Turning Research into Treatments

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Turning Research into Treatments

  1. 1. Turning Research into Treatments MDA VENTURE PHILANTHROPY, INC.
  2. 2. Muscular Dystrophy Association And MDA Venture Philanthropy MDA is a 50 year-old, $150M charity We cover 40 neuromuscular diseases Programs expenditures of $150M: clinical services, research and education MVP is MDA’s drug development arm, with the sole goal of funding the discovery and commercialization of treatments and cures for neuromuscular disease.
  3. 3. 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
  4. 4. Muscular Dystrophies Duchenne Becker Limb-Girdle Facioscapulohumeral Congenital Oculopharyngeal Distal Emery-Dreifuss Metabolic Myopathies Phosphorylase Deficiency Acid Maltase Disease Phosphofructokinase Deficiency Debrancher Enzyme Deficiency Mitochondrial Myopathy Carnitine Deficiency Carnitine Palmityl Transferase Deficiency Phosphoglycerate Kinase Deficiency Phosphoglycerate Mutase Deficiency Lactate Dehydrogenase Deficiency Myoadenylate Deaminase Deficiency Other Myopathies Myotonia Congenita Paramyotonia Congenita Central Core Disease Nemaline Myopathy Myotubular Myopathy Periodic Paralysis MDA’s Umbrella Motor Neuron Disease Amyotrophic Lateral Sclerosis Spinal Muscular Atrophy Spinal Bulbar Muscular Atrophy Inflammatory Myopathies Polymyositis Dermatomyositis Inclusion Body Myositis Diseases of Neuromuscular Junction Myasthenia Gravis Lambert-Eaton Syndrome Congenital Myasthenic Syndromes Endocrine Myopathies Hyperthyroid Myopathy Hyopthyroid Myopathy Diseases of Peripheral Nerve Charcot-Marie-Tooth Disease Friedreich’s Ataxia Dejerine-Sottas Disease
  5. 5. 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 the weakened muscles. Amyotrophic Lateral Sclerosis: Treatments: Rilutek/riluzole Originator: Sanofi-Aventis. Function: Glutamate antagonist. Launched in 21 countries. Also used to treat Huntington’s disease. On average increases survival by 3 months, no reduction in rate of progression of the disease. Spinal Muscular Atrophy: No therapies currently available. Most diseases in our program have no therapies available, and all are orphan diseases
  6. 6. Pipeline: Duchenne Muscular Dystrophy Preclinical Human TrialsDiscovery PhaseI PhaseII PhaseIII MDAcontribution Technology Company DMDalpha7 Integrin Antioxidant Exonskipping Non -viralGeneTherapy Glutamine/ creatine HDACinhibitors Growthfactors Mitochondrialpore Myostatin inhib NF-kB inhibition NOstimulation Cardiacdrugs Utrophin upregulation Pentoxifylline Oxatomide HGFmimetic RPI78M Membranesealant Glucocorticoids GeneTherapy:AAV NonsenseSuppression GeneTherapy:AD Antifibrotic Losartan MyoblastTransplant Exercise Laminin -111 MuscleGrowth StemCells GeneCorrection Utrophingenetherapy PGC-1alpha Santhera Prosensa,AVI Biopharma ,etc AMT,Genzyme,Asklepios,etc. Invivogen Fermentek Debiopharm ,Scynexis Acceleron , Orico ,PTCetc. PTCTherapeutics Children ’sResearchInstitute Children ’sResearchInstitute Salbutamol Schering -Plough Prothelia Phrixus CooperativeInstituteofNeurologicalResearch Stempath ,Stemcellsciencesetc Avicena Summit,Domain, PTCTherapeutics PTC, Combinatorx ,Prothelia etc. Angion ReceptoPharm Catabasis ,Validus, Combinatorx etc. NicOx Biglycan Tyversan TRIM -edicine Children ’sResearchInstitute ClevelandClinicFoundation UniversityofCalifornia,LosAngeles UniversityofPittsburgh McGillUniversity UniversityofBasel UniversityofMassachusettsPPARdelta Proteasome inhibition ThomasJeffersonUniversity UniversityofCalifornia,LosAngelesMuscledelivery prot . NationwideChildren ’sHospitalUDP -GalNac UTSouthwesternMedicalCenter JohnsHopkinsUniversity Orig:Schering -Plough,Shireetc. Calpain inhib . Ipsen , CepTor Preclinical Human TrialsDiscovery PhaseI PhaseII PhaseIII MDAcontribution Technology Company DMDalpha7 Integrin Antioxidant Exonskipping Non -viralGeneTherapy Glutamine/ creatine HDACinhibitors Growthfactors Mitochondrialpore Myostatin inhib NF-kB inhibition NOstimulation Cardiacdrugs Utrophin upregulation Pentoxifylline Oxatomide HGFmimetic RPI78M Membranesealant Glucocorticoids GeneTherapy:AAV NonsenseSuppression GeneTherapy:AD Antifibrotic Losartan MyoblastTransplant Exercise Laminin -111 MuscleGrowth StemCells GeneCorrection Utrophingenetherapy PGC-1alpha Santhera Prosensa,AVI Biopharma ,etc AMT,Genzyme,Asklepios,etc. Invivogen Fermentek Debiopharm ,Scynexis Acceleron , Orico ,PTCetc. PTCTherapeutics Children ’sResearchInstitute Children ’sResearchInstitute Salbutamol Schering -Plough Prothelia Phrixus CooperativeInstituteofNeurologicalResearch Stempath ,Stemcellsciencesetc Avicena Summit,Domain, PTCTherapeutics PTC, Combinatorx ,Prothelia etc. Angion ReceptoPharm Catabasis ,Validus, Combinatorx etc. NicOx Biglycan Tyversan TRIM -edicine Children ’sResearchInstitute ClevelandClinicFoundation UniversityofCalifornia,LosAngeles UniversityofPittsburgh McGillUniversity UniversityofBasel UniversityofMassachusettsPPARdelta Proteasome inhibition ThomasJeffersonUniversity UniversityofCalifornia,LosAngelesMuscledelivery prot . NationwideChildren ’sHospitalUDP -GalNac UTSouthwesternMedicalCenter JohnsHopkinsUniversity Orig:Schering -Plough,Shireetc. Calpain inhib . Ipsen , CepTorCalpain inhib . Ipsen , CepTor
