1. Examples of NINDS Funding Opportunity
Announcements
Proof
Lead Pre-clinical Phase I
Target ID Assay Screening Hit to Lead of
Optimization Safety Trials
Concept
Exploratory Translational Grants (R21)
Cooperative Program
Tool Compound (U01, U54, U44)
Discovery (R01)
Tool Compound
Optimization (R01)
David Jett Rebecca Farkas Tom Miller
Contacts: jettd@ninds.nih.gov farkasr@nih.gov tm208y@nih.gov

2. Molecular Libraries HTS Services
Screening centers offer:
 Technical assistance in adapting assays for HTS
 Technical assistance for developing screening plan
 Library screening with your assay
 Med chem optimization of your probe
… AT NO COST TO YOU!
http://mli.nih.gov/mli/
Assay Screening Hit to Lead

3. Bridging Interventional Development
Gaps (BrIDGs)
Provides FREE ACCESS to contract services for:
● Production/bulk supply
● GMP manufacturing
● Formulation
● Pharmacokinetic testing
● Animal toxicology
● Manufacture of clinical trial supplies
● Product development planning and advice in IND preparation
http://nctt.nih.gov/bridgs Pre-clinical
Safety

4. Blueprint Neurotherapeutics Network
• Goal: phase I clinical success
Principal investigator Lead
• Any nervous system indication Industry consultants Development
NIH staff
• “Virtual pharma” structure Team
Bioactivity/ Phase I
Medicinal PK/Tox GMP/
Efficacy Trials
Chemistry Formulation
Studies
U01s NIH Contracts
• Free access to drug development services + funding for biology
• For novices and experts seeking additional capacity
• PI retains control of IP
Lead Pre-clinical Phase I
Optimization Safety Trials

5. Blueprint Neurotherapeutics Network
Current Projects
Principal Investigator Institution Disorder
John Bixby University of Miami Optic Neuropathy
Raymond J. Dingledine Emory University Stroke
Marcie A. Glicksman Brigham and Women’s Hospital ALS
Michael Lark Trevena, Inc. Depression
Konstantin Petrukhin Columbia University Macular Degeneration
Edwin W. Rubel University of Washington Hearing Loss
Steven Lee Wagner UC San Diego Alzheimer’s

6. Blueprint Neurotherapeutics Network
Entry Criteria
Disease biology
• Novel target for the disease Deadline:
• Strong biological validation June 2012
• Feasible path to the clinic
Assays
• Robust in vitro assay for optimization
• Strong confirmatory assays
Compounds
Jill Heemskerk
• Amenable to chemistry
jh440o@nih.gov
• IP free of obvious roadblocks
Rebecca Farkas
http://neuroscienceblueprint.nih.gov/bpdrugs/ farkasr@nih.gov

7. ? Contact Program Staff
Provide the following info to help NIH staff help you:
• Where does your project fall within the discovery/development pipeline?
• What is the disease indication?
• What kind of therapeutic are you developing (drug, biologic, device, etc.)?
• Would you prefer to do the work yourself or utilize NIH research services?
Rebecca Farkas
farkasr@nih.gov