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Cebix Sofinnova Japan Presentation

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Cebix Presentation for Sofinnova Japan

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Cebix Sofinnova Japan Presentation

  1. 1. 1  CONFIDENTIAL  
  2. 2. Cebix  Mission   2   Cebix  will  develop  safe  and     effec?ve  therapies  for  the   treatment  of  long-­‐term     complica?ons  in  diabetes  
  3. 3. Re?nopathy  (20%)   Nephropathy   Peripheral  Neuropathy   Autonomic  Neuropathy   Long-­‐term  complica?ons  of     Type  I  Diabetes   (35%)   (50%)   (25%)   3  
  4. 4. 4   Type  I  Diabetes   Pro-­‐insulin/C-­‐pep?de   C-­‐pep?de   Insulin   Pro-­‐Insulin   4   C-­‐pep?de  is  the  second  hormone   •     Similar  to  Preglucagon      –  Glucagon      –  GLP-­‐1  and  GLP-­‐2  
  5. 5. An agent for the treatment of mild to moderate peripheral neuropathy in type 1 diabetes The therapy of choice in the treatment of this significant unmet medical need, based on improvement of sensory function Initial Target Indication 5   Product Profile C-­‐Pep?de  Replacement  
  6. 6. C-­‐pep?de  influences  major   metabolic  pathways   Ca2+   MAPK   Na+,  K+-­‐  ATPase   eNOS   Na+   K+   Ca2+   Transcrip?on     factors   NO NO  G-­‐protein  ATF-­‐1   CREB   ZEB   NFκB   PPARγ 6  
  7. 7. 35 40 45 50 55 60 65 70 0   1   2   3   4   5   6   Onset  of     diabetes   1  week   2  months   5  months   m/s Nerve  Conduc?on  Velocity   8  months   C-­‐pep?de   C-­‐pep?de   Sima  et  al  2001   Nerve  Func?on  Improved   Diabe?c  rat  model   CONFIDENTIAL   Non-­‐diabe?c  control   Diabe?c  no  treatment   Diabe?c  +  C-­‐pep?de  
  8. 8. 8   C-­‐pep?de  Replacement:   Does  it  work  in  Type  1   Diabetes  pa?ents?  
  9. 9. 9   Restores  sensory  nerve  conduc?on   Phase  2a   Ekberg  et  al,     Diabetes  2003   50 52 54 56 Baseline 6  wks 12  wks SCV  (m/s)   C-­‐pep?de  n=26 Placebo  n=23 Healthy  Controls   p<0.05   9  
  10. 10. C-­‐pep?de  Improves:  Nerve  Conduc?on,  Vibra?on   Percep?on  &  Clinical  Status   Phase  2   Ekberg  et  al,     Diab  Care  2007   Nerve  conduc:on:     %  pa8ents  >1  m/s   0 10   20   30   37  %   19  %   40    p<0.03     N=139  Placebo  n=  47   C-­‐pep?de  n=92   p<0.002   Median  differences   0.20   0.15   0.10   0.05   0   -­‐0.05   Foot    Leg                                       ns   Vibra:on  Percep:on:   Δ  VPT  (SDS)   0   0.5   1.0   1.5    Clinical  Status:   Neurological  Impairment   2.0   p<0.01  ns   Median  differences  
  11. 11. Safety  profile  of  C-­‐pep?de   •  6  safety  pharmacology  studies  completed   •  5  toxicological  studies  (monkey  and  rat)   –  Up  to  60x  higher  than  replacement  dose   •  19  clinical  studies  conducted  exposing  ~300  pa?ents   –  Replacement  of  endogenous  levels  intended:  0.4-­‐  6  nM   ⇒ Benign  safety  profile,  consistent  with  replacement  therapy   CONFIDENTIAL   11  
  12. 12. 12   Development  Path  Secured    Unmet  medical  need    Biology    -­‐    Func?on  and  Pathophysiology    Safety    Efficacy    Dose    -­‐    Replacement    Drug  manufacturing   1.  Regulatory  Path   2.  Intellectual  Property   3.  Drug  delivery  
  13. 13. Pre-­‐IND  Mee?ng  with  FDA   July  2010   •  Regulatory   –  FDA  Confirmed  qualifica?on  of  Subpart  H   •  Allows  use  of  surrogate  end  point  for  Pivotal  Phase  2b   •  Clinical   –  Nerve  conduc?on  velocity  accepted  as  the  sole   primary  endpoint  for  approval   •  Nonclinical   –  IND-­‐enabling  tox  plan  endorsed  by  FDA   13  
