Biopump, an autologous tissue-­based platform technology for the sustained production and delivery of therapeutic proteins. 3 lead products address markets >$16B/yr in anemia, hepatitis C and hemophilia. EPODURE  in  Phase  I/II  dosing  trials,  producing  EPO  in  anemia  patients  with  CKD.   INFRADURE  preparing  to  commence  Phase  I/II  trials  in  Israel  for  production  of  IFN-­a   to  treat  hepatitis  C.   HEMODURE  being  developed  as  a  sustained  Factor  VIII  therapy  for  the  prophylactic   treatment  of  hemophilia.   A single treatment in anemic patients shown to last 6-­28 months. Solid IP protection: 10 issued and 50+ pending patents. First validating pharma deal: Baxter for hemophilia, Factor VIII Biopump. Experienced management;; founded in 2000, based in Israel and U.S. 4  
  5. 5. Our Biopump Method:1. Harvest  the  tissue  by  needle  biopsy  from    2. Process tissue  into  a  drug  producing   Biopump  in  10-­14  days  by  controlled   Biopump and a toothpick transfer  of  desired  gene.  3. Measure   protein  production  level.   10  Harvests  4. Implant required  number  of  Biopumps   4  Implants    5. Reversible  by  simple  ablation.   5  
  6. 6. Repeat Bolus Injections vs. Biopump:Protein   Injection  overshoot    Adverse side effectsconcentration     EPO: Cardiovascular Riskin  serum   IFN-­a: Severe flu symptoms Therapeutic   window   ..   ..   Biopump Sustained Clinical Dose #  of  Days   Injection   undershoot   Missed  injection                Injected  dose  in  range   (No  Effect)     6  
  7. 7. Biopump Platform: EPODURE in  vitro: for anemia Sustained EPO high level production for 6+ months EPODURE long term in vitro EPO secretion 10000 Skin 1 Skin 2 1000IU / Biopump / day 100 10 1 6 9 16 25 36 46 66 80 101 122 143 164 185 T ime (Days) Ti Time to Implant in Patient 7  
  8. 8. EPODURE Replaces Injections, Elevates Hemoglobin Level>28 Months of Continuous Anemia Relief: Estimated baseline 100 days after last injection EPO Injections EPODURE   8  
  9. 9. Biopump Platform: INFRADURE in  vitro:  for Hepatitis CSustained IFN-­a high level production for 6+ months INFRADURE Long term in-vitro production 10000 1000IFN ng/Biopump/day 100 10 1 6 9 16 27 37 48 62 76 97 118 139 160 181 202 223 244 Days from harvesting Ti Time to Implant in Patient 9  
  10. 10. How the Biopump Therapeutic System Works: b   a  a    Harvest  dermis  tissue                 -­   d   c  b    Transfer  to  processing  station.   i  c    Adenoviral  gutless  vector  carrying   h              gene  for  desired  protein.     e  d    Process  each  micro-­organ  into   f              Biopump.  e    Biopump  producing  protein.     g   DermaVac Biopump and a toothpickf      Measure  daily  protein  production                per  Biopump  for  dosing.  g    Wash  several  days  to  remove                vector.    h    Re-­implant  Biopumps              subcutaneously  per  dosing.  i    Sustained  local  delivery  of  protein  for                life  of  cells  in  Biopump  (>  6  months).   BioCryo 10  
  11. 11. Unique Platform, Disruptive Potential: Platform for sustained production and delivery of therapeutic proteins Current focus anemia, hepatitis C and hemophilia: Concept proven with EPODURE EPO Biopumps in anemia patients with unprecedented results from single administration: Potentially much more cost effective treatment. Designed to be implemented using standard facilities and procedures. 11  
  12. 12. Potential Healthcare Advantages:Increased efficacy: Sustained  dose  within  therapeutic  window.  Improved safety:  not  produced  in  rodent  cells.   Fewer  side  effects  resulting  from  overdose.   The BiopumpImproved patient compliance: therapeutic system Replaces  frequent  injections.   could change the Reliable  treatment    not  dependent  on  patient.   paradigm for theReduced costs: treatment of chronic Does  not  require  an  expensive  protein    manufacturing   diseases. facility.  Reversible treatment: Simple  process  (ablation).  Extend treatment to under-­treated populations: Existing  treatment  impractical  or  too  costly.   12  
