NEW THERAPEUTIC USES (NTU) PROGRAM Christine Colvis, Ph.D. June 18, - PowerPoint PPT Presentation

NEW THERAPEUTIC USES (NTU) PROGRAM Christine Colvis, Ph.D. June 18, 2015

Accelerating Therapeutic Development New Therapies for Patients PHARMA RESEARCHERS • Create drugs • Provide new therapeutic • Provide agents AGREEMENTS use ideas • Access patient populations • Conduct COLLABORATION clinical trial FUNDING ALLIANCES NIH/NCATS • Post agent information • Develop agreement templates • Crowdsource ideas

Pilot Program Awards Issued June 2013 Disease Academic Partner Pharma Partner Alzheimer’s Disease Yale AstraZeneca Alcoholism U Rhode Island/NIAAA Pfizer Calcific Aortic Stenosis Mayo Clinic Sanofi Duchenne Muscular Dystrophy Kennedy Sanofi Krieger/UWash Lymphangioleiomyomatosis Baylor AstraZeneca Peripheral Artery Disease U Virginia AstraZeneca Smoking Cessation VCU/Pittsburgh Janssen Schizophrenia (2) Indiana U Lilly Yale Pfizer

Alzheimer’s Disease Project Timeline • 2012  June – NTU FOAs published  July – S t rit t mat t er Nat ure Neuroscience paper on Fyn in AD ePub  August – pre-applicat ion due Alzheimer amyloid- β oligomer bound to postsynaptic prion protein activates Fyn to impair neurons. • Nat Neurosci. 2012 S ep;15(9):1227-35. doi: 10.1038/ nn.3178. Epub 2012 Jul 22 . • Um JW 1 , Nygaard HB, Heiss JK, Kostylev MA, S tagi M, Vortmeyer A, Wisniewski T , Gunther EC, S trittmatter S M.

Alzheimer’s Disease Project Timeline • 2012  June – NTU FOAs published  July – S trittmatter Nature Neuroscience paper on Fyn in AD ePub  August – pre-application due • 2013  June – award made  August – first subj ect dosed for Phase 1b • 2014  Dec/ Jan – Phase 2a begins • 2015  Feb – animal data epub ahead of print  March – S en. Mikulski visits NCATS  April – human phase 1b results published  June – Presentation to council