Turning Research into Treatments

Turning Research into Treatments

MDA VENTURE PHILANTHROPY, INC.

Muscular Dystrophy AssociationAnd MDA Venture Philanthropy

MDA is a 50 year-old, $150M charity

We cover 40 neuromuscular diseases

Programs expenditures of $150M: clinical services, research andeducation

MVP is MDA’s drug development arm, with the sole goal offunding the discovery and commercialization of treatmentsand cures for neuromuscular disease.

MDA Research – Active Projects($123 million committed)

44,787,999

Muscular dystrophies

Motor Neuron diseases

Other

$49,354,168

$35,222,786

$38,762,031

Muscular DystrophiesDuchenneBeckerLimb-GirdleFacioscapulohumeralCongenitalOculopharyngealDistalEmery-Dreifuss

Metabolic MyopathiesPhosphorylase DeficiencyAcid Maltase DiseasePhosphofructokinase DeficiencyDebrancher Enzyme DeficiencyMitochondrial MyopathyCarnitine DeficiencyCarnitine Palmityl TransferaseDeficiencyPhosphoglycerate Kinase DeficiencyPhosphoglycerate Mutase DeficiencyLactate Dehydrogenase DeficiencyMyoadenylate Deaminase DeficiencyOther MyopathiesMyotonia CongenitaParamyotonia CongenitaCentral Core DiseaseNemaline MyopathyMyotubular MyopathyPeriodic Paralysis

MDA’s Umbrella

Motor Neuron DiseaseAmyotrophic Lateral SclerosisSpinal Muscular AtrophySpinal Bulbar Muscular AtrophyInflammatory MyopathiesPolymyositisDermatomyositisInclusion Body MyositisDiseases of NeuromuscularJunctionMyasthenia GravisLambert-Eaton SyndromeCongenital Myasthenic SyndromesEndocrine MyopathiesHyperthyroid MyopathyHyopthyroid MyopathyDiseases of PeripheralNerveCharcot-Marie-Tooth DiseaseFriedreich’s AtaxiaDejerine-Sottas Disease

Market Landscape

Duchenne Muscular Dystrophy: Treatments: Prednisone. Function: Corticosteroid antagonist. Launched. Prolongs

walking in patients, but has side affects including weight gain which adds stain to theweakened muscles.

Amyotrophic Lateral Sclerosis: Treatments: Rilutek/riluzole Originator: Sanofi-Aventis. Function: Glutamate

antagonist. Launched in 21 countries. Also used to treat Huntington’s disease. Onaverage increases survival by 3 months, no reduction in rate of progression of thedisease.

Spinal Muscular Atrophy: No therapies currently available.

Most diseases in our program have no therapiesavailable, and all are orphan diseases

Pipeline: Duchenne MuscularDystrophy

Preclinical Human TrialsDiscovery

Phase I Phase II Phase III

MDA contribution

Technology Company

DMDalpha7 Integrin

Antioxidant

Exon skipping

Non -viral Gene Therapy

Glutamine/ creatine

HDAC inhibitors

Growth factors

Mitochondrial pore

Myostatin inhib

NF -kB inhibition

NO stimulation

Cardiac drugs

Utrophin upregulation

Pentoxifylline

Oxatomide

HGF mimetic

RPI78M

Membrane sealant

Glucocorticoids

Gene Therapy: AAVNonsense Suppression

Gene Therapy: AD

Antifibrotic

Losartan

Myoblast Transplant

Exercise

Laminin -111

Muscle Growth

Stem Cells

Gene Correction

Utrophin gene therapy

PGC-1alpha

Santhera

Prosensa, AVI Biopharma , etc

AMT, Genzyme, Asklepios, etc.

Invivogen

Fermentek

Debiopharm , Scynexis

Acceleron , Orico , PTC etc.

PTC Therapeutics

Children ’s Research Institute


Salbutamol Schering -Plough

Prothelia

PhrixusCooperative Institute of Neurological Research

Stempath , Stem cell sciences etc

Avicena

Summit, Domain,

PTC Therapeutics

PTC, Combinatorx , Prothelia etc.

Angion

ReceptoPharm

Catabasis , Validus, Combinatorx etc.