  13. 13. Lead Products: EPODURE (anemia) Sustained EPO therapy ($9.6B/yr) could replace  $15-­30,000/yr/patient  in injections, offering: Superior  treatment  at  lower  cost:  >6-­12  months  sustained  EPO  therapy,  avoid   peak  overdose  risks,  improve  compliance  and  reliability.   Improved  hemoglobin  control,  directly  address  current  key  issues  in  anemia:     FDA  hemoglobin  safety,  CMS  reimbursement  bundling.   INFRADURE (hepatitis C) Sustained IFN-­a therapy ($2.6B/yr) could replace  $35,000/yr/patient in injections, offering: Effective  treatment  with  greatly  reduced  side  effects    safer,  patient  friendly,   lower  cost  and  unmatched  treatment  interval:  6+  months.   Cost  effective  alternative  for  interferon  therapy,  direct  antiviral  agents.   HEMODURE (hemophilia) Sustained FVIII therapy for ($4B/yr) could replace  >$100-­250,000/yr/patient  injections, potentially offering: PROPHYLACTIC TREATMENT     >  6-­12  months  sustained  FVIII  therapy  from  single  treatment.   Improved  QOL  at  a  lower  cost.   13  
  14. 14. Pipeline for Biopump Platform: Condition Protein Development stage 2009 Sales ($b)* Anemia Erythropoietin Phase I/II 9.6 Hepatitis C Interferon Alpha Preclinical 2.6 Hemophilia Factor VIII Preclinical Co-­Dvlpmt. 4.0 Growth Retardation Growth hormone Future Candidate 2.9 Multiple Sclerosis Interferon Beta Future Candidate 5.2 Diabetes Insulin Future Candidate 13.3 Arthritis IL-­1Ra Future Candidate 18.1 Wound Healing PDGF-­BB Future Candidate NA Obesity Peptide YY3-­36 Future Candidate NA Chronic Pain IL-­10 Future Candidate NA Cancer Recovery G-­CSF Future Candidate 5.2(1)  R&D  Pipeline  News,  La  Merie  Business  Intelligence,  March  10,  2010   14  
  15. 15. Value  Proposition:   Game changer Potential major win for: Patients Physicians Payors Pharma PartnersReplaces frequent Billable procedure Reduces costs Blockbuster opportunitiesInjections Improved patient flow Fewer claims No multi-­$B proteinImproves quality of life manufacturing plant Increased patient PreventivePrevents side effects compliance & treatment Superior valueMore reliable treatment reliability proposition to capture market shareSafer, better outcomesMuch more affordable 15  
  16. 16.   Business model Revenues before product approval. Platform:  Same  low-­cost  core  technology    multiple  deal  opportunities.   Major Opportunities:  Each  >$1B/year,  no  protein  factory  needed.   Other Opportunities: Niche  applications    rapid  route  to  product  approval;;  high  value-­ added.   New  proteins/markets.   Timing:  Typical  deals  at  Phase  I/II  or  Phase  II.   Early Revenue Source: Pre-­approval  milestone  payments,  typically  $100M+.   Royalties  on  product  sales,  or  transfer  price.   16  
  17. 17. Phase I/II Interim Study Conclusions:Presented at ASN November 2010 by leading authority.*EPODURE is safe and doseable;; no antigenic response.Clinical feasibility demonstrated.Single EPODURE administration can raise and maintainhemoglobin levels for up to 28 months without any injections ofESAs.EPODURE has significant potential to become an effectiveinterventional treatment a paradigm shift.* Dr. Anatole Besarab Director of Clinical Research in the Division of Nephrologyand Hypertension at Henry Ford Hospital in Detroit, Michigan. 17  
  18. 18. Hemoglobin Cycling with EPO Injectionsvs. EPODURE Response:   EPO  Injections   EPODURE   18  
  19. 19. EPO Naïve Hemoglobin Response: EPODURE   19  
  20. 20. EPODURE Mid-­dose Sustained Hemoglobin Response: EPODURE   20  
  21. 21. Route to Revenues:Regulatory: Safety  already  shown  especially  as  treatment  can  be  reversed;;   providing  clear  route  forward.   QA  designed  in:  automated  processor  using  sealed  cassettes.   Niche:  expedited  route,  small  pivotal  trial  for  approval.  Clinical Pathway Simplified: Delivers  well-­known  proteins  now  in  routine  clinical  use.   Better  delivery  and  compliance.   Better  safety:  own  protein,  no  peak  overdose  or  under-­dose   between  injections  and  ability  to  reverse  or  stop  treatment.  Scale Up: Reliable  method    >5,000  Biopumps  made.   Automated  bioprocessor  with  sealed  cassettes    in  development.  Partnering: Typically  aiming  for  Phase  I/II.   21   Potential  major  revenue  source  before  sales,  based  on  comps.  
