myelin repair open science summit 07.31.10 v2

104/08/23

The Myelin Repair Foundation

Open Science SummitJuly 31, 2010

A New Paradigm for Medical Research

04/08/23

2

Drug Discovery Today

04/08/23

3

Academic Research Academic Research Drug Drug DiscoveryDiscovery

Proposal, peer review and grant system results in:

Incremental experiments

Competition and lack of coordination between scientific disciplines

Inefficient communication process

Scientists do not specialize in diseases

Chaotic progress - no disease based research roadmap

Intellectual Property is not protected

04/08/23

4

Pharmaceutical R&D output flat despite increasing spend...

1. IMDs (Incrementally Modified Drugs) are new derivatives, formulations and combinations2. NME (New Molecular Entities) include both chemical and biological therapeutic agents, excludes contrast agents and diagnosticsSource: FDA, PhRMA 2005 Yearbook

…but NME approvals relatively flat…but NME approvals relatively flat

0

70

140

’91 ’92 ’93 ’94 ’95 ’96 ’97 ’98 ’99 ’00 ’01 ’02 ’03 ’04 ’05

No. of approvals

NMEs

INDs

R&D spending has grown steadily…R&D spending has grown steadily…

0

15

30

45

’91 ’92 ’93 ’94 ’95 ’96 ’97 ’98 ’99 ’00 ’01 ’02 ’03 ’04 ’05

($B)

CAGR11.3%

CAGR10.5%

Domestic R&D

R&D abroad

04/08/23

5

Many Players but historically no overall coordinator/manager

National Institutes of Health

Legacy non-profit disease research organizations

Academic scientists

Contract research organizations

Biotech and pharmaceutical industry

Food and Drug Administration

Medical service providers

Insurance companies

Patients and their families

04/08/23

6

The System is Broken

04/08/23

7

MRF’s Mission

To accelerate the discovery of myelin repair treatments to improve the lives of people suffering from Multiple Sclerosis (MS), and

To establish the MRF Accelerated Research Collaboration™ (ARC™) model as a new paradigm for medical research.

04/08/23

804/08/23 8

SponsoredResearch

BenBarres

RobertMiller

SteveMiller

BrianPopko

Mu

ltip

le

04/08/23

904/08/23 9

Six Areas of Research

1. Understanding how oligodendrocytes are normally generated from neural stem cells and how MS perturbs this process

2. Understanding the underlying mechanism of myelination and how it is perturbed in MS

3. Understanding how nodes of Ranvier and paranodes are normally formed and how they are perturbed in MS

4. Understanding the immune response in MS and how inflammation affects myelin repair

5. Understanding how the Blood Brain Barrier is affected in MS and its role in the disease

6. Development of better animal models for study of MS and remyelination

MRF’s Discovery Biology Research Plan

04/08/23

10

Crossing the Valley of Death – Accelerating the transition from promising targets to clinical candidates

0

200

400

600

800

1,000

Target ID Validation Screening Lead opt Pre-clin Phase I Phase II Phase III NDA Total

Percentage of total cost of success

$880M

19%

23%

5%

10%

7%

10%

11% 1%

Source: BCG economic model study in Biomedical Industry Advisory Group, 2005; BCG clinical development model, 2007

TargetValidation

Screen-ing

TargetID

Optimi-zation

Pre- clinical

PhaseI

PhaseII

PhaseIII

Filing NDA

14%

71% of industry costs

1.0 0.4 2.7 1.5 1.5 2.5 1.5 14.72.0 1.6Avg.

time (yr)

ARC II

15 - 30

Cost ($M)

Basic Research

MRF’s ARC Model Proposed ARC Expansion

04/08/23

11

First Five Years – 2004 to 2009Exceeded milestones and goals

Established Center for Myelin Repair Discovery Biology with replicable infrastructure.

Engaged leading myelin repair scientists in a managed, patient-outcome-driven research plan.

Discovered 144 putative targets.

Developed 24 new research tools with application to all neurological disease research.

(continued)

04/08/23

12

First Five Years – 2004 to 2009Exceeded milestones and goals

Awarded first patent in 2008; 18 more patent applications have been submitted or are in preparation.

Developed “virtual biotech” infrastructure for evaluating targets.

Established myelin repair as a viable alternative approach to treating multiple sclerosis

Established first-of-a-kind partnership with an pharmaceutical company to advance one or more therapeutic targets and/or models.

Raised $33M but accomplished the above for $21M.

04/08/23

13

MRF’s Second Five Years 2009 - 2014

Identify more high-quality potential therapeutic targets

Validate therapeutic targets to meet industry standards

Build industry partnerships leading to clinical trials

Identify new approaches to measuring clinical outcomes and to speed clinical trials

04/08/23

14

04/08/23 14

04/08/23

15

Strategies for Translating MRF Discoveries

Support existing pharma company pipelines• Offer access to MRF Target Validation toolkit• Provide data for “repurposing” existing clinical candidates

Contracting• Promote research collaborations with corporate entities• Fund Target Validation/Drug Discovery studies at CROs

Partnering• Offer exclusive licensing options to MRF targets/tools• Create Joint IP with biotech/pharma partners

Spinoffs• Create new corporate entities to develop innovative MRF programs

04/08/23

16

The only non-profit medical research foundation that participates in the entire continuum of drug discovery