Drug Discovery From Soup To Nuts

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Resources
For Drug Discovery and Development at NIDA
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The NIDA Mission

• NIDAs mission is to lead the nation in bringing
  the power of science to bear on drug abuse and
  addiction. This charge has two critical
  components the first is the strategic support
  and conduct of research across a broad range of
  disciplines. The second is to ensure the rapid
  and effective dissemination and use of the
  results of that research to significantly improve
  drug abuse and addiction prevention, treatment,
  and policy.

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The Division of Treatment Research Development
is looking for treatments that Block effects
Reduce craving Moderate or eliminate
withdrawal symptoms Block or reverse the
toxic effects of abused drugs Prevent
relapse of drugs of abuse (cocaine,
methamphetamine, opiates)
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Estimates of Market SizeCurrent Market for
Cocaine Treatment

• 2 million people are addicted or heavy users.
• 800,000 900,000 may enter treatment at least
  once in a given year.
• On any given day 250,000 are enrolled in
  treatment and 11,500 centers provide treatment.
• 40 are enrolled in primary treatment and 60 are
  enrolled in secondary treatment.
• 2 billion total spending per year 23 per
  patient per day enrolled (including inpatient and
  outpatient), 9 per day for non-intensive
  outpatients.

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Estimates of Price, Market Size Revenues

• A medication used by 250,000 patients, sold at
  3.30 per day, will generate 300 million in Peak
  Annual Revenue (PAR)
• A medication used by 125,000 patients, sold at
  6.60 per day, will generate 300 million PAR
• A medication used by 500,000 patients, sold for
  2.50 per day, will generate 440 million PAR
• A medication used by 1 million patients, sold at
  0.50 per day, will generate 200 million PAR

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Market Analysis Model
Factors determining profitability Development
cost Sales of the product Cost of Manufacturing,
distributing and marketing the product Duration
of the development and product/sales life
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Critical Market Barrier Perceptions
Small and uncertain market for cocaine addiction
pharmacotherapy. Substance abuse treatment
system that limits access to the
market. Limited and uncertain payment for
pharmacotherapy. Industry perception of science
base readiness.
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Government InterventionsIncreasing Profitability
Funding of basic science and clinical trials NDA
Phase Fast Track approval Orphan Drug status
(ODS) Market exclusivity other than ODS Less
stringent Phase IV clinical trial
requirements Marketing
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How Can NIDA Support Drug Discovery/Development
for Small Businesses?

• Grants
• - SBIR/STTR Program
• Development programs
• - Screening programs
• - Drug substance and drug product
• manufacturing
• - Preclinical testing
• - Clinical trials

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Areas of Programmatic InterestDivision of
Treatment Research Development

• Medicinal chemistry (novel compounds for the
  treatment of stimulant and opiate abuse)
• Pharmacology (development of preclinical
  pharmacological models of initiation, craving,
  drug-seeking behavior, dependence, withdrawal,
  relapse)
• Pharmacokinetics (PK/PD modeling of abused drugs
  and drug abuse treatments, PK and ADME studies,
  in vitro models for prediction of bioavailability
  of new treatment agents)
• Pharmaceutics (formulation studies to develop
  drug products with improved stability/bioavailabil
  ity, new dosage forms)
• Clinical studies (controlled studies to assess
  drug abuse treatment medications, controlled
  trials for the development of new treatment
  agents)

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Preclinical Resources

• Opiate Treatment Discovery Program
• Cocaine Treatment Discovery Program
• Safety Assessment Program

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Cocaine Treatment Discovery Program

• Receptor and transporter binding assays
• Locomotor activity assays (mouse)
• Drug discrimination assays (rat and monkey)
• Self-administration assays (rat and monkey)

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Opiate Treatment Discovery Program

• Opiate receptor binding assays (specifically
  looking for selective kappa antagonists)
• Models of relapse to heroin self-administration
  (rats)
• NMDA antagonists
• Glycine partial agonists
• GABA-B agonist
• Cholinergic ligands
• Alpha-2 agonists
• Enzyme modulators

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Safety Assessment and Toxicology Program

• AMES assay for mutagenicity
• Acute and repeat dose studies (2 species)
• Drug interaction studies (cocaine, ethanol,
  morphine)
• Cardiovascular (conscious dog)
• Neurotoxicity
• Metabolism

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Clinical Support(CRADA required)
Phase I (safety) Phase II (dose-finding, proof of
concept) Phase III (multi-site) Phase IV
(Clinical Trials Network)
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Chemistry Support

• Non-GMP and GMP synthesis
• Formulation
• Stability studies
• PK studies

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Uncle Sam
DOES NOT WANT YOUR INTELLECTUAL PROPERTY
RIGHTS!
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Who To Call?
Cocaine Treatment Discovery Program Jane Acri,
Ph.D. (jacri_at_nih.gov) 301-443-6270 Opiate
Treatment Discovery Program David McCann, Ph.D.
(dmccann_at_nih.gov) 301-443-6270 New chemical
entities/Treatment related grant concepts Jamie
Biswas, Ph.D. (jb168r_at_nih.gov) 301-443-5280
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www.nida.nih.gov