<ul><li>Presents Webinar on Accessing Shelved Compounds Through the  CTSA Pharmaceutical Assets Portal </li></ul><ul><li>A...
CTSA PHARMACEUTICAL ASSETS PORTAL www.ctsapharmaportal.org Kate Marusina, Ph.D., MBA  December 2009
Goals <ul><li>To improve information exchange between pharmaceutical companies and the CTSA Consortium/NIH regarding drugs...
Achievements to Date <ul><li>Dr. Francis Collins expressed his support at the CTSA Industry Forum (Feb 2010) </li></ul><ul...
What is CTSA? <ul><li>Clinical and Translational Science Award </li></ul><ul><li>The goal is to transform the local, regio...
CTSA Pharmaceutical Assets “Database” <ul><li>Inspiration: Daniel Rader and BMS-201038  (AEGR-733 ), targeted inhibition o...
Challenges of the “database” approach <ul><li>Closely guarded assets </li></ul><ul><li>Pharma just gearing up for repositi...
Recent Number of NMEs  Approved by FDA is Disappointing PhRMA R&D Productivity (Avg Annual R&D Spend Increase = 10.3%; ’90...
Research & Development Productivity Needs Attention  The Challenge – 1 of 10 is Aspirational!
…… ..but Opportunities to Improve Abound Preclinical Phase 1 Phase 2/ PoC Phase 3 Approval 65  55  25  65  85  Cumulative ...
<ul><li>Initiated in ’07 as a dedicated group of scientists focused on discovering, evaluating, and pre-clinically validat...
Indications Discovery Business Plan –  An Ever-Evolving Model DISEASE RELEVANT SCREENING HYPOTHESIS GENERATION Reg Tox thr...
Pfizer Indications Discovery – Learnings  <ul><li>Pfizer Compounds & Drug Development expertise present significant opport...
Pfizer Indications Discovery – Key Focus Area: MoA-Disease Association  <ul><li>Idea Generation </li></ul><ul><ul><li>MoA ...
An Invitation to Collaboration <ul><li>Improving health through scientific discovery is a lofty goal, in today’s economic ...
Two ways to create a match  between Academia and Pharma
From Academia to Pharma <ul><li>Known compound for  PRE-clinical studies:  http://www.pfizer.com/research/licensing/compou...
How to join the Portal (www.ctsapharmaportal.org)
Members of the Portal (as of June 09)
The Portal at a glance <ul><li>348 researchers nation-wide, including NIH </li></ul><ul><li>Main interest areas of Portal ...
The Portal at a glance <ul><li>80% of participants desire a compound at clinical stage </li></ul><ul><li>>80% desire to ru...
Specific target/disease interests (~150)
Two ways to create a match  between Academia and Pharma
From Pharma to CTSA via FoX target Disease A Disease A Disease C Disease B Disease D Disease B
FoX Pilot for 5 universities <ul><li>Obtained data from Biovista ( www.biovista.com ) </li></ul><ul><ul><li>Researcher-tar...
Data cloud for a target X
Examples of Filters <ul><li>Who published the most </li></ul><ul><li>By university </li></ul><ul><li>By disease </li></ul>...
Example of a Filter
Pharma Concerns re: research with their compounds <ul><li>Different Timing: </li></ul><ul><ul><li>Execution of the study <...
Next steps – Contractual Issues <ul><li>Workgroup  </li></ul><ul><ul><li>Guiding Principles for the MTAs for the compounds...
Call for Action to the Foundations <ul><li>Spread the word about the Portal among your constituency </li></ul><ul><li>Work...
Summary - Future Plans <ul><li>Proactively  engage the pharmaceutical industry  in providing information about the compoun...
Team : <ul><li>UC Davis – Project Management, Company Contacts </li></ul><ul><ul><li>Kate Marusina </li></ul></ul><ul><li>...
<ul><li>QUESTIONS AND ANSWERS </li></ul><ul><li>WEBINAR Accessing Shelved Compounds Through the  CTSA Pharmaceutical Asset...
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Pharma Assets Portal

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Accessing Shelved Compounds Through the CTSA Pharmaceutical Assets Portal

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  • 05/13/10
  • Killing projects early is a theme to return to later in the talk.
