Partnering with PatientsHoward Fillit, MD Executive Director , Alzheimer’s Drug Discovery Foundation Richard Insel, MD Executive Vice President, Research , Juvenile Diabetes Research Foundation

James C. O’Leary Chief Innovation Officer , Genetic Alliance Louise Perkins, PhD Chief Scientific Officer, Multiple Myeloma Research Foundation Steven Perrin, PhD Chief Executive Officer and Chief Scientific Officer , ALS Therapy Development Institute Kristin Schneeman - Chair Program Director, FasterCures  

nonprofit nonprofit think tank and think tank and catalyst for action catalyst for action that that 

works across sectors and diseases works across sectors and diseases to improve the effectiveness and efficiency of to improve the effectiveness and efficiency of 

the medical research systemthe medical research system

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

TRAIN Central Stationwww.fastercures.org/train

An effort designed to facilitate cross-sector collaborations needed to turn a scientific discovery into an accessible therapy. 

Be a part of an effort that brings together people with the expertise, experience, and creativity needed to transform the medical research system.

The MMRF and MMRC are sister organizations contributing to all phases of drug development.

• Funds worldwide research • Builds myeloma community• Partners with MMRC

• Supports 13 institutions• Conducts tissue banking• Facilitates Phase I/II trials

With over $140M raised to date, the MMRF serves as an end-to-end solution for drug development.

Implementation of business solutions to trial barriers led to MMRC trials opening 35% faster and robust trial enrollment.

0

50

100

150

200

250

300

350

400

Jan Feb Mar April May June July Aug Sep Oct Nov Dec

2007 2008 2009 2010

Trial Activation Enrollment

361

247

74

120

The MMRF Patient Navigator program aims to drive clinical trial accrual with specific benefits for late-stage trials.

Program Highlights

Up-to-date searchable database

Trained clinical specialists

Late-stage trials prioritized

Site support

Marketing

Targeted communications to stage of disease and geography

MMRF Newsletter articles, ads

Ads in MMRC, cancer publications

Social networking/communities

Accelerating Drug Discovery for Alzheimer’s Disease: The Alzheimer’s Drug Discovery Foundation

Accelerating Drug Discovery for Alzheimer’s Disease: ADDF Venture Philanthropy

Some Potential Advantages to Early Stage Biotechs of Venture Philanthropy Funding

Biotechnology Founders Program: CoMentisLeading Anti-Amyloid Therapy

Biotechnology Founders Program: CoMentisLeading Anti-Amyloid Therapy

Biotechnology Founders Program: Allon TherapeuticsLeading Anti-tangle Therapy

Listed on the Toronto Exchange (NPC.TSX) 2005, current market capitalization around $50M Listed on the Toronto Exchange (NPC.TSX) 2005, current market capitalization around $50M 

Received return of note at time of IPOReceived return of note at time of IPO

ADDF Biotechnology Founders Program: Allon Therapeutics, Leading Anti-tangle Therapy

17

– Start-up biotech company focused on development of small molecule mimetics of neurotrophins (NGF, BDNF) to treat AD, PD, and other neurodegenerative diseases

• “holy grail” of neuroprotection

– Founder is Professor Frank Longo, Chairman, Neurology and Neurosciences at Stanford

– PTX chose the ADDF Venture Philanthropy Fund as preferred founding investor

ADDF Venture Philanthropy Fund Investments

ADDF Venture Philanthropy Fund Investments

Juvenile Diabetes Research Foundation• Mission: To find a cure for type 1 diabetes and 

its complications• Strategy: Proactively accelerate the discovery, 

development, and delivery of drugs and devices to cure, better treat, or prevent type 1 diabetes and its complications

• Core Principles: People with T1DM, Pipeline, Priorities, Partners 

Beta

 Cel

lTh

erap

ies 

Imm

une 

Ther

apie

s  

DIABECELL® Encapsulated Cell Replacement Therapy

JDRF’s Industry Partners

Glu

cose

 Con

trol

 Co

mpl

icati

ons

Ther

apie

s  

JDRF’s Industry Partners

JDRF’s Clinical Trial Awareness Activities:JDRF Type 1 Diabetes Clinical Trials Connection (www.trials.jdrf.org)

