end duchenne grant award program (gap)
DESCRIPTION
End Duchenne Grant Award Program (GAP) presented by Sharon Hesterlee at PPMD's 2010 Annual Connect ConferenceTRANSCRIPT
PPMD’s Research Strategy Sharon Hesterlee, Ph.D. Sr Director Research and Advocacy
Cost of Developing a New Drug* (“New Chemical EnFty”)
All failed Drugs $400M in opportunity costs
Preclinical Development 42%
true costs
Clinical Development 58% true costs
$1B
3 in 5 Phase III Trials Succeed
* Data from Pharma drugs developed enFrely in-‐house
Adams CP, Brantner VV (2010) “Spending on New Drug Development” Health Econ. 19: 130–141 (2010)
Only 1 in 13 Candidates Succeed
*LifeSciences World 10/13/2006
Clinical Trial Costs*
Phase I: About $15K/person Phase II: About $19K/person Phase III: About $26K/person
Cost Per PaFent: DMD
Best Case Moderate Case
Worst Case
Market is 25,000 worldwide
$40,000/PaFent
Market is 10,000 worldwide
$100,000/PaFent $200,000/PaFent
Market is 5,000 worldwide
Plus ongoing manufacturing and markeFng costs plus profit margin = esFmated $500 million/year to recoup all cost and make a profit
Boblenecks in Drug Development
Defraying Clinical Development Costs
What the non-‐profit and government communiFes have done (funded): • Funding the “Valley of Death” • Developing InternaFonal PaFent Registries • Developing Clinical Research Networks • Developing and ValidaFng Clinical Endpoints • Developing Natural History to accurately power studies • Standards of Care
Where we need to improve: • We need biomarkers to decrease tesFng Fme • We need novel trial designs to reduce costs and Fme • We need centralized IRBs • We need electronic medical records • We need clinical data standards • We need harmonizaFon between the FDA and EMEA
PPMD Research Program: Strategic DirecFons
• Increase the efficiency of research
• Support crucial infrastructure
• Fund preclinical and clinical drug development “Valley of Death”
• Advocate for the above
• Leverage, leverage, leverage
PPMD Research Programs
Grant Program Goal
InvesFgators Award Fund preclinical and clinical drug development “Valley of Death”
Venture Philanthropy Corporate Grant Fund preclinical and clinical drug development “Valley of Death”
End Duchenne GAP Program Increase the efficiency of research; Fund preclinical drug development “Valley of Death”
Infrastructure Grant Support crucial infrastructure
Bridge Funds Increase the efficiency of research
63% 7%
1%
15%
12%
2%
Project Catalyst
Infrastructure
Workshops
End Duchenne GAP
Other
Fellowships
PPMD Near Term Research Expenditures: ~ $4 Million
10
High throughput screen in collaboraFon with PTC TherapeuFcs for four –six targets:
1. UpregulaFon of Utrophin
2. InhibiFon of myostaFn/IGF-‐1
3. UpregulaFon of alpha-‐7 integrin
4. UpregulaFon of utrophin
5. InhibiFon of SERCA2a/phospholambin
Drug Development: Industry
Increasing Research Efficiency: END Duchenne Grant Award Program
Concept: Provide funds (smaller dollars) to invesFgators working on therapeuFcs for DMD to improve potenFal for NIH award (big dollars)
Year One Awardees:
JusFn Fallon, Brown University: Biglycan to upregulate utrophin Krista Vandenborne, University of Florida: MRI as an endpoint Melissa Spencer, UCLA: OsteoponFn target to prevent fibrosis Carmen Bertoni, UCLA: Stop codon read-‐through drug Brad Hodges, Prothelia: Laminin 111 therapeuFc
$7M from NIH
$253,000 from NIH
PPMD InvesFgator Award: Valley of Death Dongsheng Duan, Ph.D., University of Missouri $111,000
-‐OpFmizing gene therapy vectors for cardiomyopathy
PPMD InvesFgator Award: Valley of Death Jerry Mendell, M.D., NaFonwide Children's Research InsFtute $600,000
-‐Gene Therapy trial of follistaFn for Becker muscular dystrophy
Block Grants for Travel for ACE-‐031: Bridging the Valley of Death
$5000/site for up to 20 sites in Canada in the U.S:
Will help cover
-‐gas -‐overnight accommodaFons -‐meals
First award has gone out to Alberta Children’s Hospital
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Bridging the Valley of Death
Lead Candidates OpFmizaFon Pre-‐IND I II III
Ataluren (PTC-‐124)
MyostaFn Inhibitor
Utrophin Upregulator
IGF-‐1 Upregulator
SERCA2a Upregulator
IniFal funding from PPMD,MDA, Charley’s Fund leveraged over $400 million from other sources
IniFal investment of $2.6M by PPMD led to $15.4M grant from NINDS for an academic collaboraFon to further the project
Biglycan (Tyverson)
Lamin III (Prothelia)
FollistaFn gene therapy (Jerry Mendell, NaFonwide)
ACE-‐031(Acceleron, travel awards)
CriFcal Infrastructure: DuchenneConnect
DuchenneConnect is a robust curated registry that contributes core data elements to the TREAT-‐NMD Global registry. DuchenneConnect also collects over 100 addiFonal fields of informaFon and can send out announcements in a broad or targeted fashion.
Since 2008 Duchenne Connect has alone or via TREAT-‐NMD Global Registry:
• Provided data to four companies for feasibility studies to idenFfy cohorts of eligible trial parFcipants
• Provided data for one biorepository recruitment effort
• Provided data for three pre-‐markeFng requests
• Sent out noFces for interview studies and clinical recruitment announcements
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R&D Strategy: Leverage and Gaps
d
$1.3M
$2.9M
Drug Development (Academic)
Drug Development (Industry)
Clinical Infrastructure (endpoints, biomarkers)
$0.4M
PPMD funds are applied strategically: we fill gaps and we leverage hundreds of millions of dollars from other sources.
Through MD CARE Act, leveraged an addiFonal $200M dollars from federal government since 2001
Leveraged an addiFonal $15.4 million from NINDS to support PTC
TherapeuFcs investment Leveraged an addiFonal $22M dollars from the CDC for DMD-‐
specific programs
QuesFons?
Sharon Hesterlee, Ph.D. Sr Director Research and Advocacy [email protected]
(520) 444-‐4462