  • 05/13/10
  • 05/13/10
  • Pharma Assets Portal

    1. 1. <ul><li>Presents Webinar on Accessing Shelved Compounds Through the CTSA Pharmaceutical Assets Portal </li></ul><ul><li>April 12, 2010 </li></ul><ul><li>www.fastercures.org/train </li></ul><ul><li>Kate Marusina, Ph.D., MBA, Manager, Research Facilitation and Industry Alliance, Clinical and Translational Science Center, University of California Davis School of Medicine </li></ul><ul><li>Dean J. Welsch, Ph.D., Research Fellow, Pfizer Global Research & Development, Indications Discovery Research Unit </li></ul><ul><li>Moderator: Margaret Anderson, Executive Director, FasterCures </li></ul>
    2. 2. CTSA PHARMACEUTICAL ASSETS PORTAL www.ctsapharmaportal.org Kate Marusina, Ph.D., MBA December 2009
    3. 3. Goals <ul><li>To improve information exchange between pharmaceutical companies and the CTSA Consortium/NIH regarding drugs available for repositioning </li></ul><ul><li>Specific emphasis in on drugs discontinued at clinical stage </li></ul><ul><li>Via the Portal, these unique and previously inaccessible assets will be made available to the entire CTSA academic community and the NIH intramural researchers, enabling new translational research and a considerably accelerated path to the bedside </li></ul>
    4. 4. Achievements to Date <ul><li>Dr. Francis Collins expressed his support at the CTSA Industry Forum (Feb 2010) </li></ul><ul><li>Pfizer financially supports development of the Foci-of-Expertise, for hypothesis driven identification of the repositioning opportunities (Dec 2009). </li></ul><ul><li>The Portal project was presented at the dedicated forum at the NIH: “Clinical and Translational Science Awards (CTSA) Pharmaceutical Assets Portal: Matching Academia and Industry for Drug Repositioning” (Dec 2009). </li></ul><ul><li>The Portal Project was listed in the NIH report to the Secretary of Health and Human Services </li></ul><ul><li>The Portal received press coverage in the Nature Medicine, Proto Magazine, Sacramento Bee Newspaper, Drug Repositioning Summit in Boston (October 2009). </li></ul><ul><li>The Portal begins collaboration with AUTM and The National Academies to work out the material transfer and licensing provisions for the drugs originated from the Portal. </li></ul>
    5. 5. What is CTSA? <ul><li>Clinical and Translational Science Award </li></ul><ul><li>The goal is to transform the local, regional and national environment for clinical and translational science, thereby increasing the efficiency, quality and speed of clinical and translational research </li></ul><ul><li>The largest academic integration effort to date </li></ul><ul><li>Required functions: </li></ul><ul><ul><li>Biomedical Informatics </li></ul></ul><ul><ul><li>Regulatory Knowledge and Support </li></ul></ul><ul><ul><li>Training and Education </li></ul></ul><ul><ul><li>Community Outreach </li></ul></ul><ul><ul><li>Translational Pilot studies </li></ul></ul><ul><li>Power of the network (45 - 60 universities) </li></ul>
    6. 6. CTSA Pharmaceutical Assets “Database” <ul><li>Inspiration: Daniel Rader and BMS-201038 (AEGR-733 ), targeted inhibition of the microsomal triglyceride transfer protein (MTP) </li></ul><ul><li>Project sponsored by NCRR in Sept 2008 as “Pharmaceutical Assets Database” </li></ul>JANUARY 10, 2007 Penn Researchers Demonstrate Ability of New Therapy to Treat Patients With Severely Elevated Cholesterol Levels New England Journal of Medicine Publishes Report of New Therapeutic Approach That Has Implications for People With High Cholesterol (PHILADELPHIA) - Researchers at the University of Pennsylvania School of Medicine have demonstrated the potential of a new type of therapy for patients who suffer from high cholesterol levels. In this study, patients with homozygous familial hypercholesterolemia (FH), a high-risk condition refractory to conventional therapy, had a remarkable 51% reduction in low-density lipoprotein (LDL) or “bad cholesterol” levels.
