Partnership to accelerate novel treatments for heart valve disease

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Philomena Glenn
6 years ago
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1 Partnership to accelerate novel treatments for heart valve disease Mayo Clinic, NIH/NCATS, Sanofi, University of Minnesota, Nanosyn Jordan D. Miller, PhD Innovator Presentation

2 Tweet With Us Jordan D. Miller, PhD: Mayo

3 Heart Valve Function

4 Heart Valve Function

5 Heart Valve Function Can be the earliest sign of heart valve disease

6 Heart Valve Function

7 Heart Valve Function

8 10 million people will have aortic valve stenosis

9 Current Treatment Options Valve Replacement Transcatheter Approaches

10 For the rest of the talk An unlikely story Discovery Opportunity Pivots Partnerships

11 Goals 5 Years Ago DNA

12 K99/R00 Grant Nitric oxide signaling in valve disease

13 K99/R00 Grant Nitric oxide signaling Successful Therapeutically limited * WT DDAH1 +/

14 K99/R00 Grant Nitric oxide signaling Successful Therapeutically limited Not Pharma Not medicinal chemistry group

15 Typical Academic Model DNA

16 New NIH Program New Therapeutic Uses for Existing Drugs One year proof of concept (UH2) Cell Animal Human (Phase I) Two year clinical trial (UH3) Human (Phase II)

17 New NIH Program List of ~30 drugs Provided by Pharma partners Known safety profile formulation

18 New NIH Program List of ~30 drugs What is the likelihood that one would hit our target?

19 New NIH Program List of ~30 drugs What is the likelihood that one would hit our target?

20 New NIH Program

21 New NIH Program

22 New NIH Program Nitric Oxide N O Ataciguat Ataciguat N sgc O Oxidative Stress Ataciguat Ox sgc Valve Calcification

23 Shift in Mindset DNA

24 Our New Goal DNA

25 <1 year to get to human studies Historical view Science Medicine

26 New View Diverse skillsets Diverse ways of thinking Shared common goal Patient needs

27 New View Initial UH2/UH3 Team Jordan D. Miller, PhD (PI) Scientist Hartzell V. Schaff, MD (Co-PI) Surgeon Maurice Enriquez-Sarano, MD (Co-PI) Cardiologist

28 Our Proposed Approach UH2 Phase UH3 Phase Cells Mice Human (Phase I) Human (Phase II) Proof of Concept Tolerance/ Proof of Concept Effectiveness/Efficacy

29 Historical Model PI/Mayo Sanofi NIH

30 After the Grant Started PI/Mayo Sanofi NIH

31 After the Grant Started Brought Expertise Mayo Previous experience with drug Cross-reference IND Trial design/refinement Sanofi Industry insights NIH

32 Everything was underway A new twist Launchpad for Enterprise Innovation that is Evidence-based Customer -focused

33 Two key insights After 10 weeks Repurposing has limited value Limited Patent Life New Drugs in Phase II

34 New Key Partnerships Novel drug development Academia Industry

35 UH2/UH3 Where are we now? Completing Phase I and planning Phase II Drug development Identified 2 lead compounds (one with each partner) Planning refinement

36 An expensive process $500k January 2015 Scalable Screening Assays Novel Hits Compound Refinement July 2016 $3 5M Cell based Assays Animal Models Phase I Safety Trials Phase II Efficacy Trials

37 Challenges Funding for new leads Regulatory endpoints Business development strategies Public awareness of valve disease Screening Strategies for early diagnosis

38 Heart valve disease Major unmet needs Summary

39 Summary Heart valve disease Major unmet needs Requires new strategy Multidisciplinary teams Unique partnerships Patients FDA

40 Heart valve disease Summary Major unmet needs Requires new strategy Brings forth new challenges

41 Heart valve disease Summary Major unmet needs Requires new strategy Brings forth new challenges Training current and next generation

42 Summary Tremendously exciting time for translational research Looking for new partners at all levels

43 Team Mayo Clinic Miller Laboratory Bin Zhang, MD Carolyn Roos, MS Michael Hagler Heyu Zhang, MD/PhD Nassir Thalji, MB ChB Leslie Smith Brandon Dunagan Maurice Enriquez-Sarano, MD Hartzell Schaff, MD Rakesh Suri, MD/DPhil Jenny Weiss Marcia Andresen Reid Mark Wentworth Juliana St-Helene Kraft Launchpad for Enterprise Todd Morrill, PhD NIH Christine Colvis, PhD Bonnie Dunn, PhD, Bobbie-Ann Austin, PhD Frank Evans, PhD Sanofi Julia Gershkovich Karl Denkel, PhD Michael Halpern Christoph Gaudin, MD Many others University of Minnesota IDTT Michael Walters, PhD Jon Hawkinson, PhD Henry Wong, PhD Gunda Georg, PhD Nanosyn

University of California Center for Accelerated Innovation

University of California Center for Accelerated Innovation MICHAEL PALAZZOLO April 6, 2015 Outline CAI overview and introduction RFA and important dates Selection process Pre-application Finding help 2