Pharma Assets Portal

Presents Webinar on Accessing Shelved Compounds Through the CTSA Pharmaceutical Assets Portal
April 12, 2010
www.fastercures.org/train
Kate Marusina, Ph.D., MBA, Manager, Research Facilitation and Industry Alliance, Clinical and Translational Science Center, University of California Davis School of Medicine
Dean J. Welsch, Ph.D., Research Fellow, Pfizer Global Research & Development, Indications Discovery Research Unit
Moderator: Margaret Anderson, Executive Director, FasterCures

CTSA PHARMACEUTICAL ASSETS PORTAL www.ctsapharmaportal.org Kate Marusina, Ph.D., MBA December 2009

Goals
To improve information exchange between pharmaceutical companies and the CTSA Consortium/NIH regarding drugs available for repositioning
Specific emphasis in on drugs discontinued at clinical stage
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

Achievements to Date
Dr. Francis Collins expressed his support at the CTSA Industry Forum (Feb 2010)
Pfizer financially supports development of the Foci-of-Expertise, for hypothesis driven identification of the repositioning opportunities (Dec 2009).
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).
The Portal Project was listed in the NIH report to the Secretary of Health and Human Services
The Portal received press coverage in the Nature Medicine, Proto Magazine, Sacramento Bee Newspaper, Drug Repositioning Summit in Boston (October 2009).
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.

What is CTSA?
Clinical and Translational Science Award
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
The largest academic integration effort to date
Required functions:
Biomedical Informatics
Regulatory Knowledge and Support
Training and Education
Community Outreach
Translational Pilot studies
Power of the network (45 - 60 universities)

CTSA Pharmaceutical Assets “Database”
Inspiration: Daniel Rader and BMS-201038 (AEGR-733 ), targeted inhibition of the microsomal triglyceride transfer protein (MTP)
Project sponsored by NCRR in Sept 2008 as “Pharmaceutical Assets Database”
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.

Challenges of the “database” approach
Closely guarded assets
Pharma just gearing up for repositioning
Lack of a systematic databasing in the companies
Data has not been published
External databases only capture about 50% of the assets
Needed to find an indirect way of accessing the information – via pharma champions

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

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 = 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

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
Early portfolio progress warranted build to include ability to conduct
clinical studies
Diversity of skills
Broad Disease Area focus (“indication agnostic”)
Leverages group, Pfizer Research Unit and external expertise
Provides a systematic approach to expanding the indication options
for Pfizer compounds
Goal is to deliver > 1 PoC per year
at steady state
To date, have delivered
initiation of 5 new clinical studies
Pfizer Indications Discovery Products Process Evolution

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
Project Teams
Clinical Data Mining
External Knowledge
Text
Genetics
Pathways
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

Pfizer Indications Discovery – Learnings
Pfizer Compounds & Drug Development expertise present significant opportunity
Goals change – be quick to adapt
Focus on Unmet Medical Need
Therapeutic Area agnostic
Expect challenges……….create opportunities
“ we don’t know how to do IPF” – collaborate with experts (IPFnet)
“ there’s no market opportunity” – consider Orphan drug status, Patient Advocacy Groups, Foundations
Change (Ind Disc Group) was readily embraced by broader Pfizer organization
Idea generation, scientific and technical support
Early progress critical
Required building supporting infrastructure
Stage Gates - unique
Idea Tracker (Cmpds/MoA/Disease)
Compound Book (Cmpd Data)
Understanding MoA-Disease associations is critical
The Lion King – Circle of Life
“ There's more to see than can ever be seen,
More to do than can ever be done.”
It’s time to focus!