NicOx

Biglycan Tyversan

TRIM -edicine


Cleveland Clinic Foundation

University of California, Los Angeles

University of Pittsburgh

McGill University

University of Basel

University of MassachusettsPPARdelta

Proteasome inhibition Thomas Jefferson University

University of California, Los AngelesMuscle delivery prot .

Nationwide Children ’s HospitalUDP -GalNac

UT Southwestern Medical Center

Johns Hopkins University

Orig: Schering -Plough, Shire etc.

Calpain inhib . Ipsen , CepTor

Preclinical Human TrialsDiscovery

Phase I Phase II Phase III

MDA contribution

Technology Company

DMDalpha7 Integrin

Antioxidant

Exon skipping

Non -viral Gene Therapy

Glutamine/ creatine

HDAC inhibitors

Growth factors

Mitochondrial pore

Myostatin inhib

NF -kB inhibition

NO stimulation

Cardiac drugs

Utrophin upregulation

Pentoxifylline

Oxatomide

HGF mimetic

RPI78M

Membrane sealant

Glucocorticoids

Gene Therapy: AAVNonsense Suppression

Gene Therapy: AD

Antifibrotic

Losartan

Myoblast Transplant

Exercise

Laminin -111

Muscle Growth

Stem Cells

Gene Correction

Utrophin gene therapy

PGC-1alpha

Santhera

Prosensa, AVI Biopharma , etc

AMT, Genzyme, Asklepios, etc.

Invivogen

Fermentek

Debiopharm , Scynexis

Acceleron , Orico , PTC etc.

PTC Therapeutics



Salbutamol Schering -Plough

Prothelia

PhrixusCooperative Institute of Neurological Research

Stempath , Stem cell sciences etc

Avicena

Summit, Domain,

PTC Therapeutics

PTC, Combinatorx , Prothelia etc.

Angion

ReceptoPharm

Catabasis , Validus, Combinatorx etc.

NicOx

Biglycan Tyversan

TRIM -edicine


Cleveland Clinic Foundation

University of California, Los Angeles

University of Pittsburgh

McGill University

University of Basel

University of MassachusettsPPARdelta

Proteasome inhibition Thomas Jefferson University

University of California, Los AngelesMuscle delivery prot .

Nationwide Children ’s HospitalUDP -GalNac

UT Southwestern Medical Center

Johns Hopkins University

Orig: Schering -Plough, Shire etc.

Calpain inhib . Ipsen , CepTorCalpain inhib . Ipsen , CepTor

MDA Funding Distribution

Basic Research Drug ScreeningTarget Identification

“Proof-of-Principle”Testing in Animals

Translational Research“Preclinical Drug

Development”Clinical ResearchIncluding Trials

Main ResearchProgram

MDA Funding in Millions

Active projects

MVP/ Main ResearchProgram




$67.1

$20.5$16.7

$3.9 $10.0

MDA’s Translational ResearchProgram

Started in 2004

Four major grant categories: Corporate Grant (6 projects funded) IND-Planning Grant (2 projects funded) Infrastructure Grant (9 projects funded) Clinical Research Training Grant (6 Fellows funded)

Expenditures to date: ~$13 M


Muscular Dystrophy Drugs UnderDevelopment by Industry*

No. new drugs started commercial development in muscular

dystrophies

0

1

2

3

4

5

6

7

8

1993 1994 1995 1996 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008

year

No

. n

ew

dru

g p

rod

uc

ts

MDA Translational ResearchProgram Launched

*PharmaProjects

Lead Candidate Optimization of HDAC inhibitor for Friedreich’s Ataxia

Stem cell transplantation strategy for ALS

Phase IIa Study of Iplex in Myotonic Muscular Dystrophy

Phase Ib/II Study of Mini-Dystrophin Gene in AAV Vector

PTC 124 Treatment for Duchenne Muscular Dystrophy

Phase I/II Study of Mini-Dystrophin Gene in AAV Vector

Project

$0.2MCalifornia Stem Cell, Inc.

$1.0MRepligen Corporation

$2.1MInsmed

$2.5MAsklepios Biopharmaceuticals, Inc.

$1.47MPTC Therapeutics, Inc.

$1.57MAsklepios Biopharmaceuticals, Inc.

InvestmentCompany

MDA For-Profit Investments2004- 2008

MDA VENTURE PHILANTHROPY, INC.MDA VENTURE PHILANTHROPY, INC.

Leverage….