    7. 7. Challenges of the “database” approach <ul><li>Closely guarded assets </li></ul><ul><li>Pharma just gearing up for repositioning </li></ul><ul><li>Lack of a systematic databasing in the companies </li></ul><ul><li>Data has not been published </li></ul><ul><li>External databases only capture about 50% of the assets </li></ul><ul><ul><li>Needed to find an indirect way of accessing the information – via pharma champions </li></ul></ul>
    8. 8. Recent Number of NMEs Approved by FDA is Disappointing PhRMA R&D Productivity (Avg Annual R&D Spend Increase = 10.3%; ’90-’07) Source: Pharmaceutical Research and Manufacturers of America, PhRMA Annual Membership Survey, 2008; CDER PhRMA R&D Spend (Billions of Dollars) Number of NME Approvals by FDA NMEs Approved
    9. 9. Research & Development Productivity Needs Attention The Challenge – 1 of 10 is Aspirational!
    10. 10. …… ..but Opportunities to Improve Abound Preclinical Phase 1 Phase 2/ PoC Phase 3 Approval 65 55 25 65 85 Cumulative = 5% “ In times of profound change, the learners inherit the earth, while the learned find themselves beautifully equipped to deal with a world that no longer exists.” “ It still holds true that man is most uniquely human when he turns obstacles into opportunities.” - Eric Hoffer Stage % Survival versus
    11. 11. <ul><li>Initiated in ’07 as a dedicated group of scientists focused on discovering, evaluating, and pre-clinically validating additional opportunities for active and failed Pfizer Development candidates </li></ul><ul><li>Early portfolio progress warranted build to include ability to conduct </li></ul><ul><li>clinical studies </li></ul><ul><li>Diversity of skills </li></ul><ul><li>Broad Disease Area focus (“indication agnostic”) </li></ul><ul><li>Leverages group, Pfizer Research Unit and external expertise </li></ul><ul><li>Provides a systematic approach to expanding the indication options </li></ul><ul><li>for Pfizer compounds </li></ul><ul><li>Goal is to deliver > 1 PoC per year </li></ul><ul><li>at steady state </li></ul><ul><li>To date, have delivered </li></ul><ul><ul><li>initiation of 5 new clinical studies </li></ul></ul>Pfizer Indications Discovery Products Process Evolution
    12. 12. Indications Discovery Business Plan – An Ever-Evolving Model DISEASE RELEVANT SCREENING HYPOTHESIS GENERATION Reg Tox through Phase 2 Pre-Clinical VALIDATION Endorsement to Initiate Clinical PoC Study Internal, Partner, Out-license MINE EXISTING KNOWLEDGE <ul><li>Project Teams </li></ul><ul><li>Clinical Data Mining </li></ul><ul><li>External Knowledge </li></ul><ul><ul><li>Text </li></ul></ul><ul><ul><li>Genetics </li></ul></ul><ul><ul><li>Pathways </li></ul></ul>Phase 3/Marketed CANs LDs Active Clinical Candidates Failed compounds – “RIPs” Those losing CoM patents Internal, Partner In vivo models Clinical Proof of Concept Internal, Partner, Out-license In vitro assays
    13. 13. Pfizer Indications Discovery – Learnings <ul><li>Pfizer Compounds & Drug Development expertise present significant opportunity </li></ul><ul><li>Goals change – be quick to adapt </li></ul><ul><li>Focus on Unmet Medical Need </li></ul><ul><ul><li>Therapeutic Area agnostic </li></ul></ul><ul><ul><li>Expect challenges……….create opportunities </li></ul></ul><ul><ul><ul><li>“ we don’t know how to do IPF” – collaborate with experts (IPFnet) </li></ul></ul></ul><ul><ul><ul><li>“ there’s no market opportunity” – consider Orphan drug status, Patient Advocacy Groups, Foundations </li></ul></ul></ul><ul><li>Change (Ind Disc Group) was readily embraced by broader Pfizer organization </li></ul><ul><ul><li>Idea generation, scientific and technical support </li></ul></ul><ul><ul><li>Early progress critical </li></ul></ul><ul><li>Required building supporting infrastructure </li></ul><ul><ul><li>Stage Gates - unique </li></ul></ul><ul><ul><li>Idea Tracker (Cmpds/MoA/Disease) </li></ul></ul><ul><ul><li>Compound Book (Cmpd Data) </li></ul></ul><ul><li>Understanding MoA-Disease associations is critical </li></ul><ul><li>The Lion King – Circle of Life </li></ul><ul><li>“ There's more to see than can ever be seen, </li></ul><ul><li>More to do than can ever be done.” </li></ul><ul><li>It’s time to focus! </li></ul>
