Kristin SchneemanKristin SchneemanProgram DirectorProgram Director

September 12, 2008September 12, 2008

““What You Seed Is What You Get” What You Seed Is What You Get” Making Faster Cures Making Faster Cures

the Central Organizing Principle the Central Organizing Principle of the Research Enterpriseof the Research Enterprise

Saving lives by saving timeSaving lives by saving time

Our mission is to identify and Our mission is to identify and implement global solutions to implement global solutions to

accelerate the pace of accelerate the pace of discovery and clinical development discovery and clinical development

of new therapies of new therapies for the treatment of disease.for the treatment of disease.

FasterCures’FasterCures’ Blueprint for Change Blueprint for Change

Research Leadership and Innovation• The Redstone Acceleration and Innovation

Network (TRAIN): Sharing best practices in funding and conducting innovative research

• Philanthropy Advisory Service: Develop a more transparent information marketplace to help philanthropists make informed investment decisions

• TRAIN-dustrial Relations: Facilitate partnerships between nonprofits and industry to help translate research into therapeutic breakthroughs

FasterCures’FasterCures’ Blueprint for Change Blueprint for Change

Research Tools and Resources

• Patients Helping Doctors

• BioBank Central

• Electronic health records

Research Environment

• Advocacy for change at the FDA and NIH

• Intellectual property and technology transfer

• HIPAA reform

““Entrepreneurs for Cures”Entrepreneurs for Cures”

What would your business model be if your bottom line were curing a disease?

The High Costs of Business as UsualThe High Costs of Business as Usual

We’re spending a lot on treating disease:$2.1 trillion on healthcare in 2006 – more than $7000 per person

We’re not spending much on curing disease:

And productivity is falling:

Sources: CMS, Research!America

A New Central Organizing PrincipleA New Central Organizing Principle

Our current research infrastructure has led to great advances in knowledge of human health and disease;

but it is not a good system for developing therapies for patients

studying biologystudying biology curing diseasescuring diseases

The Translation GapThe Translation Gap

Basic research is the earliest stage of research, carried out for the advancement of knowledge, without necessarily any regard to its application to practical problems.

Translational research is the process of applying ideas, insights, and discoveries generated through basic scientific inquiry to the treatment and prevention of human disease – the critical bridge between basic research and clinical research.

Clinical research is research in human subjects aiming toward approved treatments for patients.

Separating what we Separating what we need to do in scienceneed to do in science

and what we need to doand what we need to doin the culture of sciencein the culture of science

to accelerate curing diseasesto accelerate curing diseases

TRAIN: You Are Not AloneTRAIN: You Are Not Alone

The Redstone Acceleration and Innovation NetworkThe Redstone Acceleration and Innovation Network

Collaborative, mission-driven, results-oriented, andstrategic in their use of capital, these groups aremotivated solely by moving promising therapies

from the laboratory bench to the patient’s bedside asrapidly as possible – even those that do not directly

fund therapy development.

“Venture Philanthropy” → “Passion Capital”

• Strategic use of capital• Building collaborations• Streamlining the

grantmaking process• Sharing information

“…patient groups with an entrepreneurial bent have become the drug industry’s new power brokers.” - Forbes, 9/15/08

Nonprofits as “De-Riskers”Nonprofits as “De-Riskers”

• Developing pre-clinical tools• Targeting research to support translation• Funding mechanisms that bring in industry• Managing academic science• Access to patient community• New indications for existing drugs• High-throughput screening of promising

compounds• Access to scientific expertise• Advocating with FDA

New Business Models to Accelerate ResearchNew Business Models to Accelerate Research

• More than $66 million in research funding since 2005

• Actively supports more than 30 new treatments in development – 4 FDA-approved

• Partners include Vertex Pharmaceuticals, Parion Sciences, PTC Therapeutics, Inspire Pharmaceuticals, Gilead Sciences, Altus Pharmaceuticals

Research Preclinical Phase 1 Phase 2 Phase 3 To Patients

New Business Models to Accelerate ResearchNew Business Models to Accelerate Research

• Funded $126 million in research to date, two-thirds for translational research

• Funded testing of over 100 therapeutic targets for Parkinson’s Disease

• Partnership with Elan Corp. gives Elan first look at promising projects

New Business Models to Accelerate ResearchNew Business Models to Accelerate Research

• Has raised $102 million to fund 70 labs worldwide

• 4 new treatments approved in 4 years

• 10 clinical trials facilitated by MMRF’s Research Consortium

• 30 MMRF-funded compounds now in preclinical studies and clinical trials

Instead of Instead of following the money, following the money,

lead the money.lead the money.