  14. 14. Intellectual  Property   •  Seven  patents  issued:   –  Formula?on   –  Cardio  autonomic  effect   –  Ac?ve  pentapep?de   •  Three  submioed  to  US  Patent  Office  in  2009-­‐2010   –  Effect  on  erec?le  dysfunc?on   –  Effect  on  hypoglycemia   –  Subject  of  maoer  on  long-­‐ac?ng  form   •  Japan  strategy   –  Will  file  PCT  within  12  months  of  original  filing   –  Plan  to  use  the  Patent  Prosecu?on  Highway  (PPH)   14  
  15. 15. Formula?on  Criteria   15   Injec?on  volume  ≤  1  mL   1   2   3   4   5   6   7   Syringeability:      ≤  27  gauge      <  20  seconds     <20%  drug  loss  in  burst   PK  profile  consistent  with     once  weekly  dosing  
  16. 16. Selected  Formula?on  Technologies   16   PROMAXX   Atrigel   Pumps   Trans-­‐   dermal   patch   Octoplus   Eryto-­‐   pharm   Halozyme   Altus   Alkamer   Nektar   Enzon   Syringability  ≤  27  gauge     Stable  for    >  1.5  years  at  4°C   PK  profile  consistent  with  once  weekly  dosing   No  more  than  20  percent  drug  loss  in  burst   Product  load  of  at  least  1  percent  of  volume   Camurus   Flamel   Durect   Alkermes   La?tude  
  17. 17. PK  profile   17   100   1   10   C-­‐pep?de  conc  (ng/ml)   0   4   8   12   16   Time  (days)   T1/2  in  monkey  ~  3  days   Extended  Half-­‐life  Product  Depot  Product  
  18. 18. Path  Forward   Development   18  CONFIDENTIAL  
  19. 19. Cebix  Development  Program   19   2011   2012   2013  2010   Formula?on   &   CMC   6-­‐mo  interim  data:   surrogate  marker   12-­‐mo  data:   clinical  end-­‐point   Pivotal  Phase  2b  NEUROPATHY   Human  PK   pre-­‐IND  mee?ng   IND   Submission   Partnering  
  20. 20. World  Wide  Incidence  –  Type  1   CONFIDENTIAL   20  
  21. 21. C-­‐pep?de  Replacement   Ini?al  Commercial  Opportunity   Type  I  diabe?cs  (auto-­‐immune)  in  US+  EU:  4  million     Severe     neuropathy   No   neuropathy   Type  I   Diabetes   Type  II  Diabetes   50%   400,000  pa:ents            25%   penetra?on   Mild-­‐to-­‐moderate   neuropathy   21   10%   40%   90%   10%  
  22. 22. Diabetes  Market-­‐  Japan   22   50,000  pa?ents   Age  group:  under  20   Annual  incidence  rate:  1.3-­‐1.7/100.000     Diagnosed  Type  I  diabetes/  IDDM:   A  Amos  et  al  1997,  Diabe?c  Medicine  1997   Global  epidemiology,  in  The  epidemiology  of  diabetes  mellitus  2001   IDF:  Diabetes  Atlas  2000   All  diabetes:  8.7  million  pa?ents   Age  group:  older  than  20   400,000  pa?ents   5  %:  LADA,  SPIDDM,  type  1,5      Kobayashi  T  et  al     Ann  N  Y  Acad  Sci  958  :  117-­‐130,  2002  
  23. 23. C-­‐pep?de  Replacement   •  Large  unmet  need   •  Experienced,  well  funded  team   •  Convincing  biology   •  Aorac?ve  risk-­‐benefit  profile   •  Strong  proof-­‐of-­‐concept  in  mul?ple  indica?ons   •  Once-­‐weekly  dosing  formula?on  established   •  An?cipate  filing  IND  Q4  2010   23  
  24. 24. Partnering  Objec?ve   •  Cebix  is  seeking  to  partner  the  once-­‐weekly  C-­‐ pep?de  replacement  product  within  Japan  or   throughout  a  broader  region  up  to  and   including  Worldwide  opportuni?es   CONFIDENTIAL   24  
  25. 25. Contacts   James  Callaway,  PhD   President  R  &  D   Email:  jim@cebix.com   Phone  (office):  +1-­‐858-­‐729-­‐6502   Phone  (mobile):  +1-­‐858-­‐967-­‐1471   Annica  Mårtensson,  PhD,  MBA   Director,  Pharm.  Development  &  Corp.  Strategy   Email:  annica@cebix.com   Phone  (office):  +1-­‐858-­‐729-­‐6503   Phone  (mobile):  +1-­‐858-­‐366-­‐2548   CONFIDENTIAL   25  

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