  7. 7. MDA Funding Distribution Basic Research Drug Screening Target Identification “Proof-of-Principle” Testing in Animals Translational Research “Preclinical Drug Development” Clinical Research Including Trials Main Research Program MDA Funding in Millions Active projects MVP/ Main Research Program MVP/ Main Research Program MVP/ Main Research Program MVP/ Main Research Program $67.1 $20.5 $16.7 $3.9 $10.0
  8. 8. MDA’s Translational Research Program  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.
  9. 9. Muscular Dystrophy Drugs Under Development 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.newdrugproducts MDA Translational Research Program Launched *PharmaProjects
  10. 10. 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 Investments 2004- 2008 MDA VENTURE PHILANTHROPY, INC.MDA VENTURE PHILANTHROPY, INC.
  11. 11. Leverage….  In 2005, MDA invested $1.5 million into a phase I and phase IIa study of PTC 124 for Duchenne muscular dystrophy (CF Foundation also invested in this drug)  In 2008, Genzyme and PTC Therapeutics signed a co-development deal worth up to $400M to move the drug forward for four indications including Duchenne and Cystic Fibrosis MDA VENTURE PHILANTHROPY, INC.
  12. 12. Elevator Speech  Makes selective investments in companies with promising paths to market.  Conducts professional diligence in evaluating investment opportunities.  Builds on MDA’s investment of over $1 billion in science, manpower and infrastructure.  Leverages investments at strategic points in the drug development process. MDA Venture Philanthropy is a new program of MDA exclusively focused on funding the discovery and commercialization of treatments and cures for neuromuscular disease.
  13. 13. 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.
  14. 14. • 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 Partners MDA VENTURE PHILANTHROPY, INC.
  15. 15. 220 MDA Clinics Nationwide 10 networked clinical research centers MDA Clinic MDA ALS Clinic Duchenne research network ALS research network
  16. 16. 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 the group  Will be invited to annual MVP investor meeting MDA VENTURE PHILANTHROPY, INC.
  17. 17. MDA’s Venture Philanthropy Strategic Objectives
  18. 18. MDA Venture Philanthropy Business Horizons Place 3 Investments Place 4 drugs into clinical testing Place 6 drugs into clinical testing; receive at least one ROI
  19. 19. Stanley Appel, M.D. Chair, Department of Neurology Methodist Neurological Institute Cristina Csimma, PharmD, M.H.P. VP Drug Development Virdante Pharmaceuticals, Inc Merit Cudkowicz, M.D. Director Neurology Clinical Trial Unit Director MDA ALS Clinic Massachusetts General Hospital Kenneth H. Fischbeck, M.D. Neurogenetics Branch Porter Neuroscience Research Center National Institutes of Neurological Disorders and Stroke Louis M. Kunkel, Ph.D. Professor of Pediatrics Harvard Medical School/Children's Hospital Elizabeth McNally, M.D., Ph.D. Director, Cardiovascular Genetics Clinic University of Chicago Medical Center Brian A. Pollok, Ph.D. Chief Scientific Officer Head of Global Research and Development Life Technologies John D. Porter, Ph.D. Program Director, Neuromuscular Disease Channels, Synapses, and Circuits Cluster National Institutes of Neurological Disorders and Stroke Charles Thornton, M.D. Associate Professor of Neurology University of Rochester Lee Wrubel, M.D. General Partner Foundation Medical Partners Scientific Development Advisory Board
  20. 20. Cristina Csimma, PharmD, M.H.P. VP Drug Development Virdante Pharmaceuticals, Inc John E. Howell Partner Global Strategic Partners Augie Nieto Chairman, Octane Fitness MDA ALS Division Co-Chairperson Mario J. Palumbo, Jr. Partner Millenium Partners Joe Rhodes Managing Partner Stockton Road LLC Lee Wrubel, M.D. General Partner Foundation Medical Partners Business Advisory Board
  21. 21. Contact Information Jane Larkindale, Ph.D. Portfolio Director MDA Venture Philanthropy jlarkindale@mdavp.org (520) 615-6702 Sharon Hesterlee, Ph.D. Senior Vice President MDA Venture Philanthropy shesterlee@mdavp.org (520) 615-6701 MDA VENTURE PHILANTHROPY, INC.
  22. 22. Summary MVP ‘s goal is to accelerate getting therapies to the clinic for the over 40 neuromuscular diseases that have no current cures. MVP is funded through resources from MDA’s grassroots funding, and by major gifts to specific disease portfolios. MVP works by investing in therapies at early stages bridging the valley of death, reducing the costs and time of clinical trials and by facilitating interactions between researchers, companies and clinicians.

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