HEALTH RESEARCH ALLIANCEMEMBERS’ MEETING

Hosted by The Foundation for the National Institutes of Health

Hyatt Regency Bethesda | Bethesda, MarylandSeptember 6-7, 2012

A Case Study on a Ground-Breaking Public-Private Partnership:

NIH/NCATS and Eli Lilly & Company

G. Sitta Sittampalam, PhD (NCATS)Timothy Grese, PhD (Eli Lilly &Co)

Out line

National Center for Advancing Translational Sciences- Goals &Rationale for NCATS (NIH) – Lilly Collaboration- NCATS Mission- NCATS Pharmaceutical Collection (NPC)- A Case Study- The Learning Collaborative (TLC)

Eli Lilly & Co. – Open Innovation Drug Discovery- Establishment & Mission- Phenotypic Drug Discovery (PD2) - Target Drug Discovery Initiative (TargetD2)- Disease pathways of mutual interest.

- Pilot experiment to leverage assets/resources in public-private partnerships- Leverage Lilly Phenotypic Assay Panels – successful in OIDD- Benefits larger drug discovery community- Publish data jointly- build a “collaboration culture”- Collaboration culture = Competencies + Excellence in Science + Trust- Benefit TRND via repurposing NPC compounds

5,000 Years

Lilly-NCATS Collaboration- Goals & Rationale

Accomplishing this effort, even in a few years, makes a lot of sense!

• Screen NPC Collection in Lilly Phenotypic Assay Panels• Jointly publish data –availability to the broader scientific community• Understanding Systems Pharmacology of known drugs• Leverage data for The Rare & Neglected Diseases (TRND) programs

NCATSEstablished: December 23, 2011

Part of consolidated Appropriations Act 2012 (PL112-74)

NCATS Mission“To catalyze the generation of innovative methods and technologies that will enhance the development, testing, and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions

Current Organization

CAN- Cures Acceleration NetworkORDR-Office of Rare Diseases ResearchDPI- Division of Preclinical InnovationDCI- Division of Clinical Innovation

Clinical Translational Science AwardCTSA (Extramural)

NCGC (2003)

NCTT (2010)

NCATS (2012)

NPC Screens at NCGC/NCATS

• NPC Collection has been screened in over 200 assay formats

• Assays represent ~ 25 different disease pathologies

• Examples: - Chronic Lymphocytic Leukemia (CLL)- Acute Myeloid Leukemia (AML) - Niemann-Pick Disease- Type C (NPC)- Transmissible spongiform encephalopathies (TSEs, Prion Disease)- Malaria, Giardia (Neglected Diseases)- Cryptococcal Meningitis

• All projects involves external collaborations with academic/non-profit partners

Chronic Lymphocytic Leukemia

102 molecules: CLL Pan actives vs. Normal B-cells

Doug Auld PhD., Adrien Wiestner MD. et al

From screen completion to clinic in ~17 months!A Rationale for starting the Lilly-NCATS collaboration

Current status - Lilly-NCATS Collaboration

• Publication of NPC collection (Sci. Transl. Med): April 2011

• NCATS- Lilly discussions: June - July 2011

• Collaborative Research Agreement signed: December 2011

• Press release : “NIH and Lilly to generate public resource of approved and investigational medicines” – March 13, 2012

• NCATS- Lilly Scientific and Steering Committees identified – April 2012.

• Compound acquisition and distribution – April-May 2012

• Phenotypic screens initiated at Lilly: July-August 2012

Initial discussions to initiating screens: ~ 12 months.

Lilly Open Innovation Drug Discovery Program

Timothy Grese, PhD.Chief Scientific Officer—Lead Generation and External Innovation

Discovery Chemistry Research and TechnologiesLilly Research Laboratories

Supplementary slides

Therapeutics for Rare & Neglected Diseases(TRND)