The U.S. National Center for Advancing Translational Sciences CHRISTOPHER P. AUSTIN, M.D. DIRECTOR, NCATS U.S. NATIONAL INSTITUTES OF HEALTH BIO INTERNATIONAL.

Slides:

Advertisements

Similar presentations

The Drug Discovery Process

Advertisements

U.S. Food and Drug Administration Notice: Archived Document The content in this document is provided on the FDAs website for reference purposes only. It.

Challenges in new drug discovery in South Asia

Alternative Careers in Pharma and Biotechnology Douglas Buckley Ph.D. May 13, 2011 Alternative Career Symposium Baylor College of Medicine.

An Academic Model to Bridge the Valley of Death April 17, 2009 Scott Weir, PharmD, PhD Institute for Advancing Medical Innovation University of Kansas.

Influencing Change in Research, Treatment Protocols, and New Drug Development.

U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICES NATIONAL INSTITUTES OF HEALTH Working with FDA: Biological Products and Clinical Development Critical Path.

Nanotechnology in Drug Discovery- Development and Delivery

Challenges in Clinical Applications for Nanotechnology N. Tony Eissa, MD Table 3.

Cancer Nanotechnology: New Opportunities for Targeted Therapies FDA Public Meeting October 10, 2006 Piotr Grodzinski, Ph.D. Director, Nanotechnology for.

Drug-Like Properties: Optimizing Pharmacokinetics and Safety During Drug Discovery Li Di and Edward H. Kerns ACS Short Course.

Discovery of new medicines through new models of collaboration Simon Ward Professor of Medicinal Chemistry & Director of Translational Drug Discovery Group.

John H. Ferguson MD, Medical Consultant, ORDR/NCATS/NIH.

1 The UK Opportunity: what is experimental medicine? UNLOCK YOUR GLOBAL BUSINESS POTENTIAL Pre- clinical development Phase I Phase II Phase III Product.

The NIH Roadmap for Medical Research

The South African Malaria Initiative A Case Study E Jane Morris Bridging the Gap in Global Health Innovation - from Needs to.

Drug discovery and development

U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICES National Institutes of Health National Institute of Neurological Disorders and Stroke U.S. DEPARTMENT OF HEALTH.

Figure 4.1 NEW PRODUCT DEVELOPMENT PROCESS Finance Corporate strategy and portfolio decisions Regulatory affairs Marketing and sales + market research.

Johns Hopkins Technology Transfer 1 Translational Biomedical Research: Moving Discovery from Academic Centers to the Community Translational Biomedical.

Knowledgebase Creation & Systems Biology: A new prospect in discovery informatics S.Shriram, Siri Technologies (Cytogenomics), Bangalore S.Shriram, Siri.

NCI Review of the Clinical Trials Process 6 th Annual National Forum on Biomedical Imaging in Oncology James H. Doroshow M.D. April 7, 2005 Bethesda, Maryland.

Aberdeen Confidence in Concept Fund Prof Phil Hannaford Vice Principal – Research & Knowledge Exchange.

Partnering with Federal Labs: A Panel Discussion FLC Mid-Atlantic Region Annual Meeting October 24, 2007.

Marine Drug Development and Delivery Prof. Dr. Basavaraj K. Nanjwade M. Pharm., Ph. D Department of Pharmaceutics KLE University College of Pharmacy BELGAUM ,

Exploratory IND Studies

West Virginia Clinical Translational Science Institute Links Scientists and Teachers Sara Hanks, Ann Chester, Summer Kuhn.

Single Patient Use of Investigational Anticancer Agents: An Industry Perspective Gerard T. Kennealey, MD Vice President, Clinical Research, Oncology AstraZeneca.

Developing medicines for the future and why it is challenging Angela Milne.

PUTTING THE PATIENT AT THE CENTER OF HEALTHCARE RESEARCH Towards a more inclusive model.