    14. 14. Pfizer Indications Discovery – Key Focus Area: MoA-Disease Association <ul><li>Idea Generation </li></ul><ul><ul><li>MoA Compound File for Screening </li></ul></ul><ul><ul><ul><li>Internal Research Units & External Partnerships </li></ul></ul></ul><ul><ul><li>Mining Existing Knowledge </li></ul></ul><ul><ul><ul><li>Internal Research & Development-wide IdeaFarms </li></ul></ul></ul><ul><ul><ul><li>CTSA Portal - Research Compounds, Clinical Compounds, Other </li></ul></ul></ul><ul><li>Idea Validation </li></ul><ul><ul><li>Foci of Expertise (FoX) Tool </li></ul></ul><ul><ul><li>Internal Research Units & Academic Medical Centers </li></ul></ul><ul><li>Clinical Confirmation </li></ul><ul><ul><li>Expand Indications Discovery expertise/capacity </li></ul></ul><ul><ul><li>Need to leverage external expertise (e.g., IPFnet) </li></ul></ul><ul><ul><li>Biomarkers of Target MoA & Disease Efficacy </li></ul></ul><ul><ul><li>Patient Selection (e.g., Novartis’ ILARIS for Cryoptyrin-Associated Periodic Syndromes; CAPS) </li></ul></ul><ul><ul><li>Multi-site Protocols </li></ul></ul><ul><li>“ Generic Agreements” </li></ul>Collaboration
    15. 15. An Invitation to Collaboration <ul><li>Improving health through scientific discovery is a lofty goal, in today’s economic and organizational environment there are many challenges, and many exciting opportunities. </li></ul><ul><li>Pfizer’s Indications Discovery group seeks novel cross-functional partnerships that bring new medicines to patients. </li></ul><ul><ul><li>Discussions like these, and those we’ve had with NIH Chemical Genomics Center (NCGC), Foundation for NIH (FNIH), NIH Office of Technology Transfer, and CTSA, are expected to advance our common interests. </li></ul></ul><ul><ul><li>The CTSA Pharmaceutical Assets portal and Foci-of-Expertise tool are but two examples of Pfizer’s Indications Discovery efforts to expand possibilities via collaboration. </li></ul></ul><ul><li>Coming together is a beginning; keeping together is progress; working together is success. </li></ul><ul><ul><li>- Henry Ford </li></ul></ul>
    16. 16. Two ways to create a match between Academia and Pharma
    17. 17. From Academia to Pharma <ul><li>Known compound for PRE-clinical studies: http://www.pfizer.com/research/licensing/compound_gift.jsp </li></ul><ul><li>Please fill out this form http://www.ctsapharmaportal.org/files/Pfizer_Write_in_Request_Form.doc </li></ul><ul><li>2. Known compound for CLINICAL studies: http://www.pfizer.com/research/investigator/investigator_initiative.jsp Submit the request directly via the website. </li></ul><ul><li>3. Request a compound based on the mechanism of action . Fill out Indications Discovery Form (“ Dream List ”). Requests are forwarded to the Indications Discovery Unit at Pfizer for review. </li></ul>
    18. 18. How to join the Portal (www.ctsapharmaportal.org)
    19. 19. Members of the Portal (as of June 09)
    20. 20. The Portal at a glance <ul><li>348 researchers nation-wide, including NIH </li></ul><ul><li>Main interest areas of Portal participants: </li></ul>
    21. 21. The Portal at a glance <ul><li>80% of participants desire a compound at clinical stage </li></ul><ul><li>>80% desire to run a clinical trials after proof of concept is established </li></ul><ul><li>50% had prior experience in obtaining investigational drugs </li></ul><ul><ul><li>Learned about a drug from public sources </li></ul></ul><ul><ul><li>Only ½ of these attempts succeeded </li></ul></ul><ul><ul><li>Failed largely because of contractual issues (40%) </li></ul></ul>
    22. 22. Specific target/disease interests (~150)
    23. 23. Two ways to create a match between Academia and Pharma
    24. 24. From Pharma to CTSA via FoX target Disease A Disease A Disease C Disease B Disease D Disease B
