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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
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
Children ’s Research Institute
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
Children ’s Research Institute
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
Children ’s Research Institute
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
MVP/ Main ResearchProgram
MVP/ Main ResearchProgram
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
MDA VENTURE PHILANTHROPY, INC.
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
MDA VENTURE PHILANTHROPY, INC.
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
MDA VENTURE PHILANTHROPY, INC.
• 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 VENTURE PHILANTHROPY, INC.
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
MDA VENTURE PHILANTHROPY, INC.
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.