Using Predictive Classifiers in the Design of Phase III Clinical Trials Richard Simon, D.Sc. Chief, Biometric Research Branch National Cancer Institute.

The National Center for Advancing Translational Sciences Catalyzing Translational Innovation KRISHNA BALAKRISHNAN, PH.D., M.B.A. OFFICE OF STRATEGIC ALLIANCES,

FDA Office of Orphan Products Development

Why Write A Grant? Elaine M. Hylek, MD, MPH Professor of Medicine Associate Director, Education and Training Division BU CTSI Section of General Internal.

Research in the Office of Vaccines Research and Review: Vision and Overview Jesse Goodman, M.D., M.P.H. Director, Center for Biologics Evaluation and Research.

ACCELERATING CLINICAL AND TRANSLATIONAL RESEARCH Scott C. Denne, MD Associate Director, Indiana CTSI April 25, 2011 Plenary Session.

MRC Technology an independent life science medical research charity Biotech & Money 2016 Mr Andrew Mercieca, Director of Finance

Recent Studies Funded by the Progressive MS Alliance Tim Coetzee, PhD ACTRIMS 2016.

A model collaboration using the Pool Susanne Hollinger, Ph.D., J.D. Chief Intellectual Property Officer Emory University.

26th April 2016, BioTrinity - London

OVERVIEW OF THE WORKFORCE DEVELOPMENT DOMAIN TASK FORCE APRIL 16, 2015 Joan D. Nagel, M.D., M.P.H. Program Director, Division of Clinical Innovation National.

Molecular Modeling in Drug Discovery: an Overview

독성학 박 대 훈 한약재산업학과

U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICES National Institutes of Health National Institute of Neurological Disorders and Stroke Big Discoveries, Small.

Division of Cancer Treatment and Diagnosis Presented By: Michael Difilippantonio, Ph.D. October 1, 2015.

From the Valley of Death to the Bridge of Life: Models for De-Risking Early Translational Projects from Around the World.

Moxifloxacin Development in TB Dr. Martin Springsklee VP, Therapeutic Area Head, Clinical Development Anti-Infectives Bayer HealthCare AG GATB Annual Stakeholder.

Moiz Bakhiet, MD, PhD, Professor and Chairman

TITIN ANDRI WIHASTUTI SCHOOL OF NURSING FACULTY OF MEDICINE

Building a Drug Repurposing Network (DR3N) for the CTSA Consortium:

Telephone    Provider of Global Contract Research Services Accelerating Preclinical Research, Drug Discovery.

Drug Discovery &Development

American Evaluation Association, Evaluation 2016

FP6 Priority 1, Life Sciences Genomics and Biotechnology for Health

ATOM Accelerating Therapeutics for Opportunities in Medicine

Cancer Prevention Clinical Trials at [Name of Clinical Site]

Action: BM0806 Recent Advances in Histamine Receptor H4R Research

An Introduction to Medicinal Chemistry 3/e

Gestora brasileiro focada exclusivamente na área da saúde.

Darts Sciences Darts Target Identification.

SPARC – Washington University LEAP Inventor Challenge Program RFP

From Bench to Clinical Applications: Money Talks

Trial Innovation Network A new national collaborative initiative within the CTSA Program and is composed of three key organizational partners – the CTSA.

Drug Development Stages

Drug Design and Drug Discovery

FDA Tox Working Group Which goals of the FDA Roadmap are most important to FDA stakeholders? Integration of predictive technologies Replacing animal tests.

FP6 Priority 1, Life Sciences Genomics and Biotechnology for Health

Volume 21, Issue 9, Pages (September 2014)

Considerations for Successful Biopharmaceutical Product Development: Discovery to Proof of Concept -A Panel Discussion Stanley C. McDermott, PharmD, MS,

Presentation transcript:

The U.S. National Center for Advancing Translational Sciences CHRISTOPHER P. AUSTIN, M.D. DIRECTOR, NCATS U.S. NATIONAL INSTITUTES OF HEALTH BIO INTERNATIONAL CONVENTION JUNE 7, 2016

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.

