Sanjay Bidichandani, MBBS, PhDVice President for Research

Basic ResearchTarget Identification Drug Screening

“Proof-of-Principle”Testing in Animals

Translational Research“Preclinical Drug

Development”

Clinical ResearchIncluding Trials

MDA’s Research Portfolio

Total ~300 grants ~$100 M

Annually ~100 new grants ~$40 M

• Medical Advisory Committee • Scientific Advisory Committee • Translational Res Advisory Committee• Ad hoc peer-review

Muscular DystrophiesDuchenneBeckerLimb-GirdleFacioscapulohumeralCongenitalOculopharyngealDistalEmery-DreifussMyotonic dystrophy

Metabolic MyopathiesPhosphorylase DeficiencyAcid Maltase DiseasePhosphofructokinase DeficiencyDebrancher Enzyme DeficiencyMitochondrial MyopathyCarnitine DeficiencyCarnitine Palmityl Transferase DeficiencyPhosphoglycerate Kinase DeficiencyPhosphoglycerate Mutase DeficiencyLactate Dehydrogenase DeficiencyMyoadenylate Deaminase Deficiency

Other MyopathiesMyotonia CongenitaParamyotonia CongenitaCentral Core DiseaseNemaline MyopathyMyotubular MyopathyPeriodic Paralysis

Motor Neuron DiseaseAmyotrophic Lateral SclerosisSpinal Muscular AtrophySpinal Bulbar Muscular Atrophy

Inflammatory MyopathiesPolymyositisDermatomyositisInclusion Body Myositis

Neuromuscular Junction DiseasesMyasthenia GravisLambert-Eaton SyndromeCongenital Myasthenic Syndromes

Endocrine MyopathiesHyperthyroid MyopathyHyopthyroid Myopathy

Diseases of Peripheral NerveCharcot-Marie-Tooth DiseaseFriedreich AtaxiaDejerine-Sottas Disease

MDA’s Research Portfolio

Active projects (million)

Basic Research Drug ScreeningTarget Identification

“Proof-of-Principle”Testing in Animals

Translational Research“Preclinical DrugDevelopment”

Clinical ResearchIncluding Trials

Basic Research Program

Basic Research Program

Translational / Basic Research Program

Translational Translational / other

$60.8

$4.8

$14.2

$3.7 $7.2

MDA’s Research Portfolio

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35

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45

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gMDA’s Research Portfolio

FY10 / non-ARRA

**

Turning Research into Treatments

MDA VENTURE PHILANTHROPY

TargetID

HitID

LeadOptim.

Can Pre-IND

P1 P2 P3 Reg

2 yrs 1yr 1.5-2yrs 1 yr 1 yr 2 yr 2-3 yrs

RiskRisk CostCost

MDA

Academia Biotech Big Pharma

Translational research

MDA’s Drug Development Program

MVP projects by stage of development

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1

2

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animal PoC preclinical Phase 1 or 2a Phase 2

Stage (projects may overlap)

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cts

MDA’s Recent Advances In Therapy Development

Repligen Friedreich ataxia

Friedreich ataxia Repligen

Askelepios Duchenne / Becker

ReveraGen Duchenne / Becker

Duchenne PTC

Mendell LGMD2d

Miller ALS (SOD1)

PoC PreC I/IIa IIb

Repligen SMA

“It’s a great time to be a mouse with a neuromuscular disease!”

John Porter, Ph.D. (NIH / NINDS)

…and that’s a good thing…

Challenges

How to safely and effectively extend these successes to humans?

Who is going to fill the void between biotechs funded by foundations & fed - and - big pharma?

Smart partnership….

Finding Treatments: Smart Partnership

• Leveraging through MDA’s drug development program• Federal govt & other non-profits• Partnership with Pharma and Biotech companies• Venture capitalists

Enhancing Research through

Communication & Collaboration

MDA Neuron SymposiumRole of glia in motor neuron degeneration in ALS

MDA Muscle SymposiumNewborn screening for Duchenne muscular dystrophy

MDA Translational SymposiaEncouraging technology transfer from academiaGene therapy for neuromuscular disease (with AFM)

MDA Symposium Series - 2012

Getting a head start…

MDA ClinicMDA ALS ClinicDuchenne research networkALS research network

Clinics & Clinical Research Networks

Currently = 40 Trainees (~$7 million)

Historically = 377 trainees (~ $48 million)

Investment in Training Researchers….

• Basic researchers (DG)• Clinical researchers (CRTG)

MVP: Program Review

Zero trainees in translational projects!

Bridge to Industry (B2I)

• B2I postdoctoral fellowships Training future NMD translational researchers

Academia

Industry

Future Muscle Disease Researchers