In 2005, MDA invested $1.5 million into a phase Iand phase IIa study of PTC 124 for Duchennemuscular dystrophy (CF Foundation also investedin this drug)

In 2008, Genzyme and PTC Therapeutics signeda co-development deal worth up to $400M tomove the drug forward for four indicationsincluding Duchenne and Cystic Fibrosis


Elevator Speech

Makes selective investments in companies with promising pathsto market.

Conducts professional diligence in evaluating investmentopportunities.

Builds on MDA’s investment of over $1 billion in science,manpower and infrastructure.

Leverages investments at strategic points in the drugdevelopment process.

MDA Venture Philanthropy is a new program ofMDA exclusively focused on funding thediscovery and commercialization of treatmentsand cures for neuromuscular disease.

Evaluation Process

No committee decisions No grant applications Iterative process 13 week turn-around plan Sophisticated business and science due diligence Routine and regular evaluation of intellectual

property


• FDA buy-in for clinical trial endpoints

• A responsive physician network through MDA Clinics

• International scale registries with common core data sets

• Efficient trial networks established with experienced clinicians for multicenter trials using standardized, validated endpoints

• Current natural history data

•Access to world class expertise in all disease areas

MDA/MVP as PartnersMDA VENTURE PHILANTHROPY, INC.

220 MDA Clinics Nationwide10 networked clinical research centers

MDA ClinicMDA ALS ClinicDuchenne research networkALS research network

Leverage: Follow-on Funding

Council of Industry Advisors Pharmaceuticals Companies Larger Biotechnology Companies Interested VCs

Will have access to public reports from MVP

MVP will consider suggestions from thegroup

Will be invited to annual MVP investormeeting


MDA’s Venture PhilanthropyStrategic Objectives

MDA Venture PhilanthropyBusiness Horizons

Place 3 Investments Place 4 drugs intoclinical testing

Place 6 drugs intoclinical testing; receiveat least one ROI

Stanley Appel, M.D.Chair, Department of NeurologyMethodist Neurological Institute

Cristina Csimma, PharmD, M.H.P.VP Drug DevelopmentVirdante Pharmaceuticals, Inc

Merit Cudkowicz, M.D.Director Neurology Clinical Trial UnitDirector MDA ALS ClinicMassachusetts General Hospital

Kenneth H. Fischbeck, M.D.Neurogenetics BranchPorter Neuroscience Research CenterNational Institutes of Neurological Disorders andStroke

Louis M. Kunkel, Ph.D.Professor of PediatricsHarvard Medical School/Children's Hospital

Elizabeth McNally, M.D., Ph.D.Director, Cardiovascular Genetics ClinicUniversity of Chicago Medical Center

Brian A. Pollok, Ph.D.Chief Scientific OfficerHead of Global Research and DevelopmentLife Technologies

John D. Porter, Ph.D.Program Director, Neuromuscular DiseaseChannels, Synapses, and Circuits ClusterNational Institutes of Neurological Disorders andStroke

Charles Thornton, M.D.Associate Professor of NeurologyUniversity of Rochester

Lee Wrubel, M.D.General PartnerFoundation Medical Partners

Scientific Development Advisory Board

Cristina Csimma, PharmD, M.H.P.VP Drug DevelopmentVirdante Pharmaceuticals, Inc

John E. HowellPartnerGlobal Strategic Partners

Augie NietoChairman, Octane FitnessMDA ALS Division Co-Chairperson

Mario J. Palumbo, Jr.PartnerMillenium Partners

Joe RhodesManaging PartnerStockton Road LLC

Lee Wrubel, M.D.General PartnerFoundation Medical Partners

Business Advisory Board

Contact Information

Jane Larkindale, Ph.D. Portfolio Director MDA Venture Philanthropy [email protected] (520) 615-6702

Sharon Hesterlee, Ph.D. Senior Vice President MDA Venture Philanthropy [email protected] (520) 615-6701


Summary

MVP ‘s goal is to accelerate getting therapies to the clinic for the over 40neuromuscular diseases that have no current cures.

MVP is funded through resources from MDA’s grassroots funding, and by major giftsto specific disease portfolios.

MVP works by investing in therapies at early stages bridging thevalley of death, reducing the costs and time of clinical trials andby facilitating interactions between researchers, companies andclinicians.

Turning Research into Treatments

Health & Medicine

Transcript of Turning Research into Treatments