Biobank of pancreata and related tissues from cadaveric organ donors with:

• Recent onset type 1 diabetes• Established diabetes including long-standing 

(>50yrs)•  At-risk, islet autoantibody positive 

Tissues being used by > 50 groups to address key immunological, histological, viral, and metabolic questions to better understand type 1 diabetes

www.jdrfnpod.org

JDRF’s BioBank: nPOD

JDRF’s FDA Activities• FDA-NIH-JDRF Workshops:

– How Good Does a Beta Cell Have to Be: November 2009– Towards An Artificial Pancreas: July 2008– Obstacles and Opportunities on the Road to Artificial Pancreas:

Closing the Loop: December 2005• Artificial Pancreas placed on Critical Path list in 2006; 

Artificial Pancreas Review team received FDA “Leveraging/Collaboration Award” in 2007

• FDA acceptance of in silico model to test algorithms – Algorithms can be extensively tested on in silico ‘patients’ prior 

to clinical study and allows elimination of animal testing

JDRF’s Reimbursement Activities JDRF funded a multicenter phase IV clinical trial of 

effectiveness of Continuous Glucose Monitors in Type 1 Diabetes → Change in reimbursement policy (United Healthcare, Aetna, Cigna, Wellpoint, Kaiser Permanente) and clinician and patient adoption

Why Does ALS TDI Exist?

• Nature Drug Discovery, February 2010• Nature Drug Discovery, March 2010

Drug DiscoveryPre Clinical

DevelopmentPhase I Phase II Phase III

3.1 yrs 2.5 yrs 1.5 yrs 2.5yrs 3.0 yrs

Prototypical Pharmaceutical Timelines

* Average cost to commercialization: $1.8B

TargetID Hit Lead 

OptimizationPreclinicalValidation

Phase I Phase IIPhase III

SubmissionLaunch

80%

24.3

$1

1.0

3%

$94

75%

19.4

$2.5

1.5

6%

$166

85%

14.6

$10

2.0

17%

$414

69%

12.4

$5

1.0

7%

$150

54%

8.6

$15

1.5

15%

$273

34%

4.6

$40

2.5

21%

$319

70%

1.6

$150

2.5

27%

$314

91%

1.1

$40

1.5

5%

$48

P(TS)

WIP per Launch

Cost per WIP per Phase

Cycle Time (Years)

Cost per Launch

% Total Cost per NME

• P(TS) : probability of technical success• WIP : work in progress• V : value• CT : cycle time• C : cost 

R&D Productivity Model

• Nature Drug Discovery March 2010 

Preclinical Cost per NMEAverage:  $18.5MCapitalized:  $824MCycle time:  5.5 years

Clinical Cost per NMEAverage:  $245MCapitalized:  $954MCycle time: 8 years

P(TS) x WIP x VCT x C

R&D Productivity = 

ALS TDI Investment in ALS TDI 00846

Drug DiscoveryPre Clinical

DevelopmentPhase I Phase II Phase III

3.1 yrs($10M)

1.4 yrs($20M)

1.7 yrs($10M)

2.7 yrs($30M)

1.9 yrs($100M)

Discovery Tools

Pk StudiesEfficacy Studies

PDStudies

Tolerability Studies

Disease Models

Clinical Samples

Therapeutic Development

Prototypical Pharmaceutical Cost & Timelines

0.75 yrs($0.6M)

1 yr($0.8M)

1.5 yrs($1.2M)

ALS TDI Cost & Timelines

• ALS TDI 00846 preclinical development: $2.5M• 40% of investment is in therapeutic development, pk, and tolerability

ALS-TDI Facilitates Clinical TranslationDe-Risk the Opportunity & Broker the Deal