Pfizer Indications Discovery – Key Focus Area: MoA-Disease Association
Idea Generation
MoA Compound File for Screening
Internal Research Units & External Partnerships
Mining Existing Knowledge
Internal Research & Development-wide IdeaFarms
CTSA Portal - Research Compounds, Clinical Compounds, Other
Idea Validation
Foci of Expertise (FoX) Tool
Internal Research Units & Academic Medical Centers
Clinical Confirmation
Expand Indications Discovery expertise/capacity
Need to leverage external expertise (e.g., IPFnet)
Biomarkers of Target MoA & Disease Efficacy
Patient Selection (e.g., Novartis’ ILARIS for Cryoptyrin-Associated Periodic Syndromes; CAPS)
Multi-site Protocols
“ Generic Agreements”
Collaboration

An Invitation to Collaboration
Improving health through scientific discovery is a lofty goal, in today’s economic and organizational environment there are many challenges, and many exciting opportunities.
Pfizer’s Indications Discovery group seeks novel cross-functional partnerships that bring new medicines to patients.
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.
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.
Coming together is a beginning; keeping together is progress; working together is success.
- Henry Ford

Two ways to create a match between Academia and Pharma

From Academia to Pharma
Known compound for PRE-clinical studies: http://www.pfizer.com/research/licensing/compound_gift.jsp
Please fill out this form http://www.ctsapharmaportal.org/files/Pfizer_Write_in_Request_Form.doc
2. Known compound for CLINICAL studies: http://www.pfizer.com/research/investigator/investigator_initiative.jsp Submit the request directly via the website.
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.

How to join the Portal (www.ctsapharmaportal.org)

Members of the Portal (as of June 09)

The Portal at a glance
348 researchers nation-wide, including NIH
Main interest areas of Portal participants:

The Portal at a glance
80% of participants desire a compound at clinical stage
>80% desire to run a clinical trials after proof of concept is established
50% had prior experience in obtaining investigational drugs
Learned about a drug from public sources
Only ½ of these attempts succeeded
Failed largely because of contractual issues (40%)

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
Obtained data from Biovista ( www.biovista.com )
Researcher-target
Researcher-disease
By mining OMIM, KEGG etc:
target-target
target-disease
Large scale network visualization based on biological interaction between proteins using OCTRI Synergy Tool, developed by OHSU
Currently building for 45 universities

Data cloud for a target X

Examples of Filters
Who published the most
By university
By disease
Who published within last X years
Time slider – visualize new disease appearance

Example of a Filter

Pharma Concerns re: research with their compounds
Different Timing:
Execution of the study
Contract negotiation
Dialog
Appreciation and learning of what it takes to develop a drug
Unreasonable valuation of academic contribution
Early publication may affect competitive standing
Releasing “real” compounds vs “tool” compounds

Next steps – Contractual Issues
Workgroup
Guiding Principles for the MTAs for the compounds originating from the Portal (draft accessible on the website: www.ctsapharmaportal.org )
Guiding Principles for licensing for the method of use patents on the compounds originated from the Portal
Recognize that we are a small part of the drug development process
Be sensitive to “shelved” compound issues

Call for Action to the Foundations
Spread the word about the Portal among your constituency
Work with the scientists to prepare “Dream Lists” and “Dream Proposals”
Consider broad-scope translational assessments
No indication is too small!
Make a “match” via the Portal

Summary - Future Plans
Proactively engage the pharmaceutical industry in providing information about the compounds that may be available to the research community.
Actively increase awareness of availability of the Portal among CTSA Researchers and promote availability of compounds to the research community
Define the mutually agreeable “ principles or points to consider ” for materials transfer as related to drugs originated from the Portal.

Team :
UC Davis – Project Management, Company Contacts
Kate Marusina
OHSU – Foci of Expertise
Aaron Cohen, Nathan Bahr
UW – researchers outreach and survey
Doug Brock, Pamela Nagasawa, Lynn Rose
U Penn – Analysis of key MTA provisions
Terry Fadem
U of Chicago – Advisory
Eric Ginsburg
Special Thanks to Pfizer Indications Discovery Unit
Dean Welsch
Don Frail

QUESTIONS AND ANSWERS
WEBINAR Accessing Shelved Compounds Through the CTSA Pharmaceutical Assets Portal
Kate Marusina, Ph.D., MBA, Manager, Research Facilitation and Industry Alliance, Clinical and Translational Science Center, University of California Davis School of Medicine
Dean J. Welsch, Ph.D., Research Fellow, Pfizer Global Research & Development, Indications Discovery Research Unit
Moderator: Margaret Anderson, Executive Director, FasterCures

Pharma Assets Portal

by TRAIN Central Station on May 13, 2010

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Pharma Assets Portal — Presentation Transcript