  22. 22. IP Protection & Practical Application: 10 issued patents, >50 pending patents. Licenses have been acquired for Biopump and related key elements: Factor VIII license from University of Michigan. Freedom to operate: off-­patent proteins or use of cDNA. Towards scale-­up and automation: >5,000 Biopumps produced. Using reliable DermaVac harvest and implantation devices. Prototype semi-­automated processing station demonstrated. New generation processing in development. 22  
  23. 23. Experienced Management Team:   Extensive  experience  in  healthcare  industry,  founded,  operated  and  led  firms  to  M&A   totaling  billions  of  dollars.   Board of Directors: SAB/Advisors: Management:  Andrew L. Pearlman PhD Clinical  &  Regulatory:   Andrew L. Pearlman PhDPres/CEO    >25yrs  Biomed   Allen Nissenson MD  past  Pres.   Founder President & CEO RPA,    CMO  DaVita  Corp   DellioEugene Bauer MD, Exec Chm. Anatole Besarab MD  World   authority  renal  anaemia,  Dir.    RPA   Chief Operating OfficerFormer  Dean, Stanford  Med  Sch,   Xoma, NeosilConnetics,  Peplin     Bruce Bacon MD  Leading  Hep  C     authority-­  past  Pres.ASLD   Stephen Bellomo MScJoel Kanter, founding investor Andra E. Miller PhD  Former  FDA   VP Product Development &I-­Flow,  Prospect  Medical,  Prolor   cell/gene-­therapy  group  leader   IP; COO Medgenics Israel   Stephen Ettinger DVD  World  Gary Brukardt, former  CEO  Renal   renowned  veterinary  expert   Baruch Stern PhD 
 Dean Hautamaki MD  Chairman   Chief Scientific OfficerCare  Group  (sold  for  $3.5b)     Dept  of  Medicine  ,  SMH   Ehud Shoshani MDStephen McMurray MD RPA,   Strategy:   VP Clinical AffairsFresenius,  DaVita   Mr. Burt Rosen  Senior  Pharma   Quintiles Israel   Exec,    Congressional  liaison  Alastair Clemow PhD Mr. Isaac Blech  Biotech  investor  -­   Phyllis Bellin MBAJ  &  J,    Geliflex,  Prolor   Celgene  ,  ICOS,  Nova   Director Finance & Admin     Technology:   CitibankIsaac Blech-­Biotech investor Mark A. Kay MD Stanford,  ASGT   Nir Shapir PhD 
Celgene,  ICOS,  Nova   Amos Panet PhD  Virology,Hebrew   VP R&D Development Univ  -­  Hadassah  Medical  Center   Beckman-Coulter   23  
  24. 24. Milestones 2011-­2012: EPODURE: Complete  Phase I/II trial;;  seek  U.S.  IND  for  Phase IIb,   other  pre-­trial  preparations  for  launch  in  renal  anemia.   INFRADURE: approval  from  Israeli  Ministry  of  Health  to  launch  and   obtain  initial  data  from  Phase I/II trial in hepatitis C.   HEMODURE: Improve  FVIII  production  and  delivery  in  mice  and  large   animal  model,  towards  levels  sufficient  for  clinical  studies  in  hemophilic   patients.   Partnering: Pursue  strategic  alliances   Core Technology:  Further  develop  the  platform  technology.   New Applications: Initiate  development  of  additional  applications  with   other  proteins  e.g.,  niche  applications.   24  
  25. 25. Key Take-­Aways:Disruptive platform technology for >$130B protein market.Potentially better, safer and less costly.Strong IP portfolio and value proposition.Lead products focusing on $16B in anemia (EPODURE)hepatitis C (INFRADURE) and hemophilia (HEMODURE).Working in patients shown 6-­28 months from a singletreatment.Partnering validation.Experienced, proven team. 25  
  26. 26. Medgenics  BioMed   Presentation September  2011   Andrew  L.  Pearlman,  Ph.D.  President  &  CEO  NYSE  Amex:  MDGN  AIM:  MEDU,  MEDG