Clinical Developme

nt

Study Diseas

e

Pre-Clinical Developme

nt

Build Drug

Pre-Clinical Development

ClinicalBasic Research

Drug Development Gap

Find a Drug Target

• Largest Online Discussion of ALS Therapeutics• 3,000+ Members, 40,000 posts•Topics include Therapeutics & Clinical Trials to Living With ALS and Advocacy• Free, anonymous and open to the public

• Largest Online Discussion of ALS Therapeutics• 3,000+ Members, 40,000 posts•Topics include Therapeutics & Clinical Trials to Living With ALS and Advocacy• Free, anonymous and open to the public

Merit Cudkowicz, M.D.Mass General HospitalHarvard Medical School

Jonathan Katz, M.DCalifornia Pacific Medical Center(Forbes Norris)

Stanley Appel, M.D.Methodist Neuro. InstituteCornell Medical School

ALS TDIPatient Forum

PreclinicalData

ClinicalTrials

ExperimentalTherapies

PatientCare

Carey Goldberg

Boston Globe NY- Times, LA- Times, AP

ALS ForumBuild and Empower a Community

Transforming Health through Genetics

Promoting an Environment of Openness

Survey of Disease Advocacy Organizations• 37 organizations responded to the survey• 13 organizations (35%) were involved with a 

biorepository, and 15 organizations (41%)  were involved with a registry

• Half of biorepositories and 73% of registries have been in operation for more than 5 years

• Of those organizations without biorepositories or registries, 65% were interested in establishing a biorepository, and 86% were interested in establishing a registry

• Cost was the major reason for not establishing a biorepository or registry

DAO DemographicsCharacteristic N (%)Organization Age

<5 years 2 (6)5-10 years 13 (36)11-20 years 15 (42)>20 years 6 (17)

Paid EmployeesYes 28 (76)

Volunteer NetworkYes 29 (81)

Annual Budget<$100K 17 (49)$100K-$499K 11 (31)$500K-$999K 6 (17)$1M-$5M 1 (3)>$5M 1 (3)

DAO InvolvementTypes of Engagement N (%)

Established Biorepository/registry 12 (67)

Own Biorepository/registry 9 (53)

Provide Financial Support 10 (56)

Help Obtain Financial Support 10 (56)

Recruit Participants 17 (94)

Obtain Informed Consent 11 (61)

Sample TypesBiorepository Collects N (%)

Blood 13 (72)

Tissue (Frozen) 10 (56)

Tissue (Formalin) 9 (50)

Cell Blocks 5 (28)

Cell Lines 7 (39)

Other Biological Material 3 (17)

Desire to Collect other Types 13 (81)

Sample Use and StorageCharacteristic N (%)Experiments DNA/genomics Analysis 9 (64) RNA Expression 1 (7) Protein/immunohistochemistry 2 (14) Other 5 (36)Users Research Consortium 5 (31) Any Qualified Researcher 12 (75) Other 5 (31) Costs Passed on to Researcher 9 (50)Location Nonprofit Organization 3 (21) Commercial Lab 3 (21) University/research Lab 8 (57) Government Lab 1 (7) Other 3 (21)

Disease Specific OrganizationData and Sample Management

Informed ConsentRobust Clinical Information

Sample Collection & TrackingData and Sample Query

Data ManagementElectronic & Paper Records

Security & Access Control

Informed ConsentRobust Clinical Information

Sample Collection & TrackingData and Sample Query

Data ManagementElectronic & Paper Records

Security & Access Control

Patient Enrollment

Disease Sample and Clinical

Banking

Disease Sample and Clinical

Banking

Biological SamplesBiological Samples

Clinical InformationClinical Information

Web basedBoot camps

ManualsWorkbooks

WikisWebinars

Demonstrating Private Access’ Technology to Transform Patient Recruitment

Thank you• James C. O'Leary

Chief Innovation Officer

• Genetic Alliance4301 Connecticut Ave. NWSuite 404Washington, DC 20008-2369

• (202) 966-5557 x206joleary@geneticalliance.orgwww.geneticalliance.org