Sheraton University

City Hotel

Saturday, May 19, 2018

7:00 am – 4:30 pm

#PennMedMDBR2018 #GGPennRareSymposium

New and Continuing Roles for Patients in Research and Drug Development

David Fajgenbaum, M.D., MBA, MSc

Rob Lutz

Chief Business Officer

Stonebridge Biopharma

Luke Rosen

Founder, KIF1A.org

Associate Director, Patient

Engagement, Ovid Therapeutics

#PennMedMDBR2018 #GGPennRareSymposium

David Fajgenbaum, M.D., MBA, MSc

Co-founder and Executive Director, Castleman Disease Collaborative Network

Research Assistant Professor of Medicine in Translational Medicine and Human Genetics,

University of Pennsylvania

Associate Director of Patient Impact, Penn Orphan Disease Center

New and Continuing Roles for Patients in Research and Drug Development

#PennMedMDBR2018 #GGPennRareSymposium

Overview

• Introduce Penn Orphan Disease Center

• My patient journey

• Hurdles for Castleman disease and rare disease research

• Solutions for Castleman disease and rare disease research

• Lessons learned

• Intro to New and Continuing Roles for Patients in Research and Drug Development

https://globalgenes.org/raresymposium/ #GGPennRareSymposium #PennMedMDBR2017

Overview

Data

Working with FDA

Strategy

Collaboration

New and Continuing Roles for Patients in Research and Drug Development

Drug Development 101New Approaches and New Approval Pathways to Drug Development

Communicating with stakeholders

Why and How Should Our Community Start a Patient Registry

Why and How Should We Expand the Data in Our Registry?

How to Begin Research Collaborations

Expanding Your Reach: Strategically Growing Your Collaborations

Who's Who at Regulatory Agencies

When and How to Talk to Regulators

Communicating for collaboration

#PennMedMDBR2018 #GGPennRareSymposium

Luke Rosen

Founder, KIF1A.org

Associate Director, Patient Engagement,

Ovid Therapeutics

New and Continuing Roles for Patients in Research and Drug Development

8CONFIDENTIAL

DISCLAIMER

The material presented in this presentation is for informational purposes only. The views expressed in presentations are those of the speaker(s) and not, necessarily, of Ovid Therapeutics.

#PennMedMDBR2018 #GGPennRareSymposium

My Real Bio

I’m Luke, this is all very difficult and I never thought I’d be doing any of this.

I was an actor and a writer once, now I’m trying to do whatever I can to

shorten the process of drug development to save kids with rare

neurological diseases, like my daughter. I’ve met remarkably dedicated

people in the last few months and ask for help daily. I have no idea what

tomorrow will be like.

#PennMedMDBR2018 #GGPennRareSymposium

Gritty Collaboration

1. Tell the real story

• Don’t waste time.

2. Articulate unmet needs beyond a cure

• What is meaningful change?

3. Get rid of the box

• Risk. Fail. Risk again.

4. Build community and clinical cohort simultaneously

• Share information and eliminate parallel work.

#PennMedMDBR2018 #GGPennRareSymposium

Patient as Key Opinion Leader

http://www.kif1a.org/-weneedamouse.html

Dear Jax,Thirty-two families need a mouse.

#WeNeedAMouseDay 1

Day 2 Day 3 Day 4 Day 4 Day 5 Day 6 Day 7 Day 8 Day 9 Day 10 Day 11 Day 12 Day 13 Day 14

#PennMedMDBR2018 #GGPennRareSymposium

Rob Lutz

Chief Business Officer

Stonebridge Biopharma

New and Continuing Roles for Patients in Research and Drug Development

Patient Advocates and Pharmaceutical Companies

Global Genes / UPenn Symposium

May 19, 2018

13Confidential--for internal Strongbridge Biopharma uses only

My Rare Disease Journey

◼ My daughter Isabel is born with Prader Willi Syndrome (PWS) in January, 2000

◼ Joined the Board of PWSA(USA) the next year

◼ Took the opportunity to join a pharmaceutical company—Shire-- (in finance)

◼ Now Chief Business Officer at Strongbridge Biopharma plc (a rare disease focused company)

◼ 18 years later, there are FIVE late-stage clinical trials for PWS starting or about to start

◼ My daughter has significant challenges remaining but is graduating high school, is normal height/weight and LOVES horse back riding!

– Hopefully, future research and development will further improve her potential quality of life

Confidential--for internal Strongbridge Biopharma uses only 14

My Learnings: My Perspective as an Advocate and a Pharmaceutical Executive

◼ An organized, active group of patients, parents or advocates can make a difference- CONNECT THE DOTS

– Cultivate “key opinion leaders”

– Collect database of patients

– Document the disease (registry, patient-related outcomes, genetics, etc.)

– Provide grants for early scientific research (fill in gaps for NIH and pharma)

– Advocate at the FDA (they want to hear the voice of the patient)

– Attend or create conferences (both patient advocacy and medically focused)

– Collect samples (cells, brains, etc.)

– Build awareness (clinicians and pharma companies)

– YOU ARE THE EXPERTS

◼ Don’t forget to focus on yourself and the patient

Confidential--for internal Strongbridge Biopharma uses only 15