    25. 25. FoX Pilot for 5 universities <ul><li>Obtained data from Biovista ( www.biovista.com ) </li></ul><ul><ul><li>Researcher-target </li></ul></ul><ul><ul><li>Researcher-disease </li></ul></ul><ul><li>By mining OMIM, KEGG etc: </li></ul><ul><ul><li>target-target </li></ul></ul><ul><ul><li>target-disease </li></ul></ul><ul><li>Large scale network visualization based on biological interaction between proteins using OCTRI Synergy Tool, developed by OHSU </li></ul><ul><li>Currently building for 45 universities </li></ul>
    26. 26. Data cloud for a target X
    27. 27. Examples of Filters <ul><li>Who published the most </li></ul><ul><li>By university </li></ul><ul><li>By disease </li></ul><ul><li>Who published within last X years </li></ul><ul><li>Time slider – visualize new disease appearance </li></ul>
    28. 28. Example of a Filter
    29. 29. Pharma Concerns re: research with their compounds <ul><li>Different Timing: </li></ul><ul><ul><li>Execution of the study </li></ul></ul><ul><ul><li>Contract negotiation </li></ul></ul><ul><li>Dialog </li></ul><ul><ul><li>Appreciation and learning of what it takes to develop a drug </li></ul></ul><ul><li>Unreasonable valuation of academic contribution </li></ul><ul><li>Early publication may affect competitive standing </li></ul><ul><li>Releasing “real” compounds vs “tool” compounds </li></ul>
    30. 30. Next steps – Contractual Issues <ul><li>Workgroup </li></ul><ul><ul><li>Guiding Principles for the MTAs for the compounds originating from the Portal (draft accessible on the website: www.ctsapharmaportal.org ) </li></ul></ul><ul><ul><li>Guiding Principles for licensing for the method of use patents on the compounds originated from the Portal </li></ul></ul><ul><li>Recognize that we are a small part of the drug development process </li></ul><ul><li>Be sensitive to “shelved” compound issues </li></ul>
    31. 31. Call for Action to the Foundations <ul><li>Spread the word about the Portal among your constituency </li></ul><ul><li>Work with the scientists to prepare “Dream Lists” and “Dream Proposals” </li></ul><ul><li>Consider broad-scope translational assessments </li></ul><ul><li>No indication is too small! </li></ul><ul><li>Make a “match” via the Portal </li></ul>
    32. 32. Summary - Future Plans <ul><li>Proactively engage the pharmaceutical industry in providing information about the compounds that may be available to the research community. </li></ul><ul><li>Actively increase awareness of availability of the Portal among CTSA Researchers and promote availability of compounds to the research community </li></ul><ul><li>Define the mutually agreeable “ principles or points to consider ” for materials transfer as related to drugs originated from the Portal. </li></ul>
    33. 33. Team : <ul><li>UC Davis – Project Management, Company Contacts </li></ul><ul><ul><li>Kate Marusina </li></ul></ul><ul><li>OHSU – Foci of Expertise </li></ul><ul><ul><li>Aaron Cohen, Nathan Bahr </li></ul></ul><ul><li>UW – researchers outreach and survey </li></ul><ul><ul><li>Doug Brock, Pamela Nagasawa, Lynn Rose </li></ul></ul><ul><li>U Penn – Analysis of key MTA provisions </li></ul><ul><ul><li>Terry Fadem </li></ul></ul><ul><li>U of Chicago – Advisory </li></ul><ul><ul><li>Eric Ginsburg </li></ul></ul><ul><li>Special Thanks to Pfizer Indications Discovery Unit </li></ul><ul><ul><li>Dean Welsch </li></ul></ul><ul><ul><li>Don Frail </li></ul></ul>
    34. 34. <ul><li>QUESTIONS AND ANSWERS </li></ul><ul><li>WEBINAR Accessing Shelved Compounds Through the CTSA Pharmaceutical Assets Portal </li></ul><ul><li>Kate Marusina, Ph.D., MBA, Manager, Research Facilitation and Industry Alliance, Clinical and Translational Science Center, University of California Davis School of Medicine </li></ul><ul><li>Dean J. Welsch, Ph.D., Research Fellow, Pfizer Global Research & Development, Indications Discovery Research Unit </li></ul><ul><li>Moderator: Margaret Anderson, Executive Director, FasterCures </li></ul>
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