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Mission Increase the longevity and quality of life of
patients with muscular dystrophy
Lead Drug Candidate – LAM-111 Delivered systemically to MDC1A and DMD
mouse models Excellent efficacy with no toxicity Human LAM-111 in human MDC1A and DMD
cells Human LAM-111 in mouse MDC1A and DMD
muscle cells Stable and scalable human LAM-111
manufacturing process Under evaluation for treatment of
dystroglycanopathy and dysferlinopathy
Ashley
Merosin Deficient (MDC1A) mouse treated with laminin-111 (10mg LAM-111/kg/week starting at 10 days of age)
Merosin Deficient (MDC1A) mice have a reduced lifespan and normally live between 6-15 weeks of age, so 1 year is a very significant milestone
Picture provided by Prothelia collaborator Dr. Dean Burkin, University of Nevada, Reno
Adelina
Industry Science
Drug Development Program
AdvocacyGovernment
Drug manufacture
Animal testing
Clinical testing
Project coordination
Investment
Development incentives
Funding support
Regulatory guidance
Rare Disease Program (HTS
etc)
Preclinical models
Natural history
Patient identification and
accrual
Investment
Patient referral
Trial promotion
Investment
Maia
3 in 5 Phase III Trials Succeed
* Data from Pharma drugs developed entirely in-house
Adams CP, Brantner VV (2010) “Spending on New Drug Development” Health Econ. 19: 130–141 (2010)
Only 1 in 13Candidates Succeed
Pierce
*LifeSciences World 10/13/2006
Phase I: About $15K/personPhase II: About $19K/personPhase III: About $26K/person
What the non-profit and government communities have done (funded):• Funding the “Valley of Death”• Developing International Patient Registries• Developing Clinical Research Networks• Developing and Validating Clinical Endpoints• Developing Natural History to accurately power studies•Standards of Care
Areas of improvement:• We need biomarkers to decrease testing time• We need novel trial designs to reduce costs and time• We need centralized IRBs• We need electronic medical records• We need clinical data standards• We need harmonization between the FDA and EMEA
Jake
– Brad Hodges, PhD Chairman, SMAB; CSO, Prothelia, Inc.
– Dean Burkin, PhD Lead Investigator; Director, Nevada Transgenic Center for Biomedical Research Excellence,
Associate Professor, University of Nevada School of Medicine
– Ed Connor, MD Director, Office of Investigational Therapeutics at Children's National Medical Center and
Professor of Pediatrics at George Washington University School of Medicine and Health Sciences
– Eric Hoffman, PhD Chairman, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences and Director, Research Center for Genetic Medicine at Children's National Medical Center
– George Vella, PhD Director of Research and Strategic Planning,Charley’s Fund
– John M McCall, PhD President, PharMac LLC
– Markus A Ruegg, PhD Professor of Neurobiology, BiozentrumUniversity of Basel
– Steve D Hauschka, PhD Professor of Biochemistry, University of Washington
– Brad Hodges, PhD Chairman, SMAB; CSO, Prothelia, Inc.
– Dean Burkin, PhD Lead Investigator; Director, Nevada Transgenic Center for Biomedical Research Excellence,
Associate Professor, University of Nevada School of Medicine
– Ed Connor, MD Director, Office of Investigational Therapeutics at Children's National Medical Center and
Professor of Pediatrics at George Washington University School of Medicine and Health Sciences
– Eric Hoffman, PhD Chairman, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences and Director, Research Center for Genetic Medicine at Children's National Medical Center
– George Vella, PhD Director of Research and Strategic Planning,Charley’s Fund
– John M McCall, PhD President, PharMac LLC
– Markus A Ruegg, PhD Professor of Neurobiology, BiozentrumUniversity of Basel
– Steve D Hauschka, PhD Professor of Biochemistry, University of Washington
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Kyra
Patient Advocacy Groups– Cure CMD (Congenital Muscular Dystrophy)– Struggle Against Muscular Dystrophy (SAM), Ireland– Muscular Dystrophy Association (MDA) – Parent Project Muscular Dystrophy (PPMDP)– Charley’s Fund, Inc.
Government Organizations– National Institute of Health (NIH) – financial support (3
grants)– Treat-NMD (Neuromuscular Disease), European Union
Patient Advocacy Groups– Cure CMD (Congenital Muscular Dystrophy)– Struggle Against Muscular Dystrophy (SAM), Ireland– Muscular Dystrophy Association (MDA) – Parent Project Muscular Dystrophy (PPMDP)– Charley’s Fund, Inc.
Government Organizations– National Institute of Health (NIH) – financial support (3
grants)– Treat-NMD (Neuromuscular Disease), European Union
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