What is Translational Science? Translational Science is the field of investigation focused on understanding the scientific and operational principles underlying each step of the translational process. NCATS studies translation as a scientific and organizational problem.

NCATS Scientific Initiatives  Clinical Translational Science Clinical and Translational Science Awards program Rare Disease Clinical Research Network New Therapeutic Uses program  Preclinical Translational Science NCATS Chemical Genomics Center Therapeutics for Rare and Neglected Diseases program Bridging Interventional Development Gaps program  Re-engineering Translational Sciences Toxicology in the 21st Century Microphysiological Systems (Tissue Chip) program Office of Rare Diseases Research

Division of Preclinical Innovation Target Validation through Drug Development to First-in-Human Collaborative project team model  > 300 collaborations with investigators worldwide Each project customized to problem and development stage Each project is “dual use”  Solves specific block in translation  Provides insight of general utility to advance science of translation

Therapeutics for Rare/Neglected Dis (TRND) BrIDGs Stem Cell Technology Facility Tox21 (Systems Toxicology) RNAi Paradigm/Technology Development Repurposing Lead Optimization Preclinical Development Probe/Lead Development Target Validation Target FDA approval Clinical Trials I II III Project Entry Point Deliverables Repurposing Unvalidated target Validated target Lead compound Preclinical development candidate Genome-wide RNAi systems biology data Chemical genomics data Small molecule and siRNA research probes More efficient/faster/cheaper translation and therapeutic development Leads for therapeutic development Predictive in vitro toxicology profiles Approved drugs effective for new indications New drugs for untreatable diseases Novel clinical trial designs Drugs suitable for adoption for further development Assay Dev Assay, Chemistry Technologies Target assay DPI Program NCGC NCATS Division of Preclinical Innovation Stem cell tools/data

NCATS DPI Staff

CSC Developing Aldehyde Dehydrogenase Inhibitors Collaborator: Vasilis Vasiliou, Yale University Target: ALDH1A1 Therapeutic Scope: Cancer, Inflammation, Obesity, Development Objective: Identification and characterization of small molecule inhibitors of Aldehyde Dehydrogenase isoform 1A1 Chemico-Biological Interactions 202 (2013) 2–10 ALDH

Developing Aldehyde Dehydrogenase Inhibitors Developed two highly potent series of ALDH1A1 inhibitors, e.g., NCT-501, with excellent selectivity and ADME properties Demonstrated efficacy of candidate inhibitors in 3D-spheroid and organoid (cancer stem cell) models of Glioblastoma multiforme 1A11B13A1ALDH2 Permeability (PAMPA) Aqueous Solubility 40 nM>57 µM 164 (10 -6 cm/s)>60 µg/mL RLM (T 1/2 ) HLM (T 1/2 ) Caco-2: P (B-A) /P (A-B) Protein Binding 84 min> 2 h19.6/19.3 = % ALDH inhibition (IC 50 ), selectivity, and early ADME properties of NCT-501

NCATS Partnership on Sickle Cell Disease  Collaborator: AesRx, LLC  Compound: Aes-103 (5-hydroxymethyl-2-furfural) Binds to sickle hemoglobin, increases O 2 affinity  TRND partnered with AesRx to de-risk Project initiation to patients in <12 mos Animal toxicology studies CMC (Chemistry, Manufacturing, and Controls) Regulatory: interactions with FDA, IND filing Ph Ia, Ib, IIa trials  Project acquired by Baxter

ncats.nih.gov/expertise

Learn More About NCATS Website: Facebook: facebook.com/ncats.nih.gov Twitter: twitter.com/ncats_nih_gov YouTube: youtube.com/user/ncatsmedia E-Newsletter: ncats.nih.gov/news-and- events/e-news/e-news.html us!